3. 脊髄性筋萎縮症 Spinal muscular atrophy Clinical trials / Disease details


臨床試験数 : 237 薬物数 : 123 - (DrugBank : 29) / 標的遺伝子数 : 51 - 標的パスウェイ数 : 75

  
48 trials found
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1NCT05337553
(ClinicalTrials.gov)
July 6, 202214/4/2022A Study to Evaluate the Efficacy and Safety of Taldefgrobep Alfa in Participants With Spinal Muscular AtrophyA Randomized, Double-Blind, Placebo-Controlled, Study to Evaluate the Efficacy and Safety of Taldefgrobep Alfa in Ambulatory and Non-Ambulatory Participants With Spinal Muscular Atrophy With Open-Label ExtensionSpinal Muscular Atrophy;Neuromuscular Diseases;SMADrug: taldefgrobep alfa;Drug: PlaceboBiohaven Pharmaceuticals, Inc.NULLRecruiting4 Years21 YearsAll225Phase 3United States;Belgium;France;Germany;Italy;Netherlands;Poland;Spain;United Kingdom
2NCT05115110
(ClinicalTrials.gov)
June 2, 20221/11/2021A Study to Investigate the Safety and Efficacy of RO7204239 in Combination With Risdiplam (RO7034067) in Participants With Spinal Muscular AtrophyA Two-Part, Seamless, Multi-Center, Randomized, Placebo-Controlled, Double-Blind Study to Investigate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Efficacy of RO7204239 in Combination With Risdiplam (RO7034067) in Patients With Spinal Muscular AtrophySpinal Muscular Atrophy (SMA)Drug: RO7204239;Drug: Placebo;Drug: RisdiplamHoffmann-La RocheNULLRecruiting2 Years25 YearsAll259Phase 2/Phase 3United States;Belgium;Italy;Netherlands;Poland;United Kingdom;Germany
3EUCTR2021-003417-19-NL
(EUCTR)
25/05/202211/01/2022A Study to Investigate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Efficacy of RO7204239 in Combination with Risdiplam (RO7034067) in Ambulant Patients with Spinal Muscular AtrophyA TWO-PART, SEAMLESS, MULTI-CENTER, RANDOMIZED, PLACEBO-CONTROLLED, DOUBLE-BLIND STUDY TO INVESTIGATE THE SAFETY, TOLERABILITY, PHARMACOKINETICS, PHARMACODYNAMICS AND EFFICACY OF RO7204239 IN COMBINATION WITH RISDIPLAM (RO7034067) IN AMBULANT PATIENTS WITH SPINAL MUSCULAR ATROPHY Spinal Muscular Atrophy (SMA);Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]Product Name: N/A
Product Code: RO7204239/F01-01
INN or Proposed INN: N/A
Other descriptive name: GYM329
Trade Name: Evrysdi
INN or Proposed INN: Risdiplam
F.Hoffmann-La Roche LtdNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
180Phase 2;Phase 3United States;Poland;Belgium;Germany;Netherlands;United Kingdom;Italy
4EUCTR2021-005314-34-ES
(EUCTR)
11/05/202209/03/2022Phase 3 Active Treatment Trial to Evaluate the Efficacy and Safety of Apitegromab in Patients with Later-Onset Spinal Muscular Atrophy Who Are Being Treated with Nusinersen or RisdiplamPhase 3, Double-Blind, Placebo-Controlled Trial to Evaluate the Efficacy and Safety of Apitegromab (SRK-015) in Patients with Later-Onset Spinal Muscular Atrophy Receiving Background Nusinersen or Risdiplam Therapy - SAPPHIRE Spinal Muscular Atrophy (SMA)
MedDRA version: 20.1;Level: LLT;Classification code 10041583;Term: Spinal muscular atrophy, unspecified;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
Product Name: Apitegromab
Product Code: SRK-015
INN or Proposed INN: Apitegromab
Scholar Rock, Inc.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
204Phase 3France;United States;Poland;Belgium;Spain;Netherlands;Germany;United Kingdom;Italy
5NCT05156320
(ClinicalTrials.gov)
February 24, 20221/12/2021Efficacy and Safety of Apitegromab in Patients With Later-Onset Spinal Muscular Atrophy Treated With Nusinersen or RisdiplamPhase 3, Double-Blind, Placebo-Controlled Trial to Evaluate the Efficacy and Safety of Apitegromab (SRK-015) in Patients With Later-Onset Spinal Muscular Atrophy Receiving Background Nusinersen or Risdiplam TherapySpinal Muscular Atrophy;Spinal Muscular Atrophy Type 3;Spinal Muscular Atrophy Type 2;SMA;Neuromuscular Diseases;Muscular Atrophy;Atrophy;Muscular Atrophy, Spinal;Neuromuscular Manifestations;Anti-myostatinDrug: Apitegromab;Drug: PlaceboScholar Rock, Inc.NULLRecruiting2 Years21 YearsAll204Phase 3Germany;Italy;Netherlands;Poland;Spain;United Kingdom;Belgium;France;United States
6EUCTR2021-003417-19-PL
(EUCTR)
18/01/202225/10/2021A Study to Investigate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Efficacy of RO7204239 in Combination with Risdiplam (RO7034067) in Ambulant Patients with Spinal Muscular AtrophyA TWO-PART, SEAMLESS, MULTI-CENTER, RANDOMIZED, PLACEBO-CONTROLLED, DOUBLE-BLIND STUDY TO INVESTIGATE THE SAFETY, TOLERABILITY, PHARMACOKINETICS, PHARMACODYNAMICS AND EFFICACY OF RO7204239 IN COMBINATION WITH RISDIPLAM (RO7034067) IN AMBULANT PATIENTS WITH SPINAL MUSCULAR ATROPHY Spinal Muscular Atrophy (SMA);Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]Product Name: N/A
Product Code: RO7204239/F01-01
INN or Proposed INN: N/A
Other descriptive name: GYM329
Trade Name: Evrysdi
INN or Proposed INN: Risdiplam
F.Hoffmann-La Roche LtdNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
180Phase 2;Phase 3United States;Belgium;Poland;Netherlands;United Kingdom;Italy
