Clinical trial NCT04102501: A Study to Assess Efficacy, Long Term Safety and Tolerability of RT001 in Subjects With Friedreich's Ataxia
Official title: A Randomized, Double-Blind, Controlled, Phase 2/3 Study to Assess Efficacy, Long Term Safety and Tolerability of RT001 in Subjects With Friedreich's Ataxia
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE3
Conditions: Friedreich's Ataxia
Lead sponsor: Biojiva LLC
Sponsor class: INDUSTRY
First posted: 2019-09-25
Enrollment: 65
Sex: ALL
Minimum age (years): 12
Maximum age (years): 50
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

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 capsules). Sixty eligible patients will undergo various assessments at different time points during the the study. The study duration is 13 months which includes screening, treatment and safety follow up phone call.

Eligibility criteria: Key Inclusion Criteria:

1. Male or female 12 to 50 years of age
2. Medical history consistent with the symptoms of FRDA at ≤ 25 years of age
3. Detection of biallelic pathogenic variants in frataxin gene (FXN)
4. Ambulatory (with or without assistive device) and capable of performing other assessments/evaluations.
5. Must be able to walk 25 feet during the timed 1-minute walk

Key Exclusion Criteria:

1. Received treatment with other experimental therapies within the last 30 days prior to the first dose
2. Previously participated in the RT001 trial
3. Refusal to discontinue fish oils or other oil-based supplements for the duration of the study (Screening till last study procedure completed)
4. History of malignancies (other than basal cell carcinomas)
5. Inability to complete CPET protocol
6. Female who is breastfeeding or has a positive pregnancy test
7. History of uncontrolled diabetes mellitus (Type 1 or 2)

Primary outcomes:
measure: Change From Baseline to 11 Months in Maximum Consumption of Oxygen (mlO2/kg/Min) Using Cardiopulmonary Exercise Testing (CPET)
timeFrame: 11 months
Secondary outcomes:
measure: Change From Baseline in the Timed 1 Minute Walk Distance
timeFrame: 11 months
Other outcomes:
Locations:
city: Long Beach
country: United States
facility: Collaborative Neuroscience Network, LLC
state: California
city: Los Angeles
country: United States
facility: UCLA
state: California
city: Gainesville
country: United States
facility: University of Florida
state: Florida
city: Tampa
country: United States
facility: USF Ataxia Research Center
state: Florida
city: Iowa City
country: United States
facility: University of Iowa Stead Family Children's Hospital
state: Iowa
city: Philadelphia
country: United States
facility: Children's Hospital of Philadelphia
state: Pennsylvania
Study officials:
affiliation: Chief Medical Officer
name: Peter Milner, MD
role: STUDY_DIRECTOR
References: