Trial record 1 of 1 for:
04535609
An Efficacy and Safety Study of 24 Week Treatment With Mavodelpar (REN001) in Primary Mitochondrial Myopathy Patients (STRIDE)
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| ClinicalTrials.gov Identifier: NCT04535609 |
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Recruitment Status :
Active, not recruiting
First Posted : September 2, 2020
Last Update Posted : May 30, 2023
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Sponsor:
Reneo Pharma Ltd
Information provided by (Responsible Party):
Reneo Pharma Ltd
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Brief Summary:
This is a randomized, double-blind, placebo-controlled, parallel group, multi-centre, study designed to investigate the efficacy and safety of REN001 administered once daily over a 24-week period to patients with PMM.
| Condition or disease | Intervention/treatment | Phase |
|---|---|---|
| Primary Mitochondrial Myopathy | Drug: Mavodelpar Drug: Placebo | Phase 2 |
| Study Type : | Interventional (Clinical Trial) |
| Actual Enrollment : | 213 participants |
| Allocation: | Randomized |
| Intervention Model: | Parallel Assignment |
| Masking: | Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor) |
| Primary Purpose: | Treatment |
| 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 |
Resource links provided by the National Library of Medicine
MedlinePlus related topics:
Muscle Disorders
| Arm | Intervention/treatment |
|---|---|
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Experimental: Mavodelpar
Once daily
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Drug: Mavodelpar
Once daily
Other Name: REN001 |
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Placebo Comparator: Matched placebo
Once daily
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Drug: Placebo
Once daily |
Primary Outcome Measures :
- Change in distance walked during a 12 Minute Walk Test [ Time Frame: Baseline to Week 24 ]Distance walked in meters
Secondary Outcome Measures :
- Change in PROMIS Short Form - Fatigue 13a (FACIT-fatigue) scores [ Time Frame: Baseline to Week 24 ]The PROMIS is a 13-item questionnaire to describe fatigue and its impact upon daily activities and function. Each item is scored between 1=Not At All and 5=Very Much
Other Outcome Measures:
- 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 in number of sit to stands completed during a 30 second sit to stand (30STS) test [ Time Frame: Baseline to Week 24 ]Number completed
- Change in step counts as measured by a pedometer [ Time Frame: Baseline to Week 24 ]Number of steps
- Change in Modified Fatigue Impact Scale (MFIS) score [ Time Frame: Baseline to Week 24 ]The MFIS is a 21-item questionnaire to describe the impact of fatigue on physical, cognitive, and psychosocial functioning. The questionnaire includes 9 physical, 10 cognitive and 2 psychosocial items with each item scored between 0=Never and 4=Almost Always
- Change in Patient Global Impression of Severity (PGIS) score [ Time Frame: Baseline to Week 24 ]The PGIS is a 2-item questionnaire to describe the severity of fatigue and muscle symptoms. Each item is scored as Very Much Worse, Moderately Worse, Minimally Worse, No Change, Minimally Improved, Moderately Improved, or Very Much Improved
- Change in 36-Item Short Form Health Survey (SF-36) score [ Time Frame: Baseline to Week 24 ]The SF-36 is a 36-item questionnaire to describe health status and quality of life. The questionnaire includes 8 domains (vitality, physical functioning, bodily pain, general health perceptions, role limitations due to physical health, role limitations due to emotional health, social role functioning, and mental health). Items are scored, summed into domains, and transformed into a scale of 0-100
- Change in Brief Pain Inventory (BPI) score [ Time Frame: Baseline to Week 24 ]The BPI is a 15-item questionnaire to describe severity of pain and its interference on functioning. The questionnaire includes 4 pain severity items each scored between 0=No Pain and 10= Pain, and 7 pain interference items each scored between 0=Does not Interfere and 10=Completely Interferes
- Change Work Productivity and Activity Impairment Questionnaire: Specific Health Problem (WPAI:SHP) score [ Time Frame: Baseline to Week 24 ]The WPAI:SHP is a 6-item questionnaire to describe impairment in work and activities due to a certain disease. Items are scored, summed, and transformed into a scale of 0-100%
- Change in Patient Global Impression of Change (PGIC) score [ Time Frame: Baseline to Week 24 ]The PGIC is a 2-item questionnaire to describe the change in fatigue and muscle symptoms since starting the study. Each item is scored as Very Much Worse, Moderately Worse, Minimally Worse, No Change, Minimally Improved, Moderately Improved, or Very Much Improved
Information from the National Library of Medicine
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| Ages Eligible for Study: | 18 Years and older (Adult, Older Adult) |
| Sexes Eligible for Study: | All |
| Accepts Healthy Volunteers: | No |
Criteria
Inclusion Criteria:
- 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.
Exclusion:
- 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.
Information from the National Library of Medicine
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
Locations
Show 41 study locations
Sponsors and Collaborators
Reneo Pharma Ltd
Investigators
| Principal Investigator: | Amel Karaa, MD | Massachusetts General Hospital (MGH) |
| Responsible Party: | Reneo Pharma Ltd |
| ClinicalTrials.gov Identifier: | NCT04535609 |
| Other Study ID Numbers: |
REN001-201 |
| First Posted: | September 2, 2020 Key Record Dates |
| Last Update Posted: | May 30, 2023 |
| Last Verified: | May 2023 |
| 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 |
Additional relevant MeSH terms:
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Muscular Diseases Mitochondrial Myopathies Musculoskeletal Diseases Neuromuscular Diseases |
Nervous System Diseases Mitochondrial Diseases Metabolic Diseases |