7EUCTR2021-003417-19-IT
(EUCTR)
03/01/202218/10/2021A Study to Investigate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Efficacy of RO7204239 in Combination with Risdiplam (RO7034067) in Ambulant Patients with Spinal Muscular AtrophyA TWO-PART, SEAMLESS, MULTI-CENTER, RANDOMIZED, PLACEBO-CONTROLLED, DOUBLE-BLIND STUDY TO INVESTIGATE THE SAFETY, TOLERABILITY, PHARMACOKINETICS, PHARMACODYNAMICS AND EFFICACY OF RO7204239 IN COMBINATION WITH RISDIPLAM (RO7034067) IN AMBULANT PATIENTS WITH SPINAL MUSCULAR ATROPHY - NA Spinal Muscular Atrophy (SMA)
MedDRA version: 20.0;Level: LLT;Classification code 10079415;Term: Spinal muscular atrophy type III;System Organ Class: 100000004850
MedDRA version: 20.1;Classification code 10051203;Term: Spinal muscular atrophy congenital;Classification code 10041583;Term: Spinal muscular atrophy, unspecified;System Organ Class: 100000004850
MedDRA version: 20.0;Classification code 10079413;Term: Spinal muscular atrophy type I;Classification code 10079416;Term: Spinal muscular atrophy type II;Classification code 10079417;Term: Spinal muscular atrophy infantile onset;Classification code 10079419;Term: Spinal muscular atrophy pre-symptomatic;Classification code 10079418;Term: Spinal muscular atrophy later onset;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: N/A
Product Code: [RO7204239/F01-01]
Trade Name: Evrysdi
Product Name: Risdiplam
Product Code: [RO7034067]
INN or Proposed INN: Risdiplam
F. HOFFMANN - LA ROCHE LTD.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
180Phase 2;Phase 3United States;Poland;Belgium;Netherlands;United Kingdom;Italy
8ChiCTR2000041208
2020-12-302020-12-22A randomized, double-blind, placebo parallel controlled clinical trial on the efficacy and safety of salbutamol sulfate tablets in the treatment of children with spinal muscular atrophyA randomized, double-blind, placebo parallel controlled clinical trial on the efficacy and safety of salbutamol sulfate tablets in the treatment of children with spinal muscular atrophy spinal muscular atrophyexperimental group: salbutamol sulfate tablets ;control group:placebo;The Children's Hospital, Zhejiang University School of MedicineNULLRecruiting217Bothexperimental group:60;control group:30;Phase 4China
9EUCTR2016-000750-35-GB
(EUCTR)
25/11/202023/06/2016A study to investigate the safety, tolerability, pharmacokinetics, pharmacodynamics and efficacy of Risdiplam (RO7034067) in type 2 and 3 spinal muscular atrophy patientsA TWO-PART SEAMLESS, MULTI-CENTER RANDOMIZED, PLACEBO-CONTROLLED, DOUBLE BLIND STUDY TO INVESTIGATE THE SAFETY, TOLERABILITY, PHARMACOKINETICS, PHARMACODYNAMICS AND EFFICACY OF RISDIPLAM (RO7034067) IN TYPE 2 AND 3 SPINAL MUSCULAR ATROPHY PATIENTS. Spinal Muscular Atrophy (SMA) Type 2 and 3
MedDRA version: 20.1;Level: PT;Classification code 10041582;Term: Spinal muscular atrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]
Product Name: RO7034067
Product Code: RO7034067/F12
INN or Proposed INN: risdiplam
Product Name: RO7034067
Product Code: RO7034067/F13
INN or Proposed INN: risdiplam
F. Hoffmann-La Roche LtdNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
231Phase 2United States;Serbia;Spain;Turkey;Russian Federation;United Kingdom;Italy;France;Canada;Belgium;Brazil;Poland;Croatia;Bulgaria;Germany;China;Japan
10NCT03781479
(ClinicalTrials.gov)
January 21, 201918/12/2018Controlled Trial to Evaluate Amifampridine Phosphate in Spinal Muscular Atrophy Type 3 PatientsA Randomized, Placebo-Controlled, Crossover Study to Evaluate the Safety and Efficacy of Amifampridine Phosphate in Ambulatory Patients With Spinal Muscular Atrophy (SMA) Type 3Muscular Atrophy, SpinalDrug: Amifampridine Phosphate;Drug: Placebo Oral TabletCatalyst Pharmaceuticals, Inc.NULLCompleted6 Years50 YearsAll13Phase 2Italy
11EUCTR2016-000750-35-BG
(EUCTR)
20/06/201804/06/2018A study to investigate the safety, tolerability, pharmacokinetics, pharmacodynamics and efficacy of RO7034067 in type 2 and 3 spinal muscular atrophy patientsA TWO-PART SEAMLESS, MULTI-CENTER RANDOMIZED, PLACEBO-CONTROLLED, DOUBLE BLIND STUDY TO INVESTIGATE THE SAFETY, TOLERABILITY, PHARMACOKINETICS, PHARMACODYNAMICS AND EFFICACY OF RO7034067 IN TYPE 2 AND 3 SPINAL MUSCULAR ATROPHY PATIENTS. Spinal Muscular Atrophy (SMA)
MedDRA version: 20.1;Level: PT;Classification code 10041582;Term: Spinal muscular atrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]
Product Name: RO7034067
Product Code: RO7034067/F12
INN or Proposed INN: n.a
Product Name: RO7034067
Product Code: RO7034067/F13
INN or Proposed INN: n.a
F. Hoffmann-La Roche LtdNULLNot RecruitingFemale: yes
Male: yes
219Phase 2Serbia;United States;Spain;Ukraine;Turkey;Russian Federation;Italy;Switzerland;United Kingdom;France;Hungary;Mexico;Canada;Argentina;Poland;Belgium;Brazil;Romania;Croatia;Bulgaria;Germany;Japan;China
12EUCTR2017-004600-22-IT
(EUCTR)
