A Study to Assess Efficacy, Long Term Safety and Tolerability of RT001 in Subjects With Friedreich's Ataxia
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
1 other identifier
interventional
65
1 country
6
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
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at below P25 for phase_3
Started Oct 2019
6 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
Click on a node to explore related trials.
Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
September 23, 2019
CompletedFirst Posted
Study publicly available on registry
September 25, 2019
CompletedStudy Start
First participant enrolled
October 30, 2019
CompletedPrimary Completion
Last participant's last visit for primary outcome
August 23, 2021
CompletedStudy Completion
Last participant's last visit for all outcomes
August 23, 2021
CompletedResults Posted
Study results publicly available
May 27, 2022
CompletedMay 27, 2022
April 1, 2022
1.8 years
September 23, 2019
April 6, 2022
May 4, 2022
Conditions
Outcome Measures
Primary Outcomes (1)
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
11 months
Secondary Outcomes (1)
Change From Baseline in the Timed 1 Minute Walk Distance
11 months
Study Arms (2)
RT001
EXPERIMENTAL* 9 capsules daily (8.64 g total dose) given as 3 capsules three times a day (TID) with meals for the first month of treatment. * Six capsules daily (5.76 g total dose) given as 3 capsules (BID) with breakfast, and 3 capsules with dinner after the first month of treatment
Placebo
PLACEBO COMPARATOR* 9 capsules daily (8.64 g total dose) given as 3 capsules three times a day (TID) with meals for the first month of treatment. * Six capsules daily (5.76 g total dose) given as 3 capsules (BID) with breakfast, and 3 capsules with dinner after the first month of treatment
Interventions
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.
The placebo product is composed of encapsulated USP safflower oil. The placebo capsules are identical in appearance and size to RT001.
Eligibility Criteria
You may qualify if:
- 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
You may not qualify if:
- 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)
Contact the study team to confirm eligibility.
Sponsors & Collaborators
- Biojiva LLClead
Study Sites (6)
Collaborative Neuroscience Network, LLC
Long Beach, California, 90806, United States
UCLA
Los Angeles, California, 90095, United States
University of Florida
Gainesville, Florida, 32608, United States
USF Ataxia Research Center
Tampa, Florida, 33612, United States
University of Iowa Stead Family Children's Hospital
Iowa City, Iowa, 52242, United States
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104, United States
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Results Point of Contact
- Title
- Mark G. Midei, MD, VP Medical Affairs
- Organization
- Retrotope
Study Officials
- STUDY DIRECTOR
Peter Milner, MD
Chief Medical Officer
Publication Agreements
- PI is Sponsor Employee
- No
- Restrictive Agreement
- No
Study Design
- Study Type
- interventional
- Phase
- phase 3
- Allocation
- RANDOMIZED
- Masking
- QUADRUPLE
- Who Masked
- PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
- Purpose
- TREATMENT
- Intervention Model
- PARALLEL
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
September 23, 2019
First Posted
September 25, 2019
Study Start
October 30, 2019
Primary Completion
August 23, 2021
Study Completion
August 23, 2021
Last Updated
May 27, 2022
Results First Posted
May 27, 2022
Record last verified: 2022-04