DDrare: Database of Drug Development for Rare Diseases

113. 筋ジストロフィー
［臨床試験数：567，薬物数：442（DrugBank：93），標的遺伝子数：55，標的パスウェイ数：151］

Searched query = "Muscular dystrophy", "Dystrophinopathies", "Myotilinopathy", "Laminopathy", "Caveolinopathy", "LGMD1C", "Desminopathy", "Sarcoglycanopathy", "α-dystroglycanopathy", "FCMD", "Walker-Warburg syndrome", "Muscle-eye-brain disease", "Myotonic dystrophy", "Integrin α7 deficient CMD", "Rigid spine syndrome"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: 03/15/2021. Trials are sorted by Date_enrollment from most recent to oldest in the table.

Drug = (+)- Epicatechin; (+)-Epicatechin; (-)-Epicatechin; (-)-epicatechin; 12 mg/kg GSK2402968; 2011-2012 seasonal flu vaccine Intramuscular; 2011-2012 seasonal flu vaccine Subcutaneous; 29180.00.00, 34681.00.00, 34681.01.00, 34681.02.00; 3 mg/kg GSK2402968; 4-Benzyl-2-naphthalen-1-yl-1,2,4- thiadiazolidine-3,5-dione; 4-benzyl-2-naphthalen-1-yl-1,2,4-thiadiazolidine-3,5-dione; 5-(ethylsulfonyl)-2-(naphthalen-2-yl)benzo[d]oxazole; 6 mg/kg GSK2402968; 750 mg metformin and 7.5 g L-citrulline daily p.o.; 9 mg/kg GSK2402968; AAV1-gamma-sarcoglycan vector injection; ACE-031 (Extension of cohort 1 from core study, A031-03); ACE-031 (Extension of cohort 2 from core study, A031-03); ACE-031 (Extension of cohort 3 from core study, A031-03); ACE-031 0.5 mg/kg q4wk; ACE-031 1.0 mg/kg q2wk; ACE-083; ASP0367; ATALUREN; ATYR1940; AVI-4658; AVI-4658 (Eteplirsen); AVI-4658 (PMO); AVI-4658 for Injection; Acetate; Actonel Once a week 5mg film coated tablets; Adeno-associated viral vector serotype 9 containing the human mini-dystrophin gene; Albuterol; Allogeneic Cardiosphere-Derived Cells (CAP-1002); Anti-Myostatin Adnectin; Anti-myostati; Anti-myostatin Adnectin; Arginine; Aspirin; Ataluren; Ataluren 1000 mg; Ataluren 125 mg; Ataluren 250 mg; Autologous bone marrow mononuclear cell transplantation; BIO101; BLS-M22; BMN 044; BMN 044 IV 6 mg/kg; BMN 044 IV 9 mg/kg; BMN 044 SC 6 mg/kg; BMN 045; BMN 053; BMN053; BMS-986089; BMS-986089-01 (RO7239361) , anti-myostatin; BMS-986089-01 (RO7239361) Injections, 15 mg/Syringe (21.4 mg/mL); BMS-986089-01 (RO7239361) Injections, 35 mg/Syringe (50 mg/mL); BMS-986089-01 (RO7239361) Injections, 50 mg/Syringe (71.4 mg/mL); BMS-986089-01 (RO7239361) Injections, 7.5 mg/Syringe (10.7 mg/mL); BMS-986089-01 (RO7239361), anti-myostatin; BMS-986089-01 Injections, 15 mg/Syringe (21.4 mg/mL); BMS-986089-01 Injections, 35 mg/Syringe (50 mg/mL); BMS-986089-01 Injections, 50 mg/Syringe (71.4 mg/mL); BMS-986089-01 Injections, 7.5 mg/Syringe (10.7 mg/mL); BMS-986089-01, Anti-myostatin; BMS-986089-01, anti-myostatin; BOTULINUM TOXIN TYPE A; Balance; Behavioral: In-home Exercise Training; Behavioral: Regular exercise; Biological: Umbilical Cord Based Allogenic Mesenchymal Stem Cell; Bisoprolol; Bisoprolol Fumarate; Bisphosphonate treatment; Blood test; Botulinum toxin type A; CALCIFEDIOL; CAP-1002; CARVEDILOL; CASIMERSEN; CAT-1004; CRD007; Cabaletta; Calcichew D3 Forte; Calcifediol; Canakinumab; Canakinumab Injection [Ilaris]; Cardicor; Cardicor (Merck brand); Cardiovascular profile; Carvedilol; Casimersen; Chronic Corticosteroid Therapy; Cialis 10 mg film-coated tablets; Cialis 2.5 mg film-coated tablets; Cialis 20 mg film-coated tablets; Cialis 5 mg film-coated tablets; Citrulline; Coenzyme Q10; Coenzyme Q10 and Lisinopril; Cosyntropin; Cosyntropin acetate; Cr13C; Creatine; Creatine Monohydrate; Creatine monohydrate; Creatine-13C; Cyclosporin; Cyclosporin A; DEFLAN*10CPR 6MG; DEFLAZACORT; DELTACORTENE*20CPR 5MG; DHA; DILATREND; DRISAPERSEN SODIUM; DS-5141b; Decortin; Deflazacort; Deflazacort 6 mg Tablets; Dehydroepiandrosterone; Dehydroepiandrosterone 100 and 400 mg; Device: Activity Monitor; Dexmedetomidine; Diagnostic Test: 30 seconds sit to stand test; Diagnostic Test: Beck depression inventory; Diagnostic Test: Berg balance scale; Diagnostic Test: Brief Pain Inventory Short-Form; Diagnostic Test: DM1-ActivC; Diagnostic Test: Fatigue and Daytime Sleepiness Scale; Diagnostic Test: Functional Index-2; Diagnostic Test: GSGC; Diagnostic Test: Hand opening time; Diagnostic Test: Manual muscle testing; Diagnostic Test: McGill pain questionnaire; Diagnostic Test: Myotonia (from Individualised Neuromuscular Quality of Life Questionnaire); Diagnostic Test: Myotonia Behaviour scale; Diagnostic Test: Pressure pain threshold; Diagnostic Test: Quantitative muscle testing; Diagnostic Test: Quick motor function test; Diagnostic Test: R-PAct; Diagnostic Test: Scale for Assessment and Rating of Ataxia; Diagnostic Test: Six minute walking test; Dosing Extension; Drisapersen; EDASALONEXENT; EDG-5506; EGCG; EMD 87580; EMEA/H/C/000638; EMFLAZA® (DEFLAZACORT); EMFLAZA® (deflazacort); ENALAPRIL; EPA; EPA and DHA; ERX-963; ETEPLIRSEN; EV substance code: SUB188638; EXONDYS 51™; Edasalonexent; EnaHexal; EnaHexal®, 10mg; EnalHexal®, 5 mg; Enalapril; Epicatechin; Epigallocatechin; Epigallocatechin-Gallate; Eplerenone; Eteplirsen; Eteplirsen Injection; Eucardic; Exondys 51; Ezutromid; FG-3019; FTX-1821; Filgrastim; Flavocoxid; GIVINOSTAT; GIVINOSTAT (hyrochloride monohydrate); GOLODIRSEN; GSK2402968; GSK2402968 3mg/kg/week; GSK2402968 6 mg/kg/week; GSK2402968 6mg/kg/week; Genetic: RNA extraction; Gentamicin; Gentamicin infusions twice a week for six months; Givinostat; Givinostat (hydrochloride monohydrate); Givinostat (idrocloruro monoidrato); Glutamine; Gold; Golodirsen; Granulocyte colony-stimulating factor (Filgrastim); Guanidine; H44AON188; H51AON23; HIDROFEROL; HLA-identical allogeneic mesoangioblasts; HMABs; HT-100; Haemostasis tests; Hidroferol; Hidroferol®; Hormonal profile; Human Umbilical Cord Mesenchymal Stem Cells; Human umbilical cord mesenchymal stem cells; IBUPROFEN; IBUPROFEN 200MG; IDEBENONE; IGF-1; IONIS-DMPKRx; ISOSORBIDE DINITRATE; ITF2357; Ibuprofen; Idebenone; Idebenone 150 mg film-coated tablets; Ifetroban; Inflammatory profile; Isosorbide; Isosorbide Dinitrate; Isosorbide Dinitrate 20 mg; Isosorbide Dinitrate 40 mg; Isosorbide Dinitrate 60 mg; Isosorbide Dinitrate 80 mg; Isosorbide dinitrate; Ketamine; L-Arginine; L-Citrulline and Metformin & L-Citrulline; L-Glutamine; L-arginine; LOSMAPIMOD; Lentiviral Vector Trans-Skip gene modified Autologous Mesoangioblasts; Lisinopril; Lonafarnib; Losartan; Losmapimod; METFORMIN; METFORMINA - 500 30 COMPRESSE 500 MG; METOPROLOL SUCCINATE; MNK-1411; MONITOR; MYO-029; MYODM; Metabolic profile; Metformin; Metformin 500mg; Metformin and Metformin & L-Citrulline; Metformina; Methylphenidate; MetoHEXAL® Succ ® 23,75 mg Retardtabletten; MetoHEXAL® Succ ® 47,5 mg Retardtabletten; MetoHEXAL® Succ ® 95 mg Retardtabletten; Metoprolol; Mexiletine; Monocytes and megacaryocytes culture and RNA extraction; Myoblast autologous graft; Myoblast transplantation; Myoblastes autologues; N(4,5Bis methanesulfonyl2methylbenzoyl) guanidine hydrochloride monohydrate; N-(4,5-Bis methanesulfonyl-2-methyl-benzoyl)guanidine hydrochloride monohydrate; NPC-14; NS-065/NCNP-01; NS-089/NCNP-02; Nanosuspension); Nebivolol; Needle biopsy of the vastus lateralis muscle; Nitrate; Nébivolol; Oligomero morfolino fosforodiamidato per skipping dell’esone 45; Oligomero morfolino fosforodiamidato per skipping dell’esone 53; Omigapil; Other: Dystrophin levels; Other: Echocardiogram; Other: Ibuprofen and Isosorbide Dinitrate combination; Other: Magnetic Resonance Imaging (MRI); Other: Metabolic Exercise Testing using stationary bicycle; Other: Pulmonary Function Testing (PFT); Other: Standard steroid treatment; Other: beetroot juice extract; Oxatomide; Oxatomide (tinset); P-188 NF; PAMREVLUMAB; PB1046 Injection; PEMIROLAST; PF-06252616; PF-06939926; PFT; PRD2175434; PREDNISONE; PRO044; PRO044 IV; PRO044 IV 6 mg/kg; PRO044 IV 9 mg/kg; PRO044 SC; PRO044 SC 6 mg/kg; PRO045; PRO045, 0.15 mg/kg/week; PRO045, 1.0 mg/kg/week; PRO045, 3.0 mg/kg/week; PRO045, 6.0 mg/kg/week; PRO045, 9.0 mg/kg/week; PRO045, selected dose; PRO051; PRO053; PS188; PS188 (company code); PS220; PS524; PTC0161480, PTC124 Compound 1a, RPS2505, PTC-C124, 291844; PTC124; Pamrevlumab; Pamrevlumab (FG-3019); Pemirolast; Pending; Pentoxifylline; Peptide-conjugated phosphorodiamidate morpholino oligomer for exon 51 skipping; Perindopril; Perindopril 2mg Tablets; Phosphorodiamidate Morpholino Oligomer; Phosphorodiamidate morpholino oligomer for exon 45 skipping; Phosphorodiamidate morpholino oligomer for exon 53 skipping; Powder for Oral Suspension); Prednisolone; Prednison; Prednisone; Prednisone 5 mg tablets; Procedure: Increased exercise intensity; Procedure: Saline injection; Procedure: Taking of blood; Protein-carbohydrate supplementation; RAAV1.CMV.huFollistatin344; RAAV1.CMV.huFollistin344; RAAV2.5-CMV-minidystrophin (d3990); RAAV9-CK8-h-µD5; RAAVrh74.MCK.GALGT2; RAAVrh74.MCK.micro-Dystrophin; RAXONE - 150 MG- COMPRESSE RIVESTITE CON FILM- USO ORALE- FLACONE (HDPE)- 180 COMPRESSE; RO7239361; RO7239361 (BMS-986089); RO7239361 Injections, 15 mg/Syringe (21.4 mg/mL); RO7239361 Injections, 35 mg/Syringe (50 mg/mL); RO7239361 Injections, 50 mg/Syringe (71.4 mg/mL); RO7239361 Injections, 7.5 mg/Syringe (10.7 mg/mL); Ramipril; Ranolazine; Raxone; Recombinant human anti-GDF-8 antibody; Regimen Selection Phase Group 1 (COMPLETED); Regimen Selection Phase Group 2; Regimen Selection Phase Group 3; Resveratrol; Revatio; RhIGF-I/rhIGFBP-3; RhIGFBP-3; Rimeporide; Risedronate; Risedronate sodium; SGT-001; SMT C1100; SMT C1100 (F5; SMT C1100 (F5); SMT C1100 (F6; SMT C1100 (F6); SNT-MC17; SRP-4045; SRP-4053; SRP-5051; Sandimmun Optoral; ScAAV9.U7.ACCA; ScAAVrh74.tMCK.hSGCA; Sildenafil; Sleep parameters; Sodium Nitrate; Sodium Nitrate - double dose; Sodium nitrate; SomatoKine/IPLEX; Somatropin; Spironolactone; Stem Cell; Stem Cells; Sunphenon; Sunphenon EGCG; Sustanon; Sustanon (testosterone); Sustanon 250; Suvodirsen; TADALAFIL; TAMOX; TAMOXIFEN; TAMOXIFEN CITRATE; TAS-205; TETRACOSACTIDE HEXAACETATE; TRIATEC; Tacrolimus; Tadalafil; Tadalafil 20 MG; Tadalafil and Sildenafil; Tamoxifen; Tamoxifen 20mg Hexal® Filmtabletten; Tehalose 30gr; Temerit; Testosterone; Testosterone Enanthate; Testosterone decanoate; Testosterone enanthate; Testosterone isocaproate; Testosterone phenylpropionate; Testosterone propionate; Tetracosactide; Tideglusib; Tinset; Tranilast; Translarna; Translarna 1000 mg granules for oral suspension; Translarna 125 mg granules for oral suspension; Translarna 250 mg granules for oral suspension; Treatment Phase Group 4; Trehalose; Triatec; Umbilical Cord Mesenchymal Stem Cell; Umbilical Cord Mesenchymal Stem Cells; VAMOROLONE; VBP15; VILTOLARSEN; Vamarolone; Vamorolone; Vamorolone 0.25 mg/day/day; Vamorolone 0.25 mg/kg/day; Vamorolone 0.75 mg/day/day; Vamorolone 0.75 mg/kg/day; Vamorolone 2.0 mg/day/day; Vamorolone 2.0 mg/kg/day; Vamorolone 6.0 mg/day/day; Vamorolone 6.0 mg/kg/day; Viltolarsen; Vit C Vit E Zn Se; WVE-210201; WVE-210201 (suvodirsen); Xeomin; Zn; [WVE-210201]

