Clinical trials: Ataxia, Cerebellar
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.
Friedreich Ataxia
A Multiple Ascending Dose Study of DT-216P2 in Patients With Friedreich's Ataxia
The purpose of this study is to evaluate the safety and tolerability, and pharmacokinetics of DT-216P2 administered multiple ascending doses in patients with FA.
- Trial name
- NCT06874010
- Testing
- DT-216P2
- Where
- 3 sites in Australia
- Volunteers
- 20 people
- Sponsor
- Design Therapeutics, Inc.
Friedreich's Ataxia
Safety and Efficacy Study of A0001 in Subjects With Friedreich's Ataxia
This is a Phase 2a double-blind, placebo-controlled study with two dose levels of A0001 given twice daily for 28 days. Potential subjects will be screened first to determine eligibility, after which they will be randomized to…
- Trial name
- NCT01035671
- Testing
- alpha-tocopherolquinone (A0001)
- Where
- United States
- Volunteers
- 42 people
- Sponsor
- Penwest Pharmaceuticals Co.
Healthy
Assess the Effect of Zolpidem, Silenor & Placebo on Arousability, Ataxia/Balance & Cognition in Healthy Volunteers
This is a Phase IV, randomized, double-blind, placebo-controlled, four-arm crossover study. The study will assess the effects of a single dose of Silenor 6 mg compared with matching placebo and a single dose of zolpidem 10 mg…
- Trial name
- NCT02353299
- Testing
- Silenor 6 mg, zolpidem 10 mg
- Where
- United States
- Volunteers
- 52 people, healthy volunteers welcome
- Sponsor
- Currax Pharmaceuticals
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 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.
Rett Syndrome
A Safety Study of NNZ-2566 in Patients With Rett Syndrome
The purpose of this study is to determine whether NNZ-2566 is safe and well tolerated in the treatment of Rett Syndrome in adolescent and adult females.
- Trial name
- NCT01703533
- Testing
- NNZ-2566
- Where
- 3 sites in United States
- Volunteers
- 67 people
- Sponsor
- Neuren Pharmaceuticals Limited
Schizophrenia, Cerebellar Ataxia
A TAK-831-1001, Single and Multiple Rising Dose Study in Healthy Participants
The purpose of this study is to determine the safety, tolerability and pharmacokinetics (PK) of single and multiple rising doses of TAK-831 in healthy participants.
- Trial name
- NCT02566759
- Testing
- TAK-831 Oral Suspension, TAK-831 Tablet
- Where
- United Kingdom
- Volunteers
- 110 people, healthy volunteers welcome
- Sponsor
- Neurocrine Biosciences
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.
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
Ataxia Telangiectasia
Open-Label Extension of EryDex Study IEDAT-04-2022
This is an international, multi-center, prospective, open-label, non-comparative study aiming to provide access to treatment with EryDex to ataxia telangiectasia (A-T) patients who completed the IEDAT-04-2022 trial which studied…
- Trial name
- OLE_NEAT (NCT06664853)
- Testing
- Dexamethasone sodium phosphate
- Where
- 21 sites in United States, Denmark and 7 more
- Volunteers
- 101 people
- Sponsor
- Quince Therapeutics S.p.A.
Friedreich Ataxia
A Study to Learn More About the Effects and Long-Term Safety of Omaveloxolone (BIIB141) in Children and Teens With Friedreich's Ataxia
In this study, researchers will learn more about omaveloxolone, also known as BIIB141 or SKYCLARYS®. Omaveloxolone is already approved for people with Friedreich's Ataxia (FA) who are 16 years of age or older.
- Trial name
- BRAVE (NCT06953583)
- Testing
- Omaveloxolone
- Where
- 34 sites in United States, Australia and 14 more
- Volunteers
- 255 people
- Sponsor
- Biogen
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
Ataxia Telangiectasia
Comparative Bioavailability of Betamethasone Oral Solution Metered Spray (GTX-102) in Healthy Subjects
A Randomized, Open-label, Crossover Study to Evaluate the Comparative Bioavailability, Pharmacokinetics, and Safety of GTX-102 Administered as an Oral Spray Compared to Intramuscular Injection - betamethasone and an Oral Solution…
- Trial name
- NCT05531890
- Testing
- GTX-102 medium dose fast Period 1 and Period 2, GTX-102 medium dose slow Period 1 and Period 2, GTX-102 high dose fast Period 1 and Period 2
- Where
- Canada
- Volunteers
- 48 people, healthy volunteers welcome
- Sponsor
- Grace Therapeutics Inc.
