Status:
COMPLETED
A Study to Assess Efficacy, Long Term Safety and Tolerability of RT001 in Subjects With Friedreich's Ataxia
Lead Sponsor:
Biojiva LLC
Conditions:
Friedreich's Ataxia
Eligibility:
All Genders
12-50 years
Phase:
PHASE3
Brief Summary
The purpose of this study is to assess the Efficacy, Long Term Safety and Tolerability of RT001 in subjects with Friedreich's Ataxia
Detailed Description
This is a randomized, double-blind, placebo-controlled study to evaluate the efficacy, safety, tolerability, in subjects with FRDA following the oral administration of study drug (active or placebo ca...
Eligibility Criteria
Inclusion
- Key
- Male or female 12 to 50 years of age
- Medical history consistent with the symptoms of FRDA at ≤ 25 years of age
- Detection of biallelic pathogenic variants in frataxin gene (FXN)
- Ambulatory (with or without assistive device) and capable of performing other assessments/evaluations.
- Must be able to walk 25 feet during the timed 1-minute walk
- Key
Exclusion
- Received treatment with other experimental therapies within the last 30 days prior to the first dose
- Previously participated in the RT001 trial
- Refusal to discontinue fish oils or other oil-based supplements for the duration of the study (Screening till last study procedure completed)
- History of malignancies (other than basal cell carcinomas)
- Inability to complete CPET protocol
- Female who is breastfeeding or has a positive pregnancy test
- History of uncontrolled diabetes mellitus (Type 1 or 2)
Key Trial Info
Start Date :
October 30 2019
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
August 23 2021
Estimated Enrollment :
65 Patients enrolled
Trial Details
Trial ID
NCT04102501
Start Date
October 30 2019
End Date
August 23 2021
Last Update
May 27 2022
Active Locations (6)
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1
Collaborative Neuroscience Network, LLC
Long Beach, California, United States, 90806
2
UCLA
Los Angeles, California, United States, 90095
3
University of Florida
Gainesville, Florida, United States, 32608
4
USF Ataxia Research Center
Tampa, Florida, United States, 33612