04/04/201807/04/2021Clinical study evaluating the effect of Amifampridine Phosphate in Ambulatory Patients with Spinal Muscular Atrophy (SMA) Type 3.A Randomized, Placebo-Controlled, Crossover Study to Evaluate the Safety and Efficacy of Amifampridine Phosphate in Ambulatory Patients with Spinal Muscular Atrophy (SMA) Type 3 - SMA-001 Spinal Muscular Atrophy (SMA) Type 3
MedDRA version: 20.1;Level: PT;Classification code 10041582;Term: Spinal muscular atrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]
Trade Name: FIRDAPSE - 10 MG - COMPRESSE - USO ORALE - BLISTER(ALU/PVC/PVDC) 100 X 1 COMPRESSE
Product Name: Amifampridina fosfato
Product Code: [Amifampridina fosfato]
INN or Proposed INN: AMIFAMPRIDINA
Other descriptive name: 3,4-Diaminopyridine Phosphate
CATALYST PHARMACEUTICALS INC.NULLNot RecruitingFemale: yes
Male: yes
12Phase 2Italy
13JPRN-JapicCTI-173722
03/4/201804/10/2017A Study to Investigate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Efficacy of RO7034067 in Type 2 and 3 Spinal Muscular Atrophy Participants (Sunfish)A Two-Part Seamless, Multi-Center Randomized, Placebo-Controlled, Double-blind Study to Investigate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Efficacy of RO7034067 in Type 2 and 3 Spinal Muscular Atrophy Patients Spinal Muscular AtrophyIntervention name : RO7034067
INN of the intervention : risdiplam
Dosage And administration of the intervention : po
Control intervention name : Placebo
INN of the control intervention : -
Dosage And administration of the control intervention : po
CHUGAI PHARMACEUTICAL CO., LTD.NULLcomplete225BOTH168Phase 2Japan, Asia except Japan, North America, South America, Europe
14EUCTR2016-000750-35-HR
(EUCTR)
27/03/201812/04/2018A study to investigate the safety, tolerability, pharmacokinetics, pharmacodynamics and efficacy of Risdiplam (RO7034067) in type 2 and 3 spinal muscular atrophy patientsA TWO-PART SEAMLESS, MULTI-CENTER RANDOMIZED, PLACEBO-CONTROLLED, DOUBLE BLIND STUDY TO INVESTIGATE THE SAFETY, TOLERABILITY, PHARMACOKINETICS, PHARMACODYNAMICS AND EFFICACY OF RO7034067 IN TYPE 2 AND 3 SPINAL MUSCULAR ATROPHY PATIENTS. Spinal Muscular Atrophy (SMA) Type 2 and 3
MedDRA version: 20.1;Level: PT;Classification code 10041582;Term: Spinal muscular atrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]
Product Name: RO7034067
Product Code: RO7034067/F12
INN or Proposed INN: risdiplam
Product Name: RO7034067
Product Code: RO7034067/F13
INN or Proposed INN: risdiplam
F. Hoffmann-La Roche LtdNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
219Phase 2United States;Serbia;Spain;Ukraine;Turkey;Russian Federation;United Kingdom;Switzerland;Italy;France;Hungary;Mexico;Canada;Argentina;Poland;Belgium;Brazil;Romania;Croatia;Bulgaria;Germany;China;Japan
15EUCTR2016-000750-35-DE
(EUCTR)
09/11/201613/07/2016A Study to investigate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics And Efficacy of Risdiplam (RO7034067) in Type 2 and 3 Spinal Muscular Atrophy PatientsA TWO-PART SEAMLESS, MULTI-CENTER RANDOMIZED, PLACEBO-CONTROLLED, DOUBLE BLIND STUDY TO INVESTIGATE THE SAFETY, TOLERABILITY, PHARMACOKINETICS, PHARMACODYNAMICS AND EFFICACY OF RO7034067 IN TYPE 2 AND 3 SPINAL MUSCULAR ATROPHY PATIENTS. Spinal Muscular Atrophy (SMA) Type 2 and 3
MedDRA version: 20.1;Level: PT;Classification code 10041582;Term: Spinal muscular atrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]
Product Name: RO7034067
Product Code: RO7034067/F12
INN or Proposed INN: Risdiplam
Product Name: RO7034067
Product Code: RO7034067/F13
INN or Proposed INN: Risdiplam
F. Hoffmann-La Roche LtdNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
219Phase 2Serbia;United States;Spain;Ukraine;Turkey;Russian Federation;United Kingdom;Switzerland;Italy;France;Hungary;Mexico;Canada;Argentina;Poland;Brazil;Belgium;Romania;Croatia;Bulgaria;Germany;China;Japan
16NCT02908685
(ClinicalTrials.gov)
October 20, 201619/9/2016A Study to Investigate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Efficacy of Risdiplam (RO7034067) in Type 2 and 3 Spinal Muscular Atrophy (SMA) ParticipantsA Two Part Seamless, Multi-Center Randomized, Placebo-Controlled, Double-Blind Study to Investigate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Efficacy of Risdiplam (RO7034067) in Type 2 and 3 Spinal Muscular Atrophy PatientsMuscular Atrophy, SpinalDrug: Placebo;Drug: RisdiplamHoffmann-La RocheNULLActive, not recruiting2 Years25 YearsAll231Phase 2/Phase 3United States;Belgium;Brazil;Canada;China;Croatia;France;Germany;Italy;Japan;Poland;Russian Federation;Serbia;Spain;Turkey;United Kingdom;Argentina;Australia;Sweden;Switzerland;Taiwan
17EUCTR2016-000750-35-BE
(EUCTR)
06/09/201612/07/2016A study to investigate the safety, tolerability, pharmacokinetics, pharmacodynamics and efficacy of Risdiplam (RO7034067) in type 2 and 3 spinal muscular atrophy patientsA TWO-PART SEAMLESS, MULTI-CENTER RANDOMIZED, PLACEBO-CONTROLLED, DOUBLE BLIND STUDY TO INVESTIGATE THE SAFETY, TOLERABILITY, PHARMACOKINETICS, PHARMACODYNAMICS AND EFFICACY OF RO7034067 IN TYPE 2 AND 3 SPINAL MUSCULAR ATROPHY PATIENTS. Spinal Muscular Atrophy (SMA) Type 2 and 3
