A Study of the Efficacy and Safety of 24 Week Treatment With REN001 in Patients With Primary Mitochondrial Myopathy (STRIDE)
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|ClinicalTrials.gov Identifier: NCT04535609|
Recruitment Status : Recruiting
First Posted : September 2, 2020
Last Update Posted : December 8, 2022
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|Condition or disease||Intervention/treatment||Phase|
|Primary Mitochondrial Myopathy||Drug: REN001 Drug: Placebo||Phase 2|
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||200 participants|
|Intervention Model:||Parallel Assignment|
|Masking:||Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)|
|Official Title:||A Double-blind, Placebo-controlled Study to Evaluate the Efficacy and Safety of 24 Weeks Treatment With REN001 in Patients With Primary Mitochondrial Myopathy|
|Actual Study Start Date :||May 21, 2021|
|Estimated Primary Completion Date :||September 2023|
|Estimated Study Completion Date :||October 2023|
Placebo Comparator: Matched placebo
- Change from Baseline in distance walked during the Walk Test [ Time Frame: Week 24 ]Distance walked in meters
- Change from Baseline at Week 24 in the Modified Fatigue Impact Scale (MFIS) physical sub-scale score [ Time Frame: Week 24 ]Physical Scale 0-36 scoring
- Patient Global Impression of Change (PGIC) score at Week 24 [ Time Frame: Week 24 ]7 point scale: Very much improved, Moderately improved, Minimally improved, No change, Minimally worse, Moderately worse, Very much worse
- Incidence and severity of adverse events, serious adverse events, and withdrawals due to adverse events [ Time Frame: Baseline to Week 24 ]Number and Severity
- Change from Baseline in number of sit to stands in the sit to stand test [ Time Frame: Week 24 ]Individual counts
- Change from Baseline in number of steps [ Time Frame: Week 24 ]Step count from pedometer
- Change from Baseline in Patient Global Impression of Severity (PGIS) score [ Time Frame: Week 24 ]5 point scale: Absent, Mild, Moderate, Severe, Very Severe
- Change from Baseline in MFIS total, cognitive and psychosocial sub-scale scores [ Time Frame: Week 24 ]Total MFIS 0-84, cognitive 0-40, psychosocial 0-8
- Change from Baseline in Brief Pain Inventory pain severity and pain interference scores [ Time Frame: Week 24 ]Numerical rating scale 0-10
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|Ages Eligible for Study:||18 Years and older (Adult, Older Adult)|
|Sexes Eligible for Study:||All|
|Accepts Healthy Volunteers:||No|
- Subjects age 18 years or older with PMM as defined by the International Workshop: Outcome measures and clinical trial readiness in primary mitochondrial myopathies in children and adult (Mancuso et al 2017).
- A confirmed PMM diagnosis due to known pathogenic gene mutation or deletion of the mitochondrial genome. The Sponsor may authorize local genetic testing at Screening, if required, but results must be available prior to randomization of the subject.
- Documented PMM primarily characterized by exercise intolerance or active muscle pain.
- Subjects must be ambulatory and able to perform the walking tests independently (walking aids are allowed).
- Have no changes to any therapeutic exercise regimen within 30 days prior to Day 1 and be willing to remain on the same therapeutic exercise regimen for the duration of the study.
- Females should be either of non-child-bearing potential or must agree to use highly effective methods of contraception from Screening through to 30 days after last dose in the study. Males with partners who are WOCBP must also use contraception.
- Concomitant medications (including supplements) must be stable for at least 1 month prior to enrolment and throughout participation in the study.
- Evidence of a personally signed and dated informed consent document indicating that the subject has been informed of all pertinent aspects of the study.
- Participation in a prior REN001 (previously known as HPP-593) study.
- Currently taking or anticipated to need a PPAR agonist during the study.
- Subjects with bone deformities or motor abnormalities other than related to the mitochondrial myopathy that may interfere with the outcome measures.
- Clinically significant kidney disease or impairment calculated as eGFR Grade 2 or above <60ml/min/1.73m2 using the Chronic Kidney Disease Epidemiology Collaboration (CKD-EPI) creatinine equation at Screening.
- Clinically significant liver disease or impairment of AST or ALT Grade 2 or above (>2.5 x ULN), or Total bilirubin > 1.6 x ULN or >ULN with other signs and symptoms of hepatotoxicity at Screening.
- Subjects with uncontrolled diabetes and/or a Screening HbA1c of ≥11%.
- Evidence of significant concomitant clinical disease that may need a change in management during the study or could interfere with the conduct or safety of this study. (Stable well-controlled chronic conditions such hypercholesterolemia, gastroesophageal reflux, or depression under control with medication (other than tricyclic antidepressants), are acceptable provided the symptoms and medications would not be predicted to compromise safety or interfere with the tests and interpretations of this study.)
- Subjects with a history of cancer. A history of in situ basal cell carcinoma in the skin is allowed.
- Clinically significant cardiac disease and/or clinically significant ECG abnormalities such as 2nd degree heart block, symptomatic tachyarrhythmia or unstable arrythmia (right bundle branch block, left fascicular block and long PR interval are not excluded) that in the opinion of the Investigator should exclude the subject from completing exercise tests.
- Evidence of hospitalization for rhabdomyolysis within the year prior to enrolment.
- Pregnant or nursing females.
- History of sensitivity to PPAR agonists.
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): NCT04535609
|Contact: Clinical Trial Coordinator||+44 (0) 1304 firstname.lastname@example.org|
|Principal Investigator:||Amel Karaa, MD||Massachusetts General Hospital (MGH)|
|Responsible Party:||Reneo Pharma Ltd|
|Other Study ID Numbers:||
|First Posted:||September 2, 2020 Key Record Dates|
|Last Update Posted:||December 8, 2022|
|Last Verified:||December 2022|
|Individual Participant Data (IPD) Sharing Statement:|
|Plan to Share IPD:||No|
|Studies a U.S. FDA-regulated Drug Product:||Yes|
|Studies a U.S. FDA-regulated Device Product:||No|
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