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_ gender	Target_ size	Phase	Countries
1	EUCTR2017-004554-42-FR (EUCTR)	26/03/2020	17/08/2018	The study examines boys suffering from Duchenne muscular dystrophy. We are carrying out this study to examine the effect and tolerance of Tamoxifen in this disease.	Tamoxifen in Duchenne muscular dystrophy - TAMDMDA multicentre, randomised, double-blind, placebo-controlled, phase 3 safety and efficacy 48-week trial	Duchenne muscular dystrophy 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] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]	Trade Name: Tamoxifen 20mg Hexal® Filmtabletten INN or Proposed INN: Tamoxifen Other descriptive name: TAMOXIFEN CITRATE	University of Basel Children's Hospital, Division of Neuropediatrics	NULL	Authorised-recruitment may be ongoing or finished	Female: no Male: yes	100	Phase 3	France;Belgium;Spain;Turkey;Netherlands;Germany;United Kingdom;Switzerland
2	EUCTR2017-004554-42-GB (EUCTR)	29/01/2019	03/08/2018	The study examines boys suffering from Duchenne muscular dystrophy. We are carrying out this study to examine the effect and tolerance of Tamoxifen in this disease.	Tamoxifen in Duchenne muscular dystrophy - TAMDMDA multicentre, randomised, double-blind, placebo-controlled, phase 3 safety and efficacy 48-week trialTamoxifen in Duchenne muscular dystrophy: A 48-week open labelextension of a multicentre, randomised, double-blind, placebo-controlled,phase 3 safety and efficacy trial	Duchenne muscular dystrophy 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] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]	Trade Name: Tamoxifen 20mg Hexal® Filmtabletten INN or Proposed INN: Tamoxifen Other descriptive name: TAMOXIFEN CITRATE	University of Basel Children's Hospital, Division of Neuropediatrics	NULL	Authorised-recruitment may be ongoing or finished	Female: no Male: yes	100	Phase 3	France;Belgium;Spain;Netherlands;Germany;Switzerland;United Kingdom
3	EUCTR2017-004554-42-BE (EUCTR)		30/04/2019	The study examines boys suffering from Duchenne muscular dystrophy. We are carrying out this study to examine the effect and tolerance of Tamoxifen in this disease.	Tamoxifen in Duchenne muscular dystrophy:A multicenter, randomised, double-blind, placebo-controlled, phase 3 safety and efficacy 48-week trialTamoxifen in Duchenne muscular dystrophy: A 48-week open label extension of a multicentre, randomised, double-blind, placebo-controlled, phase 3 safety and efficacy trial	Duchenne muscular dystrophy 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] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]	Trade Name: Tamoxifen 20mg Hexal® Filmtabletten INN or Proposed INN: Tamoxifen Other descriptive name: TAMOXIFEN CITRATE	University of Basel Children's Hospital, Division of Neuropediatrics	NULL	NA	Female: no Male: yes	100	Phase 3	France;Spain;Belgium;Turkey;Netherlands;Germany;United Kingdom;Switzerland

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

EUCTR2017-004554-42-FR
(EUCTR)

26/03/2020

17/08/2018

The study examines boys suffering from Duchenne muscular dystrophy. We are carrying out this study to examine the effect and tolerance of Tamoxifen in this disease.

Tamoxifen in Duchenne muscular dystrophy - TAMDMDA multicentre, randomised, double-blind, placebo-controlled, phase 3 safety and efficacy 48-week trial

Duchenne muscular dystrophy
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] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]

Trade Name: Tamoxifen 20mg Hexal® Filmtabletten
INN or Proposed INN: Tamoxifen
Other descriptive name: TAMOXIFEN CITRATE

University of Basel Children's Hospital, Division of Neuropediatrics

NULL

Authorised-recruitment may be ongoing or finished

Female: no
Male: yes

100

Phase 3

France;Belgium;Spain;Turkey;Netherlands;Germany;United Kingdom;Switzerland

EUCTR2017-004554-42-GB
(EUCTR)

29/01/2019

03/08/2018

The study examines boys suffering from Duchenne muscular dystrophy. We are carrying out this study to examine the effect and tolerance of Tamoxifen in this disease.

Tamoxifen in Duchenne muscular dystrophy - TAMDMDA multicentre, randomised, double-blind, placebo-controlled, phase 3 safety and efficacy 48-week trialTamoxifen in Duchenne muscular dystrophy: A 48-week open labelextension of a multicentre, randomised, double-blind, placebo-controlled,phase 3 safety and efficacy trial

Trade Name: Tamoxifen 20mg Hexal® Filmtabletten
INN or Proposed INN: Tamoxifen
Other descriptive name: TAMOXIFEN CITRATE

University of Basel Children's Hospital, Division of Neuropediatrics

NULL

Authorised-recruitment may be ongoing or finished

Female: no
Male: yes

100

Phase 3

France;Belgium;Spain;Netherlands;Germany;Switzerland;United Kingdom

EUCTR2017-004554-42-BE
(EUCTR)

30/04/2019

The study examines boys suffering from Duchenne muscular dystrophy. We are carrying out this study to examine the effect and tolerance of Tamoxifen in this disease.

Tamoxifen in Duchenne muscular dystrophy:A multicenter, randomised, double-blind, placebo-controlled, phase 3 safety and efficacy 48-week trialTamoxifen in Duchenne muscular dystrophy: A 48-week open label extension of a multicentre, randomised, double-blind, placebo-controlled, phase 3 safety and efficacy trial

Trade Name: Tamoxifen 20mg Hexal® Filmtabletten
INN or Proposed INN: Tamoxifen
Other descriptive name: TAMOXIFEN CITRATE

University of Basel Children's Hospital, Division of Neuropediatrics

NULL

Female: no
Male: yes

100

Phase 3

France;Spain;Belgium;Turkey;Netherlands;Germany;United Kingdom;Switzerland