Clinical trials: Ataxia
Advanced Solid Tumor
Berzosertib Human Mass Balance Study (DDRiver Solid Tumors 208)
The study was conducted in two periods, Period 1 (mass balance) and Period 2 (extension). The purpose of Period 1 of this study was to provide a quantitative characterization of the mass balance, rates and routes of elimination…
- Trial name
- NCT05246111
- Testing
- [14C]Berzosertib, Berzosertib, Topotecan
- Where
- 2 sites in Hungary
- Volunteers
- 6 people
- Sponsor
- Merck Healthcare KGaA, Darmstadt, Germany, an affiliate of Merck KGaA, Darmstadt, Germany
Solid Tumor
Tuvusertib (M1774) Human Mass Balance and Absolute Bioavailability Study (DDRIVER Solid Tumors 303)
This is a single sequence 2-period open label study in participants with advanced solid tumors. The purpose of Period 1 of this study is to assess the mass balance to determine drug-related entities present in circulation and…
- Trial name
- NCT06308263
- Testing
- Tuvusertib [14C]Tuvusertib microtracer, Tuvusertib, Tuvusertib + [14C]Tuvusertib microdose bolus injection
- Where
- Hungary
- Volunteers
- 12 people
- Sponsor
- Merck Healthcare KGaA, Darmstadt, Germany, an affiliate of Merck KGaA, Darmstadt, Germany
FXN Gene, FRDA
Biomarker for Friedreich's Ataxia (BioFridA)
International, multicenter, observational, longitudinal monitoring study to identify biomarker/s for Friedreich's Ataxia and to explore the clinical robustness, specificity, and long-term variability of these biomarker/s
- Conditions
- FXN Gene, FRDA, Hereditary Diseases
- Trial name
- BioFridA (NCT04548921)
- Where
- Lebanon
- Volunteers
- 1,000 people
- Sponsor
- CENTOGENE GmbH Rostock
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
Friedreich's Ataxia
A First in Human Study of RT001 in Patients With Friedreich's Ataxia
The purpose of this study is to evaluate the safety, tolerability and pharmacokinetics of RT001 in patients with Friedreich's ataxia.
- Trial name
- NCT02445794
- Testing
- Low dose cohort, High dose cohort
- Where
- 2 sites in United States
- Volunteers
- 19 people
- Sponsor
- Biojiva LLC
Friedreich's Ataxia
A Study Investigating the Safety and Tolerability of Deferiprone in Patients With Friedreich's Ataxia
The primary objective of this study is to demonstrate the safety and tolerability of deferiprone in subjects with Friedreich's ataxia (FRDA).
- Trial name
- NCT00530127
- Testing
- deferiprone
- Where
- 6 sites in Australia, Belgium and 4 more
- Volunteers
- 80 people
- Sponsor
- ApoPharma
Ataxia Telangiectasia, Genetic Syndrome
Extension Treatment Using EryDex System in Patients With AT Who Participated in the ATTeST-IEDAT-02-2015 Study
Primary Objective To monitor and evaluate the long-term safety and tolerability of EDS-EP in AT patients. Secondary Objective To evaluate the long-term effect of EDS-EP on health-related Quality of Life (QoL; EQ-5D-5L scale).
- Trial name
- OLE-IEDAT (NCT03563053)
- Testing
- EryDex System
- Where
- 17 sites in United States, Australia and 9 more
- Volunteers
- 104 people
- Sponsor
- Quince Therapeutics S.p.A.
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 (SCA)
Multi-Modal Digital Monitoring of Disease Symptoms in Spinocerebellar Ataxia
This is an observational study. Subjects with a documented diagnosis of spinocerebellar ataxia (SCA) will be screened and recruited to participate at two clinical sites: Johns Hopkins University (JHU) and Columbia University…
- Trial name
- NCT07851376
- Where
- 2 sites in United States
- Volunteers
- 25 people
- Sponsor
- BioSensics
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
Ataxia Telangiectasia
N-Acetyl-L-Leucine for Ataxia-Telangiectasia (A-T)
This is a multinational, multicenter, open-label, rater-blinded prospective Phase II study which will assess the safety and efficacy of N-Acetyl-L-Leucine (IB1001) for the treatment of Ataxia-Telangiectasia (A-T).
- Trial name
- NCT03759678
- Testing
- IB1001
- Where
- 4 sites in United States, Germany and 2 more
- Volunteers
- 17 people
- Sponsor
- IntraBio Inc
Ataxia Telangiectasia
International Ataxia Rating Scale in Younger Patients
The project will collect information on the mapping of clinical ratings on a number of scales that are used in the assessment of patients with ataxias.
- Trial name
- NCT01942850
- Where
- 5 sites in United States, India and 2 more
- Volunteers
- 63 people
- Sponsor
- Quince Therapeutics S.p.A.
Friedreich Ataxia
Efficacy, Tolerability, and Pharmacokinetics of Multiple Doses of Oral TAK-831 in Adults With Friedreich Ataxia
The purpose of this study is to evaluate the efficacy of TAK-831 versus placebo on upper extremity (arm and hands) motor function and manual dexterity.
- Trial name
- NCT03214588
- Testing
- TAK-831
- Where
- 6 sites in United States
- Volunteers
- 67 people
- Sponsor
- Neurocrine Biosciences
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
Mitochondrial Diseases
EPI-743 for Mitochondrial Respiratory Chain Diseases
This study will evaluate the safety and efficacy of EPI-743 in participants with severe mitochondrial respiratory chain diseases who are considered to be within 90 days of end-of-life care.
- Trial name
- NCT01370447
- Testing
- EPI-743
- Where
- 16 sites in United States
- Sponsor
- PTC Therapeutics
Friedreich Ataxia
Study to Evaluate DT-216 in Adult Patients With Friedreich Ataxia
The purpose of this study is to evaluate the safety, tolerability, pharmacokinetic and pharmacodynamic effects of intravenous DT-216 in adult patients with Friedreich Ataxia.
- Trial name
- NCT05285540
- Testing
- DT-216
- Where
- United States
- Volunteers
- 39 people
- Sponsor
- Design Therapeutics, Inc.
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
Friedreich's Ataxia
Safety and Pharmacology Study of VP 20629 in Adults With Friedreich's Ataxia
The objectives of the study are: To evaluate the safety and tolerability of single and multiple oral doses of VP 20629 in subjects with Friedreich's ataxia (FA).
- Trial name
- NCT01898884
- Testing
- VP 20629
- Where
- 5 sites in United States
- Volunteers
- 46 people
- Sponsor
- Shire
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
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.