No.	TrialID	Date_ enrollment	Date_ registration	Public_title	Scientific_title	Condition	Intervention	Primary_ sponsor	Secondary_ sponsor	Recruitment_ Status	Inclusion_ agemin	Inclusion_ agemax	Inclusion_ gender	Target_ size	Phase	Countries
1	EUCTR2016-000602-10-BG (EUCTR)	10/03/2020	07/01/2020	A clinical study to assess how effective and safe is idebenone treatment in patients with Duchenne Muscular Dystrophy (DMD) who are currently receiving Glucocorticoid steroids	A Phase III Double-blind, Randomized, Placebo-Controlled Study assessing the Efficacy, Safety and Tolerability of Idebenone in Patients with Duchenne Muscular Dystrophy Receiving Glucocorticoid Steroids - SIDEROS	Duchenne Muscular Dystrophy (DMD) MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]	Trade Name: Raxone Product Name: Idebenone INN or Proposed INN: IDEBENONE Other descriptive name: IDEBENONE	Santhera Pharmaceuticals (Switzerland) Limited	NULL	Not Recruiting			Female: no Male: yes	266	Phase 3	United States;Spain;Ireland;Austria;Israel;United Kingdom;Italy;Switzerland;France;Hungary;Belgium;Bulgaria;Germany;Netherlands;Sweden
2	EUCTR2017-004279-30-NL (EUCTR)	23/01/2020	16/05/2018	A clinical study to assess the long-term safety and efficacy of idebenone treatment in patients with Duchenne Muscular Dystrophy (DMD) who completed the SIDEROS study.	A Phase III Open-Label Extension Study to Assess the Long-Term Safety and Efficacy of Idebenone in Patients with Duchenne Muscular Dystrophy (DMD) who completed the SIDEROS study - SIDEROS-E	Duchenne Muscular Dystrophy (DMD) MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]	Trade Name: Raxone Product Name: Idebenone INN or Proposed INN: IDEBENONE Other descriptive name: IDEBENONE	Santhera Pharmaceuticals (Switzerland) Limited	NULL	Not Recruiting			Female: no Male: yes	266	Phase 3	United States;France;Spain;Belgium;Austria;Israel;Germany;Netherlands;United Kingdom;Switzerland;Italy;Sweden
3	EUCTR2017-004279-30-AT (EUCTR)	15/02/2019	04/01/2019	A clinical study to assess the long-term safety and efficacy of idebenone treatment in patients with Duchenne Muscular Dystrophy (DMD) who completed the SIDEROS study.	A Phase III Open-Label Extension Study to Assess the Long-Term Safety and Efficacy of Idebenone in Patients with Duchenne Muscular Dystrophy (DMD) who completed the SIDEROS study - SIDEROS-E	Duchenne Muscular Dystrophy (DMD) MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]		Santhera Pharmaceuticals (Switzerland) Limited	NULL	Authorised-recruitment may be ongoing or finished			Female: no Male: yes	266	Phase 3	France;United States;Belgium;Spain;Austria;Israel;Netherlands;Germany;Italy;United Kingdom;Switzerland;Sweden
4	EUCTR2017-004279-30-ES (EUCTR)	21/12/2018	16/11/2018	A clinical study to assess the long-term safety and efficacy of idebenone treatment in patients with Duchenne Muscular Dystrophy (DMD) who completed the SIDEROS study.	A Phase III Open-Label Extension Study to Assess the Long-Term Safety and Efficacy of Idebenone in Patients with Duchenne Muscular Dystrophy (DMD) who completed the SIDEROS study - SIDEROS-E	Duchenne Muscular Dystrophy (DMD) MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]		Santhera Pharmaceuticals (Switzerland) Limited	NULL	Authorised-recruitment may be ongoing or finished			Female: no Male: yes	266	Phase 3	France;United States;Belgium;Spain;Austria;Israel;Netherlands;Germany;Italy;United Kingdom;Switzerland;Sweden
5	EUCTR2016-000602-10-HU (EUCTR)	18/12/2018	05/11/2018	A clinical study to assess how effective and safe is idebenone treatment in patients with Duchenne Muscular Dystrophy (DMD) who are currently receiving Glucocorticoid steroids	A Phase III Double-blind, Randomized, Placebo-Controlled Study assessing the Efficacy, Safety and Tolerability of Idebenone in Patients with Duchenne Muscular Dystrophy Receiving Glucocorticoid Steroids - SIDEROS	Duchenne Muscular Dystrophy (DMD) MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]	Trade Name: Raxone Product Name: Idebenone INN or Proposed INN: IDEBENONE Other descriptive name: IDEBENONE	Santhera Pharmaceuticals (Switzerland) Limited	NULL	Not Recruiting			Female: no Male: yes	266	Phase 3	United States;Spain;Ireland;Austria;Israel;United Kingdom;Italy;Switzerland;France;Hungary;Belgium;Bulgaria;Germany;Netherlands;Sweden
No.	TrialID	Date_ enrollment	Date_ registration	Public_title	Scientific_title	Condition	Intervention	Primary_ sponsor	Secondary_ sponsor	Recruitment_ Status	Inclusion_ agemin	Inclusion_ agemax	Inclusion_ gender	Target_ size	Phase	Countries
6	EUCTR2017-004279-30-GB (EUCTR)	26/10/2018	09/05/2018	A clinical study to assess the long-term safety and efficacy of idebenone treatment in patients with Duchenne Muscular Dystrophy (DMD) who completed the SIDEROS study.	A Phase III Open-Label Extension Study to Assess the Long-Term Safety and Efficacy of Idebenone in Patients with Duchenne Muscular Dystrophy (DMD) who completed the SIDEROS study - SIDEROS-E	Duchenne Muscular Dystrophy (DMD) MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]	Trade Name: Raxone Product Name: Idebenone INN or Proposed INN: IDEBENONE Other descriptive name: IDEBENONE	Santhera Pharmaceuticals (Switzerland) Limited	NULL	Not Recruiting			Female: no Male: yes	266	Phase 3	United States;France;Spain;Belgium;Austria;Israel;Netherlands;Germany;Switzerland;Italy;United Kingdom;Sweden
7	NCT03603288 (ClinicalTrials.gov)	July 4, 2018	31/5/2018	Phase III Study With Idebenone in Patients With Duchenne Muscular Dystrophy (SIDEROS-E)	A Phase III Open-Label Extension Study to Assess the Long-Term Safety and Efficacy of Idebenone in Patients With Duchenne Muscular Dystrophy (DMD) Who Completed the SIDEROS Study	Duchenne Muscular Dystrophy	Drug: idebenone 150 mg film-coated tablets	Santhera Pharmaceuticals	NULL	Recruiting	11 Years	N/A	Male	266	Phase 3	United States;Austria;Belgium;France;Germany;Italy;Spain;Switzerland;United Kingdom
8	EUCTR2017-004279-30-BE (EUCTR)	14/06/2018	04/05/2018	A clinical study to assess the long-term safety and efficacy of idebenone treatment in patients with Duchenne Muscular Dystrophy (DMD) who completed the SIDEROS study.	A Phase III Open-Label Extension Study to Assess the Long-Term Safety and Efficacy of Idebenone in Patients with Duchenne Muscular Dystrophy (DMD) who completed the SIDEROS study - SIDEROS-E	Duchenne Muscular Dystrophy (DMD) MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]	Trade Name: Raxone Product Name: Idebenone INN or Proposed INN: IDEBENONE Other descriptive name: IDEBENONE	Santhera Pharmaceuticals (Switzerland) Limited	NULL	Not Recruiting			Female: no Male: yes	266	Phase 3	United States;France;Spain;Belgium;Austria;Israel;Netherlands;Germany;United Kingdom;Switzerland;Italy;Sweden
9	EUCTR2016-000602-10-NL (EUCTR)	26/04/2017	20/09/2016	A clinical study to assess how effective and safe is idebenone treatment in patients with Duchenne Muscular Dystrophy (DMD) who are currently receiving Glucocorticoid steroids	A Phase III Double-blind, Randomized, Placebo-Controlled Study assessing the Efficacy, Safety and Tolerability of Idebenone in Patients with Duchenne Muscular Dystrophy Receiving Glucocorticoid Steroids - SIDEROS	Duchenne Muscular Dystrophy (DMD) MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]	Trade Name: Raxone Product Name: Idebenone INN or Proposed INN: IDEBENONE Other descriptive name: IDEBENONE	Santhera Pharmaceuticals (Switzerland) Limited	NULL	Not Recruiting			Female: no Male: yes	266	Phase 3	United States;Spain;Ireland;Austria;Israel;Italy;Switzerland;United Kingdom;France;Hungary;Belgium;Bulgaria;Netherlands;Germany;Sweden
10	EUCTR2016-000602-10-GB (EUCTR)	28/12/2016	23/09/2016	A clinical study to assess how effective and safe is idebenone treatment in patients with Duchenne Muscular Dystrophy (DMD) who are currently receiving Glucocorticoid steroids	A Phase III Double-blind, Randomized, Placebo-Controlled Study assessing the Efficacy, Safety and Tolerability of Idebenone in Patients with Duchenne Muscular Dystrophy Receiving Glucocorticoid Steroids - SIDEROS	Duchenne Muscular Dystrophy (DMD) MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]	Trade Name: Raxone Product Name: Idebenone INN or Proposed INN: IDEBENONE Other descriptive name: IDEBENONE	Santhera Pharmaceuticals (Switzerland) Limited	NULL	Not Recruiting			Female: no Male: yes	266	Phase 3	United States;Spain;Ireland;Austria;Israel;United Kingdom;Italy;Switzerland;France;Hungary;Belgium;Bulgaria;Netherlands;Germany;Sweden
No.	TrialID	Date_ enrollment	Date_ registration	Public_title	Scientific_title	Condition	Intervention	Primary_ sponsor	Secondary_ sponsor	Recruitment_ Status	Inclusion_ agemin	Inclusion_ agemax	Inclusion_ gender	Target_ size	Phase	Countries
11	EUCTR2016-000602-10-AT (EUCTR)	21/12/2016	01/12/2016	A clinical study to assess how effective and safe is idebenone treatment in patients with Duchenne Muscular Dystrophy (DMD) who are currently receiving Glucocorticoid steroids	A Phase III Double-blind, Randomized, Placebo-Controlled Study assessing the Efficacy, Safety and Tolerability of Idebenone in Patients with Duchenne Muscular Dystrophy Receiving Glucocorticoid Steroids - SIDEROS	Duchenne Muscular Dystrophy (DMD) MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]	Trade Name: Raxone Product Name: Idebenone INN or Proposed INN: IDEBENONE Other descriptive name: IDEBENONE	Santhera Pharmaceuticals (Switzerland) Limited	NULL	Authorised-recruitment may be ongoing or finished			Female: no Male: yes	266	Phase 3	United States;Spain;Ireland;Austria;Israel;Italy;Switzerland;United Kingdom;France;Hungary;Belgium;Bulgaria;Germany;Netherlands;Sweden
