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

Primary Purpose

Friedreich's Ataxia

Status

Completed

Phase

Phase 3

Locations

United States

Study Type

Interventional

Intervention

RT001

Placebo

About this trial

This is an interventional treatment trial for Friedreich's Ataxia

Eligibility Criteria

12 Years - 50 Years (Child, Adult)All SexesDoes not accept healthy volunteers

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)

Sites / Locations

Collaborative Neuroscience Network, LLC
UCLA
University of Florida
USF Ataxia Research Center
University of Iowa Stead Family Children's Hospital
Children's Hospital of Philadelphia

Arms of the Study

Arm 1

Arm 2

Arm Type

Experimental

Placebo Comparator

Arm Label

RT001

Placebo

Arm Description

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

Outcomes

Primary Outcome Measures

Change From Baseline to 11 Months in Maximum Consumption of Oxygen (mlO2/kg/Min) Using Cardiopulmonary Exercise Testing (CPET)

To measure the change in MVO2 from baseline to Month 11 using CPET

Secondary Outcome Measures

Change From Baseline in the Timed 1 Minute Walk Distance

Change from baseline Distance walked in 1 minute at 11 months

Full Information

NCT ID

NCT04102501

First Posted

September 23, 2019

Last Updated

May 4, 2022

Sponsor

Retrotope, Inc.

1. Study Identification

Unique Protocol Identification Number

NCT04102501

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

Study Type

Interventional

2. Study Status

Record Verification Date

April 2022

Overall Recruitment Status

Completed

Study Start Date

October 30, 2019 (Actual)

Primary Completion Date

August 23, 2021 (Actual)

Study Completion Date

August 23, 2021 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Sponsor

Name of the Sponsor

Retrotope, Inc.

4. Oversight

Studies a U.S. FDA-regulated Drug Product

Yes

Studies a U.S. FDA-regulated Device Product

Data Monitoring Committee

Yes

5. 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

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

Friedreich's Ataxia

7. Study Design

Primary Purpose

Treatment

Study Phase

Phase 3

Interventional Study Model

Parallel Assignment

Masking

ParticipantCare ProviderInvestigatorOutcomes Assessor

Allocation

Randomized

Enrollment

65 (Actual)

8. Arms, Groups, and Interventions

Arm Title

RT001

Arm Type

Experimental

Arm Description

Arm Title

Placebo

Arm Type

Placebo Comparator

Arm Description

Intervention Type

Drug

Intervention Name(s)

RT001

Intervention Description

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.

Intervention Type

Drug

Intervention Name(s)

Placebo

Intervention Description

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

Primary Outcome Measure Information:

Title

Change From Baseline to 11 Months in Maximum Consumption of Oxygen (mlO2/kg/Min) Using Cardiopulmonary Exercise Testing (CPET)

Description

To measure the change in MVO2 from baseline to Month 11 using CPET

Time Frame

11 months

Secondary Outcome Measure Information:

Title

Change From Baseline in the Timed 1 Minute Walk Distance

Description

Change from baseline Distance walked in 1 minute at 11 months

Time Frame

11 months

10. Eligibility

Sex

All

Minimum Age & Unit of Time

12 Years

Maximum Age & Unit of Time

50 Years

Accepts Healthy Volunteers

Eligibility Criteria

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)

Overall Study Officials:

First Name & Middle Initial & Last Name & Degree

Peter Milner, MD

Organizational Affiliation

Chief Medical Officer

Official's Role

Study Director

Facility Information:

Facility Name

Collaborative Neuroscience Network, LLC

City

Long Beach

State/Province

California

ZIP/Postal Code

90806

Country

United States

Facility Name

UCLA

City

Los Angeles

State/Province

California

ZIP/Postal Code

90095

Country

United States

Facility Name

University of Florida

City

Gainesville

State/Province

Florida

ZIP/Postal Code

32608

Country

United States

Facility Name

USF Ataxia Research Center

City

Tampa

State/Province

Florida

ZIP/Postal Code

33612

Country

United States

Facility Name

University of Iowa Stead Family Children's Hospital

City

Iowa City

State/Province

Iowa

ZIP/Postal Code

52242

Country

United States

Facility Name

Children's Hospital of Philadelphia

City

Philadelphia

State/Province

Pennsylvania

ZIP/Postal Code

19104

Country

United States

12. IPD Sharing Statement

Learn more about this trial

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

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