MedDRA version: 20.1;Level: PT;Classification code 10041582;Term: Spinal muscular atrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]
Product Name: RO7034067
Product Code: RO7034067/F12
INN or Proposed INN: risdiplam
Product Name: RO7034067
Product Code: RO7034067/F13
INN or Proposed INN: risdiplam
F. Hoffmann-La Roche LtdNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
219Phase 2United States;Serbia;Spain;Ukraine;Turkey;Russian Federation;United Kingdom;Switzerland;Italy;France;Hungary;Mexico;Canada;Argentina;Poland;Belgium;Brazil;Romania;Croatia;Bulgaria;Germany;China;Japan
18EUCTR2016-000750-35-IT
(EUCTR)
29/08/201628/06/2016A Study to investigate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics And Efficacy of RO7034067 in Type 2 and 3 Spinal Muscular Atrophy PatientsA TWO-PART SEAMLESS, MULTI-CENTER RANDOMIZED, PLACEBO-CONTROLLED, DOUBLE BLIND STUDY TO INVESTIGATE THE SAFETY, TOLERABILITY, PHARMACOKINETICS, PHARMACODYNAMICS AND EFFICACY OF RO7034067 IN TYPE 2 AND 3 SPINAL MUSCULAR ATROPHY PATIENTS. Spinal Muscular Atrophy (SMA)
MedDRA version: 19.0;Level: PT;Classification code 10041582;Term: Spinal muscular atrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]
Product Name: RO7034067
Product Code: RO7034067/F06 with solvent (RO7034067/F08)
INN or Proposed INN: n.a
Product Name: RO7034067
Product Code: RO7034067/F07 with solvent (RO7034067/F09)
INN or Proposed INN: n.a
F. Hoffmann-La Roche LtdNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
186Phase 2United States;Spain;Turkey;Italy;Switzerland;United Kingdom;France;Canada;Belgium;Australia;Germany;Netherlands;Sweden
19EUCTR2014-003626-40-BE
(EUCTR)
09/06/201630/03/2016Nivolumab versus placebo after surgery in subjects with urinary tract cancerA Phase 3 Randomized, Double-blind, Multi-center Study of Adjuvant Nivolumab versus Placebo in Subjects with High Risk Invasive Urothelial Carcinoma - CheckMate 274 Spinal Muscular Atrophy
MedDRA version: 20.1;Level: LLT;Classification code 10051203;Term: Spinal muscular atrophy congenital;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]
Product Code: LMI070
Other descriptive name: LMI070
Bristol-Myers Squibb International CorporationNULLNot RecruitingFemale: yes
Male: yes
800Phase 1;Phase 2Netherlands;Germany;Italy;United States;Czech Republic;Hungary;Poland;Belgium;Denmark;Russian Federation;Bulgaria
20NCT02644668
(ClinicalTrials.gov)
January 14, 201623/12/2015A Study of CK-2127107 in Patients With Spinal Muscular AtrophyA Phase 2, Double-Blind, Randomized, Placebo-Controlled, Multiple Dose Study of CK-2127107 in Two Ascending Dose Cohorts of Patients With Spinal Muscular AtrophySpinal Muscular AtrophyDrug: Placebo;Drug: Reldesemtiv 150 mg;Drug: Reldesemtiv 450 mgCytokineticsAstellas Pharma Global Development, Inc.Completed12 YearsN/AAll70Phase 2United States;Canada
21NCT02633709
(ClinicalTrials.gov)
January 7, 201615/12/2015A Study to Investigate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Risdiplam (RO7034067) Given by Mouth in Healthy VolunteersA Single-Center, Randomized, Investigator/Subject-Blind, Adaptive Single-Ascending-Dose(SAD), Placebo-Controlled, Parallel Study to Investigate the Safety, Tolerability, Pharmacokinetics (Including the Effect of Food and the Effect of Itraconazole on the Pharmacokinetics of a Single Oral Dose of RO7034067), and Pharmacodynamics of RO7034067 Following Oral Administration in Healthy SubjectsSpinal Muscular AtrophyDrug: Itraconazole;Other: Placebo;Drug: RisdiplamHoffmann-La RocheNULLCompleted18 Years45 YearsMale33Phase 1Netherlands
22NCT02941328
(ClinicalTrials.gov)
December 201530/9/2016SPACE Trial: Pyridostigmine vs Placebo in SMA Types 2, 3 and 4A Phase II, Mono-center, Placebo-controlled, Double-blind, Crossover Trial to Investigate Effect and Efficacy of Pyridostigmine in Dutch Patients With Spinal Muscular Atrophy Types 2, 3 and 4Spinal Muscular Atrophy;SMA;Kugelberg-Welander DiseaseDrug: Pyridostigmine;Drug: PlaceboUMC UtrechtNULLCompleted12 YearsN/AAll39Phase 2Netherlands
23EUCTR2011-004369-34-NL
(EUCTR)
22/04/201503/11/2014 SPACE trial SMA and Pyridostigmine in Adults and Children; Experimental trial to assess effect of pyridostigmine compared to placebo in patients with spinal muscular atrophy types 2, 3 and 4 SPACE trial SMA and Pyridostigmine in Adults and Children; Efficacy trial Phase II, mono-center, doubleblind, placebo-controlled, crossover trial to assess efficacy of pyridostigmine in patients with spinal muscular atrophy types 2, 3 and 4 - SPACE trial Proximal spinal muscular atrophy (SMA) is characterized by weakness of predominantly axial and proximal muscle groups and is caused by homozygous deletion of the survival motor neuron 1 (SMN1)-gene. There are 4 SMA types (type 1-4), with a descending order of severity. Age at onset and achieved motor milestones are the characteristics to define severity. Treatment of SMA is exclusively supportive.
