Primary Mitochondrial MyopathyPhase 2STRIDENCT04535609
An Efficacy and Safety Study of 24 Week Treatment With Mavodelpar (REN001) in Primary Mitochondrial Myopathy Patients
A Phase 2 study testing Mavodelpar in people with Primary Mitochondrial Myopathy, run by Reneo Pharma Ltd at 41 sites in 15 countries.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Primary Mitochondrial Myopathy
- What is being tested?
- Mavodelpar
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 213 participants
- Where
- 41 sitesin 15 countries
- Study dates
- 21 May 2021to 5 Oct 2023
- Phase
- Phase 2
- 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 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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.
Who cannot join
- 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%.
Show all 12Show fewer
- 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.
Trial locations and contacts
41 sites in 15 countries.
41 sites in 15 countries
United States9 sites
Akron Children's Hospital
Akron, Ohio, 44308
Massachusetts General Hospital
Boston, Massachusetts, 02114
University of Texas SouthWestern Medical Center
Dallas, Texas, 75390
Myology Institute, University of Florida
Gainesville, Florida, 32608
Centre for the Treatment of Pediatric Neurodegenerative Disease, University of Texas McGovern Medical School
Houston, Texas, 77025
University of California, San Diego
La Jolla, California, 92093
Columbia University Irving Medical Center
New York, New York, 10032
The Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104
UPMC Children's Hospital of Pittsburgh
Pittsburgh, Pennsylvania, 15224
France6 sites
Centre Hospitalier Universitaire d' Angers
Angers, Pays de la Loire Region, 49933
Hôpital Neurologique Pierre Wertheimer
Bron, 69599
Hôpital Roger Salengro
Lille, Hauts-de-France, 59037
CHU de Nice
Nice, 06202
Hôpital Pitié-Salpêtrière
Paris, Île-de-France Region, 75651
Hôpitaux Universitaires de Strasbourg
Strasbourg, Grand Est, 67200
Italy5 sites
IRCCS Istituto delle Scienze Neurologiche
Bologna, 40139
A.O.U Policlinico di Messina U.O.C Neurologia e Malattie Neuromuscolari
Messina, Sicily, 98125
Fondazione IRCCS Istituto Neurologico "Carlo Besta" UOC Genetica Medica e Neurogenetica
Milan, Lombardy, 20126
Azienda Ospedaliero-Universitaria Pisana Dipartimento di specialita' mediche UOC Neurologia
Pisa, Tuscany, 56126
Fondazione Policlinico Universitario Agostino Gemelli IRCCS Neurophysiopathology Unit
Rome, Lazio, 00168
Australia3 sites
PARC Clinical Research
Adelaide, South Australia, 5000
The Alfred Hospital
Melbourne, Victoria, 3004
Royal North Shore Hospital
St Leonards, New South Wales, 2065
Spain3 sites
Hospital Clinic de Barcelona
Barcelona, 8036
Hospital Universitario 12 de Octubre
Madrid, 08041
Hospital Universitari i Politècnic La Fe
Valencia, 46026
United Kingdom3 sites
Queen Square Centre for Neuromuscular Diseases
London, Greater London, WC1N 3BG
The Newcastle upon Tyne Hospitals NHS Foundation Trust
Newcastle upon Tyne, Tyne and Wear, NE1 4LP
Salford Royal NHS Foundation Trust
Salford, Greater Manchester, M6 8HD
Canada2 sites
M.A.G.I.C. Clinic (Metabolics and Genetics in Calgary)
Calgary, Alberta, T2E 7Z4
Adult Metabolic Diseases Clinic, Vancouver General Hospital
Vancouver, British Columbia, V5Z 1M9
Germany2 sites
University Hospital Bonn Clinic and Polyclinic for Neurology
Bonn, 53127
Medical Center of the University of Munich Friedrich Baur Institute at the Neurological Clinic and Polyclinic
Munich, 80336
Hungary2 sites
Semmelweis University Insitute of Genomics and Rare Disorders
Budapest, 1082
University of Pécs Clinical Centre, Department of Neurology
Pécs, 7623
About this study
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
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.
- Conditions
- Primary Mitochondrial Myopathy
- Being tested
- Mavodelpar, Placebo
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
MavodelparDrug
Also called REN001
Once daily
PlaceboDrug
Once daily
| Study group | Role | What they receive |
|---|---|---|
| Mavodelpar | Experimental | Once daily Receives: Drug: Mavodelpar |
| Matched placebo | Placebo comparator | Once daily Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change in Distance Walked During a 12 Minute Walk Test
Distance walked in meters
Measured: Baseline to Week 24
Secondary outcomes (1)
Change in PROMIS Short Form - Fatigue 13a (FACIT-fatigue) Scores
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
Measured: Baseline to Week 24
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted2 Sep 2020
- Study start21 May 2021
- Primary completion12 Sep 2023
- Study completion5 Oct 2023
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 Primary Mitochondrial MyopathyStudies 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 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 213 participants (actual)
Sponsor and investigators
- Lead sponsor
- Reneo Pharma Ltd
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Amel Karaa, MD, Principal investigator, Massachusetts General Hospital (MGH)
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04535609
- Sponsor's protocol ID
- REN001-201
- Acronym
- STRIDE
- Status last verified
- Apr 2024
- First submitted
- 27 Aug 2020
- Last updated
- 8 May 2024
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 8 May 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT04535609
- Acronym / sponsor ID
- STRIDE / REN001-201
- Conditions
- Primary Mitochondrial Myopathy
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 213 participants (actual)
- Lead sponsor
- Reneo Pharma LtdIndustry (pharma, biotech, devices)
- Study start
- 21 May 2021 (actual)
- Primary completion
- 12 Sep 2023 (actual)
- Study completion
- 5 Oct 2023 (actual)
- First posted
- 2 Sep 2020 (actual)
- Results
- Posted 3 Apr 2024
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
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 |
|---|---|---|
| Mavodelpar | Experimental | Drug: MavodelparOnce daily |
| Matched placebo | Placebo comparator | Drug: PlaceboOnce daily |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (8 listed)
- 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.
Key exclusion criteria (12 listed)
- 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.
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 |
|---|---|---|
| MavodelparAlso: REN001Once daily | Drug | Mavodelpar |
| PlaceboOnce daily | Drug | Matched placebo |
Primary outcome
Change in Distance Walked During a 12 Minute Walk Test
Time frame: Baseline to Week 24
Distance walked in meters
1 secondary outcome
Change in PROMIS Short Form - Fatigue 13a (FACIT-fatigue) Scores
Time frame: Baseline to Week 24
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 8 May 2024.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Individual participant dataSharing planned: No
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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.
Amel Karaa, MD principal investigator
Massachusetts General Hospital (MGH)
41 sites in 15 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Mavodelpar in people with Primary Mitochondrial Myopathy, run by Reneo Pharma Ltd at 41 sites in 15 countries.
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 8 inclusion and 12 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 8 May 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 41 sites in United States, France, Italy, Australia, Spain and 10 more countries. 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 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
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 3 Apr 2024). 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 in Distance Walked During a 12 Minute Walk Test (time frame: Baseline to Week 24). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 8 May 2024.