A Phase 2 interventional study of Mavodelpar and Placebo in Primary Mitochondrial Myopathy, sponsored by Reneo Pharma Ltd. Completed at 41 sites in 15 countries. Open to participants aged 18 Years and older. Per ClinicalTrials.gov, last updated 2024-05-08.
Sponsored by Reneo Pharma Ltd · Phase 2, Interventional, and Treatment
Among 19 studies in Mitochondrial Myopathies with a site in United States — 6 open to participants now, 8 under way, none of them phase 3. Mitochondrial Myopathies · United States.
Among the 19 studies in Mitochondrial Myopathies with a site in United States, this one has the largest enrollment of the 12 interventional ones that give a count, is the only one that started in 2021, is one of 5 in phase 2, and was the 10th registered.
46 studies on the registry are indexed under Mitochondrial Myopathies; 14 are open to participants now.
Browse Mitochondrial Myopathies studies →6 of the 19 studies in Mitochondrial Myopathies with a site in United States are open now. Recruiting first, the newest first:
The POLG Foundation · Sites in United States, Australia, Denmark and 6 more countries
Children's Hospital of Philadelphia · Sites in United States
Ages 18–65 · Healthy volunteers
Children's Hospital of Philadelphia · Sites in United States
Ages 10–60 · Healthy volunteers
Children's Hospital of Philadelphia · Sites in United States
Ages 18–65 · Healthy volunteers
Children's Hospital of Philadelphia · Sites in United States
Ages 0–100 · Healthy volunteers
A study can be open while its sites in United States have not opened yet or have stopped. Only those recruiting at a site in United States →
Of the 13 Mitochondrial Myopathies studies recruiting now, 8 (62%) set no upper age limit and 4 (31%) stop at 75 or younger; 6 take children under 18; 6 take healthy volunteers.
This study: 18 Years and older · all sexes · no healthy volunteers.
The full table on the hub →This tab as a report to print or save as PDF → With an account.
19 studies; the count open now beside each. This study's row is marked.
A study in two phases is counted once above. Listed by phase, each with its combined phases: Phase 1 5 · Phase 2 7 · Phase 3 1
First in 1978; this study’s year is marked. 1 started in 2021.
By drug class, each counting the studies that test any of its medicines:
This study's enrollment of 213 is above the median of 24 across 31 interventional studies indexed under Mitochondrial Myopathies.
Median enrollment 50 across the 12 interventional studies, 215 across the 7 observational. Of the 10 that completed or were terminated, 4 (40%) have results posted.
This tab as a report to print or save as PDF → With an account.
6 of 19 have an industry lead sponsor. The 10 most frequent:
Reneo Pharma Ltd is the lead sponsor of 6 studies on the registry; none are open to participants now.
Reneo Pharma Ltd has 3 studies filed under Mitochondrial Myopathies — fewer than ten, so there is no comparison to draw.
5 of the studies in Mitochondrial Myopathies with a site in United States that are under way reach primary completion within a year, 0 of them phase 3. Phase 3 first, then by date:
| Study | Primary completion |
|---|---|
| NCT05590468Ralitza GavrilovaA Study to Evaluate Vitamin B3 Derivative to Treat Mitochondrial Myopathy | 2026-11 |
| NCT01694940Columbia UniversityNorth American Mitochondrial Disease Consortium Patient Registry and Biorepository (NAMDC) | 2026-12-31 |
| NCT04086329Children's Hospital of PhiladelphiaValidation of Oxygen Nanosensor in Mitochondrial Myopathy | 2027-08-01 |
| NCT06819683Children's Hospital of PhiladelphiaValidation of Nanosensor Oxygen Measurement | 2027-09 |
| NCT05569122Children's Hospital of PhiladelphiaApplying pGz in Mitochondrial Disease | 2027-09 |
Among the 19 studies in Mitochondrial Myopathies with a site in United States, from their registry updates:
This tab as a report to print or save as PDF → With an account.
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.
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
Exclusion:
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Able to become pregnant: from the first period until menopause, unless permanently unable to through surgery (removal of the womb, or of both ovaries or both fallopian tubes).
Criteria for people of childbearing potential usually ask for a negative pregnancy test and for contraception during the study and for a time after it.
Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.
Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Judged by the study doctor to matter for your health or for the study. It is a doctor’s decision, so ask the study team if you are unsure whether something counts.
