Clinical trials: Spinocerebellar Ataxia
Spinocerebellar Ataxia Type 3
Study to Determine the Safety and Tolerability of Varenicline (Chantix®) in Treating Spinocerebellar Ataxia Type 3
Spinocerebellar ataxia (SCA) is a group of inherited disorders characterized by cerebellar degeneration leading to imbalance, incoordination, speech difficulties and problems with walking.
- Trial name
- NCT00992771
- Testing
- varenicline
- Where
- 3 sites in United States
- Volunteers
- 20 people
- Sponsor
- University of South Florida
Spinocerebellar Ataxia Type 2
Clinical Trial With Riluzole in Spinocerebellar Ataxia Type 2 (ATRIL)
ATRIL is a multi-centric, double-blind randomized, two-arm controlled study. 42 SpinoCerebellar Ataxia type 2 (SCA2) patients, both gender, at least 18 years of age will be included.
- Trial name
- ATRIL (NCT03347344)
- Testing
- Riluzole
- Where
- France
- Volunteers
- 42 people
- Sponsor
- Assistance Publique - Hôpitaux de Paris
Friedreich Ataxia
A Study to Assess the Safety and Efficacy of Vatiquinone in Participants With Friedreich Ataxia
The primary objective of this study is to assess the long-term safety of vatiquinone in participants with Friedreich ataxia (FA) previously exposed to vatiquinone.
- Trial name
- NCT05515536
- Testing
- Vatiquinone
- Where
- 14 sites in United States, Australia and 7 more
- Volunteers
- 130 people
- Sponsor
- PTC Therapeutics
Friedreich Ataxia
FRDA Investigator Initiated Study (IIS) With Elamipretide
To evaluate the safety, tolerability, and activity of Elamipretide in treating vision loss in Friedreich Ataxia (FRDA).
- Trial name
- ELViS-FA (NCT05168774)
- Testing
- Elamipretide
- Where
- United States
- Volunteers
- 20 people
- Sponsor
- Children's Hospital of Philadelphia
Ataxia Telangiectasia
Susceptibility to Infections, Tumor Risk and Liver Disease in Patients With Ataxia Telangiectasia
Ataxia telangiectasia (A-T) is a rare devastating human recessive disorder characterized by progressive cerebellar ataxia, immunodeficiency, chromosomal instability and cancer susceptibility.
- Trial name
- NCT03357978
- Testing
- bioelectrical impedance analysis, blood draw, transient elastography (FibroScan)
- Where
- Germany
- Volunteers
- 30 people
- Sponsor
- Johann Wolfgang Goethe University Hospital
Friedreich's Ataxia
Cardiac Output and Fatigue in Friedreich's Ataxia
This is a clinical trial examining to impact of aerobic training plus omaveloxolone in FRDA. Thirty individuals with FRDA will be recruited; 20 individuals will be on omaveloxolone treatment whereas the other ten individuals will…
- Trial name
- NCT07444333
- Testing
- Aerobic Exercise
- Where
- United States
- Volunteers
- 30 people
- Sponsor
- Scott Barbuto
Ataxia Telangiectasia
Body Composition and Hormonal Status in Ataxia Telangiectasia
Ataxia telangiectasia (A-T) is a rare devastating human recessive disorder characterized by progressive cerebellar ataxia, immunodeficiency, chromosomal instability, and cancer susceptibility.
- Trial name
- NCT02345200
- Testing
- bioelectrical impedance analysis, blood draw
- Volunteers
- 52 people, healthy volunteers welcome
- Sponsor
- Johann Wolfgang Goethe University Hospital
Friedreich's Ataxia
A Study to Characterize the Cardiac Phenotype of Individuals With Friedreich's Ataxia (CARFA Study)
Friedreich's ataxia (FA) is an autosomal recessive disease with an incidence of 1/50,000 in the Caucasian population. The main manifestations of FA are progressive sensory and cerebellar ataxia and cardiomyopathy (CM).
- Trial name
- NCT02840669
- Testing
- Cardiac magnetic resonance imaging (CMR), Exercise-stress test, Echocardiography (ECHO)
- Where
- France
- Volunteers
- 40 people, healthy volunteers welcome
- Sponsor
- Adverum Biotechnologies, Inc.
Friedreich Ataxia, Rare Diseases
Understanding the Natural History Early in the Course or Presentation of Friedreich Ataxia
Multicenter, prospective, observational natural history and outcome measure study of children and young adults with Friedreich ataxia.
- Trial name
- EARLY-FA (NCT06560346)
- Testing
- Geneticlly confirmed disease causing FXN mutatuion, Healthy Control
- Where
- 7 sites in United States, Australia and 3 more
- Volunteers
- healthy volunteers welcome
- Sponsor
- Friedreich's Ataxia Research Alliance
Friedreich's Ataxia
Long-Term Safety Extension Study of ACTIMMUNE® (Interferon γ-1b) in Children and Young Adults With Friedreich's Ataxia
The purpose of this long term extension study is to evaluate the long-term safety of ACTIMMUNE® (interferon-γ 1b) in participants with Friedreich's Ataxia (FA).
