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Fatty Acid Oxidation Inhibitor

REN001 for Mitochondrial Myopathy

Phase 2 & 3

Waitlist Available

Led By Grainne Gorman, MD

Research Sponsored by Reneo Pharma Ltd

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Have PMM which continues to be primarily characterized by exercise intolerance or active muscle pain.

Willing and able to swallow the REN001 gelatin capsules.

Timeline

Screening 3 weeks

Treatment Varies

Follow Up baseline to month 24

Awards & highlights

Study Summary

This trial evaluates the long-term safety and tolerability of a drug for people with PMM due to mtDNA or nDNA mutations. Includes both those already on the drug and those trying it for the first time.

Who is the study for?

This trial is for adults with Primary Mitochondrial Myopathy (PMM) due to genetic mutations, who have completed prior REN001 studies or are new to the treatment. Participants must be able to swallow capsules, maintain stable medication regimens, and use effective contraception if of childbearing potential. Exclusions include significant health risks, unstable chronic conditions, substance dependencies, severe liver or kidney disease, uncontrolled diabetes, anticipated need for PPAR agonists other than REN001, a history of cancer except certain skin cancers.Check my eligibility

What is being tested?

The study tests the long-term safety and tolerability of daily doses of REN001 in patients with PMM caused by mitochondrial DNA (mtDNA) or nuclear DNA (nDNA) mutations. It includes those previously treated in related studies as well as REN001-naïve subjects with nDNA mutations.See study design

What are the potential side effects?

While specific side effects are not listed here, participants will be monitored for any adverse reactions due to long-term use of REN001. These may include typical drug-related side effects such as digestive issues, allergic reactions or sensitivities specific to this medication's profile.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

Select...

I experience significant muscle pain or struggle with exercise due to my PMM.

Select...

I can swallow gelatin capsules.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ baseline to month 24

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~baseline to month 24

This trial's timeline: 3 weeks for screening, Varies for treatment, and baseline to month 24 for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.

Primary outcome measures

Incidence and severity of adverse events, serious adverse events, and withdrawals due to adverse events

Secondary outcome measures

Absolute values, changes from baseline, and incidence of potentially clinically significant changes in laboratory safety tests, electrocardiograms, supine vital signs, and eye assessments

Change Work Productivity and Activity Impairment Questionnaire: Specific Health Problem (WPAI:SHP) score

Change in 36-Item Short Form Health Survey (SF-36) score

+6 more

Trial Design

1Treatment groups

Experimental Treatment

Group I: REN001Experimental Treatment1 Intervention

100 mg once daily

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

ReN001

Not yet FDA approved

0 / 2Eligibility criteria met

Find a Location

Who is running the clinical trial?

Reneo Pharma LtdLead Sponsor

5 Previous Clinical Trials

340 Total Patients Enrolled

Grainne Gorman, MDPrincipal InvestigatorNewcastle Hospital NHS Foundation Trust

2 Previous Clinical Trials

63 Total Patients Enrolled

Media Library

REN001 (Fatty Acid Oxidation Inhibitor) Clinical Trial Eligibility Overview. Trial Name: NCT05267574 — Phase 2 & 3

Mitochondrial Myopathy Research Study Groups: REN001

Mitochondrial Myopathy Clinical Trial 2023: REN001 Highlights & Side Effects. Trial Name: NCT05267574 — Phase 2 & 3

REN001 (Fatty Acid Oxidation Inhibitor) 2023 Treatment Timeline for Medical Study. Trial Name: NCT05267574 — Phase 2 & 3

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

Is there an opportunity to join this research endeavor currently?

"According to the data hosted on clinicaltrials.gov, it is clear that this medical trial has ended its recruitment phase - having started February 1st 2022 and last edited June 8th 2023. Nevertheless, there are 37 other trials actively seeking participants at present."

Answered by AI

Are there many venues administering this clinical trial in North America?

"The Alfred Hospital in Melbourne, University Hospital Leuven in Leuven, and M.A.G.I.C Clinic (Metabolics and Genetics in Calgary) in Calgary are only a few of the 29 sites currently recruiting for this study across various countries."

Answered by AI

Recent research and studies

clinicaltrials.govStudy

An Open Label, Long Term Safety Study of REN001 in Primary Mitochondrial Myopathy Patients (Stride Ahead)

2022. "An Open Label, Long Term Safety Study of REN001 in Primary Mitochondrial Myopathy Patients (Stride Ahead)". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT05267574.