12	EUCTR2016-000602-10-ES (EUCTR)	20/12/2016	18/11/2016	A clinical study to assess how effective and safe is idebenone treatment in patients with Duchenne Muscular Dystrophy (DMD) who are currently receiving Glucocorticoid steroids	A Phase III Double-blind, Randomized, Placebo-Controlled Study assessing the Efficacy, Safety and Tolerability of Idebenone in Patients with Duchenne Muscular Dystrophy Receiving Glucocorticoid Steroids - SIDEROS	Duchenne Muscular Dystrophy (DMD) MedDRA version: 19.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]	Trade Name: Raxone Product Name: Idebenone INN or Proposed INN: IDEBENONE Other descriptive name: IDEBENONE	Santhera Pharmaceuticals (Switzerland) Limited	NULL	Authorised-recruitment may be ongoing or finished			Female: no Male: yes	266	Phase 3	France;United States;Belgium;Spain;Austria;Netherlands;Germany;Italy;United Kingdom;Switzerland;Sweden
13	EUCTR2016-000602-10-FR (EUCTR)	25/11/2016	12/01/2017	A clinical study to assess how effective and safe is idebenone treatment in patients with Duchenne Muscular Dystrophy (DMD) who are currently receiving Glucocorticoid steroids	A Phase III Double-blind, Randomized, Placebo-Controlled Study assessing the Efficacy, Safety and Tolerability of Idebenone in Patients with Duchenne Muscular Dystrophy Receiving Glucocorticoid Steroids - SIDEROS	Duchenne Muscular Dystrophy (DMD) MedDRA version: 19.1;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]	Trade Name: Raxone Product Name: Idebenone INN or Proposed INN: IDEBENONE Other descriptive name: IDEBENONE	Santhera Pharmaceuticals (Switzerland) Limited	NULL	Not Recruiting			Female: no Male: yes	266	Phase 3	United States;Spain;Ireland;Austria;Switzerland;United Kingdom;Italy;France;Hungary;Belgium;Bulgaria;Germany;Netherlands;Sweden
14	EUCTR2016-000602-10-DE (EUCTR)	17/11/2016	30/05/2016	A clinical study to assess how effective and safe is idebenone treatment in patients with Duchenne Muscular Dystrophy (DMD) who are currently receiving Glucocorticoid steroids	A Phase III Double-blind, Randomized, Placebo-Controlled Study assessing the Efficacy, Safety and Tolerability of Idebenone in Patients with Duchenne Muscular Dystrophy Receiving Glucocorticoid Steroids - SIDEROS	Duchenne Muscular Dystrophy (DMD) MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]	Trade Name: Raxone Product Name: Idebenone INN or Proposed INN: IDEBENONE Other descriptive name: IDEBENONE	Santhera Pharmaceuticals (Switzerland) Limited	NULL	Not Recruiting			Female: no Male: yes	266	Phase 3	United States;Spain;Ireland;Austria;Israel;Italy;Switzerland;United Kingdom;France;Hungary;Belgium;Bulgaria;Germany;Netherlands;Sweden
15	EUCTR2016-000602-10-IT (EUCTR)	20/10/2016	06/02/2018	A clinical study to assess how effective and safe is idebenone treatment inpatients with Duchenne Muscular Dystrophy (DMD) who are currentlyreceiving Glucocorticoid steroids	A Phase III Double-blind, Randomized, Placebo-Controlled Studyassessing the Efficacy, Safety and Tolerability of Idebenone inPatients with Duchenne Muscular Dystrophy Receiving GlucocorticoidSteroids - SIDEROS	Duchenne Muscular Dystrophy (DMD) MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]	Trade Name: RAXONE - 150 MG- COMPRESSE RIVESTITE CON FILM- USO ORALE- FLACONE (HDPE)- 180 COMPRESSE INN or Proposed INN: IDEBENONE Other descriptive name: IDEBENONE	SANTHERA PHARMACEUTICALS	NULL	Authorised-recruitment may be ongoing or finished			Female: no Male: yes	266	Phase 3	France;United States;Belgium;Spain;Austria;Netherlands;Germany;United Kingdom;Switzerland;Italy;Sweden
No.	TrialID	Date_ enrollment	Date_ registration	Public_title	Scientific_title	Condition	Intervention	Primary_ sponsor	Secondary_ sponsor	Recruitment_ Status	Inclusion_ agemin	Inclusion_ agemax	Inclusion_ gender	Target_ size	Phase	Countries
16	EUCTR2016-000602-10-BE (EUCTR)	02/09/2016	19/07/2016	A clinical study to assess how effective and safe is idebenone treatment in patients with Duchenne Muscular Dystrophy (DMD) who are currently receiving Glucocorticoid steroids	A Phase III Double-blind, Randomized, Placebo-Controlled Study assessing the Efficacy, Safety and Tolerability of Idebenone in Patients with Duchenne Muscular Dystrophy Receiving Glucocorticoid Steroids - SIDEROS	Duchenne Muscular Dystrophy (DMD) MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]	Trade Name: Raxone Product Name: Idebenone INN or Proposed INN: IDEBENONE Other descriptive name: IDEBENONE	Santhera Pharmaceuticals (Switzerland) Limited	NULL	Not Recruiting			Female: no Male: yes	266	Phase 3	United States;Spain;Ireland;Austria;Israel;Italy;Switzerland;United Kingdom;France;Hungary;Belgium;Bulgaria;Germany;Netherlands;Sweden
17	NCT02814019 (ClinicalTrials.gov)	September 2016	17/6/2016	A Phase III Double-blind Study With Idebenone in Patients With Duchenne Muscular Dystrophy (DMD) Taking Glucocorticoid Steroids	A Phase III Double-blind, Randomized, Placebo-Controlled Study Assessing the Efficacy, Safety and Tolerability of Idebenone in Patients With Duchenne Muscular Dystrophy Receiving Glucocorticoid Steroids	Duchenne Muscular Dystrophy (DMD)	Drug: Idebenone 150 mg film-coated tablets;Drug: placebo	Santhera Pharmaceuticals	NULL	Active, not recruiting	10 Years	N/A	Male	255	Phase 3	United States;Austria;Belgium;Bulgaria;France;Germany;Hungary;Ireland;Israel;Italy;Netherlands;Spain;Sweden;Switzerland;United Kingdom
18	NCT03433807 (ClinicalTrials.gov)	January 29, 2013	8/2/2018	Expanded Access Program for Idebenone in Participants With Duchenne Muscular Dystrophy (DMD)	Expanded Access Protocol (EAP) of Idebenone in Patients With Duchenne Muscular Dystrophy	Duchenne Muscular Dystrophy	Drug: Idebenone	Santhera Pharmaceuticals	NULL	Temporarily not available	8 Years	N/A	All			United States
19	EUCTR2009-012037-30-ES (EUCTR)	27/06/2012	29/03/2012	Hereditary neuromuscular disease	A Phase III Double-Blind, Randomised, Placebo-Controlled Study of the Efficacy, Safety and Tolerability of Idebenone in 10 ? 18 Year Old Patients with Duchenne Muscular Dystrophy - DELOS	Duchenne Muscular Dystrophy MedDRA version: 14.1;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]	Product Name: Idebenone Product Code: SNT-MC17 INN or Proposed INN: IDEBENONE	Santhera Pharmaceuticals (Switzerland) Limited	NULL	Not Recruiting			Female: no Male: yes	280	Phase 3	France;United States;Belgium;Spain;Austria;Netherlands;Germany;Italy;Switzerland;Sweden
20	EUCTR2009-012037-30-IT (EUCTR)	27/03/2012	26/04/2012	(DELOS) DuchEnne MD Long-term IdebenOne Study	A Phase III Double-Blind, Randomised, Placebo-Controlled Study of the Efficacy, Safety and Tolerability of Idebenone in 10 – 18 Year Old Patients with Duchenne Muscular Dystrophy - DELOS	Duchenne Muscular Dystrophy MedDRA version: 14.1;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]	Product Name: Idebenone Product Code: NA INN or Proposed INN: IDEBENONE	SANTHERA PHARMACEUTICALS (SWITZERLAND) LTD	NULL	Not Recruiting			Female: no Male: yes	280	Phase 3	France;United States;Canada;Spain;Belgium;Austria;Netherlands;Germany;Italy;Sweden
No.	TrialID	Date_ enrollment	Date_ registration	Public_title	Scientific_title	Condition	Intervention	Primary_ sponsor	Secondary_ sponsor	Recruitment_ Status	Inclusion_ agemin	Inclusion_ agemax	Inclusion_ gender	Target_ size	Phase	Countries
21	EUCTR2009-012037-30-NL (EUCTR)	03/06/2010	17/12/2009	Hereditary neuromuscular disease	A Phase III Double-Blind, Randomised, Placebo-Controlled Study of the Efficacy, Safety and Tolerability of Idebenone in 10 – 18 Year Old Patients with Duchenne Muscular Dystrophy - DELOS	Duchenne Muscular Dystrophy MedDRA version: 17.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]	Product Name: Idebenone Product Code: SNT-MC17 INN or Proposed INN: IDEBENONE	Santhera Pharmaceuticals (Switzerland) Limited	NULL	Authorised-recruitment may be ongoing or finished			Female: no Male: yes	266	Phase 3	France;United States;Belgium;Spain;Austria;Germany;Netherlands;Italy;Switzerland;Sweden
22	EUCTR2009-012037-30-AT (EUCTR)	10/03/2010	10/02/2010	Hereditary neuromuscular disease	A Phase III Double-Blind, Randomised, Placebo-Controlled Study of the Efficacy, Safety and Tolerability of Idebenone in 10 – 18 Year Old Patients with Duchenne Muscular Dystrophy - DELOS	Duchenne Muscular Dystrophy MedDRA version: 17.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]	Product Name: Idebenone Product Code: SNT-MC17 INN or Proposed INN: IDEBENONE	Santhera Pharmaceuticals (Switzerland) Limited	NULL	Not Recruiting			Female: no Male: yes	266	Phase 3	France;United States;Belgium;Spain;Austria;Netherlands;Germany;Italy;Switzerland;Sweden
23	EUCTR2009-012037-30-SE (EUCTR)	23/02/2010	28/12/2009	Hereditary neuromuscular disease	A Phase III Double-Blind, Randomised, Placebo-Controlled Study of the Efficacy, Safety and Tolerability of Idebenone in 10 – 18 Year Old Patients with Duchenne Muscular Dystrophy - DELOS	Duchenne Muscular Dystrophy MedDRA version: 17.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]	Product Name: Idebenone Product Code: SNT-MC17 INN or Proposed INN: IDEBENONE	Santhera Pharmaceuticals (Switzerland) Limited	NULL	Not Recruiting			Female: no Male: yes	266	Phase 3	France;United States;Belgium;Spain;Austria;Netherlands;Germany;Italy;Switzerland;Sweden
