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.

Tracking Information

• First Submitted Date: September 23, 2019
• First Posted Date: September 25, 2019
• Results First Submitted Date: April 6, 2022
• Results First Posted Date: May 27, 2022
• Last Update Posted Date: May 27, 2022
• Actual Study Start Date: October 30, 2019
• Actual Primary Completion Date: August 23, 2021 (Final data collection date for primary outcome measure)

Current Primary Outcome Measures (submitted: May 4, 2022)

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

Original Primary Outcome Measures (submitted: September 23, 2019)

Peak workload change from baseline to 9 months using cardiopulmonary exercise testing (CPET) [ Time Frame: 9 months ]

To measure the percentage change in peak workload from baseline to Month 9 using CPET

Current Secondary Outcome Measures (submitted: May 4, 2022)

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

Original Secondary Outcome Measures (submitted: September 23, 2019)

• Distance walked during a 1-minute walk (T1MW) [ Time Frame: 9 months ]
  The T1MW is a quantitative measure of lower extremity function. The distance walked over 1 minute is measured
• Effects of RT001 on a Fatigue scale [ Time Frame: 9 months ]
  To measure the change in score from baseline to Month 9 on a fatigue scale.
• Effects of RT001 on Clinical Global Impression [CGI] [ Time Frame: 9 months ]
  The CGI is a 3-item observer rated scale that measures illness severity, global improvement and therapeutic response.
• Effects of RT001 on Visual Analogue Scale [VAS] [ Time Frame: 9 months ]
  VAS for Improvement of FA Symptoms and a VAS for fatigue.

• Current Other Pre-specified Outcome Measures: Not Provided
• Original Other Pre-specified Outcome Measures: Not Provided

Descriptive Information

• Brief Title: 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
• 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.
• Study Type: Interventional
• Study Phase: Phase 3
• Study Design: Allocation: Randomized; Intervention Model: Parallel Assignment; Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor); Primary Purpose: Treatment
• Condition: Friedreich's Ataxia

Intervention

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

Study Arms

• 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
  Intervention: Drug: 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
  Intervention: Drug: Placebo

• Publications *: Not Provided

Recruitment Information

• Recruitment Status: Completed
• Actual Enrollment (submitted: December 3, 2020): 65
• Original Estimated Enrollment (submitted: September 23, 2019): 60
• Actual Study Completion Date: August 23, 2021
• Actual Primary Completion Date: August 23, 2021 (Final data collection date for primary outcome measure)

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)

Sex/Gender

• Sexes Eligible for Study: All

• Ages: 12 Years to 50 Years (Child, Adult)
• Accepts Healthy Volunteers: No
• Contacts: Contact information is only displayed when the study is recruiting subjects
• Listed Location Countries: United States

Administrative Information

• NCT Number: NCT04102501
• Other Study ID Numbers: RT001-006
• Has Data Monitoring Committee: Yes

U.S. FDA-regulated Product

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

• IPD Sharing Statement: Not Provided
• Current Responsible Party: Retrotope, Inc.
• Original Responsible Party: Same as current
• Current Study Sponsor: Retrotope, Inc.
• Original Study Sponsor: Same as current
• Collaborators: Not Provided

Investigators

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

• PRS Account: Retrotope, Inc.
• Verification Date: April 2022