Phase 3 N=65 Randomized Quadruple-blind Treatment

A Study to Assess Efficacy, Long Term Safety and Tolerability of RT001 in Subjects With Friedreich's Ataxia

Friedreich's Ataxia

Bottom Line

View on ClinicalTrials.gov: NCT04102501 ↗

Enrolled (actual)

Serious AEs

3.1%

Results posted

May 2022

Primary outcome: Primary: 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

Study Design & Population

Study type: Interventional
Phase: Phase 3
Interventions: RT001 (Drug); Placebo (Drug)
Age: Pediatric, Adult · 12+ yrs
Sex: All
Sponsor: Biojiva LLC
Primary completion: Aug 2021

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

Summary

The purpose of this study is to assess the Efficacy, Long Term Safety and Tolerability of RT001 in subjects with Friedreich's Ataxia

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)

View full record on ClinicalTrials.gov →

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.