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

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ClinicalTrials.gov Identifier: NCT04102501
Recruitment Status : Recruiting
First Posted : September 25, 2019
Last Update Posted : January 10, 2020
Sponsor:
Information provided by (Responsible Party):
Retrotope, Inc.

Tracking Information
First Submitted Date  ICMJE September 23, 2019
First Posted Date  ICMJE September 25, 2019
Last Update Posted Date January 10, 2020
Actual Study Start Date  ICMJE October 30, 2019
Estimated Primary Completion Date February 2021   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: November 1, 2019)
Peak workload change from baseline to 11 months using cardiopulmonary exercise testing (CPET) [ Time Frame: 11 months ]
To measure the percentage change in peak workload from baseline to Month 11 using CPET
Original Primary Outcome Measures  ICMJE
 (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
Change History Complete list of historical versions of study NCT04102501 on ClinicalTrials.gov Archive Site
Current Secondary Outcome Measures  ICMJE
 (submitted: November 1, 2019)
  • Effect of RT001 on modified Friedreich Ataxia Rating Scale (mFARS) [ Time Frame: 11 months ]
    The modified FARS is derived from the FARS Neuro Scale. The FARS Neuro Scale is based on the neurological examination of the Bulbar System, Upper Limb Coordination, Lower Limb Coordination, Peripheral Nervous System and Upright Stability Function. The mFARS will not include sub-scores from Peripheral Nervous System.
  • Distance walked during a 1-minute walk (T1MW) [ Time Frame: 11 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: 11 months ]
    To measure the change in score from baseline to Month 11 on a fatigue scale. This scale rates your fatigue over the last 7 days and is rated on a 5 point scale with a range of responses from not at all, a little bit, some what, quite a bit and very much.
  • Effects of RT001 on Clinical Global Impression [CGI] [ Time Frame: 11 months ]
    The CGI is a 3-item observer rated scale that measures illness severity, global improvement and therapeutic response.The illness severity is rated on a 7-point scale, with the severity of illness scale using a range of responses from 1 (normal) through 7 (amongst the most severely ill patients). The global improvement scores range from 1 (very much improved) through to 7 (very much worse). Treatment response ratings should take account of both therapeutic efficacy and treatment-related adverse events and range from 0 (marked improvement and no side-effects) and 4 (unchanged or worse and side-effects outweigh the therapeutic effects). Each component of the CGI is rated separately; the instrument does not yield a global score.
Original Secondary Outcome Measures  ICMJE
 (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  ICMJE A Study to Assess Efficacy, Long Term Safety and Tolerability of RT001 in Subjects With Friedreich's Ataxia
Official Title  ICMJE 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  ICMJE Interventional
Study Phase  ICMJE Phase 3
Study Design  ICMJE Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Condition  ICMJE Friedreich's Ataxia
Intervention  ICMJE
  • 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  ICMJE
  • 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

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Recruiting
Estimated Enrollment  ICMJE
 (submitted: September 23, 2019)
60
Original Estimated Enrollment  ICMJE Same as current
Estimated Study Completion Date  ICMJE April 2021
Estimated Primary Completion Date February 2021   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 12 Years to 50 Years   (Child, Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE
Contact: Jayshree Krishnaswami 8583424498 jayshree@retrotope.com
Contact: Mark Midei, MD 4088345729 mark@retrotope.com
Listed Location Countries  ICMJE United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT04102501
Other Study ID Numbers  ICMJE 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  ICMJE Not Provided
Responsible Party Retrotope, Inc.
Study Sponsor  ICMJE Retrotope, Inc.
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Study Director: Peter Milner, MD Chief Medical Officer
PRS Account Retrotope, Inc.
Verification Date November 2019

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP