Clinical trials: Machado-Joseph Disease
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
Spinocerebellar Ataxias, Spinocerebellar Ataxia Type 1
Study of CAD-1883 for Spinocerebellar Ataxia
This is a randomized, double-blind, placebo-controlled Phase 2 study evaluating oral administration of CAD-1883 in the treatment of adults with a genotypic diagnosis of Spinocerebellar Ataxia (SCA).
- Conditions
- Spinocerebellar Ataxias, Spinocerebellar Ataxia Type 1, Spinocerebellar Ataxia Type 2, Spinocerebellar Ataxia Type 3
- Trial name
- Synchrony-1 (NCT04301284)
- Testing
- CAD-1883
- Where
- 6 sites in United States
- Sponsor
- Cadent Therapeutics
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.
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
Identification of Biomarkers in Spinocerebellar Ataxia 3
The purpose of this study is to examine the differences in cerebral spinal fluid (CSF) and blood of patients with spinocerebellar ataxias and healthy volunteers.
- Trial name
- SCA3 (NCT03885167)
- Testing
- Specimen Collection
- Where
- United States
- Volunteers
- 22 people, healthy volunteers welcome
- Sponsor
- University of Michigan
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
Spinocerebellar Ataxia Type 1, Spinocerebellar Ataxia Type 3
A Safety and Pharmacokinetics Trial of VO659 in SCA1, SCA3 and HD
The goal of this first-in-human clinical trial is to assess the safety and tolerability of four doses of a new study drug called VO659 in people with genetic disorders called spinocerebellar ataxia type 1, type 3 or Huntington's…
- Conditions
- Spinocerebellar Ataxia Type 1, Spinocerebellar Ataxia Type 3, Huntington Disease
- Trial name
- NCT05822908
- Testing
- VO659
- Where
- 14 sites in Denmark, France and 4 more
- Volunteers
- 68 people
- Sponsor
- Vico Therapeutics B. V.
Spinocerebellar Ataxia
Measuring Neurological Impairment and Functional Visual Assessment In Spinocerebellar Ataxias
Measuring the various difficulties patients with spinocerebellar ataxias (SCA) report in an accurate manner is important to be able to test any therapy that may be developed.
- Trial name
- NCT00654251
- Where
- 2 sites in United States
- Volunteers
- 20 people
- Sponsor
- University of Mississippi Medical Center
Neurodegenerative Disorders
Development of IPS from Donated Somatic Cells of Patients with Neurological Diseases
Human fibroblasts and possibly other human somatic cells may be reprogrammed into induced pluripotent stem (iPS) cells by the forced expression of transcription factors (1-5).
- Trial name
- NCT00874783
- Where
- Israel
- Volunteers
- 120 people, healthy volunteers welcome
- Sponsor
- Hadassah Medical Organization
Machado-Joseph Disease
Weight in Lower Limbs Improves Gait Ataxia of in Machado-Joseph Disease Patients
Few studies evaluated the efficacy of adding weights on the lower limbs in patients with ataxic disorders. There is no current evidence on which would be the most appropriate weight for use in this context.
- Trial name
- NCT02906046
- Testing
- Weight in Lower Limbs
- Volunteers
- 20 people
- Sponsor
- Federal University of Health Science of Porto Alegre
Ataxia, Spinocerebellar Ataxias
Cerebello-Spinal tDCS as Rehabilitative Intervention in Neurodegenerative Ataxia
Neurodegenerative cerebellar ataxias represent a group of disabling disorders which currently lack effective therapies. Cerebellar transcranial direct current stimulation (tDCS) is a non-invasive technique, which has been…
- Conditions
- Ataxia, Spinocerebellar Ataxias, Spinocerebellar Ataxia Type 1, Spinocerebellar Ataxia Type 2
- Trial name
- SCA02 (NCT04153110)
- Testing
- Anodal cerebellar and cathodal spinal tDCS
- Where
- Italy
- Volunteers
- 61 people
- Sponsor
- Azienda Socio Sanitaria Territoriale degli Spedali Civili di Brescia
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 Ataxias, Spinocerebellar Ataxia Type 1
Open Pilot Trial of BHV-4157
24 adults, between the ages of 18 and 75 years, with cerebellar ataxia will be enrolled in a 12 week trial of BHV-4157 for treatment of ataxia.
