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

• ClinicalTrials.gov Identifier: NCT04102501
• Recruitment Status: Completed
• First Posted: September 25, 2019
• Results First Posted: May 27, 2022
• Last Update Posted: May 27, 2022
• Sponsor: Retrotope, Inc.
• Information provided by (Responsible Party): Retrotope, Inc.

Study Description

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

Condition or disease	Intervention/treatment	Phase
Friedreich's Ataxia	Drug: RT001; Drug: Placebo	Phase 3

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.

Study Design

• Study Type: Interventional (Clinical Trial)
• Actual Enrollment: 65 participants
• Allocation: Randomized
• Intervention Model: Parallel Assignment
• Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
• Primary Purpose: Treatment
• 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
• Actual Study Start Date: October 30, 2019
• Actual Primary Completion Date: August 23, 2021
• Actual Study Completion Date: August 23, 2021

Arms and Interventions

Arm	Intervention/treatment
Experimental: RT001; 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	Drug: RT001; 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.
Placebo Comparator: Placebo; 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	Drug: Placebo; The placebo product is composed of encapsulated USP safflower oil. The placebo capsules are identical in appearance and size to RT001.

Outcome Measures

Primary Outcome Measures :

1. Change From Baseline to 11 Months in Maximum Consumption of Oxygen (mlO2/kg/Min) Using Cardiopulmonary Exercise Testing (CPET) [ Time Frame: 11 months ]
   To measure the change in MVO2 from baseline to Month 11 using CPET

Secondary Outcome Measures :

1. Change From Baseline in the Timed 1 Minute Walk Distance [ Time Frame: 11 months ]
   Change from baseline Distance walked in 1 minute at 11 months

Eligibility Criteria

• Ages Eligible for Study: 12 Years to 50 Years (Child, Adult)
• Sexes Eligible for Study: All
• Accepts Healthy Volunteers: No

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)

Contacts and Locations

Locations

United States, California

Collaborative Neuroscience Network, LLC

Long Beach, California, United States, 90806

UCLA

Los Angeles, California, United States, 90095

United States, Florida

University of Florida

Gainesville, Florida, United States, 32608

USF Ataxia Research Center

Tampa, Florida, United States, 33612

United States, Iowa

University of Iowa Stead Family Children's Hospital

Iowa City, Iowa, United States, 52242

United States, Pennsylvania

Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, United States, 19104

Sponsors and Collaborators

Retrotope, Inc.

Investigators

• Study Director: Peter Milner, MD; Chief Medical Officer

  Study Documents (Full-Text)

Documents provided by Retrotope, Inc.:

Study Protocol and Statistical Analysis Plan  [PDF] November 3, 2021

More Information

• Responsible Party: Retrotope, Inc.
• ClinicalTrials.gov Identifier: NCT04102501
• Other Study ID Numbers: RT001-006
• First Posted: September 25, 2019
• Results First Posted: May 27, 2022
• Last Update Posted: May 27, 2022
• Last Verified: April 2022

• Studies a U.S. FDA-regulated Drug Product: Yes
• Studies a U.S. FDA-regulated Device Product: No

Additional relevant MeSH terms:

Ataxia; Cerebellar Ataxia; Friedreich Ataxia; Dyskinesias; Neurologic Manifestations; Nervous System Diseases; Cerebellar Diseases; Brain Diseases; Central Nervous System Diseases; Spinocerebellar Degenerations; Spinal Cord Diseases; Heredodegenerative Disorders, Nervous System; Neurodegenerative Diseases; Genetic Diseases, Inborn; Mitochondrial Diseases; Metabolic Diseases