Clinical trials: Spinocerebellar Ataxias
Friedreich Ataxia
Micronised Resveratrol as a Treatment for Friedreich Ataxia
The aim of this study is to assess the efficacy of micronised resveratrol as a treatment for FRDA, in terms of reducing the severity of ataxia symptoms at 24 weeks, through a randomised blinded, placebo controlled crossover trial.
- Trial name
- NCT03933163
- Testing
- Resveratrol
- Where
- 4 sites in Australia
- Volunteers
- 25 people
- Sponsor
- Murdoch Childrens Research Institute
Spinocerebellar Ataxias
Stemchymal® for 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
- NCT06397274
- Testing
- Stemchymal
- Volunteers
- 20 people
- Sponsor
- Steminent US, Inc.
Ataxia, Spinocerebellar, Insulin-Like Growth Factor I
Chinese Medicine WT for Spinocerebellar Ataxia Type 3
Spinocerebellar ataxia type 3 (SCA3) is one of autosomal dominant hereditary ataxias. Standing imbalance, unsteady gait, dysmetria, fatigue, and depression would occur gradually.
- Conditions
- Ataxia, Spinocerebellar, Insulin-Like Growth Factor I, Medicine, Chinese Traditional
- Trial name
- NCT05038306
- Testing
- Chinese medicine WT
- Where
- Taiwan
- Volunteers
- 33 people
- Sponsor
- Changhua Christian Hospital
Friedreich's Ataxia
(+) Epicatechin to Treat Friedreich's Ataxia
This 24-week study will test the safety and effectiveness of synthetically produced (+) Epicatechin in treating patients who have Friedreich's Ataxia, a neurological disorder.
- Trial name
- NCT02660112
- Testing
- (+)-Epicatechin
- Where
- United States
- Volunteers
- 10 people
- Sponsor
- Ralitza Gavrilova
Ataxia-Telangiectasia
Study for Treatment of Cancer in Children With Ataxia-telangiectasia
This is a pilot/feasibility study designed to investigate the feasibility of treating children with Ataxia-Telangiectasia (A-T) and cancer with regimens nearly as intense as non-A-T patients with cancer would receive.
- Trial name
- NCT00187057
- Testing
- vinblastine, vincristine, prednisone, daunorubicin, doxorubicin, methotrexate, cyclophosphamide, L-asparaginase, etoposide, cytarabine, mercaptopurine
- Where
- United States
- Volunteers
- 6 people
- Sponsor
- St. Jude Children's Research Hospital
Ataxia Telangiectasia
Evaluate the Neurological Effects of EryDex on Subjects With A-T
This is an international, multi-center, randomized, prospective, double-blind, placebo-controlled, Phase 3 study, designed to assess the effect of EryDex (dexamethasone sodium phosphate [DSP] in autologous erythrocytes)…
- Trial name
- NEAT (NCT06193200)
- Testing
- Dexamethasone sodium phosphate
- Where
- 19 sites in United States, Denmark and 7 more
- Volunteers
- 105 people
- Sponsor
- Quince Therapeutics S.p.A.
Ataxia, Spinocerebellar, Ataxias, Hereditary
Functional Trunk Training in Ataxia Patients
The study is to examine the effect of functional trunk training on trunk control and upper extremity functions in patients with autosomal recessive ataxia.
- Trial name
- NCT04740359
- Testing
- Functional training, Routine training
- Where
- Turkey (Türkiye)
- Volunteers
- 20 people
- Sponsor
- Hasan Kalyoncu University
Friedreich Ataxia
A Study to Assess Variation in Potential Biomarkers in Friedreich Ataxia
To test the variability of specific ribonucleic acid (RNA) and proteins as well as frataxin levels in samples of blood and buccal cells taken directly from patients with Friedreich's ataxia (FRDA) in order to confirm potential…
- Trial name
- NCT04255680
- Testing
- Buccal Swabs and Blood Draws
- Where
- United States
- Volunteers
- 20 people
- Sponsor
- Larimar Therapeutics, Inc.
Friedreich's Ataxia
GV101 for Friedreich's Ataxia (FA) Treatment
This is a dose-ranging, randomized, double-blind, placebo-controlled 12-week Phase 2a trial with an up to two week loading dose period.
- Trial name
- NCT07870590
- Testing
- GV101
- Where
- United States
- Volunteers
- 48 people
- Sponsor
- Graviton Bioscience Corporation
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.
