RO7034067
( DrugBank: - / KEGG DRUG: - )
1 disease
| 告示番号 | 疾患名（ページ内リンク） | 臨床試験数 |
|---|---|---|
| 3 | 脊髄性筋萎縮症 | 37 |
3. 脊髄性筋萎縮症
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
Showing 1 to 10 of 37 trials
| 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 | NCT05115110 (ClinicalTrials.gov) | June 2, 202220220602 | 1/11/202120211101 | A Study to Investigate the Safety and Efficacy of RO7204239 in Combination With Risdiplam (RO7034067) in Participants With Spinal Muscular Atrophy A Study to Investigate the Safety and Efficacy of RO7204239 in Combination With Risdiplam (RO7034067 ... | A 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 Atrophy A Two-Part, Seamless, Multi-Center, Randomized, Placebo-Controlled, Double-Blind Study to Investigat ... | Spinal Muscular Atrophy (SMA) | Drug: RO7204239;Drug: Placebo;Drug: Risdiplam | Hoffmann-La Roche | NULL | Recruiting | 2 Years | 25 Years | All | 259 | Phase 2/Phase 3 | United States;Belgium;Italy;Netherlands;Poland;United Kingdom;Germany |
| 2 | EUCTR2021-003417-19-NL (EUCTR) | 25/05/202220220525 | 11/01/202220220111 | A Study to Investigate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Efficacy of RO7204239 in Combination with Risdiplam (RO7034067) in Ambulant Patients with Spinal Muscular Atrophy A Study to Investigate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Efficacy of ... | A 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 A TWO-PART, SEAMLESS, MULTI-CENTER, RANDOMIZED, PLACEBO-CONTROLLED, DOUBLE-BLIND STUDY TO INVESTIGAT ... | 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 Product Name: N/A Product Code: RO7204239/F01-01 INN or Proposed INN: N/A Other descriptive name: GYM32 ... | F.Hoffmann-La Roche Ltd | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 180 | Phase 2;Phase 3 | United States;Poland;Belgium;Germany;Netherlands;United Kingdom;Italy | ||
| 3 | EUCTR2021-003417-19-PL (EUCTR) | 18/01/202220220118 | 25/10/202120211025 | A Study to Investigate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Efficacy of RO7204239 in Combination with Risdiplam (RO7034067) in Ambulant Patients with Spinal Muscular Atrophy A Study to Investigate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Efficacy of ... | A 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 A TWO-PART, SEAMLESS, MULTI-CENTER, RANDOMIZED, PLACEBO-CONTROLLED, DOUBLE-BLIND STUDY TO INVESTIGAT ... | 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 Product Name: N/A Product Code: RO7204239/F01-01 INN or Proposed INN: N/A Other descriptive name: GYM32 ... | F.Hoffmann-La Roche Ltd | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 180 | Phase 2;Phase 3 | United States;Belgium;Poland;Netherlands;United Kingdom;Italy | ||
| 4 | EUCTR2021-003417-19-IT (EUCTR) | 03/01/202220220103 | 18/10/202120211018 | A Study to Investigate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Efficacy of RO7204239 in Combination with Risdiplam (RO7034067) in Ambulant Patients with Spinal Muscular Atrophy A Study to Investigate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Efficacy of ... | A 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 A TWO-PART, SEAMLESS, MULTI-CENTER, RANDOMIZED, PLACEBO-CONTROLLED, DOUBLE-BLIND STUDY TO INVESTIGAT ... | 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] Spinal Muscular Atrophy (SMA) MedDRA version: 20.0;Level: LLT;Classification code 10079415;Term: Spi ... | Product Name: N/A Product Code: [RO7204239/F01-01] Trade Name: Evrysdi Product Name: Risdiplam Product Code: [RO7034067] INN or Proposed INN: Risdiplam Product Name: N/A Product Code: [RO7204239/F01-01] Trade Name: Evrysdi Product Name: Risdiplam Product C ... | F. HOFFMANN - LA ROCHE LTD. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 180 | Phase 2;Phase 3 | United States;Poland;Belgium;Netherlands;United Kingdom;Italy | ||
