Clinical trials: Spinocerebellar Ataxias Type 1
Inclusion Body Myositis, Myotonic Dystrophy 1
The Use of Assistive Gait Devices Can Reduce the Risk of Falls in Patients With Neuromuscular Diseases Following a Training Period.
The planned project is an intervention study to assess the risk of falling after adaptation of an assistive gait devices in patients with the following neuromuscular diseases: Inclusion body myositis, myotonic dystrophy, limb…
- Conditions
- Inclusion Body Myositis, Myotonic Dystrophy 1, Myotonic Dystrophy 2, Facio-Scapulo-Humeral Dystrophy
- Trial name
- Risk of fall (NCT07072676)
- Testing
- Assistive gait devices combined with physiotherapy
- Where
- Germany
- Volunteers
- 30 people
- Sponsor
- LMU Klinikum
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.
Spinocerebellar Ataxia Type 3
A Pharmacokinetics and Safety Study of BIIB132 in Adults With Spinocerebellar Ataxia 3
The primary objective of this study is to evaluate the safety and tolerability of multiple ascending doses of BIIB132 administered via intrathecal (IT) injection to participants with spinocerebellar ataxia type 3 (SCA3).
- Trial name
- NCT05160558
- Testing
- BIIB132
- Where
- 20 sites in United States, Germany and 4 more
- Volunteers
- 8 people
- Sponsor
- Biogen
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
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
Solid Tumors
A Safety, Pharmacokinetic and Pharmacodynamic Study of Kevetrin in Patients With Advanced Solid Tumors
In the laboratory, Kevetrin activates p53, a tumor suppressor protein that has an important role in protecting the body. p53 functions by activating proteins that repair DNA and kill cells that have genetic mutations such as in…
- Trial name
- NCT01664000
- Testing
- thioureidobutyronitrile
- Where
- United States
- Volunteers
- 48 people
- Sponsor
- Cellceutix Corporation
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 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
Spinocerebellar Ataxias Type 1, Spinocerebellar Ataxias Type 2
Dalfampridine and Gait in Spinocerebellar Ataxias
Investigators expect there will be improvement in walking speed and steadiness after taking Dalfampridine, thereby improving activities of daily living and enhancing social and occupational functions for patients with…
- Conditions
- Spinocerebellar Ataxias Type 1, Spinocerebellar Ataxias Type 2, Spinocerebellar Ataxias Type 3, Spinocerebellar Ataxias Type 6
- Trial name
- NCT01811706
- Testing
- Dalfampridine
- Where
- United States
- Volunteers
- 20 people
- Sponsor
- University of Florida
Spinocerebellar Ataxia Genotype Type 1, Spinocerebellar Ataxia Genotype Type 3
Cerebellar DBS in SCA1 and SCA3 Study
The purpose of this study is to evaluate the safety and feasibility of deep brain stimulation (DBS) in the deep cerebellar nuclei (DCN) in patients with Spinocerebellar Ataxia (SCA) types 1 and 3.
- Trial name
- NCT07752095
- Testing
- UG3NS140720 Deep Brain Stimulation System
- Where
- 2 sites in United States
- Volunteers
- 12 people
- Sponsor
- Gordon H. Baltuch
Non Invasive Prenatal Diagnosis
NIPD on CFTC for Triplet Repeat Diseases
The purpose of this study is to develop and validate an analytical and clinical NIPD test for triplet repeat diseases by isolated circulating fetal trophoblastic cells (CFTC) analysis from maternal blood, searching for the…
- Trial name
- DIACCIMEX (NCT03087526)
- Testing
- Non invasive prenatal diagnosis
- Where
- 8 sites in France
- Volunteers
- 60 people, healthy volunteers welcome
- Sponsor
- University Hospital, Montpellier
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 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
Neuromuscular Diseases, Obesity (Disorder)
Gait Analysis Parameter and Upper Limb Evaluation in Adult Patients With Neurological or Metabolic Pathology
The ActiLiège-Adult study is a prospective, longitudinal, observational study designed to collect natural history data on adult patients with neurological or metabolic diseases affecting movement.
- Conditions
- Neuromuscular Diseases, Obesity (Disorder), Myotonic Dystrophy 1, Myasthenic Syndrome
- Trial name
- Acti-Adult (NCT07136844)
- Testing
- Syde, Dynamometric measurements of muscle strength, The 6-minute walk test (6MWT)
- Where
- Belgium
- Volunteers
- 300 people
- Sponsor
- Centre Hospitalier Universitaire de Liege
Spinocerebellar Ataxia Type I
Lithium Treatment for Patients With Spinocerebellar Ataxia Type I
This study will evaluate the side effects and tolerability of the drug lithium in patients with spinocerebellar ataxia type I (SCA1) an inherited disorder caused by loss of nerve cells in parts of the brain.
- Trial name
- NCT00683943
- Testing
- Lithium Carbonate
- Where
- United States
- Volunteers
- 13 people
- Sponsor
- National Institute of Neurological Disorders and Stroke (NINDS)
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 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 1, Spinocerebellar Ataxia Type 2
Biomarkers in Autosomal Dominant Cerebellar Ataxia
Autosomal dominant cerebellar ataxias (ADCA) are a group of neurodegenerative disorders that are clinically and genetically various.
- Conditions
- Spinocerebellar Ataxia Type 1, Spinocerebellar Ataxia Type 2, Spinocerebellar Ataxia, Autosomal Recessive 3, Episodic Ataxia, Type 7
- Trial name
- BIOSCA (NCT01470729)
- Testing
- metabolic and imaging biomarkers in SCA1,2,3 and 7 patients
- Where
- France
- Volunteers
- 102 people, healthy volunteers welcome
- Sponsor
- Assistance Publique - Hôpitaux de Paris
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.
Spinocerebellar Ataxia Type 2
Study of ARO-ATXN2 Injection in Adults With Spinocerebellar Ataxia Type 2
Adult participants with spinocerebellar ataxia type 2 (SCA2) who carry ≥33 cytosine, adenine, guanine (CAG) repeats in the ATXN2 gene, and who have met all protocol eligibility criteria will be randomized to receive a single dose…
- Trial name
- NCT06672445
- Testing
- ARO-ATXN2 Injection
- Where
- 16 sites in Australia, Canada and 6 more
- Volunteers
- 39 people
- Sponsor
- Arrowhead Pharmaceuticals