Clinical trials: Cerebellar Ataxia
Friedreich Ataxia
A Study of Vatiquinone for the Treatment of Participants With Friedreich Ataxia
The primary objective of the study is to assess the pharmacokinetics (PK) and safety of vatiquinone administered in participants with Friedreich ataxia (FA) younger than 7 years.
- Trial name
- NCT05485987
- Testing
- Vatiquinone
- Where
- United States
- Volunteers
- 5 people
- Sponsor
- PTC Therapeutics
Spinocerebellar Ataxias, Spinocerebellar Ataxia Genotype Type 1
Trial in Adult Participants With Spinocerebellar Ataxia (SCA)
The primary purpose of this study was to compare the efficacy of BHV-4157 (Troriluzole) 140 milligrams (mg) once daily versus placebo after 8 weeks of treatment in participants with spinocerebellar ataxia (SCA).
- Conditions
- Spinocerebellar Ataxias, Spinocerebellar Ataxia Genotype Type 1, Spinocerebellar Ataxia Genotype Type 2, Spinocerebellar Ataxia Genotype Type 3
- Trial name
- NCT02960893
- Testing
- Troriluzole
- Where
- 18 sites in United States
- Volunteers
- 141 people
- Sponsor
- Biohaven Pharmaceuticals, Inc.
Pain
Evaluation of the Tolerability of Switching Subjects on Chronic ATC Opioid Therapy to Buprenorphine HCl Buccal Film
The primary aim of this study is to determine if chronic pain subjects on around-the-clock opioids who are receiving 100 to 220 mg oral morphine sulfate equivalent (MSE) can be safely transitioned on to buprenorphine…
- Trial name
- NCT01871285
- Testing
- Buprenorphine, Oxycodone, Morphine sulfate
- Where
- 3 sites in United States
- Volunteers
- 39 people
- Sponsor
- BioDelivery Sciences International
SCA, Spinocerebellar Ataxias
Expanded Access Protocol of Troriluzole in Patients With Spinocerebellar Ataxia (SCA)
The purpose of this expanded access protocol is to provide access to the investigational drug troriluzole in patients with spinocerebellar ataxia (SCA).
- Trial name
- NCT06034886
- Testing
- Troriluzole
- Sponsor
- Biohaven Pharmaceuticals, Inc.
Friedreich Ataxia
Efficacy, Tolerability, and Pharmacokinetics of Multiple Doses of Oral TAK-831 in Adults With Friedreich Ataxia
The purpose of this study is to evaluate the efficacy of TAK-831 versus placebo on upper extremity (arm and hands) motor function and manual dexterity.
- Trial name
- NCT03214588
- Testing
- TAK-831
- Where
- 6 sites in United States
- Volunteers
- 67 people
- Sponsor
- Neurocrine Biosciences
Myoclonus Epilepsies, Progressive
Safety, Blood Levels and Effects of AUT00201 in Patients With MEAK
A randomized, double-blind, placebo-controlled, crossover study to assess the safety, tolerability, and pharmacokinetics of single doses of AUT00201 at 100 mg or matching placebo in patients with myoclonus epilepsy and ataxia due…
- Trial name
- AUT022201 (NCT05873062)
- Testing
- AUT00201
- Where
- United States
- Volunteers
- 6 people
- Sponsor
- Autifony Therapeutics Limited
Spinocerebellar Degeneration
C-Trelin Orally Disintegrated(OD) Tablet 5mg in Ataxia Due to Spinocerebellar Degeneration
The aim of this study is to evaluate the efficacy and safety of C-Trelin OD Tablet 5mg(Taltirelin Hydrate) in Multicenter, randomized, double-blind, placebo-controlled clinical trial in patients with ataxia induced by…
- Trial name
- NCT04107740
- Testing
- C-Trelin OD Tab(5mg Taltirelin Hydrate)
- Where
- 8 sites in South Korea
- Volunteers
- 160 people
- Sponsor
- HLB Pharmaceutical Co., Ltd.
Friedreich Ataxia, Cardiomyopathies
A Study to Investigate the Safety, Tolerability, and Pharmacokinetics of ATI-105 in Healthy Adults
The goal of this clinical trial is to learn how ATI-105 administered to healthy participants at works in the body at different doses and with or without food.
- Conditions
- Friedreich Ataxia, Cardiomyopathies, Central Nervous System Diseases, Neuromuscular Diseases
- Trial name
- NCT07869654
- Testing
- ATI-105
- Where
- United States
- Volunteers
- 60 people, healthy volunteers welcome
- Sponsor
- Adjuvia Therapeutics, Inc.
Spinocerebellar Ataxias, Spinocerebellar Ataxia Type 1
Troriluzole in Adult Participants With Spinocerebellar Ataxia
The purpose of this study is to compare the efficacy of Troriluzole (200 mg once daily) versus placebo after 48 weeks of treatment in subjects with spinocerebellar ataxia (SCA).
- Conditions
- Spinocerebellar Ataxias, Spinocerebellar Ataxia Type 1, Spinocerebellar Ataxia Type 2, Spinocerebellar Ataxia Type 3
- Trial name
- NCT03701399
- Testing
- troriluzole
- Where
- 23 sites in United States, China
- Volunteers
- 299 people
- Sponsor
- Biohaven Pharmaceuticals, Inc.
Hereditary Ataxia
Safety and Efficacy of Umbilical Cord Mesenchymal Stem Cell Therapy for Patients With Hereditary Ataxia
The Hereditary Ataxias are a group of genetic disorders characterized by slowly progressive incoordination of gait and often associated with poor coordination of hands, speech, and eye movements.