Spinocerebellar Degeneration
C-Trelin Orally Disintegrated(OD) Tablet 5mg in Ataxia Due to Spinocerebellar Degeneration
The aim of this study is to evaluate the efficacy and safety of C-Trelin OD Tablet 5mg(Taltirelin Hydrate) in Multicenter, randomized, double-blind, placebo-controlled clinical trial in patients with ataxia induced by…
- Trial name
- NCT04107740
- Testing
- C-Trelin OD Tab(5mg Taltirelin Hydrate)
- Where
- 8 sites in South Korea
- Volunteers
- 160 people
- Sponsor
- HLB Pharmaceutical Co., Ltd.
Friedreich's Ataxia
A Study of Efficacy, Safety and Tolerability of Idebenone in the Treatment of Friedreich's Ataxia (FRDA) Patients
The purpose of this trial is to study the efficacy, safety and tolerability of idebenone in 12 months of treatment in children and adults with Friedreich's Ataxia.
- Trial name
- MICONOS (NCT00905268)
- Testing
- idebenone
- Where
- 13 sites in Austria, Belgium and 4 more
- Volunteers
- 232 people
- Sponsor
- Santhera Pharmaceuticals
Friedreich Ataxia
A Study to Learn About the Effects and Safety of RTA 408 (Omaveloxolone) in People Aged 16 to 40 With Friedreich's Ataxia
In this study, researchers are learning more about RTA 408, also known as omaveloxolone, BIIB141, or SKYCLARYS®. The main goal of this study is to learn more about the safety of RTA 408 and how it affects physical effort…
- Trial name
- NCT02255435
- Testing
- Omaveloxolone Capsules, 2.5 mg, Omaveloxolone Capsules, 5 mg, Omaveloxolone Capsules, 10 mg
- Where
- 11 sites in United States, Australia and 3 more
- Volunteers
- 172 people
- Sponsor
- Biogen
Phosphomannomutase 2 Deficiency, PMM2-CDG
A Study to Assess the Efficacy and Safety of Weekly Doses of GLM101 in Participants With PMM2-CDG
This study is evaluating the safety, effectiveness, and how the body absorbs, distributes, and eliminates GLM101, for participants with PMM2-CDG, including children, adolescents, and adults.
- Trial name
- NCT06892288
- Testing
- GLM101 (Part A, Double-blind), GLM101 (Part B, Open-label)
- Where
- 15 sites in United States, Belgium and 8 more
- Volunteers
- 50 people
- Sponsor
- Glycomine, Inc.
Myoclonus Epilepsies, Progressive
Safety, Blood Levels and Effects of AUT00201 in Patients With MEAK
A randomized, double-blind, placebo-controlled, crossover study to assess the safety, tolerability, and pharmacokinetics of single doses of AUT00201 at 100 mg or matching placebo in patients with myoclonus epilepsy and ataxia due…
- Trial name
- AUT022201 (NCT05873062)
- Testing
- AUT00201
- Where
- United States
- Volunteers
- 6 people
- Sponsor
- Autifony Therapeutics Limited
Spinocerebellar Ataxia - All Sub-types
Evaluation of the Safety and Preliminary Efficacy of Neuroinduced Mesenchymal Stem Cells and Exosome Therapy in Patients With Spinocerebellar Palsy
This clinical study aims to evaluate the safety and preliminary therapeutic potential of mesenchymal stem cell-derived therapies in patients diagnosed with spinocerebellar palsy.
- Trial name
- NEURO-SCP (NCT07467733)
- Testing
- Intrathecal infusion, Intranasal infusion
- Where
- Poland
- Volunteers
- 90 people
- Sponsor
- Biocells Medical