Friedreich's AtaxiaPhase 3NCT04102501

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

A Phase 3 study testing RT001 in people with Friedreich's Ataxia, run by Biojiva LLC at 6 sites in United States.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Trial summary

The purpose of this study is to assess the Efficacy, Long Term Safety and Tolerability of RT001 in subjects with Friedreich's Ataxia Read more about this study

Who is it for?: Ages 12 to 50 years
Conditions the study is for: Friedreich's Ataxia
What is being tested?: RT001
Type of treatment: Pill or oral medicine
Could I receive a placebo?: Yes, some participants may receive a placebo
How many people: 65 participants
Where: 6 sitesUnited States
Study dates: 30 Oct 2019to 23 Aug 2021
Phase: Phase 3
Are there results?: Yes, posted in the registrySummary in the doctors' view

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

Ages 12 to 50 years
All sexes
No healthy volunteers
Child, Adult

Who may be able to join

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

Who cannot join

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)

Trial locations and contacts

6 sites in 1 country.

City, postcode or hospital

6 sites in 1 country

United States6 sites

University of Florida
Gainesville, Florida, 32608
University of Iowa Stead Family Children's Hospital
Iowa City, Iowa, 52242
Collaborative Neuroscience Network, LLC
Long Beach, California, 90806
UCLA
Los Angeles, California, 90095
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104
USF Ataxia Research Center
Tampa, Florida, 33612

Other trials in United States

About this study

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

The purpose of this study is to assess the Efficacy, Long Term Safety and Tolerability of RT001 in subjects with Friedreich's Ataxia

Read the 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.

Conditions: Friedreich's Ataxia
Being tested: RT001, Placebo
Medical subject headings: Friedreich Ataxia

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

RT001Drug
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.
PlaceboDrug
The placebo product is composed of encapsulated USP safflower oil. The placebo capsules are identical in appearance and size to RT001.

Study groups in NCT04102501
Study group	Role	What they receive
RT001	Experimental	* 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 Receives: Drug: RT001
Placebo	Placebo comparator	* 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 Receives: Drug: Placebo

What is being measured

The primary outcome is the main question the study is designed to answer.

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
Measured: 11 months

Secondary outcomes (1)

Change From Baseline in the Timed 1 Minute Walk Distance
Change from baseline Distance walked in 1 minute at 11 months
Measured: 11 months

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

30 Oct 201923 Aug 2021

First posted25 Sep 2019
Study start30 Oct 2019
Primary completion23 Aug 2021
Study completion23 Aug 2021

Resources

Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
Other trials for Friedreich's AtaxiaStudies of the same condition, open and under way.
What the trial phases meanHow a treatment moves from first tests in people to approval.

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design

Study type: Interventional
Phase: Phase 3
Primary purpose: Treatment
Allocation: Randomised
Intervention model: Parallel groups
Masking: Quadruple blind
Who is masked: Participant, Care provider, Investigator, Outcomes assessor
Enrolment: 65 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT04102501
Sponsor's protocol ID: RT001-006
Status last verified: Apr 2022
First submitted: 23 Sep 2019
Last updated: 27 May 2022
Results posted: Yes, on ClinicalTrials.gov
Data monitoring committee: Yes
FDA-regulated drug: Yes
FDA-regulated device: No

Want to know more?

The sponsor has not listed a contact. The official record may have more details.

Talk to your doctor first and take this page with you. Only the study team can confirm whether you can join.

Questions to ask the study team

What is the study trying to find out?
What would I need to do, and how often would I visit?
Could I receive a placebo instead of the treatment?
What are the possible risks and benefits for me?
Will it cost me anything? Are travel or time paid for?
Can I keep seeing my own doctor during the study?
What happens when the study ends?

See locations

RecruitingObservational study
Rare Disease Patient Registry & Natural History Study - Coordination of Rare Diseases at Sanford
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An Open-Label Study of CTI-1601 in Subjects With Friedreich's Ataxia
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Not yet recruitingObservational study
A Study to Learn More About Friedrich's Ataxia in People Living in the Gulf Cooperation Council (GCC) Region of the World
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RecruitingObservational study
Clinical Course Of Disease In Participants With FA-CM
Friedreich Ataxia, Cardiomyopathy
Not yet recruitingPhase 2
GV101 for Friedreich's Ataxia (FA) Treatment
Friedreich's Ataxia

All Friedreich's Ataxia trials

Study at a glance

From the registry record, last updated by the sponsor on 27 May 2022. Recruitment status is as the sponsor reported it: confirm with the study team.