- Trial name
- NCT01360164
- Testing
- human umbilical cord mesenchymal stem cells
- Where
- China
- Volunteers
- 20 people
- Sponsor
- Shenzhen Beike Bio-Technology Co., Ltd.
Friedreich Ataxia, Cardiomyopathy, Secondary
Study of LX2006 Gene Therapy in Friedreich Ataxia Cardiomyopathy
The purpose of Study LX2006-03, a multicenter, Phase 2, open-label, randomized, controlled study, is to evaluate the efficacy and safety of LX2006 gene therapy in participants with Friedreich ataxia (FA) cardiomyopathy (CM).
- Trial name
- SUNRISE-FA 2 (NCT07721025)
- Testing
- LX2006, Usual Care
- Where
- United States
- Volunteers
- 26 people
- Sponsor
- Lexeo Therapeutics
Phosphomannomutase 2 Deficiency, PMM2-CDG
A Study to Assess the Efficacy and Safety of Weekly Doses of GLM101 in Participants With PMM2-CDG
This study is evaluating the safety, effectiveness, and how the body absorbs, distributes, and eliminates GLM101, for participants with PMM2-CDG, including children, adolescents, and adults.
- Trial name
- NCT06892288
- Testing
- GLM101 (Part A, Double-blind), GLM101 (Part B, Open-label)
- Where
- 15 sites in United States, Belgium and 8 more
- Volunteers
- 50 people
- Sponsor
- Glycomine, Inc.
Friedreich Ataxia
Single Ascending Dose Study of CTI-1601 Versus Placebo in Subjects With Friedreich's Ataxia
To evaluate the safety and tolerability of single ascending doses of CTI-1601 in participants with Friedreich's ataxia
- Trial name
- NCT04176991
- Testing
- CTI-1601
- Where
- United States
- Volunteers
- 28 people
- Sponsor
- Larimar Therapeutics, Inc.
Ataxia
Special Drug Use Investigation (Retrospective) for Arixtra® (Fondaparinux) Venous Thromboembolism Treatment (Over 100kg)
The purpose of this post-marketing surveillance study is to collect and assess information retrospectively on safety and effectiveness of fondaparinux injection in patients with venous thromboembolism (VTE) whose body weight is…
- Trial name
- NCT01428544
- Testing
- Fondaparinux sodium
- Volunteers
- 5 people
- Sponsor
- GlaxoSmithKline
Advanced Cancer, Metastatic Cancer
A Study of ART0380 for the Treatment of Advanced or Metastatic Solid Tumors
This clinical trial is evaluating a drug called ART0380 in participants with advanced or metastatic solid tumors. The main goals of this study are to: Find the recommended dose of ART0380 that can be given safely to participants…
- Conditions
- Advanced Cancer, Metastatic Cancer, Ovarian Cancer, Primary Peritoneal Cancer
- Trial name
- NCT04657068
- Testing
- ART0380, Gemcitabine, Irinotecan
- Where
- 88 sites in United States, France and 2 more
- Volunteers
- 542 people
- Sponsor
- Artios Pharma Ltd
Ataxia Telangiectasia
Comparative Bioavailability of Betamethasone Oral Solution Metered Spray (GTX-102) in Healthy Subjects
A Randomized, Open-label, Crossover Study to Evaluate the Comparative Bioavailability, Pharmacokinetics, and Safety of GTX-102 Administered as an Oral Spray Compared to Intramuscular Injection - betamethasone and an Oral Solution…
- Trial name
- NCT05531890
- Testing
- GTX-102 medium dose fast Period 1 and Period 2, GTX-102 medium dose slow Period 1 and Period 2, GTX-102 high dose fast Period 1 and Period 2
- Where
- Canada
- Volunteers
- 48 people, healthy volunteers welcome
- Sponsor
- Grace Therapeutics Inc.
Rett Syndrome
A Safety Study of NNZ-2566 in Patients With Rett Syndrome
The purpose of this study is to determine whether NNZ-2566 is safe and well tolerated in the treatment of Rett Syndrome in adolescent and adult females.
- Trial name
- NCT01703533
- Testing
- NNZ-2566
- Where
- 3 sites in United States
- Volunteers
- 67 people
- Sponsor
- Neuren Pharmaceuticals Limited
Friedreich's Ataxia
Safety and Efficacy of EPI-743 in Patients With Friedreich's Ataxia
The purpose of this study is to examine the effects of EPI-743 on visual function and neurologic function in patients with Friedreich's ataxia.
- Trial name
- NCT01728064
- Testing
- EPI-743 400 mg, EPI-743 200 mg
- Where
- 3 sites in United States
- Volunteers
- 60 people
- Sponsor
- PTC Therapeutics
Friedreich's Ataxia
Study to Assess the Safety and Tolerability of Idebenone in the Treatment of Friedreich's Ataxia Patients
This study is meant to assess the safety and tolerability of idebenone in patients with Friedreich's Ataxia over a 12 months period.
- Trial name
- IONIA-E (NCT00697073)
- Testing
- Idebenone
- Where
- 2 sites in United States
- Volunteers
- 68 people
- Sponsor
- Santhera Pharmaceuticals
Friedreich Ataxia
A Single Ascending Dose Study of DT-216P2 in Normal Healthy Participants
This purpose of the study is to evaluate the safety, tolerability and pharmacokinetics of single ascending doses of DT-216P2 administered either subcutaneously (SC) and intravenously (IV) in normal healthy participants.
- Trial name
- NCT06772870
- Testing
- DT-216P2, Saline
- Where
- Australia
- Volunteers
- 36 people, healthy volunteers welcome
- Sponsor
- Design Therapeutics, Inc.