Clinical trials: Spinocerebellar Ataxia Type 3
CACNA1A, Spinocerebellar Ataxia Type 6
Pivotal Study of N-acetyl-L-leucine for CACNA1A
A pivotal, randomized, double-blind, placebo-controlled, multi-center therapeutic study for patients age 4 and older with a confirmed diagnosis of CACNA1A.
- Conditions
- CACNA1A, Spinocerebellar Ataxia Type 6, Episodic Ataxia Type 2, Familial Hemiplegic Migraine-1
- Trial name
- NCT07221292
- Testing
- N-Acetyl-L-Leucine
- Where
- 11 sites in United States, Austria and 5 more
- Volunteers
- 60 people
- Sponsor
- IntraBio Inc
Spinocerebellar Ataxia Type 1, Spinocerebellar Ataxia Type 2
Natural History Study of and Genetic Modifiers in Spinocerebellar Ataxias
Spinocerebellar ataxias (SCA) are genetic neurological diseases that cause imbalance, poor coordination, and speech difficulties.
- Conditions
- Spinocerebellar Ataxia Type 1, Spinocerebellar Ataxia Type 2, Spinocerebellar Ataxia Type 3, Spinocerebellar Ataxia Type 6
- Trial name
- NCT01060371
- Testing
- Genetic Testing, Blood Collection, Magnetic Resonance Imaging (MRI) Scan
- Where
- 17 sites in United States, Canada
- Volunteers
- 1,400 people, healthy volunteers welcome
- Sponsor
- Lauren Moore
Spinocerebellar Ataxia Type 1, Spinocerebellar Ataxia Type 2
Umbilical Cord Mesenchymal Stem Cells Therapy (19#iSCLife®-SA) for Patients With Spinocerebellar Ataxia
The purpose of this study is verify the safety and efficacy of Human Umbilical Cord Mesenchymal Stem Cells (UC-MSC) therapy for patients with Spinocerebellar Ataxia, and in addition, explore the possible mechanisms of UC-MSC…
- Conditions
- Spinocerebellar Ataxia Type 1, Spinocerebellar Ataxia Type 2, Spinocerebellar Ataxia Type 3, Spinocerebellar Ataxia Type 6
- Trial name
- NCT03378414
- Testing
- Intravenous infusion, Intrathecal injection, umbilical cord mesenchymal stem cell
- Volunteers
- 45 people
- Sponsor
- Sclnow Biotechnology Co., Ltd.
Rare Disorders, Undiagnosed Disorders
Rare Disease Patient Registry & Natural History Study - Coordination of Rare Diseases at Sanford
CoRDS, or the Coordination of Rare Diseases at Sanford, is based at Sanford Research in Sioux Falls, South Dakota. It provides researchers with a centralized, international patient registry for all rare diseases.
- Conditions
- Rare Disorders, Undiagnosed Disorders, Disorders of Unknown Prevalence, Cornelia De Lange Syndrome
- Trial name
- CoRDS (NCT01793168)
- Where
- 2 sites in United States, Australia
- Volunteers
- 20,000 people
- Sponsor
- Sanford Health
Spinocerebellar Ataxia Type 1, Spinocerebellar Ataxia Type 2
Instrumented Data Exchange for Ataxia Study
This research study is testing body-worn sensors to measure movement during simple tests of coordination, in order to evaluate the progression and severity of ataxia.
- Conditions
- Spinocerebellar Ataxia Type 1, Spinocerebellar Ataxia Type 2, Spinocerebellar Ataxia Type 3, Spinocerebellar Ataxia Type 6
- Trial name
- IDEA (NCT04268147)
- Where
- 5 sites in United States
- Volunteers
- 128 people, healthy volunteers welcome
- Sponsor
- University of Chicago
Spinocerebellar Ataxia Type 3, Machado Joseph Disease
Safety and Efficacy of Lithium Carbonate in Patients With Spinocerebellar Ataxia Type 3
Design: Phase II-III, double-blind, parallel, placebo controlled randomized Clinical trial Background: Spinocerebellar ataxia type 3 (SCA-3) is an autosomal dominant adult-onset neurodegenerative disorder for which there is no…
- Trial name
- NCT01096082
- Testing
- Lithium Carbonate
- Where
- Brazil
- Volunteers
- 62 people
- Sponsor
- Hospital de Clinicas de Porto Alegre
Spinocerebellar Ataxia Type 3
Pilot Study of Safety and Efficacy of Sodium Phenylbutyrate in Spinocerebellar Ataxia Type 3
DESIGN: Pilot, Phase II, double-blind, placebo-controlled study JUSTIFICATION: In the literature one does not find a pharmacological treatment that changes the natural history of Spinocerebellar ataxtia type 3 (SCA3).
- Trial name
- NCT01096095
- Testing
- Sodium Phenylbutyrate
- Where
- Brazil
- Sponsor
- Hospital de Clinicas de Porto Alegre