24	EUCTR2009-012037-30-FR (EUCTR)	19/02/2010	17/12/2009	A Phase III Double-Blind, Randomised, Placebo-Controlled Study of the Efficacy, Safety and Tolerability of Idebenone in 10 – 18 Year Old Patients with Duchenne Muscular Dystrophy - DELOS	A Phase III Double-Blind, Randomised, Placebo-Controlled Study of the Efficacy, Safety and Tolerability of Idebenone in 10 – 18 Year Old Patients with Duchenne Muscular Dystrophy - DELOS	Duchenne Muscular Dystrophy MedDRA version: 12.0;Level: LLT;Classification code 10013801;Term: Duchenne muscular dystrophy	Product Name: Idebenone Product Code: SNT-MC17 INN or Proposed INN: IDEBENONE	Santhera Pharmaceuticals (Switzerland) Limited	NULL	Authorised-recruitment may be ongoing or finished			Female: no Male: yes	240	Phase 3	Germany;Netherlands;Belgium;France;Spain;Italy;Austria;Sweden
25	EUCTR2009-012037-30-DE (EUCTR)	08/10/2009	07/08/2009	Hereditary neuromuscular disease	A Phase III Double-Blind, Randomised, Placebo-Controlled Study of the Efficacy, Safety and Tolerability of Idebenone in 10 – 18 Year Old Patients with Duchenne Muscular Dystrophy - DELOS	Duchenne Muscular Dystrophy MedDRA version: 17.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]	Product Name: Idebenone Product Code: SNT-MC17 INN or Proposed INN: IDEBENONE	Santhera Pharmaceuticals (Switzerland) Limited	NULL	Not Recruiting			Female: no Male: yes	266	Phase 3	United States;France;Spain;Belgium;Austria;Netherlands;Germany;Switzerland;Italy;Sweden
No.	TrialID	Date_ enrollment	Date_ registration	Public_title	Scientific_title	Condition	Intervention	Primary_ sponsor	Secondary_ sponsor	Recruitment_ Status	Inclusion_ agemin	Inclusion_ agemax	Inclusion_ gender	Target_ size	Phase	Countries
26	EUCTR2009-012037-30-BE (EUCTR)	14/07/2009	27/05/2009	Hereditary neuromuscular disease	A Phase III Double-Blind, Randomised, Placebo-Controlled Study of the Efficacy, Safety and Tolerability of Idebenone in 10 – 18 Year Old Patients with Duchenne Muscular Dystrophy - DELOS	Duchenne Muscular Dystrophy MedDRA version: 17.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]	Product Name: Idebenone Product Code: SNT-MC17 INN or Proposed INN: IDEBENONE	Santhera Pharmaceuticals (Switzerland) Limited	NULL	Authorised-recruitment may be ongoing or finished			Female: no Male: yes	266	Phase 3	France;United States;Spain;Belgium;Austria;Netherlands;Germany;Italy;Switzerland;Sweden
27	NCT01027884 (ClinicalTrials.gov)	July 2009	8/12/2009	Phase III Study of Idebenone in Duchenne Muscular Dystrophy (DMD)	A Phase III Double-Blind, Randomised, Placebo-Controlled Study of the Efficacy, Safety and Tolerability of Idebenone in 10-18 Year Old Patients With Duchenne Muscular Dystrophy	Muscular Dystrophy, Duchenne;Ambulatory Care	Drug: Placebo;Drug: Idebenone	Santhera Pharmaceuticals	NULL	Completed	10 Years	18 Years	Male	65	Phase 3	United States;Austria;Belgium;France;Germany;Italy;Netherlands;Spain;Sweden;Switzerland
28	NCT00758225 (ClinicalTrials.gov)	September 2008	23/9/2008	Long-term Safety, Tolerability and Efficacy of Idebenone in Duchenne Muscular Dystrophy (DELPHI Extension)	A Phase II Open-label Extension Study to Obtain Long-term Safety, Tolerability and Efficacy Data of Idebenone in the Treatment of Duchenne Muscular Dystrophy - Extension to Study SNT-II-001	Duchenne Muscular Dystrophy	Drug: Idebenone	Santhera Pharmaceuticals	NULL	Completed	N/A	N/A	Male	21	Phase 2	Belgium
29	EUCTR2007-007752-34-BE (EUCTR)	14/08/2008	26/06/2008	A Phase II open-label extension study to obtain long-term safety, tolerability and efficacy data of Idebenone in the treatment of Duchenne Muscular Dystrophy	A Phase II open-label extension study to obtain long-term safety, tolerability and efficacy data of Idebenone in the treatment of Duchenne Muscular Dystrophy	Duchennes Muscular Dystrophy MedDRA version: 12.0;Level: LLT;Classification code 10013801;Term: Duchenne muscular dystrophy	Product Name: Idebenone Product Code: SNT-MC17 INN or Proposed INN: Idebenone	Santhera Pharmaceuticals (Switzerland) Ltd	NULL	Not Recruiting			Female: no Male: yes		Phase 2	Belgium
30	NCT00654784 (ClinicalTrials.gov)	October 2005	3/4/2008	Efficacy and Tolerability of Idebenone in Boys With Cardiac Dysfunction Associated With Duchenne Muscular Dystrophy	A Phase IIa Double Blind, Randomised, Placebo Controlled, Single Centre Study at the University of Leuven to Assess the Efficacy and Tolerability of Idebenone in 8 - 16 Year Old Males With Cardiac Dysfunction Associated With Duchenne Muscular Dystrophy	Duchenne Muscular Dystrophy (DMD)	Drug: idebenone;Drug: placebo	Santhera Pharmaceuticals	NULL	Completed	8 Years	16 Years	Male	21	Phase 2	Belgium
No.	TrialID	Date_ enrollment	Date_ registration	Public_title	Scientific_title	Condition	Intervention	Primary_ sponsor	Secondary_ sponsor	Recruitment_ Status	Inclusion_ agemin	Inclusion_ agemax	Inclusion_ gender	Target_ size	Phase	Countries
31	EUCTR2005-002520-33-BE (EUCTR)	06/09/2005	04/10/2005	A Phase IIa double blind, randomised, placebo controlled, single centre study at the university of Leuven to assess the efficacy and tolerability of idebenone in 10 - 16 year old males with cardiac dysfunction associated with Duchenne Muscular Dystrophy	A Phase IIa double blind, randomised, placebo controlled, single centre study at the university of Leuven to assess the efficacy and tolerability of idebenone in 10 - 16 year old males with cardiac dysfunction associated with Duchenne Muscular Dystrophy	Duchenne Muscular Dystrophy	Product Name: idebenone	Santhera Pharmaceuticals LLC	NULL	Not Recruiting			Female: no Male: yes	21	Phase 2a	Belgium
32	EUCTR2016-000602-10-IE (EUCTR)		06/02/2018	A clinical study to assess how effective and safe is idebenone treatment in patients with Duchenne Muscular Dystrophy (DMD) who are currently receiving Glucocorticoid steroids	A Phase III Double-blind, Randomized, Placebo-Controlled Study assessing the Efficacy, Safety and Tolerability of Idebenone in Patients with Duchenne Muscular Dystrophy Receiving Glucocorticoid Steroids - SIDEROS	Duchenne Muscular Dystrophy (DMD) MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]	Trade Name: Raxone Product Name: Idebenone INN or Proposed INN: IDEBENONE Other descriptive name: IDEBENONE	Santhera Pharmaceuticals (Switzerland) Limited	NULL	Not Recruiting			Female: no Male: yes	266	Phase 3	United States;Spain;Ireland;Austria;Israel;Switzerland;United Kingdom;Italy;France;Hungary;Belgium;Bulgaria;Germany;Netherlands;Sweden
33	EUCTR2017-004279-30-SE (EUCTR)		15/05/2018	A clinical study to assess the long-term safety and efficacy of idebenone treatment in patients with Duchenne Muscular Dystrophy (DMD) who completed the SIDEROS study.	A Phase III Open-Label Extension Study to Assess the Long-Term Safety and Efficacy of Idebenone in Patients with Duchenne Muscular Dystrophy (DMD) who completed the SIDEROS study - SIDEROS-E	Duchenne Muscular Dystrophy (DMD) MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]	Trade Name: Raxone Product Name: Idebenone INN or Proposed INN: IDEBENONE Other descriptive name: IDEBENONE	Santhera Pharmaceuticals (Switzerland) Limited	NULL	Not Recruiting			Female: no Male: yes	266	Phase 3	United States;France;Spain;Belgium;Austria;Israel;Netherlands;Germany;United Kingdom;Switzerland;Italy;Sweden
34	EUCTR2016-000602-10-SE (EUCTR)		22/09/2016	A clinical study to assess how effective and safe is idebenone treatment in patients with Duchenne Muscular Dystrophy (DMD) who are currently receiving Glucocorticoid steroids	A Phase III Double-blind, Randomized, Placebo-Controlled Study assessing the Efficacy, Safety and Tolerability of Idebenone in Patients with Duchenne Muscular Dystrophy Receiving Glucocorticoid Steroids - SIDEROS	Duchenne Muscular Dystrophy (DMD) MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]	Trade Name: Raxone Product Name: Idebenone INN or Proposed INN: IDEBENONE Other descriptive name: IDEBENONE	Santhera Pharmaceuticals (Switzerland) Limited	NULL	Not Recruiting			Female: no Male: yes	266	Phase 3	United States;Spain;Ireland;Austria;Israel;Italy;Switzerland;United Kingdom;France;Hungary;Belgium;Bulgaria;Germany;Netherlands;Sweden
35	EUCTR2017-004279-30-DE (EUCTR)		11/06/2018	A clinical study to assess the long-term safety and efficacy of idebenone treatment in patients with Duchenne Muscular Dystrophy (DMD) who completed the SIDEROS study.	A Phase III Open-Label Extension Study to Assess the Long-Term Safety and Efficacy of Idebenone in Patients with Duchenne Muscular Dystrophy (DMD) who completed the SIDEROS study - SIDEROS-E	Duchenne Muscular Dystrophy (DMD) MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]	Trade Name: Raxone Product Name: Idebenone INN or Proposed INN: IDEBENONE Other descriptive name: IDEBENONE	Santhera Pharmaceuticals (Switzerland) Limited	NULL	Not Recruiting			Female: no Male: yes	266	Phase 3	United States;France;Spain;Belgium;Austria;Israel;Netherlands;Germany;United Kingdom;Switzerland;Italy;Sweden
No.	TrialID	Date_ enrollment	Date_ registration	Public_title	Scientific_title	Condition	Intervention	Primary_ sponsor	Secondary_ sponsor	Recruitment_ Status	Inclusion_ agemin	Inclusion_ agemax	Inclusion_ gender	Target_ size	Phase	Countries
36	EUCTR2017-004279-30-FR (EUCTR)		14/05/2018	A clinical study to assess the long-term safety and efficacy of idebenone treatment in patients with Duchenne Muscular Dystrophy (DMD) who completed the SIDEROS study.	A Phase III Open-Label Extension Study to Assess the Long-Term Safety and Efficacy of Idebenone in Patients with Duchenne Muscular Dystrophy (DMD) who completed the SIDEROS study - SIDEROS-E	Duchenne Muscular Dystrophy (DMD) MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]	Trade Name: Raxone Product Name: Idebenone INN or Proposed INN: IDEBENONE Other descriptive name: IDEBENONE	Santhera Pharmaceuticals (Switzerland) Limited	NULL	NA			Female: no Male: yes	266	Phase 3	United States;France;Spain;Belgium;Austria;Israel;Netherlands;Germany;United Kingdom;Switzerland;Italy;Sweden