MedDRA version: 17.1;Level: LLT;Classification code 10068209;Term: Spinal muscular atrophy adult onset;System Organ Class: 100000004850
MedDRA version: 17.1;Classification code 10051203;Term: Spinal muscular atrophy congenital;System Organ Class: 100000004850 ;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]
Trade Name: pyridostigmine bromide
Product Name: Mestinon
Product Code: RVG 03820
Universtiy Medical Center UtrechtNULLNot Recruiting Female: yes
Male: yes
45 Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): noNetherlands
24EUCTR2014-002246-41-ES
(EUCTR)
15/04/201503/03/2015A Study of RO6885247 in Adult and Pediatric Patients with Spinal Muscular Atrophy (MOONFISH)A MULTICENTER, RANDOMIZED, DOUBLE-BLIND, PLACEBO-CONTROLLED, MULTIPLE-DOSE STUDY TO INVESTIGATE THE SAFETY, TOLERABILITY, PHARMACOKINETICS, AND PHARMACODYNAMICS OF RO6885247 FOLLOWING 12 WEEKS OF TREATMENT IN ADULT AND PEDIATRIC PATIENTS WITH SPINAL MUSCULAR ATROPHY (MOONFISH) - MOONFISH Spinal Muscular Atrophy
MedDRA version: 18.0;Level: PT;Classification code 10041582;Term: Spinal muscular atrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]
Product Code: RO6885247/F03
INN or Proposed INN: n.a.
Other descriptive name: n.a.
Product Code: RO6885247/F02
INN or Proposed INN: n.a.
Other descriptive name: n.a.
Roche Farma S.A., en nombre de F. Hoffmann-La Roche Ltd.NULLNot RecruitingFemale: yes
Male: yes
64Phase 1France;United States;Spain;Turkey;Netherlands;Italy;United Kingdom;Switzerland;Sweden
25NCT02240355
(ClinicalTrials.gov)
November 201411/9/2014A Study of RO6885247 in Adult and Pediatric Patients With Spinal Muscular Atrophy (MOONFISH)A Multicenter, Randomized, Double Blind, Placebo Controlled, Multiple Dose Study to Investigate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of RO6885247 Following 12 Weeks of Treatment in Adult and Pediatric Patients With Spinal Muscular Atrophy (MOONFISH).Muscular Atrophy, SpinalDrug: RO6885247;Drug: placeboHoffmann-La RocheNULLTerminatedN/A55 YearsBoth9Phase 1United States;Canada;France;Italy;Netherlands;Sweden;Switzerland;Turkey;United Kingdom;Czech Republic
26EUCTR2014-002246-41-IT
(EUCTR)
23/10/201409/09/2014A Study of RO6885247 in Adult and Pediatric Patients with Spinal Muscular Atrophy (MOONFISH)A MULTICENTER, RANDOMIZED, DOUBLE-BLIND, PLACEBO-CONTROLLED, MULTIPLE-DOSE STUDY TO INVESTIGATE THE SAFETY, TOLERABILITY, PHARMACOKINETICS, AND PHARMACODYNAMICS OF RO6885247 FOLLOWING 12 WEEKS OF TREATMENT IN ADULT AND PEDIATRIC PATIENTS WITH SPINAL MUSCULAR ATROPHY (MOONFISH) - MOONFISH Spinal Muscular Atrophy
MedDRA version: 17.0;Level: PT;Classification code 10041582;Term: Spinal muscular atrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]
Product Code: RO6885247/F03
INN or Proposed INN: n.a.
Other descriptive name: n.a.
F. Hoffmann-La Roche Ltd.NULLNot RecruitingFemale: yes
Male: yes
48Phase 1United States;Spain;Netherlands;United Kingdom;Switzerland;Italy
27NCT01671384
(ClinicalTrials.gov)
August 201313/8/2012Valproate and Levocarnitine in Children With Spinal Muscular AtrophyRandomized Placebo Controlled Trial of Valproate and Levocarnitine in Children With Spinal Muscular Atrophy Aged 2-15 YearsSpinal Muscular AtrophyDrug: Valproate, Levocarnitine;Drug: PlaceboAll India Institute of Medical Sciences, New DelhiNULLRecruiting2 Years15 YearsBoth60Phase 3India
28NCT01645787
(ClinicalTrials.gov)
June 20125/7/2012Short and Long Term Treatment With 4-AP in Ambulatory SMA PatientsColumbia SMA Project: 4-AP as a Potential SMA Therapeutic Agent and Biological Mechanisms of ActionSpinal Muscular AtrophyDrug: 4-aminopyridine;Drug: PlaceboColumbia UniversityNULLCompleted18 Years50 YearsBoth11Phase 2/Phase 3United States
29EUCTR2010-020386-24-GB
(EUCTR)
31/01/201108/10/2010Safety and Efficacy of Olesoxime (TRO19622) in 3-25 yrs SMA patientsPhase II, multicenter, randomized, adaptive, double-blind, placebo controlled study to assess safety and efficacy of olesoxime (TRO19622) in 3-25 year old Spinal Muscular Atrophy (SMA) patients. Spinal Muscular Atrophy (type 2 or type 3 non ambulant patients aged 3-25 years).
MedDRA version: 14.0;Level: PT;Classification code 10041582;Term: Spinal muscular atrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: OLESOXIME
Product Code: TRO19622
INN or Proposed INN: olesoxime
Other descriptive name: 4-cholesten-3-one, oxime
TROPHOS SANULLNot Recruiting Female: yes
Male: yes
150 Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): noFrance;Belgium;Germany;Netherlands;Italy;United Kingdom
30EUCTR2010-020386-24-DE
(EUCTR)
29/12/201028/07/2010Phase II, multicenter, randomized, adaptive, double-blind, placebo controlled study to assess safety and efficacy of olesoxime (TRO19622) in 3-25 year old Spinal Muscular Atrophy (SMA) patients.Phase II, multicenter, randomized, adaptive, double-blind, placebo controlled study to assess safety and efficacy of olesoxime (TRO19622) in 3-25 year old Spinal Muscular Atrophy (SMA) patients. Spinal Muscular Atrophy (type 2 or type 3 non ambulant patients aged 3-25 years).
