GYM329 ( DrugBank: - )


1 disease
IDDisease name (Link within this page)Number of trials
3Spinal muscular atrophy4

3. Spinal muscular atrophy


Clinical trials : 237 Drugs : 123 - (DrugBank : 29) / Drug target genes : 51 - Drug target pathways : 75
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
1EUCTR2021-003417-19-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
2EUCTR2021-003417-19-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
3EUCTR2021-003417-19-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
4EUCTR2021-003417-19-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