No.

TrialID

Date_
enrollment

Date_
registration

Public_title

Scientific_title

Condition

Intervention

Primary_
sponsor

Secondary_
sponsor

Recruitment_
Status

Inclusion_
agemin

Inclusion_
agemax

Inclusion_
gender

Target_
size

Phase

Countries

EUCTR2016-000602-10-BG
(EUCTR)

10/03/2020

07/01/2020

A clinical study to assess how effective and safe is idebenone treatment in patients with Duchenne Muscular Dystrophy (DMD) who are currently receiving Glucocorticoid steroids

A Phase III Double-blind, Randomized, Placebo-Controlled Study assessing the Efficacy, Safety and Tolerability of Idebenone in Patients with Duchenne Muscular Dystrophy Receiving Glucocorticoid Steroids - SIDEROS

Duchenne Muscular Dystrophy (DMD)
MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]

Trade Name: Raxone
Product Name: Idebenone
INN or Proposed INN: IDEBENONE
Other descriptive name: IDEBENONE

Santhera Pharmaceuticals (Switzerland) Limited

NULL

Not Recruiting

Female: no
Male: yes

266

Phase 3

United States;Spain;Ireland;Austria;Israel;United Kingdom;Italy;Switzerland;France;Hungary;Belgium;Bulgaria;Germany;Netherlands;Sweden

EUCTR2017-004279-30-NL
(EUCTR)

23/01/2020

16/05/2018

A clinical study to assess the long-term safety and efficacy of idebenone treatment in patients with Duchenne Muscular Dystrophy (DMD) who completed the SIDEROS study.

