EVRYSDI ( DrugBank: - )


1 disease
IDDisease name (Link within this page)Number of trials
3Spinal muscular atrophy6

3. Spinal muscular atrophy


Clinical trials : 217 Drugs : 149 - (DrugBank : 33) / Drug target genes : 54 - Drug target pathways : 80
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
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PhaseCountries
1EUCTR2021-003417-19-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
2EUCTR2021-003417-19-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
3EUCTR2018-002087-12-PL
(EUCTR)
21/03/201905/12/2018A Study of Risdiplam in Infants with Genetically Diagnosed and Presymptomatic Spinal Muscular AtrophyAN OPEN-LABEL STUDY OF RISDIPLAM IN INFANTS WITH GENETICALLY DIAGNOSED AND PRESYMPTOMATIC SPINAL MUSCULAR ATROPHY 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
MedDRA version: 20.0;Level: LLT;Classification code 10079419;Term: Spinal muscular atrophy pre-symptomatic;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]
Trade Name: EVRYSDI
Product Name: RO7034067
Product Code: RO7034067/F13
INN or Proposed INN: Risdiplam
Other descriptive name: RO7034067
Trade Name: EVRYSDI
Product Name: RO7034067
Product Code: RO7034067/F12
INN or Proposed INN: Risdiplam
Other descriptive name: RO7034067
F. Hoffmann-La Roche LtdNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
25Phase 2United States;Saudi Arabia;Belgium;Brazil;Poland;Australia;Russian Federation;Italy;China
4EUCTR2018-002087-12-BE
(EUCTR)
07/12/201823/10/2018A Study of Risdiplam in Infants with Genetically Diagnosed and Presymptomatic Spinal Muscular AtrophyAN OPEN-LABEL STUDY OF RISDIPLAM IN INFANTS WITH GENETICALLY DIAGNOSED AND PRESYMPTOMATIC SPINAL MUSCULAR ATROPHY 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
MedDRA version: 20.0;Level: LLT;Classification code 10079419;Term: Spinal muscular atrophy pre-symptomatic;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]
Trade Name: EVRYSDI
Product Name: RO7034067
Product Code: RO7034067/F13
INN or Proposed INN: Risdiplam
Other descriptive name: RO7034067
Trade Name: EVRYSDI
Product Name: RO7034067
Product Code: RO7034067/F12
INN or Proposed INN: Risdiplam
Other descriptive name: RO7034067
F. Hoffmann-La Roche LtdNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
25Phase 2United States;Saudi Arabia;Poland;Brazil;Belgium;Australia;Russian Federation;Italy;China
5EUCTR2021-003417-19-NL
(EUCTR)
11/01/2022A 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 LtdNULLNAFemale: yes
Male: yes
180Phase 2;Phase 3United States;Poland;Belgium;Netherlands;United Kingdom;Italy
6EUCTR2021-003417-19-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);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 3Belgium;Netherlands;United Kingdom;Italy;Poland;United States