Clinical trials: Spinocerebellar Degenerations
Advanced Solid Tumors
A Study of BMS-986484 Alone and Combination Therapy in Participants With Advanced Solid Tumors
The purpose of this study is to assess the safety and tolerability of BMS-986484 administered alone, in combination with nivolumab in participants with advanced/metastatic solid tumors including non-small cell lung cancer…
- Trial name
- NCT06544655
- Testing
- BMS-986484, Nivolumab, Oxaliplatin
- Where
- 9 sites in United States, Australia and 1 more
- Volunteers
- 213 people
- Sponsor
- Bristol-Myers Squibb
Friedreich's Ataxia
Safety and Efficacy of EPI-743 in Patients With Friedreich's Ataxia
The purpose of this study is to examine the effects of EPI-743 on visual function and neurologic function in patients with Friedreich's ataxia.
- Trial name
- NCT01728064
- Testing
- EPI-743 400 mg, EPI-743 200 mg
- Where
- 3 sites in United States
- Volunteers
- 60 people
- Sponsor
- PTC Therapeutics
Friedreich Ataxia
Multiple Ascending Dose Study of CTI-1601 Versus Placebo in Subjects With Friedreich's Ataxia
To evaluate the safety and tolerability of multiple ascending doses of CTI-1601 in participants with Friedreich's ataxia
- Trial name
- NCT04519567
- Testing
- CTI-1601
- Where
- United States
- Volunteers
- 27 people
- Sponsor
- Larimar Therapeutics, Inc.
Colon Cancer
Neoadjuvant Pembrolizumab and Porustobart in Resectable pMMR/MSS Colon Cancer
The goal of this clinical trial is to learn if giving two medicines together, porustobart and pembrolizumab, before surgery can treat a common type of colon cancer called microsatellite stable (MSS) colon cancer in adults.
- Trial name
- NCT07808151
- Testing
- Pembrolizumab, Porustobart
- Where
- 30 sites in United States, Belgium and 4 more
- Volunteers
- 100 people
- Sponsor
- Solstice Oncology, Inc.
Friedreich's Ataxia
A Study to Assess Efficacy, Long Term Safety and Tolerability of RT001 in Subjects With Friedreich's Ataxia
The purpose of this study is to assess the Efficacy, Long Term Safety and Tolerability of RT001 in subjects with Friedreich's Ataxia
- Trial name
- NCT04102501
- Testing
- RT001
- Where
- 6 sites in United States
- Volunteers
- 65 people
- Sponsor
- Biojiva LLC
Spinocerebellar Ataxias
Real-World Data Study of Troriluzole-Treated Patients With Spinocerebellar Ataxia (SCA) Compared to a Matched Natural History Control
The purpose of this study is to leverage two sources of real-world data (RWD) to assess the effectiveness of troriluzole after three years of treatment in patients with SCA by comparison to an external control of untreated…
- Trial name
- NCT06529146
- Testing
- BHV-4157
- Where
- United States
- Volunteers
- 909 people
- Sponsor
- Biohaven Therapeutics Ltd.
Hereditary Ataxia
Safety and Efficacy of Umbilical Cord Mesenchymal Stem Cell Therapy for Patients With Hereditary Ataxia
The Hereditary Ataxias are a group of genetic disorders characterized by slowly progressive incoordination of gait and often associated with poor coordination of hands, speech, and eye movements.
- Trial name
- NCT01360164
- Testing
- human umbilical cord mesenchymal stem cells
- Where
- China
- Volunteers
- 20 people
- Sponsor
- Shenzhen Beike Bio-Technology Co., Ltd.
Friedreich Ataxia, Cardiomyopathy, Secondary
Study of LX2006 Gene Therapy in Friedreich Ataxia Cardiomyopathy
The purpose of Study LX2006-03, a multicenter, Phase 2, open-label, randomized, controlled study, is to evaluate the efficacy and safety of LX2006 gene therapy in participants with Friedreich ataxia (FA) cardiomyopathy (CM).
