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Friedreich's Ataxia clinical trials at University of California Health

7 in progress, 2 open to eligible people

Showing trials for
  • Gene Therapy for Cardiomyopathy Associated With Friedreich's Ataxia

    open to eligible people ages 18-50

    This is a Phase 1/2, open-label, dose-ascending, multicenter study of the safety and efficacy of LX2006 for participants who have Friedreich's Ataxia with evidence of cardiomyopathy. The study will evaluate up to three doses of single administration of LX2006 (AAVrh.10hFXN), an adeno-associated virus (AAV) gene therapy designed to intravenously deliver the human frataxin (hFXN) gene to cardiac cells over a 52-week period. Long-term safety and efficacy will be evaluated for an additional 4-years for a total of 5-years post LX2006 treatment.

    at UCLA

  • Friedreich Ataxia Global Clinical Consortium UNIFIED Natural History Study

    open to all eligible people

    This project is a global, multicenter, prospective, longitudinal, observational natural history study that can be used to understand the disease progression and support the development of safe and effective drugs and biological products for Friedreich ataxia.

    at UCLA

  • Vatiquinone in Participants With Friedreich Ataxia

    Sorry, accepting new patients by invitation only

    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.

    at UCLA

  • CTI-1601 in Subjects With Friedreich's Ataxia

    Sorry, accepting new patients by invitation only

    This is an open-label extension (OLE) study designed to evaluate the long-term safety, tolerability, pharmacokinetics (PK), pharmacodynamics (PD), and clinical effects of subcutaneous (SC) administration of CTI-1601, also known as nomlabofusp, in subjects with Friedreich's ataxia (FRDA). The objectives of this OLE study are: - To evaluate the safety of long-term subcutaneous (SC) administration of CTI-1601 in subjects with FRDA - To evaluate the PK of long-term subcutaneous (SC) administration of CTI-1601 in subjects with FRDA - To evaluate the effect of long-term subcutaneous (SC) administration of CTI-1601 in subjects with FRDA on: - Tissue FXN concentrations - Clinical evaluations of FRDA - Gene Expression and select lipids

    at UCLA

  • RTA 408 Capsules in Patients With Friedreich's Ataxia - MOXIe

    Sorry, in progress, not accepting new patients

    Friedreich's ataxia is an autosomal recessive cerebellar ataxia caused by triplet-repeat expansions. The causative mutation is a trinucleotide (GAA) repeat expansion in the first intron of the frataxin gene, leading to impaired transcription of frataxin. The pathological consequences of frataxin deficiency include a severe disruption of iron-sulfur cluster biosynthesis, mitochondrial iron overload coupled to cellular iron dysregulation, and an increased sensitivity to oxidative stress. A hallmark of Friedreich's ataxia is impairment of antioxidative defense mechanisms, which play a major role in disease progression. Studies have demonstrated that nuclear factor erythroid-derived 2-related factor 2 (Nrf2) signaling is grossly impaired in participants with Friedreich's ataxia. Therefore, the ability of omaveloxolone (RTA 408) to activate Nrf2 and induce antioxidant target genes is hypothesized to be therapeutic in participants with Friedreich's ataxia. This 2-part study will evaluate the efficacy, safety, and pharmacodynamics of omaveloxolone (RTA 408) in the treatment of participants with Friedreich's ataxia. Part 1: The first part of this study will be a randomized, placebo-controlled, double-blind, dose-escalation study to evaluate the safety of omaveloxolone (RTA 408) at various doses in participants with Friedreich's ataxia. Part 2: The second part of this study is a randomized, placebo-controlled, double-blind, parallel-group study to evaluate the safety and efficacy of omaveloxolone (RTA 408) 150 mg in participants with Friedreich's ataxia. Participants enrolled in Part 2 will be randomized 1:1 to receive omaveloxolone (RTA 408) 150 mg or placebo. Extension: The extension will assess long-term safety and tolerability of omaveloxolone (RTA 408) in qualified participants with Friedreich's ataxia following completion of Part 1 or Part 2. Participants will not be unblinded to study treatment in Part 1 or Part 2 upon entering the extension study. Participants will receive open-label omaveloxolone (RTA 408) at 150 mg once daily.

    at UCLA

  • FA Clinical Outcome Measures

    Sorry, not currently recruiting here

    This multicenter natural history study aims to expand the network of clinical research centers in FA, and to provide a framework for facilitating therapeutic interventions. In addition, this study will lead to the development of valid yet sensitive clinical measures crucial to outcome assessment of patients with Friedreich's Ataxia. This study will support genetic modifier studies, biomarker studies, and frataxin protein level assessments by building a sample repository.

    at UCLA

  • Instrumented Data Exchange for Ataxia Study

    Sorry, in progress, not accepting new patients

    This research study is testing body-worn sensors to measure movement during simple tests of coordination, in order to evaluate the progression and severity of ataxia.

    at UCLA

Our lead scientists for Friedreich's Ataxia research studies include .

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