Clinical Trial Details

Overview

Research Study Summary

A Phase 2 Study of the Safety, Efficacy, and Pharmacodynamics of RTA 408 in the Treatment of Friedreich's Ataxia (MOXIe)

Purpose

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 patients with Friedreich's ataxia. Therefore, the ability of RTA 408 to activate Nrf2 and induce antioxidant target genes is hypothesized to be therapeutic in patients with Friedreich's ataxia.

This 2-part study will evaluate the efficacy, safety, and pharmacodynamics of RTA 408 in the treatment of patients 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 RTA 408 at various doses in patients with Friedreich's ataxia.

Part 2: The second part of this study is a randomized, placebo-controlled, double-blind, parallel study to evaluate the safety, efficacy, and pharmacodynamics of up to 2 dose levels of RTA 408 in patients with Friedreich's ataxia. Eligible patients in Part 2 will be randomized 1:1:1 to receive RTA 408 (at one of 2 doses chosen from Part 1), or placebo.

To Learn more

CW ID: 209281
Date Last Changed: March 8, 2016

Clinical Trial Snapshot

Phase
2
Gender
Both Male and Female
Age
16 to 40 Years
Overall Status
Recruiting
Lead Sponsor
Reata Pharmaceuticals, Inc.
Duration
12 Weeks
Facility Type
Out-Patient

Eligibility

Inclusion Criteria:

  • Have genetically confirmed Friedreich's ataxia
  • Have a modified FARS score ≥10 and ≤80
  • Be male or female and ≥16 years of age and ≤40 years of age
  • Have no changes to exercise regimen within 30 days prior to Study Day 1 and be willing to remain on the same exercise regimen during the 16-week study period
  • Have the ability to complete maximal exercise testing
  • Be able to swallow capsules

Exclusion Criteria:

  • Have uncontrolled diabetes (HbA1c >11.0%)
  • Have B-type natriuretic peptide value >200 pg/mL
  • Have a history of clinically significant left-sided heart disease and/or clinically significant cardiac disease
  • Have known active fungal, bacterial, and/or viral infection, including human immunodeficiency virus or hepatitis virus (B or C)
  • Have known or suspected active drug or alcohol abuse
  • Have clinically significant abnormalities of clinical hematology or biochemistry, including but not limited to elevations greater than 1.5 times the upper limit of normal of aspartate aminotransferase, alanine aminotransferase or creatinine
  • Have any abnormal laboratory test value or serious pre-existing medical condition that, in the opinion of the investigator, would put the patient at risk by study enrollment
  • Have taken any of the following drugs within 7 days prior to Study Day 1 or plan to take any of these drugs during the time of study participation:
    • Sensitive substrates for cytochrome P450 2C8 or 3A4 (e.g., repaglinide, midazolam, sildenafil)
    • Substrates for p-glycoprotein transporter (e.g., ambrisentan, digoxin)
  • Have participated in any other interventional clinical study within 30 days prior to Study Day 1
  • Have a cognitive impairment that may preclude ability to comply with study procedures

More Info

Protocol number: 408-C-1402

Contact

Kevin Allison
University of South Florida- Morsani Center for Advanced Healthcare
13330 USF Laurel Drive
Tampa, FL 33613
Phone: 813-974-9915

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