Phase 3
Completed N=65
A Study to Assess Efficacy, Long Term Safety and Tolerability of RT001 in Subjects With Friedreich's Ataxia
Source: ClinicalTrials.gov NCT04102501 ↗Enrolled (actual)
65
Serious AEs
3.1%
Results posted
May 2022
Primary outcomePrimary: Change From Baseline to 11 Months in Maximum Consumption of Oxygen (mlO2/kg/Min) Using Cardiopulmonary Exercise Testing (CPET) — 0.05; 0.08 ml O2/min/kg — p=0.5858
◆ Published Evidence
No publication linked
No peer-reviewed publication reporting this trial's results has been linked yet. This can indicate results are unpublished — a known publication-bias signal. We re-check periodically.
Summary
The purpose of this study is to assess the Efficacy, Long Term Safety and Tolerability of RT001 in subjects with Friedreich's Ataxia
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Change From Baseline to 11 Months in Maximum Consumption of Oxygen (mlO2/kg/Min) Using Cardiopulmonary Exercise Testing (CPET) |
0.05; 0.08 | 0.5858 |
| SECONDARY Change From Baseline in the Timed 1 Minute Walk Distance |
-193.83; -220.42 | 0.8061 |
Eligibility Criteria
Key Inclusion Criteria
- 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 Criteria
- 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)
Data sourced from ClinicalTrials.gov (NCT04102501). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication. Informational only — not medical advice.