A Phase III Open-Label Extension Study to Assess the Long-Term Safety and Efficacy of Idebenone in Patients with Duchenne Muscular Dystrophy (DMD) who completed the SIDEROS study - SIDEROS-E

Trade Name: Raxone
Product Name: Idebenone
INN or Proposed INN: IDEBENONE
Other descriptive name: IDEBENONE

Santhera Pharmaceuticals (Switzerland) Limited

NULL

Not Recruiting

Female: no
Male: yes

266

Phase 3

United States;France;Spain;Belgium;Austria;Israel;Germany;Netherlands;United Kingdom;Switzerland;Italy;Sweden

EUCTR2017-004279-30-AT
(EUCTR)

15/02/2019

04/01/2019

A clinical study to assess the long-term safety and efficacy of idebenone treatment in patients with Duchenne Muscular Dystrophy (DMD) who completed the SIDEROS study.

A Phase III Open-Label Extension Study to Assess the Long-Term Safety and Efficacy of Idebenone in Patients with Duchenne Muscular Dystrophy (DMD) who completed the SIDEROS study - SIDEROS-E

Duchenne Muscular Dystrophy (DMD)
MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]

Santhera Pharmaceuticals (Switzerland) Limited

NULL

Authorised-recruitment may be ongoing or finished

Female: no
Male: yes

266

Phase 3

France;United States;Belgium;Spain;Austria;Israel;Netherlands;Germany;Italy;United Kingdom;Switzerland;Sweden

EUCTR2017-004279-30-ES
(EUCTR)

21/12/2018

16/11/2018

A clinical study to assess the long-term safety and efficacy of idebenone treatment in patients with Duchenne Muscular Dystrophy (DMD) who completed the SIDEROS study.

A Phase III Open-Label Extension Study to Assess the Long-Term Safety and Efficacy of Idebenone in Patients with Duchenne Muscular Dystrophy (DMD) who completed the SIDEROS study - SIDEROS-E

Duchenne Muscular Dystrophy (DMD)
MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]

Santhera Pharmaceuticals (Switzerland) Limited

NULL

Authorised-recruitment may be ongoing or finished

Female: no
Male: yes

266

Phase 3

France;United States;Belgium;Spain;Austria;Israel;Netherlands;Germany;Italy;United Kingdom;Switzerland;Sweden

EUCTR2016-000602-10-HU
(EUCTR)

18/12/2018

05/11/2018

A clinical study to assess how effective and safe is idebenone treatment in patients with Duchenne Muscular Dystrophy (DMD) who are currently receiving Glucocorticoid steroids

Trade Name: Raxone
Product Name: Idebenone
INN or Proposed INN: IDEBENONE
Other descriptive name: IDEBENONE

Santhera Pharmaceuticals (Switzerland) Limited

NULL

Not Recruiting

Female: no
Male: yes

266

Phase 3

United States;Spain;Ireland;Austria;Israel;United Kingdom;Italy;Switzerland;France;Hungary;Belgium;Bulgaria;Germany;Netherlands;Sweden

No.

TrialID

Date_
enrollment

Date_
registration

Public_title

Scientific_title

Condition

Intervention

Primary_
sponsor

Secondary_
sponsor

Recruitment_
Status

Inclusion_
agemin

Inclusion_
agemax

Inclusion_
gender

Target_
size

Phase

Countries

EUCTR2017-004279-30-GB
(EUCTR)

26/10/2018

09/05/2018

A clinical study to assess the long-term safety and efficacy of idebenone treatment in patients with Duchenne Muscular Dystrophy (DMD) who completed the SIDEROS study.

A Phase III Open-Label Extension Study to Assess the Long-Term Safety and Efficacy of Idebenone in Patients with Duchenne Muscular Dystrophy (DMD) who completed the SIDEROS study - SIDEROS-E

Trade Name: Raxone
Product Name: Idebenone
INN or Proposed INN: IDEBENONE
Other descriptive name: IDEBENONE

Santhera Pharmaceuticals (Switzerland) Limited

NULL

Not Recruiting

Female: no
Male: yes

266

Phase 3

United States;France;Spain;Belgium;Austria;Israel;Netherlands;Germany;Switzerland;Italy;United Kingdom;Sweden

NCT03603288
(ClinicalTrials.gov)

July 4, 2018

31/5/2018

Phase III Study With Idebenone in Patients With Duchenne Muscular Dystrophy (SIDEROS-E)

A Phase III Open-Label Extension Study to Assess the Long-Term Safety and Efficacy of Idebenone in Patients With Duchenne Muscular Dystrophy (DMD) Who Completed the SIDEROS Study

Duchenne Muscular Dystrophy

Drug: idebenone 150 mg film-coated tablets

Santhera Pharmaceuticals

NULL

Recruiting

11 Years

N/A

Male

266

Phase 3

United States;Austria;Belgium;France;Germany;Italy;Spain;Switzerland;United Kingdom

EUCTR2017-004279-30-BE
(EUCTR)

14/06/2018

04/05/2018

A clinical study to assess the long-term safety and efficacy of idebenone treatment in patients with Duchenne Muscular Dystrophy (DMD) who completed the SIDEROS study.

A Phase III Open-Label Extension Study to Assess the Long-Term Safety and Efficacy of Idebenone in Patients with Duchenne Muscular Dystrophy (DMD) who completed the SIDEROS study - SIDEROS-E

Trade Name: Raxone
Product Name: Idebenone
INN or Proposed INN: IDEBENONE
Other descriptive name: IDEBENONE

Santhera Pharmaceuticals (Switzerland) Limited

NULL

Not Recruiting

Female: no
Male: yes

266

Phase 3

United States;France;Spain;Belgium;Austria;Israel;Netherlands;Germany;United Kingdom;Switzerland;Italy;Sweden

EUCTR2016-000602-10-NL
(EUCTR)

26/04/2017

20/09/2016

A clinical study to assess how effective and safe is idebenone treatment in patients with Duchenne Muscular Dystrophy (DMD) who are currently receiving Glucocorticoid steroids

Trade Name: Raxone
Product Name: Idebenone
INN or Proposed INN: IDEBENONE
Other descriptive name: IDEBENONE

Santhera Pharmaceuticals (Switzerland) Limited

NULL

Not Recruiting

Female: no
Male: yes

266

Phase 3

United States;Spain;Ireland;Austria;Israel;Italy;Switzerland;United Kingdom;France;Hungary;Belgium;Bulgaria;Netherlands;Germany;Sweden

EUCTR2016-000602-10-GB
(EUCTR)

28/12/2016

23/09/2016

A clinical study to assess how effective and safe is idebenone treatment in patients with Duchenne Muscular Dystrophy (DMD) who are currently receiving Glucocorticoid steroids

Trade Name: Raxone
Product Name: Idebenone
INN or Proposed INN: IDEBENONE
Other descriptive name: IDEBENONE

Santhera Pharmaceuticals (Switzerland) Limited

NULL

Not Recruiting

Female: no
Male: yes

266

Phase 3

United States;Spain;Ireland;Austria;Israel;United Kingdom;Italy;Switzerland;France;Hungary;Belgium;Bulgaria;Netherlands;Germany;Sweden

No.

