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 :
Active, not recruiting
First Posted : September 25, 2019
Last Update Posted : March 9, 2021
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Condition or disease | Intervention/treatment | Phase |
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Friedreich's Ataxia | Drug: RT001 Drug: Placebo | Phase 3 |
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 |
Estimated Primary Completion Date : | November 2021 |
Estimated Study Completion Date : | December 2021 |

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

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Ages Eligible for Study: | 12 Years to 50 Years (Child, Adult) |
Sexes Eligible for Study: | All |
Accepts Healthy Volunteers: | No |
Key Inclusion Criteria:
- 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
Key Exclusion Criteria:
- 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)

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
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 |
Study Director: | Peter Milner, MD | Chief Medical Officer |
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: | March 9, 2021 |
Last Verified: | December 2020 |
Studies a U.S. FDA-regulated Drug Product: | Yes |
Studies a U.S. FDA-regulated Device Product: | No |
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 |