Clinical trials: Spino Cerebellar Degeneration
Friedreich Ataxia
A Multiple Ascending Dose Study of DT-216P2 in Patients With Friedreich's Ataxia
The purpose of this study is to evaluate the safety and tolerability, and pharmacokinetics of DT-216P2 administered multiple ascending doses in patients with FA.
- Trial name
- NCT06874010
- Testing
- DT-216P2
- Where
- 3 sites in Australia
- Volunteers
- 20 people
- Sponsor
- Design Therapeutics, Inc.
Spinocerebellar Degeneration
A Confirmatory Study of KPS-0373 in Patients With Spinocerebellar Degeneration (SCD)
The purpose of this study is to investigate the superiority of KPS-0373 to placebo, and evaluate the safety and pharmacokinetics of KPS-0373 in SCD patients.
- Trial name
- NCT01970098
- Testing
- KPS-0373, High dose, KPS-0373, Low dose
- Where
- Japan
- Volunteers
- 374 people
- Sponsor
- Kissei Pharmaceutical Co., Ltd.
Friedreich Ataxia
A Study to Learn More About the Long-Term Safety of BIIB141 (Omaveloxolone) in Participants With Friedreich's Ataxia Who Are Prescribed it by Their Own Doctors
In this study, researchers will learn more about the safety of BIIB141, also known as omaveloxolone or SKYCLARYS®. This is a drug available for doctors to prescribe for people with Friedreich's Ataxia, also known as FA.
- Trial name
- NCT06623890
- Testing
- Omaveloxolone
- Where
- 14 sites in United States, Austria and 5 more
- Volunteers
- 300 people
- Sponsor
- Biogen
Friedreich's Ataxia
Long-Term Efficacy Study of Vatiquinone for the Treatment of Friedreich's Ataxia (FA)
The purpose of this study is to confirm the treatment effects of vatiquinone on the key measures of FA disease progression.
- Trial name
- PROVE-FA (NCT07681713)
- Testing
- Vatiquinone
- Where
- 9 sites in United States, Belgium and 4 more
- Volunteers
- 120 people
- Sponsor
- PTC Therapeutics
Spinocerebellar Degeneration
A Long-Term Study of KPS-0373 in Patients With Spinocerebellar Degeneration (SCD)
The purpose of this study is to evaluate the long-term safety, efficacy, and pharmacokinetics of KPS-0373 in SCD patients
- Trial name
- NCT01970124
- Testing
- KPS-0373, High dose, KPS-0373, Low dose
- Where
- Japan
- Sponsor
- Kissei Pharmaceutical Co., Ltd.
Friedreich's Ataxia
Patient Reported Outcomes in Friedreich's Ataxia Patients After Withdrawal From Treatment With Idebenone (PROTI)
This is a Phase IIIb Double-Blind, Randomised, Placebo-Controlled Study. The aim is to further investigate the effects of idebenone in patients with Friedreich's ataxia.
- Trial name
- PROTI (NCT01303406)
- Testing
- Idebenone
- Where
- 6 sites in Austria, Germany and 2 more
- Volunteers
- 29 people
- Sponsor
- Santhera Pharmaceuticals
Spinocerebellar Ataxia Type 3
STRIDES - a Clinical Research Study of an Investigational New Drug to Treat Spinocerebellar Ataxia
Phase 2b/3 double blind, randomized, placebo-controlled trial to assess safety and efficacy of SLS-005 (trehalose injection, 90.5 mg/mL for intravenous infusion) for the treatment of adults with spinocerebellar ataxia).
- Trial name
- STRIDES (NCT05490563)
- Testing
- SLS-005
- Where
- 23 sites in United States, Australia and 6 more
- Volunteers
- 23 people
- Sponsor
- Seelos Therapeutics, Inc.
Spinocerebellar Degeneration
Confirmatory Study 3 of KPS-0373 in Patients With Spinocerebellar Degeneration
This is a multicenter, randomized, double-blind, placebo-controlled, parallel-group, phase III study to evaluate the efficacy and safety of KPS-0373 given once daily after breakfast in 142 patients with spinocerebellar…
- Trial name
- NCT07040137
- Testing
- KPS-0373
- Where
- Japan
- Volunteers
- 142 people
- Sponsor
- Kissei Pharmaceutical Co., Ltd.
Friedreich Ataxia
A Clinical Study to Evaluate the Effect of MIN-102 on the Progression of Friedreich's Ataxia in Male and Female Patients
Randomized, double-blind, placebo-controlled study on the effects of MIN-102 on Biochemical, Imaging, neurophysiological, and clinical markers in patients with Friedreich's Ataxia
- Trial name
- FRAMES (NCT03917225)
- Testing
- MIN-102
- Where
- 5 sites in Belgium, France and 2 more
- Volunteers
- 39 people
- Sponsor
- Minoryx Therapeutics, S.L.
Friedreich Ataxia
A Study to Assess the Efficacy and Safety of Vatiquinone for the Treatment of Participants With Friedreich Ataxia
The primary objective of the study is to evaluate the efficacy (using the modified Friedreich Ataxia Rating Scale [mFARS]) and safety of vatiquinone in participants with Friedreich ataxia (FA).
