drugset
drugset / Trial / NCT04102501

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

NCT04102501 ↗

Phase 3 Completed 65 enrolled Biojiva LLC
RandomizedParallel-groupQuadruple-blindTreatment

Summary

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

Timeline

Start
2019-10-30
Primary completion
2021-08-23
Completion
2021-08-23

Outcome

Missed primary endpoint

registry analysis (superiority test); RT001 vs Placebo; p = 0.5858; Mixed Models Analysis NCT04102501 ↗

Drugs

EvaluationDrugModalityDoseRoute
Subject RT001 Protein / enzyme biologic 5.76 g Oral

Indications

Friedreich ataxia