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

Biojiva LLC65 enrolled

Overview

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

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.

Study Type

INTERVENTIONAL

Allocation

RANDOMIZED

Purpose

TREATMENT

Masking

QUADRUPLE

Enrollment

Conditions

Friedreich's Ataxia

Interventions

RT001DRUG

RT001 is encapsulated 9-cis, 12-cis-11,11-D2-linoleic acid ethyl ester, which is a site specific (C11) di-deutero synthetic homologue of LA ethyl ester. Each capsule contains 960 mg of RT001.

PlaceboDRUG

The placebo product is composed of encapsulated USP safflower oil. The placebo capsules are identical in appearance and size to RT001.

Eligibility

Sex: ALLMin age: 12 YearsMax age: 50 Years

Medical Language ↔ Plain English

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)

Locations (6)

Collaborative Neuroscience Network, LLC

Long Beach, California, United States

UCLA

Los Angeles, California, United States

University of Florida

Gainesville, Florida, United States

USF Ataxia Research Center

Tampa, Florida, United States

Outcomes

Primary Outcomes

Change From Baseline to 11 Months in Maximum Consumption of Oxygen (mlO2/kg/Min) Using Cardiopulmonary Exercise Testing (CPET)

To measure the change in MVO2 from baseline to Month 11 using CPET

Time frame: 11 months

Secondary Outcomes

Change From Baseline in the Timed 1 Minute Walk Distance

Change from baseline Distance walked in 1 minute at 11 months

Time frame: 11 months

Data from ClinicalTrials.gov

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