MedDRA version: 12.1;Level: LLT;Classification code 10041582;Term: Spinal muscular atrophy
Product Name: OLESOXIME
Product Code: TRO19622
INN or Proposed INN: olesoxime
Other descriptive name: 4-cholesten-3-one, oxime
TROPHOS SANULLNot RecruitingFemale: yes
Male: yes
150Phase 2Belgium;Netherlands;Germany;United Kingdom;Italy
31EUCTR2010-020386-24-NL
(EUCTR)
18/11/201028/06/2010Phase II, multicenter, randomized, adaptive, double-blind, placebo controlled study to assess safety and efficacy of olesoxime (TRO19622) in 3-25 year old Spinal Muscular Atrophy (SMA) patients.Phase II, multicenter, randomized, adaptive, double-blind, placebo controlled study to assess safety and efficacy of olesoxime (TRO19622) in 3-25 year old Spinal Muscular Atrophy (SMA) patients. Spinal Muscular Atrophy (type 2 or type 3 non ambulant patients aged 3-25 years).
MedDRA version: 12.1;Level: LLT;Classification code 10041582;Term: Spinal muscular atrophy
Product Name: OLESOXIME
Product Code: TRO19622
INN or Proposed INN: olesoxime
Other descriptive name: 4-cholesten-3-one, oxime
TROPHOS SANULLNot RecruitingFemale: yes
Male: yes
150Phase 2Belgium;Germany;Netherlands;United Kingdom;Italy
32NCT01302600
(ClinicalTrials.gov)
November 201018/2/2011Safety and Efficacy of Olesoxime (TRO19622) in 3-25 Years SMA Patients.Phase II, Multicenter, Randomized, Adaptive, Double-blind, Placebo Controlled Study to Assess Safety and Efficacy of Olesoxime (TRO19622) in 3-25 Year Old Spinal Muscular Atrophy (SMA) Patients.Spinal Muscular Atrophy Type II;Spinal Muscular Atrophy Type III Non AmbulantDrug: Olesoxime;Drug: PlaceboHoffmann-La RocheAssociation Française contre les Myopathies (AFM), ParisCompleted3 Years25 YearsBoth165Phase 2Belgium;France;Germany;Italy;Netherlands;Poland;United Kingdom
33EUCTR2010-020386-24-BE
(EUCTR)
29/10/201027/07/2010Phase II, multicenter, randomized, adaptive, double-blind, placebo controlled study to assess safety and efficacy of olesoxime (TRO19622) in 3-25 year old Spinal Muscular Atrophy (SMA) patients.Phase II, multicenter, randomized, adaptive, double-blind, placebo controlled study to assess safety and efficacy of olesoxime (TRO19622) in 3-25 year old Spinal Muscular Atrophy (SMA) patients. Spinal Muscular Atrophy (type 2 or type 3 non ambulant patients aged 3-25 years).
MedDRA version: 12.1;Level: LLT;Classification code 10041582;Term: Spinal muscular atrophy
Product Name: OLESOXIME
Product Code: TRO19622
INN or Proposed INN: olesoxime
Other descriptive name: 4-cholesten-3-one, oxime
TROPHOS SANULLNot RecruitingFemale: yes
Male: yes
150Phase 2Belgium;Germany;Netherlands;United Kingdom;Italy
34EUCTR2007-001088-32-IT
(EUCTR)
29/07/200818/07/2008A PHASE II RANDOMIZED, DOUBLE-BLIND STUDY VS. PLACEBO FOR THE EVALUATION OF EFFICACY AND TOLERABILITY OF SALBUTAMOL ADMINISTERED BY ORAL ROUTE IN PATIENTS AFFECTED BY SPINAL MUSCULAR ATHROPHY. - NDA PHASE II RANDOMIZED, DOUBLE-BLIND STUDY VS. PLACEBO FOR THE EVALUATION OF EFFICACY AND TOLERABILITY OF SALBUTAMOL ADMINISTERED BY ORAL ROUTE IN PATIENTS AFFECTED BY SPINAL MUSCULAR ATHROPHY. - ND Patients affected by SMA
MedDRA version: 9.1;Level: LLT;Classification code 10041582;Term: Spinal muscular atrophy
Trade Name: VOLMAX*30CPR 4MG R.P.
