Risdiplam ( DrugBank: Risdiplam )
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 | 36 |
3. 脊髄性筋萎縮症
臨床試験数 : 217 / 薬物数 : 149 - (DrugBank : 33) / 標的遺伝子数 : 54 - 標的パスウェイ数 : 80
Showing 1 to 10 of 36 diseases
| 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 | NCT05232929 (ClinicalTrials.gov) | March 31, 2022 | 31/1/2022 | Long-Term Follow-Up Study of Risdiplam in Participants With Spinal Muscular Atrophy (SMA) | Long-Term Follow-Up Study of Patients With Spinal Muscular Atrophy Receiving Risdiplam Treatment | Spinal Muscular Atrophy | Drug: Risdiplam | Genentech, Inc. | NULL | Recruiting | 2 Months | N/A | All | 500 | Phase 4 | United States |
| 2 | NCT05115110 (ClinicalTrials.gov) | March 31, 2022 | 1/11/2021 | A Study to Investigate the Safety and Efficacy of RO7204239 in Combination With Risdiplam (RO7034067) in Ambulatory Children 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 Ambulant 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 | Not yet recruiting | 2 Years | 10 Years | All | 180 | Phase 2/Phase 3 | United States;Belgium;Germany;Italy;Netherlands;Poland;United Kingdom |
| 3 | NCT05067790 (ClinicalTrials.gov) | January 21, 2022 | 24/9/2021 | A Study to Evaluate Higher Dose (HD) Nusinersen (BIIB058) in Participants With Spinal Muscular Atrophy Previously Treated With Risdiplam A Study to Evaluate Higher Dose (HD) Nusinersen (BIIB058) in Participants With Spinal Muscular Atrop ... | A Phase 3b Study to Evaluate Higher Dose Nusinersen (BIIB058) in Patients With Spinal Muscular Atrophy Previously Treated With Risdiplam A Phase 3b Study to Evaluate Higher Dose Nusinersen (BIIB058) in Patients With Spinal Muscular Atrop ... | Spinal Muscular Atrophy | Drug: Nusinersen | Biogen | NULL | Recruiting | 5 Years | 39 Years | All | 135 | Phase 3 | United States |
| 4 | EUCTR2021-003417-19-PL (EUCTR) | 18/01/2022 | 25/10/2021 | 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 | ||
| 5 | EUCTR2021-003417-19-IT (EUCTR) | 03/01/2022 | 18/10/2021 | 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 | ||
| 6 | EUCTR2016-000750-35-GB (EUCTR) | 25/11/2020 | 23/06/2016 | 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 ... | ||
| 7 | NCT03779334 (ClinicalTrials.gov) | August 7, 2019 | 17/12/2018 | A Study of Risdiplam in Infants With Genetically Diagnosed and Presymptomatic Spinal Muscular Atrophy A Study of Risdiplamin Infants With Genetically Diagnosed and Presymptomatic Spinal Muscular Atrophy ... | An Open-Label Study of Risdiplam in Infants With Genetically Diagnosed and Presymptomatic Spinal Muscular Atrophy An Open-Label Study of Risdiplamin Infants With Genetically Diagnosed and Presymptomatic Spinal Musc ... | Muscular Atrophy, Spinal | Drug: Risdiplam | Hoffmann-La Roche | NULL | Active, not recruiting | N/A | 6 Weeks | All | 25 | Phase 2 | United States;Australia;Belgium;Brazil;Canada;China;Italy;Poland;Russian Federation;Saudi Arabia;Taiwan United States;Australia;Belgium;Brazil;Canada;China;Italy;Poland;Russian Federation;Saudi Arabia;Tai ... |
| 8 | NCT03988907 (ClinicalTrials.gov) | June 18, 2019 | 14/6/2019 | A Drug-drug Interaction Study With Risdiplam Multiple Dose and Midazolam in Healthy Participants | A Phase I, 2-Part, Open-Label Study to Investigate the Safety, Tolerability, and Pharmacokinetics of Multiple Doses of Risdiplam and the Effect of Risdiplam on the Pharmacokinetics of Midazolam Following Oral Administration in Healthy Participants A Phase I, 2-Part, Open-Label Study to Investigate the Safety, Tolerability, and Pharmacokinetics of ... | Spinal Muscular Atrophy | Drug: Risdiplam;Drug: Midazolam | Hoffmann-La Roche | NULL | Completed | 18 Years | 55 Years | All | 35 | Phase 1 | United States |
| 9 | EUCTR2016-004184-39-NL (EUCTR) | 08/05/2019 | 20/11/2018 | 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 | ||
| 10 | EUCTR2018-002087-12-PL (EUCTR) | 21/03/2019 | 05/12/2018 | A Study of Risdiplam in Infants with Genetically Diagnosed and Presymptomatic Spinal Muscular Atrophy A Study of Risdiplamin Infants with Genetically Diagnosed and Presymptomatic Spinal Muscular Atrophy ... | AN OPEN-LABEL STUDY OF RISDIPLAM IN INFANTS WITH GENETICALLY DIAGNOSED AND PRESYMPTOMATIC SPINAL MUSCULAR ATROPHY AN OPEN-LABEL STUDY OF RISDIPLAMIN INFANTS WITH GENETICALLY DIAGNOSED AND PRESYMPTOMATIC SPINAL MUSC ... | 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 |