Spinocerebellar Ataxias
RISCA : Prospective Study of Individuals at Risk for SCA1, SCA2, SCA3, SCA6, SCA7
The spinocerebellar ataxias (SCAs) are a clinically and genetically heterogeneous group of autosomal dominantly inherited progressive ataxia disorders.
- Trial name
- RISCA (NCT01037777)
- Where
- France
- Volunteers
- 37 people
- Sponsor
- Institut National de la Santé Et de la Recherche Médicale, France
Inherited Movement Disorders, Spinocerebellar Ataxias
Biomarker Research in Inherited Movement Disorders
Inherited movement disorders are rare conditions, whose cumulative prevalence are in the order of 5-10/100,000 inhabitants, in most cases progressive and can lead to a significant loss of autonomy after one or more decades of…
- Conditions
- Inherited Movement Disorders, Spinocerebellar Ataxias, Hyperkinetic Disorders
- Trial name
- BIOMOV (NCT05034172)
- Testing
- Clinical follow-up
- Where
- France
- Volunteers
- 4,000 people, healthy volunteers welcome
- Sponsor
- Assistance Publique - Hôpitaux de Paris
Spinocerebellar Ataxia Type 3
Efficacy Of Oral Trehalose In Spinocerebellar Ataxia 3
This study evaluates the effectiveness of oral trehalose in alleviating the neuropathological and motor behaviour deficits among patients with SCA3.
- Trial name
- NCT04399265
- Testing
- Trehalose, Maltose
- Where
- Malaysia
- Volunteers
- 20 people
- Sponsor
- National University of Malaysia
Friedreich Ataxia
Multiple Ascending Dose Study of CTI-1601 Versus Placebo in Subjects With Friedreich's Ataxia
To evaluate the safety and tolerability of multiple ascending doses of CTI-1601 in participants with Friedreich's ataxia
- Trial name
- NCT04519567
- Testing
- CTI-1601
- Where
- United States
- Volunteers
- 27 people
- Sponsor
- Larimar Therapeutics, Inc.
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
Parkinson's Disease, Parkinsonian Disorders
Gait Analysis in Neurological Disease
The purpose of this study is to investigate whether speed-dependent measures of gait (so called gait signatures) can be identified in patients with neurological conditions that affect gait, particularly in subjects with…
- Conditions
- Parkinson's Disease, Parkinsonian Disorders, Atypical Parkinson Disease, Progressive Supranuclear Palsy
- Trial name
- NCT02994719
- Testing
- Anti-Parkinson medication, Deep Brain Stimulation
- Where
- United States
- Volunteers
- 120 people, healthy volunteers welcome
- Sponsor
- Beth Israel Deaconess Medical Center
Spinocerebellar Ataxias, Tai Chi
Could Tai-chi Help Maintain Balance of Spinocerebellar Ataxia Patients
Spinocerebellar atrophy is the most common autosomal dominant inherited ataxia. There are over thirty subtypes, which characterize neurologic features differently.
- Trial name
- NCT03687190
- Testing
- Tai chi, conventional medicine
- Volunteers
- 21 people
- Sponsor
- Changhua Christian Hospital
Ataxia Telangiectasia
Safety and Efficacy of Mutation-targeted Precision Genetic Therapy for Ataxia-Telangiectasia (A-T)
This project aims to evaluate the safety and efficacy of precision genetic therapy for patients with Ataxia-telangiectasia (A-T), a rare neurodegenerative disease caused by mutations in the ATM gene.
- Trial name
- NCT07215416
- Testing
- Antisense oligonucleotide targeting the ATM gene
- Where
- United States
- Volunteers
- 10 people
- Sponsor
- Timothy Yu
Friedreich Ataxia
Rosuvastatin (Crestor) in Friedreich Ataxia
This study is an exploratory open-label clinical trial of Rosuvastatin in patients with Friedreich ataxia (FRDA). This is an outpatient trial with the goal of enrolling 10 evaluable adults with genetically confirmed FRDA who are…
- Trial name
- NCT02705547
- Testing
- Rosuvastatin
- Where
- United States
- Volunteers
- 12 people
- Sponsor
- Children's Hospital of Philadelphia
SCA38
Docosahexaenoic Acid (DHA) Replacement for Treatment in Spinocerebellar Ataxia 38
The project will study a therapeutic approach in Spinocerebellar Ataxia (SCA38) by DHA replacement. SCA38 is caused by missense mutations in the ELOVL5 (Elongation of very long chain fatty acids protein 5) gene.
- Trial name
- SCA38DHA (NCT03109626)
- Testing
- DHA
- Where
- Italy
- Volunteers
- 10 people
- Sponsor
- Barbara Borroni