TrialID

Date_
enrollment

Date_
registration

Public_title

Scientific_title

Condition

Intervention

Primary_
sponsor

Secondary_
sponsor

Recruitment_
Status

Inclusion_
agemin

Inclusion_
agemax

Inclusion_
gender

Target_
size

Phase

Countries

EUCTR2016-000602-10-AT
(EUCTR)

21/12/2016

01/12/2016

A clinical study to assess how effective and safe is idebenone treatment in patients with Duchenne Muscular Dystrophy (DMD) who are currently receiving Glucocorticoid steroids

Trade Name: Raxone
Product Name: Idebenone
INN or Proposed INN: IDEBENONE
Other descriptive name: IDEBENONE

Santhera Pharmaceuticals (Switzerland) Limited

NULL

Authorised-recruitment may be ongoing or finished

Female: no
Male: yes

266

Phase 3

United States;Spain;Ireland;Austria;Israel;Italy;Switzerland;United Kingdom;France;Hungary;Belgium;Bulgaria;Germany;Netherlands;Sweden

EUCTR2016-000602-10-ES
(EUCTR)

20/12/2016

18/11/2016

A clinical study to assess how effective and safe is idebenone treatment in patients with Duchenne Muscular Dystrophy (DMD) who are currently receiving Glucocorticoid steroids

Duchenne Muscular Dystrophy (DMD)
MedDRA version: 19.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]

Trade Name: Raxone
Product Name: Idebenone
INN or Proposed INN: IDEBENONE
Other descriptive name: IDEBENONE

Santhera Pharmaceuticals (Switzerland) Limited

NULL

Authorised-recruitment may be ongoing or finished

Female: no
Male: yes

266

Phase 3

France;United States;Belgium;Spain;Austria;Netherlands;Germany;Italy;United Kingdom;Switzerland;Sweden

EUCTR2016-000602-10-FR
(EUCTR)

25/11/2016

12/01/2017

A clinical study to assess how effective and safe is idebenone treatment in patients with Duchenne Muscular Dystrophy (DMD) who are currently receiving Glucocorticoid steroids

Duchenne Muscular Dystrophy (DMD)
MedDRA version: 19.1;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]

Trade Name: Raxone
Product Name: Idebenone
INN or Proposed INN: IDEBENONE
Other descriptive name: IDEBENONE

Santhera Pharmaceuticals (Switzerland) Limited

NULL

Not Recruiting

Female: no
Male: yes

266

Phase 3

United States;Spain;Ireland;Austria;Switzerland;United Kingdom;Italy;France;Hungary;Belgium;Bulgaria;Germany;Netherlands;Sweden

EUCTR2016-000602-10-DE
(EUCTR)

17/11/2016

30/05/2016

A clinical study to assess how effective and safe is idebenone treatment in patients with Duchenne Muscular Dystrophy (DMD) who are currently receiving Glucocorticoid steroids

Trade Name: Raxone
Product Name: Idebenone
INN or Proposed INN: IDEBENONE
Other descriptive name: IDEBENONE

Santhera Pharmaceuticals (Switzerland) Limited

NULL

Not Recruiting

Female: no
Male: yes

266

Phase 3

United States;Spain;Ireland;Austria;Israel;Italy;Switzerland;United Kingdom;France;Hungary;Belgium;Bulgaria;Germany;Netherlands;Sweden

EUCTR2016-000602-10-IT
(EUCTR)

20/10/2016

06/02/2018

A clinical study to assess how effective and safe is idebenone treatment inpatients with Duchenne Muscular Dystrophy (DMD) who are currentlyreceiving Glucocorticoid steroids

A Phase III Double-blind, Randomized, Placebo-Controlled Studyassessing the Efficacy, Safety and Tolerability of Idebenone inPatients with Duchenne Muscular Dystrophy Receiving GlucocorticoidSteroids - SIDEROS

Trade Name: RAXONE - 150 MG- COMPRESSE RIVESTITE CON FILM- USO ORALE- FLACONE (HDPE)- 180 COMPRESSE
INN or Proposed INN: IDEBENONE
Other descriptive name: IDEBENONE

SANTHERA PHARMACEUTICALS

NULL

Authorised-recruitment may be ongoing or finished

Female: no
Male: yes

266

Phase 3

France;United States;Belgium;Spain;Austria;Netherlands;Germany;United Kingdom;Switzerland;Italy;Sweden

No.

TrialID

Date_
enrollment

Date_
registration

Public_title

Scientific_title

Condition

Intervention

Primary_
sponsor

Secondary_
sponsor

Recruitment_
Status

Inclusion_
agemin

Inclusion_
agemax

Inclusion_
gender

Target_
size

Phase

Countries

EUCTR2016-000602-10-BE
(EUCTR)

02/09/2016

19/07/2016

A clinical study to assess how effective and safe is idebenone treatment in patients with Duchenne Muscular Dystrophy (DMD) who are currently receiving Glucocorticoid steroids

Trade Name: Raxone
Product Name: Idebenone
INN or Proposed INN: IDEBENONE
Other descriptive name: IDEBENONE

Santhera Pharmaceuticals (Switzerland) Limited

NULL

Not Recruiting

Female: no
Male: yes

266

Phase 3

United States;Spain;Ireland;Austria;Israel;Italy;Switzerland;United Kingdom;France;Hungary;Belgium;Bulgaria;Germany;Netherlands;Sweden

NCT02814019
(ClinicalTrials.gov)

September 2016

17/6/2016

A Phase III Double-blind Study With Idebenone in Patients With Duchenne Muscular Dystrophy (DMD) Taking Glucocorticoid Steroids

A Phase III Double-blind, Randomized, Placebo-Controlled Study Assessing the Efficacy, Safety and Tolerability of Idebenone in Patients With Duchenne Muscular Dystrophy Receiving Glucocorticoid Steroids

Duchenne Muscular Dystrophy (DMD)

Drug: Idebenone 150 mg film-coated tablets;Drug: placebo

Santhera Pharmaceuticals

NULL

Active, not recruiting

10 Years

N/A

Male

255

Phase 3

United States;Austria;Belgium;Bulgaria;France;Germany;Hungary;Ireland;Israel;Italy;Netherlands;Spain;Sweden;Switzerland;United Kingdom

NCT03433807
(ClinicalTrials.gov)

January 29, 2013

8/2/2018

Expanded Access Program for Idebenone in Participants With Duchenne Muscular Dystrophy (DMD)

Expanded Access Protocol (EAP) of Idebenone in Patients With Duchenne Muscular Dystrophy

Duchenne Muscular Dystrophy

Drug: Idebenone

Santhera Pharmaceuticals

NULL

Temporarily not available

8 Years

N/A

All

United States

EUCTR2009-012037-30-ES
(EUCTR)

27/06/2012

29/03/2012

Hereditary neuromuscular disease

A Phase III Double-Blind, Randomised, Placebo-Controlled Study of the Efficacy, Safety and Tolerability of Idebenone in 10 ? 18 Year Old Patients with Duchenne Muscular Dystrophy - DELOS

Duchenne Muscular Dystrophy
MedDRA version: 14.1;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]

Product Name: Idebenone
Product Code: SNT-MC17
INN or Proposed INN: IDEBENONE

Santhera Pharmaceuticals (Switzerland) Limited

NULL

Not Recruiting

Female: no
Male: yes

280

Phase 3

France;United States;Belgium;Spain;Austria;Netherlands;Germany;Italy;Switzerland;Sweden

EUCTR2009-012037-30-IT
(EUCTR)

27/03/2012

26/04/2012

(DELOS) DuchEnne MD Long-term IdebenOne Study

A Phase III Double-Blind, Randomised, Placebo-Controlled Study of the Efficacy, Safety and Tolerability of Idebenone in 10 – 18 Year Old Patients with Duchenne Muscular Dystrophy - DELOS

Product Name: Idebenone
Product Code: NA
INN or Proposed INN: IDEBENONE

SANTHERA PHARMACEUTICALS (SWITZERLAND) LTD

NULL

Not Recruiting

Female: no
Male: yes

280

Phase 3

France;United States;Canada;Spain;Belgium;Austria;Netherlands;Germany;Italy;Sweden

No.

TrialID

Date_
enrollment

Date_
registration

Public_title

Scientific_title

Condition

Intervention

Primary_
sponsor

Secondary_
sponsor

Recruitment_
Status

Inclusion_
agemin

Inclusion_
agemax

Inclusion_
gender

Target_
size

Phase

Countries

EUCTR2009-012037-30-NL
(EUCTR)

03/06/2010

17/12/2009

Hereditary neuromuscular disease

A Phase III Double-Blind, Randomised, Placebo-Controlled Study of the Efficacy, Safety and Tolerability of Idebenone in 10 – 18 Year Old Patients with Duchenne Muscular Dystrophy - DELOS

Duchenne Muscular Dystrophy
MedDRA version: 17.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]

Product Name: Idebenone
Product Code: SNT-MC17
INN or Proposed INN: IDEBENONE

Santhera Pharmaceuticals (Switzerland) Limited

NULL

Authorised-recruitment may be ongoing or finished

Female: no
Male: yes

266

Phase 3

France;United States;Belgium;Spain;Austria;Germany;Netherlands;Italy;Switzerland;Sweden

EUCTR2009-012037-30-AT
(EUCTR)

10/03/2010

10/02/2010

Hereditary neuromuscular disease

A Phase III Double-Blind, Randomised, Placebo-Controlled Study of the Efficacy, Safety and Tolerability of Idebenone in 10 – 18 Year Old Patients with Duchenne Muscular Dystrophy - DELOS

Product Name: Idebenone
Product Code: SNT-MC17
INN or Proposed INN: IDEBENONE

Santhera Pharmaceuticals (Switzerland) Limited

NULL

Not Recruiting

Female: no
Male: yes

266

Phase 3

France;United States;Belgium;Spain;Austria;Netherlands;Germany;Italy;Switzerland;Sweden

EUCTR2009-012037-30-SE
(EUCTR)

23/02/2010

28/12/2009

Hereditary neuromuscular disease

A Phase III Double-Blind, Randomised, Placebo-Controlled Study of the Efficacy, Safety and Tolerability of Idebenone in 10 – 18 Year Old Patients with Duchenne Muscular Dystrophy - DELOS

Product Name: Idebenone
Product Code: SNT-MC17
INN or Proposed INN: IDEBENONE

Santhera Pharmaceuticals (Switzerland) Limited

NULL

Not Recruiting

Female: no
Male: yes

266

Phase 3

France;United States;Belgium;Spain;Austria;Netherlands;Germany;Italy;Switzerland;Sweden

EUCTR2009-012037-30-FR
(EUCTR)

19/02/2010

17/12/2009

A Phase III Double-Blind, Randomised, Placebo-Controlled Study of the Efficacy, Safety and Tolerability of Idebenone in 10 – 18 Year Old Patients with Duchenne Muscular Dystrophy - DELOS

Duchenne Muscular Dystrophy
MedDRA version: 12.0;Level: LLT;Classification code 10013801;Term: Duchenne muscular dystrophy

Product Name: Idebenone
Product Code: SNT-MC17
INN or Proposed INN: IDEBENONE

Santhera Pharmaceuticals (Switzerland) Limited

NULL

Authorised-recruitment may be ongoing or finished

Female: no
Male: yes

240

Phase 3

Germany;Netherlands;Belgium;France;Spain;Italy;Austria;Sweden

EUCTR2009-012037-30-DE
(EUCTR)

08/10/2009

07/08/2009

Hereditary neuromuscular disease

A Phase III Double-Blind, Randomised, Placebo-Controlled Study of the Efficacy, Safety and Tolerability of Idebenone in 10 – 18 Year Old Patients with Duchenne Muscular Dystrophy - DELOS

Product Name: Idebenone
Product Code: SNT-MC17
INN or Proposed INN: IDEBENONE

Santhera Pharmaceuticals (Switzerland) Limited

NULL

Not Recruiting

Female: no
Male: yes

266

Phase 3

United States;France;Spain;Belgium;Austria;Netherlands;Germany;Switzerland;Italy;Sweden

No.