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
ClinicalTrials.gov ID: NCT04102501
Acronym / sponsor ID: RT001-006
Conditions: Friedreich's Ataxia
Phase: Phase 3
Study type: Interventional
Recruitment status: Completed
Enrolment: 65 participants (actual)
Lead sponsor: Biojiva LLCIndustry (pharma, biotech, devices)
Study start: 30 Oct 2019 (actual)
Primary completion: 23 Aug 2021 (actual)
Study completion: 23 Aug 2021 (actual)
First posted: 25 Sep 2019 (actual)
Results: Posted 27 May 2022

Clinical summary

The research question as the sponsor registered it. This describes what the study plans to test, not what it found.

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

Study design

Primary purpose: Treatment
Allocation: Randomised
Intervention model: Parallel groups
Masking: Quadruple blind (participant, care provider, investigator, outcomes assessor)
Number of arms: 2

Arm	Type	Receives
RT001	Experimental	Drug: 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
Placebo	Placebo comparator	Drug: 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

Who may qualify

Ages: 12 to 50 years
Sex: All
Healthy volunteers: Not accepted
Age groups: Child, Adult

Key inclusion criteria (5 listed)

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 (7 listed)

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

Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.

Interventions

Intervention	Type	Arms
RT001RT001 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	RT001
PlaceboThe placebo product is composed of encapsulated USP safflower oil. The placebo capsules are identical in appearance and size to RT001.	Drug	Placebo

Primary outcome

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

1 secondary outcome

Change From Baseline in the Timed 1 Minute Walk Distance
Time frame: 11 months

Results and safety

Results posted to the registry on 27 May 2022, as reported by the sponsor. Figures are copied from the record, not re-analysed. Full results on ClinicalTrials.gov.

Participant flow

Group	Started	Completed
RT001	33	25
Placebo	32	20

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

Time frame: 11 months. Mean (Standard Error). Unit: ml O2/min/kg

Measure	RT001 n = 25	Placebo n = 20
Value	0.05 (0.04)	0.08 (0.04)

RT001 vs Placebo: p = 0.5858, Mixed Models Analysis

1 secondary outcome is reported in the full results.

Adverse events

Time frame: 12 months. Other adverse events reported at 5% frequency or more

Group	Serious adverse events	Other adverse events	Deaths
RT001	1 of 33 (3%)	18 of 33 (54.5%)	0 of 33
Placebo	1 of 32 (3.1%)	19 of 32 (59.4%)	0 of 32

Loading the posted results from ClinicalTrials.gov…

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 27 May 2022.
Posted resultsParticipant flow, baseline, all outcome measures and adverse events.

Related trials for Friedreich's Ataxia

RecruitingObservational study
Rare Disease Patient Registry & Natural History Study - Coordination of Rare Diseases at Sanford
Rare Disorders, Undiagnosed Disorders
RecruitingPhase 2
An Open-Label Study of CTI-1601 in Subjects With Friedreich's Ataxia
Friedreich Ataxia
Not yet recruitingObservational study
A Study to Learn More About Friedrich's Ataxia in People Living in the Gulf Cooperation Council (GCC) Region of the World
Friedreich's Ataxia
RecruitingObservational study
Clinical Course Of Disease In Participants With FA-CM
Friedreich Ataxia, Cardiomyopathy
Not yet recruitingPhase 2
GV101 for Friedreich's Ataxia (FA) Treatment
Friedreich's Ataxia

Referral and contact

AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.

Peter Milner, MD study director

Chief Medical Officer

6 sites in 1 country: see every site and its status.

Questions about this trial

What is this clinical trial studying?

A Phase 3 study testing RT001 in people with Friedreich's Ataxia, run by Biojiva LLC at 6 sites in United States.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 5 inclusion and 7 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 27 May 2022. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 6 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

Yes. Results are posted in the registry (first posted 27 May 2022). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Change From Baseline to 11 Months in Maximum Consumption of Oxygen (mlO2/kg/Min) Using Cardiopulmonary Exercise Testing (CPET) (time frame: 11 months). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT04102501.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 27 May 2022; AzTrials last refreshed its copy on 31 Oct 2026.