18. 脊髄小脳変性症（多系統萎縮症を除く。） Spinocerebellar degeneration Clinical trials / Disease details
臨床試験数 : 76 / 薬物数 : 98 - (DrugBank : 31) / 標的遺伝子数 : 44 - 標的パスウェイ数 : 65
| 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 | NCT05515536 (ClinicalTrials.gov) | December 8, 2022 | 23/8/2022 | A Study to Assess the Safety and Efficacy of Vatiquinone in Participants With Friedreich Ataxia | Long-Term Open-Label Study to Assess the Safety and Efficacy of Vatiquinone in Patients With Friedreich Ataxia | Friedreich Ataxia | Drug: Vatiquinone | PTC Therapeutics | NULL | Enrolling by invitation | N/A | N/A | All | 140 | Phase 3 | United States;Australia;Brazil;Canada;France;Germany;Italy;New Zealand;Spain |
| 2 | NCT05485987 (ClinicalTrials.gov) | August 15, 2022 | 1/8/2022 | A Study of Vatiquinone for the Treatment of Participants With Friedreich Ataxia | An Open-Label Study to Evaluate Pharmacokinetics, Safety, and Efficacy of Vatiquinone in Children With Friedreich Ataxia Younger Than 7 Years of Age | Friedreich Ataxia | Drug: Vatiquinone | PTC Therapeutics | NULL | Recruiting | N/A | 6 Years | All | 5 | Phase 2 | United States |
| 3 | EUCTR2020-002812-36-ES (EUCTR) | 02/06/2021 | 19/11/2020 | A study to determine if Vatiquinone, the study drug, is safe and effective to treat a neurological condition called Friedreich ataxia | A Randomized, Parallel-Arm, Double-Blind, Placebo-Controlled Study with Open-Label Extension to Assess the Efficacy and Safety of Vatiquinone for the Treatment of Friedreich ataxia (MOVE-FA) - MOVE-FA | Friedreich Ataxia (FA) MedDRA version: 20.0;Level: PT;Classification code 10017374;Term: Friedreich's ataxia;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Product Name: Vatiquinone Product Code: PTC743 INN or Proposed INN: VATIQUINONE Other descriptive name: alpha-tocotrienolquinone | PTC Therapeutics, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 126 | Phase 2;Phase 3 | United States;France;Canada;Brazil;Spain;Australia;Germany;Italy | ||
| 4 | EUCTR2020-002812-36-IT (EUCTR) | 14/04/2021 | 04/06/2021 | A study to determine if Vatiquinone, the study drug, is safe and effective to treat a neurological condition called Friedreich ataxia | A Randomized, Parallel-Arm, Double-Blind, Placebo-Controlled Study with Open-Label Extension to Assess the Efficacy and Safety of Vatiquinone for the Treatment of Friedreich ataxia (MOVE-FA) - MOVE-FA | Friedreich Ataxia (FA) MedDRA version: 20.0;Level: PT;Classification code 10017374;Term: Friedreich's ataxia;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Product Name: Vatiquinone Product Code: [PTC743] INN or Proposed INN: Vatiquinone | PTC THERAPEUTICS INC. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 126 | Phase 2;Phase 3 | France;United States;Canada;Spain;Brazil;Australia;Germany;Italy | ||
| 5 | EUCTR2020-002812-36-FR (EUCTR) | 18/03/2021 | 15/01/2021 | A study to determine if Vatiquinone, the study drug, is safe and effective to treat a neurological condition called Friedreich ataxia | A Randomized, Parallel-Arm, Double-Blind, Placebo-Controlled Study with Open-Label Extension to Assess the Efficacy and Safety of Vatiquinone for the Treatment of Friedreich ataxia (MOVE-FA) - MOVE-FA | Friedreich Ataxia (FA) MedDRA version: 20.0;Level: PT;Classification code 10017374;Term: Friedreich's ataxia;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Product Name: Vatiquinone Product Code: PTC743 INN or Proposed INN: VATIQUINONE Other descriptive name: alpha-tocotrienolquinone | PTC Therapeutics, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 126 | Phase 2;Phase 3 | United States;France;Canada;Spain;Brazil;Australia;Germany;Italy;New Zealand | ||
| 6 | EUCTR2020-002812-36-DE (EUCTR) | 18/02/2021 | 06/11/2020 | A study to determine if Vatiquinone, the study drug, is safe and effective to treat a neurological condition called Friedreich ataxia | A Randomized, Parallel-Arm, Double-Blind, Placebo-Controlled Study with Open-Label Extension to Assess the Efficacy and Safety of Vatiquinone for the Treatment of Friedreich ataxia (MOVE-FA) - MOVE-FA | Friedreich Ataxia (FA) MedDRA version: 20.0;Level: PT;Classification code 10017374;Term: Friedreich's ataxia;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Product Name: Vatiquinone Product Code: PTC743 INN or Proposed INN: VATIQUINONE Other descriptive name: alpha-tocotrienolquinone | PTC Therapeutics, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 126 | Phase 2;Phase 3 | France;United States;Canada;Spain;Brazil;Australia;Germany;Italy;New Zealand | ||
| 7 | NCT04577352 (ClinicalTrials.gov) | December 17, 2020 | 30/9/2020 | A Study to Assess the Efficacy and Safety of Vatiquinone for the Treatment of Participants With Friedreich Ataxia | A Randomized, Parallel-Arm, Double-Blind, Placebo-Controlled Study With Open-Label Extension to Assess the Efficacy and Safety of Vatiquinone for the Treatment of Friedreich Ataxia (MOVE-FA) | Friedreich Ataxia | Drug: Vatiquinone;Drug: Placebo | PTC Therapeutics | NULL | Active, not recruiting | 7 Years | N/A | All | 146 | Phase 2/Phase 3 | United States;Australia;Brazil;Canada;France;Germany;Italy;New Zealand;Spain |