TrialID

Date_
enrollment

Date_
registration

Public_title

Scientific_title

Condition

Intervention

Primary_
sponsor

Secondary_
sponsor

Recruitment_
Status

Inclusion_
agemin

Inclusion_
agemax

Inclusion_
gender

Target_
size

Phase

Countries

EUCTR2009-012037-30-BE
(EUCTR)

14/07/2009

27/05/2009

Hereditary neuromuscular disease

A Phase III Double-Blind, Randomised, Placebo-Controlled Study of the Efficacy, Safety and Tolerability of Idebenone in 10 – 18 Year Old Patients with Duchenne Muscular Dystrophy - DELOS

Product Name: Idebenone
Product Code: SNT-MC17
INN or Proposed INN: IDEBENONE

Santhera Pharmaceuticals (Switzerland) Limited

NULL

Authorised-recruitment may be ongoing or finished

Female: no
Male: yes

266

Phase 3

France;United States;Spain;Belgium;Austria;Netherlands;Germany;Italy;Switzerland;Sweden

NCT01027884
(ClinicalTrials.gov)

July 2009

8/12/2009

Phase III Study of Idebenone in Duchenne Muscular Dystrophy (DMD)

A Phase III Double-Blind, Randomised, Placebo-Controlled Study of the Efficacy, Safety and Tolerability of Idebenone in 10-18 Year Old Patients With Duchenne Muscular Dystrophy

Muscular Dystrophy, Duchenne;Ambulatory Care

Drug: Placebo;Drug: Idebenone

Santhera Pharmaceuticals

NULL

Completed

10 Years

18 Years

Male

Phase 3

United States;Austria;Belgium;France;Germany;Italy;Netherlands;Spain;Sweden;Switzerland

NCT00758225
(ClinicalTrials.gov)

September 2008

23/9/2008

Long-term Safety, Tolerability and Efficacy of Idebenone in Duchenne Muscular Dystrophy (DELPHI Extension)

A Phase II Open-label Extension Study to Obtain Long-term Safety, Tolerability and Efficacy Data of Idebenone in the Treatment of Duchenne Muscular Dystrophy - Extension to Study SNT-II-001

Duchenne Muscular Dystrophy

Drug: Idebenone

Santhera Pharmaceuticals

NULL

Completed

N/A

Male

Phase 2

Belgium

EUCTR2007-007752-34-BE
(EUCTR)

14/08/2008

26/06/2008

A Phase II open-label extension study to obtain long-term safety, tolerability and efficacy data of Idebenone in the treatment of Duchenne Muscular Dystrophy

Duchennes Muscular Dystrophy
MedDRA version: 12.0;Level: LLT;Classification code 10013801;Term: Duchenne muscular dystrophy

Product Name: Idebenone
Product Code: SNT-MC17
INN or Proposed INN: Idebenone

Santhera Pharmaceuticals (Switzerland) Ltd

NULL

Not Recruiting

Female: no
Male: yes

Phase 2

Belgium

NCT00654784
(ClinicalTrials.gov)

October 2005

3/4/2008

Efficacy and Tolerability of Idebenone in Boys With Cardiac Dysfunction Associated With Duchenne Muscular Dystrophy

A Phase IIa Double Blind, Randomised, Placebo Controlled, Single Centre Study at the University of Leuven to Assess the Efficacy and Tolerability of Idebenone in 8 - 16 Year Old Males With Cardiac Dysfunction Associated With Duchenne Muscular Dystrophy

Duchenne Muscular Dystrophy (DMD)

Drug: idebenone;Drug: placebo

Santhera Pharmaceuticals

NULL

Completed

8 Years

16 Years

Male

Phase 2

Belgium

No.

TrialID

Date_
enrollment

Date_
registration

Public_title

Scientific_title

Condition

Intervention

Primary_
sponsor

Secondary_
sponsor

Recruitment_
Status

Inclusion_
agemin

Inclusion_
agemax

Inclusion_
gender

Target_
size

Phase

Countries

EUCTR2005-002520-33-BE
(EUCTR)

06/09/2005

04/10/2005

A Phase IIa double blind, randomised, placebo controlled, single centre study at the university of Leuven to assess the efficacy and tolerability of idebenone in 10 - 16 year old males with cardiac dysfunction associated with Duchenne Muscular Dystrophy

Duchenne Muscular Dystrophy

Product Name: idebenone

Santhera Pharmaceuticals LLC

NULL

Not Recruiting

Female: no
Male: yes

Phase 2a

Belgium

EUCTR2016-000602-10-IE
(EUCTR)

06/02/2018

A clinical study to assess how effective and safe is idebenone treatment in patients with Duchenne Muscular Dystrophy (DMD) who are currently receiving Glucocorticoid steroids

Trade Name: Raxone
Product Name: Idebenone
INN or Proposed INN: IDEBENONE
Other descriptive name: IDEBENONE

Santhera Pharmaceuticals (Switzerland) Limited

NULL

Not Recruiting

Female: no
Male: yes

266

Phase 3

United States;Spain;Ireland;Austria;Israel;Switzerland;United Kingdom;Italy;France;Hungary;Belgium;Bulgaria;Germany;Netherlands;Sweden

EUCTR2017-004279-30-SE
(EUCTR)

15/05/2018

A clinical study to assess the long-term safety and efficacy of idebenone treatment in patients with Duchenne Muscular Dystrophy (DMD) who completed the SIDEROS study.

A Phase III Open-Label Extension Study to Assess the Long-Term Safety and Efficacy of Idebenone in Patients with Duchenne Muscular Dystrophy (DMD) who completed the SIDEROS study - SIDEROS-E

Trade Name: Raxone
Product Name: Idebenone
INN or Proposed INN: IDEBENONE
Other descriptive name: IDEBENONE

Santhera Pharmaceuticals (Switzerland) Limited

NULL

Not Recruiting

Female: no
Male: yes

266

Phase 3

United States;France;Spain;Belgium;Austria;Israel;Netherlands;Germany;United Kingdom;Switzerland;Italy;Sweden

EUCTR2016-000602-10-SE
(EUCTR)

22/09/2016

A clinical study to assess how effective and safe is idebenone treatment in patients with Duchenne Muscular Dystrophy (DMD) who are currently receiving Glucocorticoid steroids

Trade Name: Raxone
Product Name: Idebenone
INN or Proposed INN: IDEBENONE
Other descriptive name: IDEBENONE

Santhera Pharmaceuticals (Switzerland) Limited

NULL

Not Recruiting

Female: no
Male: yes

266

Phase 3

United States;Spain;Ireland;Austria;Israel;Italy;Switzerland;United Kingdom;France;Hungary;Belgium;Bulgaria;Germany;Netherlands;Sweden

EUCTR2017-004279-30-DE
(EUCTR)

11/06/2018

A clinical study to assess the long-term safety and efficacy of idebenone treatment in patients with Duchenne Muscular Dystrophy (DMD) who completed the SIDEROS study.

A Phase III Open-Label Extension Study to Assess the Long-Term Safety and Efficacy of Idebenone in Patients with Duchenne Muscular Dystrophy (DMD) who completed the SIDEROS study - SIDEROS-E

Trade Name: Raxone
Product Name: Idebenone
INN or Proposed INN: IDEBENONE
Other descriptive name: IDEBENONE

Santhera Pharmaceuticals (Switzerland) Limited

NULL

Not Recruiting

Female: no
Male: yes

266

Phase 3

United States;France;Spain;Belgium;Austria;Israel;Netherlands;Germany;United Kingdom;Switzerland;Italy;Sweden

No.

TrialID

Date_
enrollment

Date_
registration

Public_title

Scientific_title

Condition

Intervention

Primary_
sponsor

Secondary_
sponsor

Recruitment_
Status

Inclusion_
agemin

Inclusion_
agemax

Inclusion_
gender

Target_
size

Phase

Countries

EUCTR2017-004279-30-FR
(EUCTR)

14/05/2018

A clinical study to assess the long-term safety and efficacy of idebenone treatment in patients with Duchenne Muscular Dystrophy (DMD) who completed the SIDEROS study.

A Phase III Open-Label Extension Study to Assess the Long-Term Safety and Efficacy of Idebenone in Patients with Duchenne Muscular Dystrophy (DMD) who completed the SIDEROS study - SIDEROS-E

Trade Name: Raxone
Product Name: Idebenone
INN or Proposed INN: IDEBENONE
Other descriptive name: IDEBENONE

Santhera Pharmaceuticals (Switzerland) Limited

NULL

Female: no
Male: yes

266

Phase 3

United States;France;Spain;Belgium;Austria;Israel;Netherlands;Germany;United Kingdom;Switzerland;Italy;Sweden