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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.

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.

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 60 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
Estimated Primary Completion Date : February 2021
Estimated Study Completion Date : April 2021


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.




Primary Outcome Measures :
  1. 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


Secondary Outcome Measures :
  1. 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.

  2. 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

  3. 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.

  4. 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.



Information from the National Library of Medicine

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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)

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04102501


Contacts
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Contact: Jayshree Krishnaswami 8583424498 jayshree@retrotope.com
Contact: Mark Midei, MD 4088345729 mark@retrotope.com

Locations
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United States, California
Collaborative Neuroscience Network, LLC Recruiting
Long Beach, California, United States, 90806
UCLA Not yet recruiting
Los Angeles, California, United States, 90095
United States, Florida
USF Ataxia Research Center Recruiting
Tampa, Florida, United States, 33612
United States, Iowa
University of Iowa Stead Family Children's Hospital Not yet recruiting
Iowa City, Iowa, United States, 52242
United States, Pennsylvania
Children's Hospital of Philadelphia Recruiting
Philadelphia, Pennsylvania, United States, 19104
Sponsors and Collaborators
Retrotope, Inc.
Investigators
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Study Director: Peter Milner, MD Chief Medical Officer

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Responsible Party: Retrotope, Inc.
ClinicalTrials.gov Identifier: NCT04102501    
Other Study ID Numbers: RT001-006
First Posted: September 25, 2019    Key Record Dates
Last Update Posted: January 10, 2020
Last Verified: November 2019

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
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Ataxia
Cerebellar Ataxia
Friedreich Ataxia
Dyskinesias
Neurologic Manifestations
Nervous System Diseases
Signs and Symptoms
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