INN or Proposed INN: Salbutamol
ISTITUTO NEUROLOGICO CARLO BESTANULLNot RecruitingFemale: yes
Male: yes
Phase 2Italy
35NCT00533221
(ClinicalTrials.gov)
October 200712/9/2007Pilot Study of Growth Hormon to Treat SMA Typ II and IIICan Treatment With Human Growth Hormone Increase Strength in Spinal Muscular Atrophy Type II and III?Muscular Atrophy, SpinalDrug: somatotropin;Drug: PlaceboUniversity Hospital FreiburgNovo Nordisk A/SCompleted6 Years35 YearsBoth20Phase 2Germany
36NCT00481013
(ClinicalTrials.gov)
July 200730/5/2007Valproic Acid in Ambulant Adults With Spinal Muscular AtrophyProspective Controlled Trial of Valproic Acid in Ambulant Adults With Spinal Muscular Atrophy (VALIANTSMA) StudySpinal Muscular AtrophyDrug: Valproic Acid (VPA);Drug: PlaceboUniversity of UtahFamilies of Spinal Muscular Atrophy;AbbottCompleted18 Years60 YearsBoth33Phase 2United States
37NCT00485511
(ClinicalTrials.gov)
June 200711/6/2007A Trial of Hydroxyurea in Spinal Muscular AtrophyA Randomized, Double-Blind, Placebo-Controlled Trial of Hydroxyurea in Spinal Muscular AtrophySpinal Muscular AtrophyDrug: HydroxyureaKaohsiung Medical University Chung-Ho Memorial HospitalNULLCompleted4 YearsN/ABothPhase 2/Phase 3Taiwan
38NCT00774423
(ClinicalTrials.gov)
January 200616/10/2008Study to Evaluate the Efficacy of Riluzole in Children and Young Adults With Spinal Muscular Atrophy (SMA)Multicentric, Randomized, Double-blind Study Versus Placebo, With Two Parallel Groups Treated to Evaluate the Efficacy and the Tolerance of Riluzole in Children and Young Adults (6 to 20 Years of Age) With SMA. (Type II and Type III)SMADrug: RiluzoleAssistance Publique - Hôpitaux de ParisNULLCompleted6 Years20 YearsBoth141Phase 2/Phase 3France
39NCT00227266
(ClinicalTrials.gov)
September 200523/9/2005Valproic Acid and Carnitine in Patients With Spinal Muscular AtrophyMulti-center Phase II Trial of Valproic Acid and Carnitine in Patients With Spinal Muscular Atrophy (SMA CARNI-VAL Trial)Spinal Muscular AtrophyDrug: Valproic Acid and Levocarnitine;Drug: PlaceboUniversity of UtahFamilies of Spinal Muscular Atrophy;Leadiant Biosciences, Inc.;AbbottCompleted2 Years17 YearsAll94Phase 2United States;Canada
40NCT00568802
(ClinicalTrials.gov)
January 20044/12/2007A Pilot Therapeutic Trial Using Hydroxyurea in Type II and Type III Spinal Muscular Atrophy PatientsA Pilot Therapeutic Trial Using Hydroxyurea in Type II and Type III Spinal Muscular Atrophy PatientsMuscular Atrophy, SpinalDrug: Hydroxyurea;Drug: Placebo to match hydroxyureaStanford UniversityNULLCompleted1 Year10 YearsAll27Phase 1/Phase 2United States
41NCT00568698
(ClinicalTrials.gov)
January 20044/12/2007A Pilot Therapeutic Trial Using Hydroxyurea in Type I Spinal Muscular Atrophy PatientsA Pilot Therapeutic Trial Using Hydroxyurea in Type I Spinal Muscular Atrophy PatientsMuscular Atrophy, SpinalDrug: Hydroxyurea;Drug: Placebo to match hydroxyureaStanford UniversityNULLCompletedN/A2 YearsAll29Phase 1/Phase 2United States
42EUCTR2016-000750-35-FR
(EUCTR)
26/01/2018A Study to investigate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics And Efficacy of RO7034067 in Type 2 and 3 Spinal Muscular Atrophy PatientsA TWO-PART SEAMLESS, MULTI-CENTER RANDOMIZED, PLACEBO-CONTROLLED, DOUBLE BLIND STUDY TO INVESTIGATE THE SAFETY, TOLERABILITY, PHARMACOKINETICS, PHARMACODYNAMICS AND EFFICACY OF RO7034067 IN TYPE 2 AND 3 SPINAL MUSCULAR ATROPHY PATIENTS. Spinal Muscular Atrophy (SMA)
MedDRA version: 20.1;Level: PT;Classification code 10041582;Term: Spinal muscular atrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]
Product Name: RO7034067
Product Code: RO7034067/F06 with solvent (RO7034067/F08)
INN or Proposed INN: n.a
Product Name: RO7034067
Product Code: RO7034067/F07 with solvent (RO7034067/F09)
INN or Proposed INN: n.a
F. Hoffmann-La Roche LtdNULLNAFemale: yes
Male: yes
186Phase 2United States;Spain;Turkey;Switzerland;United Kingdom;Italy;France;Canada;Belgium;Australia;Germany;Netherlands;Sweden
43EUCTR2021-003417-19-DE
(EUCTR)
18/11/2021A Study to Investigate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Efficacy of RO7204239 in Combination with Risdiplam (RO7034067) in Ambulant Patients with Spinal Muscular AtrophyA TWO-PART, SEAMLESS, MULTI-CENTER, RANDOMIZED, PLACEBO-CONTROLLED, DOUBLE-BLIND STUDY TO INVESTIGATE THE SAFETY, TOLERABILITY, PHARMACOKINETICS, PHARMACODYNAMICS AND EFFICACY OF RO7204239 IN COMBINATION WITH RISDIPLAM (RO7034067) IN AMBULANT PATIENTS WITH SPINAL MUSCULAR ATROPHY Spinal Muscular Atrophy (SMA)
MedDRA version: 20.1;Level: LLT;Classification code 10051203;Term: Spinal muscular atrophy congenital;System Organ Class: 100000004850
MedDRA version: 20.1;Classification code 10041583;Term: Spinal muscular atrophy, unspecified;System Organ Class: 100000004850
MedDRA version: 20.0;Classification code 10079413;Term: Spinal muscular atrophy type I;Classification code 10079415;Term: Spinal muscular atrophy type III;Classification code 10079416;Term: Spinal muscular atrophy type II;Classification code 10079417;Term: Spinal muscular atrophy infantile onset;Classification code 10079418;Term: Spinal muscular atrophy later onset;Classification code 10079419;Term: Spinal muscular atrophy pre-symptomatic;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: N/A
Product Code: RO7204239/F01-01
INN or Proposed INN: N/A
Other descriptive name: GYM329
Trade Name: Evrysdi
INN or Proposed INN: Risdiplam
F.Hoffmann-La Roche LtdNULLNAFemale: yes
Male: yes
180Phase 2;Phase 3United States;Poland;Belgium;Netherlands;Germany;United Kingdom;Italy
44EUCTR2010-020386-24-FR
(EUCTR)
01/06/2010Phase II, multicenter, randomized, adaptive, double-blind, placebo controlled study to assess safety and efficacy of olesoxime (TRO19622) in 3-25 year old Spinal Muscular Atrophy (SMA) patients.Phase II, multicenter, randomized, adaptive, double-blind, placebo controlled study to assess safety and efficacy of olesoxime (TRO19622) in 3-25 year old Spinal Muscular Atrophy (SMA) patients. Spinal Muscular Atrophy (type 2 or type 3 non ambulant patients aged 3-25 years)
MedDRA version: 12.1;Level: LLT;Classification code 10041582;Term: Spinal muscular atrophy
Product Name: OLESOXIME
Product Code: TRO19622
INN or Proposed INN: olesoxime
Other descriptive name: 4-cholesten-3-one, oxime
TROPHOS SANULLNAFemale: yes
Male: yes
Phase 2France;Belgium;Germany;Netherlands;United Kingdom;Italy
45EUCTR2016-000750-35-PL
(EUCTR)
20/02/2018A study to investigate the safety, tolerability, pharmacokinetics, pharmacodynamics and efficacy of RO7034067 in type 2 and 3 spinal muscular atrophy patientsA TWO-PART SEAMLESS, MULTI-CENTER RANDOMIZED, PLACEBO-CONTROLLED, DOUBLE BLIND STUDY TO INVESTIGATE THE SAFETY, TOLERABILITY, PHARMACOKINETICS, PHARMACODYNAMICS AND EFFICACY OF RO7034067 IN TYPE 2 AND 3 SPINAL MUSCULAR ATROPHY PATIENTS. Spinal Muscular Atrophy (SMA)
MedDRA version: 20.1;Level: PT;Classification code 10041582;Term: Spinal muscular atrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]
Product Name: RO7034067
Product Code: RO7034067/F12
INN or Proposed INN: risdiplam
Product Name: RO7034067
Product Code: RO7034067/F13
INN or Proposed INN: risdiplam
F. Hoffmann-La Roche LtdNULLNAFemale: yes
Male: yes
219Phase 2United States;Serbia;Spain;Ukraine;Turkey;Russian Federation;United Kingdom;Switzerland;Italy;France;Hungary;Mexico;Canada;Argentina;Poland;Belgium;Brazil;Romania;Croatia;Bulgaria;Germany;China;Japan
46EUCTR2021-005314-34-BE
(EUCTR)
11/02/2022Phase 3 Active Treatment Trial to Evaluate the Efficacy and Safety of Apitegromab in Patients with Later-Onset Spinal Muscular Atrophy Who Are Being Treated with Nusinersen or RisdiplamPhase 3, Double-Blind, Placebo-Controlled Trial to Evaluate the Efficacy and Safety of Apitegromab (SRK-015) in Patients with Later-Onset Spinal Muscular Atrophy Receiving Background Nusinersen or Risdiplam Therapy - SAPPHIRE Spinal Muscular Atrophy (SMA)
MedDRA version: 20.1;Level: LLT;Classification code 10041583;Term: Spinal muscular atrophy, unspecified;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
Product Name: Apitegromab
Product Code: SRK-015
INN or Proposed INN: Apitegromab
Scholar Rock, Inc.NULLNAFemale: yes
Male: yes
204Phase 3United States;France;Spain;Poland;Belgium;Netherlands;Germany;United Kingdom;Italy
47EUCTR2021-003417-19-BE
(EUCTR)
15/12/2021A Study to Investigate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Efficacy of RO7204239 in Combination with Risdiplam (RO7034067) in Ambulant Patients with Spinal Muscular AtrophyA TWO-PART, SEAMLESS, MULTI-CENTER, RANDOMIZED, PLACEBO-CONTROLLED, DOUBLE-BLIND STUDY TO INVESTIGATE THE SAFETY, TOLERABILITY, PHARMACOKINETICS, PHARMACODYNAMICS AND EFFICACY OF RO7204239 IN COMBINATION WITH RISDIPLAM (RO7034067) IN AMBULANT PATIENTS WITH SPINAL MUSCULAR ATROPHY Spinal Muscular Atrophy (SMA)
MedDRA version: 20.1;Level: LLT;Classification code 10051203;Term: Spinal muscular atrophy congenital;System Organ Class: 100000004850
MedDRA version: 20.1;Classification code 10041583;Term: Spinal muscular atrophy, unspecified;System Organ Class: 100000004850
MedDRA version: 20.0;Classification code 10079413;Term: Spinal muscular atrophy type I;Classification code 10079415;Term: Spinal muscular atrophy type III;Classification code 10079416;Term: Spinal muscular atrophy type II;Classification code 10079417;Term: Spinal muscular atrophy infantile onset;Classification code 10079418;Term: Spinal muscular atrophy later onset;Classification code 10079419;Term: Spinal muscular atrophy pre-symptomatic;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: N/A
Product Code: RO7204239/F01-01
INN or Proposed INN: N/A
Other descriptive name: GYM329
Trade Name: Evrysdi
INN or Proposed INN: Risdiplam
F.Hoffmann-La Roche LtdNULLNAFemale: yes
Male: yes
180Phase 2;Phase 3United States;Poland;Belgium;Germany;Netherlands;Italy;United Kingdom
48EUCTR2021-005314-34-NL
(EUCTR)
10/03/2022Phase 3 Active Treatment Trial to Evaluate the Efficacy and Safety of Apitegromab in Patients with Later-Onset Spinal Muscular Atrophy Who Are Being Treated with Nusinersen or RisdiplamPhase 3, Double-Blind, Placebo-Controlled Trial to Evaluate the Efficacy and Safety of Apitegromab (SRK-015) in Patients with Later-Onset Spinal Muscular Atrophy Receiving Background Nusinersen or Risdiplam Therapy - SAPPHIRE Spinal Muscular Atrophy (SMA)
MedDRA version: 20.1;Level: LLT;Classification code 10041583;Term: Spinal muscular atrophy, unspecified;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
Product Name: Apitegromab
Product Code: SRK-015
INN or Proposed INN: Apitegromab
Scholar Rock, Inc.NULLNAFemale: yes
Male: yes
204Phase 3France;United States;Spain;Poland;Belgium;Germany;Netherlands;United Kingdom;Italy