- Trial name
- SUNRISE-FA 2 (NCT07721025)
- Testing
- LX2006, Usual Care
- Where
- United States
- Volunteers
- 26 people
- Sponsor
- Lexeo Therapeutics
Friedreich Ataxia
A Single Ascending Dose Study of DT-216P2 in Normal Healthy Participants
This purpose of the study is to evaluate the safety, tolerability and pharmacokinetics of single ascending doses of DT-216P2 administered either subcutaneously (SC) and intravenously (IV) in normal healthy participants.
- Trial name
- NCT06772870
- Testing
- DT-216P2, Saline
- Where
- Australia
- Volunteers
- 36 people, healthy volunteers welcome
- Sponsor
- Design Therapeutics, Inc.
Spinocerebellar Degeneration
An Extension Study of KPS-0373 in Patients With Spinocerebellar Degeneration (SCD)
The purpose of this study is to evaluate the long-term safety, efficacy, and pharmacokinetics of KPS-0373 in SCD patients.
- Trial name
- NCT01970111
- Testing
- KPS-0373, High dose, KPS-0373, Low dose
- Where
- Japan
- Sponsor
- Kissei Pharmaceutical Co., Ltd.
Friedreich's Ataxia
Study to Assess the Efficacy, Safety and Tolerability of Idebenone in the Treatment of Friedreich's Ataxia
This study is meant to assess the effectiveness of idebenone on neurological outcome measures in patients with Friedreich's Ataxia over a 6 months period.
- Trial name
- IONIA (NCT00537680)
- Testing
- Idebenone
- Where
- 2 sites in United States
- Volunteers
- 70 people
- Sponsor
- Santhera Pharmaceuticals
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
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 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.
Friedreich Ataxia
A Study to Assess Nomlabofusp in Adolescents and Children With Friedreich's Ataxia
The goal of this clinical trial is to evaluate the safety and tolerability of nomlabofusp (CTI-1601) in adolescents and children with Friedreich's ataxia (FRDA).
- Trial name
- NCT06681766
- Testing
- Nomlabofusp
- Where
- United States
- Volunteers
- 18 people
- Sponsor
- Larimar Therapeutics, Inc.
Friedreich's Ataxia
Safety Study of Carbamylated Erythropoietin to Treat Patients With the Neurodegenerative Disorder Friedreich's Ataxia
The primary purpose of the study is to determine whether carbamylated erythropoietin is a safe treatment for patients who suffer from Friedreich's Ataxia.
- Trial name
- NCT01016366
- Testing
- Lu AA24493
- Where
- 7 sites in Austria, Germany and 1 more
- Volunteers
- 36 people
- Sponsor
- H. Lundbeck A/S
Friedreich Ataxia
Study to Evaluate Multiple Ascending Dose and Multi-Dose of DT-216 in Adult Patients With Friedreich Ataxia
The purpose of this study is to evaluate the safety, tolerability, pharmacokinetic, and pharmacodynamic effects of multiple doses of intravenous DT-216 in adult patients with Friedrich Ataxia.
- Trial name
- NCT05573698
- Testing
- DT-216
- Where
- United States
- Volunteers
- 32 people
- Sponsor
- Design Therapeutics, Inc.
Friedreich's Ataxia
A Study Investigating the Long-term Safety and Efficacy of Deferiprone in Patients With Friedreich's Ataxia
The primary objective of this study is to evaluate the long-term safety and tolerability of deferiprone in subjects with Friedreich's ataxia (FRDA).
- Trial name
- NCT00897221
- Testing
- Deferiprone oral solution 100mg/mL, Deferiprone oral solution 100 mg/mL
- Where
- 4 sites in Belgium, France and 2 more
- Volunteers
- 36 people
- Sponsor
- ApoPharma
Friedreich Ataxia
Efficacy of EGb761 in Patients Suffering From Friedreich Ataxia
The purpose of this protocol is to determine the efficacy of EGb 761 120 mg bid versus placebo in patients suffering from Friedreich Ataxia
- Trial name
- NCT00824512
- Testing
- EGb 761 120 mg
- Where
- France
- Volunteers
- 22 people
- Sponsor
- Ipsen
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.