Efficacy, Tolerability, and Pharmacokinetics of Multiple Doses of Oral TAK-831 in Adults With Friedreich...
Friedreich AtaxiaThe 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. This study will also evaluate the efficacy of TAK-831 versus placebo on activities of daily living (ADL) and other secondary assessments.
Multiple Ascending Dose Study of CTI-1601 Versus Placebo in Subjects With Friedreich's Ataxia
Friedreich AtaxiaTo evaluate the safety and tolerability of multiple ascending doses of CTI-1601 in participants with Friedreich's ataxia
Study to Evaluate DT-216 in Adult Patients With Friedreich Ataxia
Friedreich AtaxiaThe purpose of this study is to evaluate the safety, tolerability, pharmacokinetic and pharmacodynamic effects of intravenous DT-216 in adult patients with Friedreich Ataxia. This single ascending dose study is randomized, double-blind, placebo-controlled.
A First in Human Study of RT001 in Patients With Friedreich's Ataxia
Friedreich's AtaxiaThe purpose of this study is to evaluate the safety, tolerability and pharmacokinetics of RT001 in patients with Friedreich's ataxia.
Methylprednisolone Treatment of Friedreich Ataxia
Friedreich AtaxiaThis study will explore whether methylprednisolone treatment is safe, well-tolerated, and beneficial in patients that are diagnosed with Friedreich Ataxia (FRDA). The study will also explore if methylprednisolone has any effects on biomarkers associated with FRDA. All subjects in the study will receive the same steroid treatment.
(+) Epicatechin to Treat Friedreich's Ataxia
Friedreich's AtaxiaThis 24-week study will test the safety and effectiveness of synthetically produced (+) Epicatechin in treating patients who have Friedreich's Ataxia, a neurological disorder.
Study to Assess the Safety and Tolerability of Idebenone in the Treatment of Friedreich's Ataxia...
Friedreich's AtaxiaThis study is meant to assess the safety and tolerability of idebenone in patients with Friedreich's Ataxia over a 12 months period.
A Phase IIa Trial to Test Safety and Efficacy Interferon Gamma Treatment in Elevating Frataxin Levels...
Friedreich AtaxiaThe primary objective of this study is to investigate whether the treatment with IFN gamma can induce significant accumulation of frataxin in FRDA patients, a possibility suggested by pre-clinical evidence in an animal model of the disease.
Rosuvastatin (Crestor) in Friedreich Ataxia
Friedreich AtaxiaThis study is an exploratory open-label clinical trial of Rosuvastatin in patients with Friedreich ataxia (FRDA). This is an outpatient trial with the goal of enrolling 10 evaluable adults with genetically confirmed FRDA who are between the ages of 18-65. Subjects will receive 10mg of oral Rosuvastatin daily for three months.
A Study to Assess the Efficacy and Safety of Vatiquinone for the Treatment of Participants With...
Friedreich AtaxiaThe primary objective of the study is to evaluate the efficacy (using the modified Friedreich Ataxia Rating Scale [mFARS]) and safety of vatiquinone in participants with Friedreich ataxia (FA).