| 5 | EUCTR2016-000750-35-GB (EUCTR) | 25/11/202020201125 | 23/06/201620160623 | A study to investigate the safety, tolerability, pharmacokinetics, pharmacodynamics and efficacy of Risdiplam (RO7034067) in type 2 and 3 spinal muscular atrophy patients A study to investigate the safety, tolerability, pharmacokinetics, pharmacodynamics and efficacy of ... | A 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. A TWO-PART SEAMLESS, MULTI-CENTER RANDOMIZED, PLACEBO-CONTROLLED, DOUBLE BLIND STUDY TO INVESTIGATE ... | 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] Spinal Muscular Atrophy (SMA) Type 2 and 3 MedDRA version: 20.1;Level: PT;Classification code 100415 ... | Product Name: RO7034067 Product Code: RO7034067/F12 INN or Proposed INN: risdiplam Product Name: RO7034067 Product Code: RO7034067/F13 INN or Proposed INN: risdiplam Product Name: RO7034067 Product Code: RO7034067/F12 INN or Proposed INN: risdiplam Product Name: RO7034 ... | F. Hoffmann-La Roche Ltd | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 231 | Phase 2 | United States;Serbia;Spain;Turkey;Russian Federation;United Kingdom;Italy;France;Canada;Belgium;Brazil;Poland;Croatia;Bulgaria;Germany;China;Japan United States;Serbia;Spain;Turkey;Russian Federation;United Kingdom;Italy;France;Canada;Belgium;Braz ... | ||
| 6 | EUCTR2016-004184-39-NL (EUCTR) | 08/05/201920190508 | 20/11/201820181120 | A study to investigate the safety, tolerability, and pharmacokinetics/pharmacodynamics of Risdiplam (RO7034067) in adult and pediatric patients with spinal muscular atrophy A study to investigate the safety, tolerability, and pharmacokinetics/pharmacodynamics of Risdiplam ... | AN OPEN-LABEL STUDY TO INVESTIGATE THE SAFETY, TOLERABILITY, AND PHARMACOKINETICS/PHARMACODYNAMICS OF RISDIPLAM (RO7034067) IN ADULT AND PEDIATRIC PATIENTS WITH SPINAL MUSCULAR ATROPHY AN OPEN-LABEL STUDY TO INVESTIGATE THE SAFETY, TOLERABILITY, AND PHARMACOKINETICS/PHARMACODYNAMICS O ... | 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] Spinal Muscular Atrophy (SMA) MedDRA version: 20.1;Level: PT;Classification code 10041582;Term: Spin ... | Product Name: RO7034067 Product Code: RO7034067/F13 INN or Proposed INN: Risdiplam | F. Hoffmann-La Roche Ltd | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 180 | Phase 2 | France;United States;Poland;Belgium;Germany;Netherlands;United Kingdom;Switzerland;Italy | ||
| 7 | EUCTR2018-002087-12-PL (EUCTR) | 21/03/201920190321 | 05/12/201820181205 | A Study of Risdiplam in Infants with Genetically Diagnosed and Presymptomatic Spinal Muscular Atrophy A Study of Risdiplam in Infants with Genetically Diagnosed and Presymptomatic Spinal Muscular Atroph ... | AN OPEN-LABEL STUDY OF RISDIPLAM IN INFANTS WITH GENETICALLY DIAGNOSED AND PRESYMPTOMATIC SPINAL MUSCULAR ATROPHY AN OPEN-LABEL STUDY OF RISDIPLAM IN INFANTS WITH GENETICALLY DIAGNOSED AND PRESYMPTOMATIC SPINAL MUS ... | 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] Spinal Muscular Atrophy (SMA) MedDRA version: 20.1;Level: PT;Classification code 10041582;Term: Spin ... | 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 Trade Name: EVRYSDI Product Name: RO7034067 Product Code: RO7034067/F13 INN or Proposed INN: Risdiplam O ... | F. Hoffmann-La Roche Ltd | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 25 | Phase 2 | United States;Saudi Arabia;Belgium;Brazil;Poland;Australia;Russian Federation;Italy;China | ||