- Trial name
- STEADFAST (NCT02797080)
- Testing
- interferon γ-1b
- Where
- 4 sites in United States
- Volunteers
- 38 people
- Sponsor
- Amgen
Advanced Solid Tumors
Study to Test How Well Patients With Advanced Solid Tumors Respond to Treatment With the Elimusertib in Combination With Pembrolizumab, to Find the Optimal Dose for Patients, How the Drug is Tolerated and the Way the Body Absorbs, Distributes and Discharges the Drug
The purpose of the study is to test how well patients with advanced solid tumors respond to treatment with elimusertib (BAY1895344) in combination with pembrolizumab.
- Trial name
- NCT04095273
- Testing
- Elimusertib (BAY1895344), Pembrolizumab (Keytruda®)
- Where
- 16 sites in United States, Germany and 3 more
- Volunteers
- 56 people
- Sponsor
- Bayer
Spinocerebellar Ataxia Type 3
STRIDES - a Clinical Research Study of an Investigational New Drug to Treat Spinocerebellar Ataxia
Phase 2b/3 double blind, randomized, placebo-controlled trial to assess safety and efficacy of SLS-005 (trehalose injection, 90.5 mg/mL for intravenous infusion) for the treatment of adults with spinocerebellar ataxia).
- Trial name
- STRIDES (NCT05490563)
- Testing
- SLS-005
- Where
- 23 sites in United States, Australia and 6 more
- Volunteers
- 23 people
- Sponsor
- Seelos Therapeutics, 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 Ataxias
Use of a New Smartphone Application to Determine Changes in Eyeblink Conditioning From Home Training in Individuals With Spinocerebellar Ataxia
Spinocerebellar ataxias are a group of disorders that cause severe disability and can be fatal. There are currently no known disease-modifying treatments available for use, and there is a critical need to find treatments that…
- Trial name
- NCT06177626
- Testing
- Aerobic Training, Balance Training
- Volunteers
- healthy volunteers welcome
- Sponsor
- Columbia University
Friedreich Ataxia
A Clinical Study to Evaluate the Effect of MIN-102 on the Progression of Friedreich's Ataxia in Male and Female Patients
Randomized, double-blind, placebo-controlled study on the effects of MIN-102 on Biochemical, Imaging, neurophysiological, and clinical markers in patients with Friedreich's Ataxia
- Trial name
- FRAMES (NCT03917225)
- Testing
- MIN-102
- Where
- 5 sites in Belgium, France and 2 more
- Volunteers
- 39 people
- Sponsor
- Minoryx Therapeutics, S.L.
Friedreich Ataxia
Safety and Efficacy of Etravirine in Friedreich Ataxia Patients
A drug repositioning effort provided evidence supporting the possible use of Etravirine, a drug approved for the treatment of HIV infections in patients starting from 2 years of age, as a treatment for FA.
- Trial name
- FAEST1 (NCT04273165)
- Testing
- Etravirine Tablets
- Where
- Italy
- Volunteers
- 30 people
- Sponsor
- IRCCS Eugenio Medea
Ataxia Telangiectasia
The Validity of Forced Expiratory Maneuvers in Ataxia Telangiectasia Studied Longitudinally
Rationale: Forced spirometry maneuvers are not routinely performed in patients with Ataxia Telangiectasia (A-T), even though they suffer from respiratory illnesses.
- Trial name
- NCT00951886
- Where
- Israel
- Volunteers
- 28 people
- Sponsor
- Sheba Medical Center
Spinocerebellar Ataxia Type 3, Multiple System Atrophy-cerebellar Type
Treatment of Transcranial Alternating Current Stimulation（tACS）on Cerebellar Ataxia
This is a longitudinal, triple-blind, randomized-controlled, prospective interventional study assessing patients with cerebellar ataxia, including spinocerebellar ataxia type 3 (SCA3) and multiple system atrophy-cerebellar type…
- Trial name
- NCT05557786
- Testing
- Transcranial Alternating Current Stimulation
- Where
- China
- Volunteers
- 164 people
- Sponsor
- First Affiliated Hospital of Fujian Medical University
Friedreich Ataxia
Transitional Life Events in Patients With Friedreich's Ataxia: Implications for Genetic Counseling
The purposes of this study are to learn about significant life changes for people with Friedreich's ataxia and about patients' experiences with health care providers.
- Trial name
- NCT00056186
- Where
- United States
- Volunteers
- 40 people
- Sponsor
- National Human Genome Research Institute (NHGRI)
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