- Trial name
- MOVE-FA (NCT04577352)
- Testing
- Vatiquinone
- Where
- 14 sites in United States, Australia and 7 more
- Volunteers
- 146 people
- Sponsor
- PTC Therapeutics
Friedreich Ataxia, Cardiomyopathy, Secondary
Gene Therapy for Cardiomyopathy Associated With Friedreich's Ataxia
This is a Phase 1/2, open-label, dose-ascending, multicenter study of the safety and efficacy of LX2006 for participants who have Friedreich's Ataxia with evidence of cardiomyopathy.
- Trial name
- NCT05445323
- Testing
- Low dose LX2006, Mid Dose LX2006, High Dose LX2006
- Where
- 3 sites in United States
- Volunteers
- 8 people
- Sponsor
- Lexeo Therapeutics
Friedreich Ataxia
An Open-Label Study of CTI-1601 in Subjects With Friedreich's Ataxia
An open label study designed to evaluate the safety, PK, PD, and clinical effects of long-term daily administration of CTI-1601 enrolling adolescent and adult patients with FRDA who have participated in a prior clinical study of…
- Trial name
- NCT06447025
- Testing
- CTI-1601
- Where
- 10 sites in United States
- Volunteers
- 85 people
- Sponsor
- Larimar Therapeutics, Inc.
SCD
A Phase II Double Blind Comparative Study of KPS-0373 in Patients With Spinocerebellar Degeneration (SCD)
The purpose of this study is to evaluate the efficacy and safety of KPS-0373 compared to placebo in patients with Spinocerebellar Degeneration (SCD).
- Trial name
- NCT01384435
- Testing
- KPS-0373
- Where
- Japan
- Volunteers
- 200 people
- Sponsor
- Kissei Pharmaceutical Co., Ltd.
Cerebellar Ataxia
Efficacy and Safety Study of Stemchymal® in Polyglutamine Spinocerebellar Ataxia
The purpose of the clinical trial is to study the therapeutic efficacy and safety of Stemchymal® infusions for polyglutamine spinocerebellar ataxia treatment by a randomized, double-blind, placebo-controlled study design.
- Trial name
- NCT02540655
- Testing
- Stemchymal®, Excipients
- Where
- Taiwan
- Volunteers
- 56 people
- Sponsor
- Steminent Biotherapeutics Inc.
Mitochondrial Diseases
EPI-743 for Mitochondrial Respiratory Chain Diseases
This study will evaluate the safety and efficacy of EPI-743 in participants with severe mitochondrial respiratory chain diseases who are considered to be within 90 days of end-of-life care.
- Trial name
- NCT01370447
- Testing
- EPI-743
- Where
- 16 sites in United States
- Sponsor
- PTC Therapeutics
Friedreich's Ataxia
A Study of Efficacy, Safety and Tolerability of Idebenone in the Treatment of Friedreich's Ataxia (FRDA) Patients
The purpose of this trial is to study the efficacy, safety and tolerability of idebenone in 12 months of treatment in children and adults with Friedreich's Ataxia.
- Trial name
- MICONOS (NCT00905268)
- Testing
- idebenone
- Where
- 13 sites in Austria, Belgium and 4 more
- Volunteers
- 232 people
- Sponsor
- Santhera Pharmaceuticals
Symptomatic Neurogenic Orthostatic Hypotension (NOH), Parkinson's Disease
Fipamezole in Neurogenic Orthostatic Hypotension
The purpose of this study is to determine whether Fipamezole is effective in the treatment of orthostatic hypotension and related symptoms in multiple system atrophy and Parkinson's disease.
- Conditions
- Symptomatic Neurogenic Orthostatic Hypotension (NOH), Parkinson's Disease, Multiple System Atrophy
- Trial name
- Foehn (NCT00758849)
- Testing
- Fipamezole
- Where
- 4 sites in France, Portugal
- Volunteers
- 24 people
- Sponsor
- Juvantia Pharma Ltd
Friedreich's Ataxia
Safety, Tolerability and Efficacy of ACTIMMUNE® Dose Escalation in Friedreich's Ataxia
The purpose of this phase 3 randomized, multi-center, double-blind, placebo-controlled study is to evaluate the efficacy and safety of ACTIMMUNE® (interferon-γ 1b) in the treatment of Friedreich's Ataxia (FA) and to evaluate the…
- Trial name
- STEADFAST (NCT02415127)
- Testing
- Interferon γ-1b
- Where
- 4 sites in United States
- Volunteers
- 92 people
- Sponsor
- Amgen
Friedreich's Ataxia
Safety and Efficacy Study of A0001 in Subjects With Friedreich's Ataxia
This is a Phase 2a double-blind, placebo-controlled study with two dose levels of A0001 given twice daily for 28 days. Potential subjects will be screened first to determine eligibility, after which they will be randomized to…
- Trial name
- NCT01035671
- Testing
- alpha-tocopherolquinone (A0001)
- Where
- United States
- Volunteers
- 42 people
- Sponsor
- Penwest Pharmaceuticals Co.
Friedreich Ataxia, Friedreich's Ataxia
A Study to Evaluate the Efficacy and Safety of Subcutaneous Nomlabofusp in Subjects With Friedreich's Ataxia
To evaluate the efficacy and safety of subcutaneous nomlabofusp in adult and pediatric subjects with Friedreich's ataxia
- Trial name
- FORWARD-FA (NCT07778836)
- Testing
- Nomlabofusp
- Where
- 4 sites in United States
- Volunteers
- 150 people
- Sponsor
- Larimar Therapeutics, Inc.