| 8 | EUCTR2018-002087-12-IT (EUCTR) | 05/02/201920190205 | 17/06/202120210617 | A Study of Risdiplam in Infants with Genetically Diagnosed and Presymptomatic Spinal Muscular Atrophy A Study of Risdiplam in Infants with Genetically Diagnosed and Presymptomatic Spinal Muscular Atroph ... | AN OPEN-LABEL STUDY OF RISDIPLAM IN INFANTS WITH GENETICALLY DIAGNOSED AND PRESYMPTOMATIC SPINAL MUSCULAR ATROPHY - RAINBOWFISH AN OPEN-LABEL STUDY OF RISDIPLAM IN INFANTS WITH GENETICALLY DIAGNOSED AND PRESYMPTOMATIC SPINAL MUS ... | 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] Spinal Muscular Atrophy (SMA) MedDRA version: 20.1;Level: PT;Classification code 10041582;Term: Spin ... | Product Name: RO7034067 Product Code: [RO7034067/F13] INN or Proposed INN: Risdiplam Product Name: RO7034067 Product Code: [RO7034067/F12] INN or Proposed INN: Risdiplam Product Name: RO7034067 Product Code: [RO7034067/F13] INN or Proposed INN: Risdiplam Product Name: RO70 ... | F. HOFFMANN - LA ROCHE LTD. | NULL | Not Recruiting | Female: yes Male: yes | 25 | Phase 2 | United States;Saudi Arabia;Poland;Belgium;Brazil;Australia;Russian Federation;China;Italy | ||
| 9 | EUCTR2018-002087-12-BE (EUCTR) | 07/12/201820181207 | 23/10/201820181023 | A Study of Risdiplam in Infants with Genetically Diagnosed and Presymptomatic Spinal Muscular Atrophy A Study of Risdiplam in Infants with Genetically Diagnosed and Presymptomatic Spinal Muscular Atroph ... | AN OPEN-LABEL STUDY OF RISDIPLAM IN INFANTS WITH GENETICALLY DIAGNOSED AND PRESYMPTOMATIC SPINAL MUSCULAR ATROPHY AN OPEN-LABEL STUDY OF RISDIPLAM IN INFANTS WITH GENETICALLY DIAGNOSED AND PRESYMPTOMATIC SPINAL MUS ... | 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] Spinal Muscular Atrophy (SMA) MedDRA version: 20.1;Level: PT;Classification code 10041582;Term: Spin ... | 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 Trade Name: EVRYSDI Product Name: RO7034067 Product Code: RO7034067/F13 INN or Proposed INN: Risdiplam O ... | F. Hoffmann-La Roche Ltd | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 25 | Phase 2 | United States;Saudi Arabia;Poland;Brazil;Belgium;Australia;Russian Federation;Italy;China | ||
| 10 | EUCTR2016-000778-40-HR (EUCTR) | 15/08/201820180815 | 07/12/201820181207 | A Study to Investigate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Efficacy of RO7034067 in Infants with Type1 Spinal Muscular Atrophy A Study to Investigate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Efficacy of ... | A TWO PART SEAMLESS, OPEN-LABEL, MULTICENTER STUDY TO INVESTIGATE THE SAFETY, TOLERABILITY, PHARMACOKINETICS, PHARMACODYNAMICS AND EFFICACY OF RO7034067 IN INFANTS WITH TYPE1 SPINAL MUSCULAR ATROPHY A TWO PART SEAMLESS, OPEN-LABEL, MULTICENTER STUDY TO INVESTIGATE THE SAFETY, TOLERABILITY, PHARMACO ... | Type 1 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] Type 1 Spinal Muscular Atrophy (SMA) MedDRA version: 20.1;Level: PT;Classification code 10041582;Ter ... | Product Name: RO7034067 Product Code: RO7034067/F12 INN or Proposed INN: risdiplam Product Name: RO7034067 Product Code: RO7034067/F13 INN or Proposed INN: risdiplam Other descriptive name: RO7034067 Product Name: RO7034067 Product Code: RO7034067/F12 INN or Proposed INN: risdiplam Product Name: RO7034 ... | F. Hoffmann-La Roche Ltd | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 64 | Phase 2 | United States;Serbia;Saudi Arabia;Spain;Ukraine;Lebanon;Turkey;Russian Federation;United Kingdom;Switzerland;Italy;France;Canada;Belgium;Brazil;Poland;Croatia;Germany;China;Japan United States;Serbia;Saudi Arabia;Spain;Ukraine;Lebanon;Turkey;Russian Federation;United Kingdom;Swi ... |