- Conditions
- Spinocerebellar Ataxias, Spinocerebellar Ataxia Type 1, Spinocerebellar Ataxia Type 2, Spinocerebellar Ataxia 3
- Trial name
- NCT03408080
- Testing
- BHV-4157
- Where
- United States
- Volunteers
- 24 people
- Sponsor
- University of California, Los Angeles
Spinocerebellar Ataxia Type 3
Repetitive Transcranial Magnetic Stimulation in SCA3 Patients
Machado-Joseph Disease (MJD) or spinocerebellar ataxia type 3 (SCA3) is the most common spinocerebellar ataxia worldwide.Repetitive transcranial magnetic stimulation (rTMS) is a form of brain stimulation therapy used to treat…
- Trial name
- NCT05502432
- Testing
- Active repetitive transcranial magnetic stimulation
- Where
- China
- Volunteers
- 39 people
- Sponsor
- Ning Wang, MD., PhD.
Spinocerebellar Ataxia Type 3, Machado-Joseph Disease
Natural History of Oculomotor Neurophysiology in Ataxic and Pre-ataxic Carriers of SCA3/MJD
The study will consist of a prospective observation of subjects in a natural history design. Disease progression will be monitored through clinical scales and video-oculography.
- Trial name
- BIGPRO (NCT04229823)
- Testing
- Video-oculography, Clinical Scales, Genotyping
- Where
- Brazil
- Volunteers
- 95 people, healthy volunteers welcome
- Sponsor
- Hospital de Clinicas de Porto Alegre
Spinocerebellar Ataxia Type 1, Spinocerebellar Ataxia 3
Clinical Trial Readiness for SCA1 and SCA3
The investigators plan to fill the gap between the current state of clinical trial readiness and the optimal one for SCA1 and SCA3, which are fatal rare diseases with no treatments.
- Trial name
- READISCA (NCT03487367)
- Where
- United States
- Volunteers
- 200 people, healthy volunteers welcome
- Sponsor
- The Methodist Hospital Research Institute
Ataxia, Cerebellar, Cerebellar Ataxia
Rehabilitative Trial With Cerebello-Spinal tDCS in Neurodegenerative Ataxia
Neurodegenerative cerebellar ataxias represent a group of disabling disorders for which we currently lack effective therapies. Cerebellar transcranial direct current stimulation (tDCS) is a non-invasive technique, which has been…
- Conditions
- Ataxia, Cerebellar, Cerebellar Ataxia, Spinocerebellar Ataxias, Ataxia, Spinocerebellar
- Trial name
- CStDCSAtaxia (NCT03120013)
- Testing
- Anodal cerebellar and cathodal spinal tDCS
- Where
- Italy
- Volunteers
- 21 people
- Sponsor
- Azienda Socio Sanitaria Territoriale degli Spedali Civili di Brescia
Machado-Joseph Disease / Spinocerebellar Ataxia 3
Study To Assess Safety, Tolerability and Efficacy of Intravenous Cabaletta in Patients With Machado-Joseph Disease
This is an exploratory, randomized, parallel-group, dose escalation and dose-controlled study without a placebo arm. Eligible patients will be randomized in a 1:1 ratio (double-blind) to receive Cabaletta in 2 doses, once weekly…
- Trial name
- NCT02147886
- Testing
- Cabaletta for IV infusion once weekly during 24 weeks
- Where
- Israel
- Volunteers
- 15 people
- Sponsor
- Bioblast Pharma Ltd.
Spinocerebellar Ataxia Type 3, Machado-Joseph Disease
Astrocytic Markers and the Pre-ataxic Period of SCA3/MJD - BIGPRO Study Astrocytes
The study will consist of a prospective observation of subjects in a natural history design. The investigators will monitor changes of clinical scales, quality of life, messenger ribonucleic acid (mRNA) of candidate genes (CCL11…
- Conditions
- Spinocerebellar Ataxia Type 3, Machado-Joseph Disease, SCA3, MJD
- Trial name
- NCT04419974
- Testing
- Molecular Diagnosis, Clinical Scales - Baseline, Blood Draw - Baseline
- Where
- Brazil
- Volunteers
- 95 people, healthy volunteers welcome
- Sponsor
- Hospital de Clinicas de Porto Alegre
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.