GFR (glomerular filtration rate) is how much blood the kidneys filter each minute. It is usually estimated from a blood test — eGFR, by formulas such as CKD-EPI or MDRD — and some studies ask for it to be measured directly instead. Usually given in mL/min/1.73 m².
Check the method, the units and the minimum the study states. A value of 90 or above is in the normal-or-high range, but kidney damage can show in other tests too.
Two enzymes measured in a routine blood test of the liver. Higher levels can mean the liver is under strain or damaged. SGPT and SGOT are older names for ALT and AST.
Criteria usually give a limit as a multiple of the upper limit of normal (ULN), often higher when cancer has spread to the liver.
The top of the normal range for a blood test, as set by the laboratory that runs it. Each lab prints its own range next to your result.
“≤ 2.5 × ULN” means the result can be up to two and a half times that top value.
A yellow substance made when red blood cells are broken down, which the liver clears. A high level can mean a problem with the liver or the bile ducts.
Criteria often allow more for people with Gilbert’s syndrome, a harmless inherited condition that raises bilirubin.
A blood test of average blood sugar over the past two to three months, given as a percentage or in mmol/mol. 6.5% (48 mmol/mol) or more is the usual threshold for diabetes, normally confirmed by a second test.
A recording of the heart’s electrical activity, taken with stickers on the chest, arms and legs. It takes a few minutes and does not hurt.
Childbearing potential
Able to become pregnant: from the first period until menopause, unless permanently unable to through surgery (removal of the womb, or of both ovaries or both fallopian tubes).
Criteria for people of childbearing potential usually ask for a negative pregnancy test and for contraception during the study and for a time after it.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Contraception
Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.
Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Informed consent
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Clinically significant
Judged by the study doctor to matter for your health or for the study. It is a doctor’s decision, so ask the study team if you are unsure whether something counts.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
GFR and eGFR (kidney filtering)
GFR (glomerular filtration rate) is how much blood the kidneys filter each minute. It is usually estimated from a blood test — eGFR, by formulas such as CKD-EPI or MDRD — and some studies ask for it to be measured directly instead. Usually given in mL/min/1.73 m².
Check the method, the units and the minimum the study states. A value of 90 or above is in the normal-or-high range, but kidney damage can show in other tests too.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
ALT and AST (liver enzymes)
Two enzymes measured in a routine blood test of the liver. Higher levels can mean the liver is under strain or damaged. SGPT and SGOT are older names for ALT and AST.
Criteria usually give a limit as a multiple of the upper limit of normal (ULN), often higher when cancer has spread to the liver.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
ULN (upper limit of normal)
The top of the normal range for a blood test, as set by the laboratory that runs it. Each lab prints its own range next to your result.
“≤ 2.5 × ULN” means the result can be up to two and a half times that top value.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Bilirubin
A yellow substance made when red blood cells are broken down, which the liver clears. A high level can mean a problem with the liver or the bile ducts.
Criteria often allow more for people with Gilbert’s syndrome, a harmless inherited condition that raises bilirubin.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
HbA1c
A blood test of average blood sugar over the past two to three months, given as a percentage or in mmol/mol. 6.5% (48 mmol/mol) or more is the usual threshold for diabetes, normally confirmed by a second test.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
ECG (electrocardiogram)
A recording of the heart’s electrical activity, taken with stickers on the chest, arms and legs. It takes a few minutes and does not hurt.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Once daily
Drug: Mavodelpar
Once daily
Drug: Placebo
Once daily
Also known as: REN001
Once daily
Change in Distance Walked During a 12 Minute Walk Test
Distance walked in meters
Time frame: Baseline to Week 24
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
Time frame: Baseline to Week 24
| Milestone | Mavodelpar | Matched Placebo |
|---|---|---|
| Started | 108 | 104 |
| Completed | 100 | 98 |
| Not completed | 8 | 6 |
Distance walked in meters
| meters | Mavodelpar | Matched Placebo |
|---|---|---|
| Change in Distance Walked During a 12 Minute Walk Test | 26.75 (12.97 to 40.53) | 30.89 (15.03 to 46.74) |
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
| score on a scale | Mavodelpar | Matched Placebo |
|---|---|---|
| Change in PROMIS Short Form - Fatigue 13a (FACIT-fatigue) Scores | -0.98 (-2.03 to 0.08) | -2.60 (-3.80 to -1.40) |
Collected over 24 weeks. Non-serious events are listed at a 5% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| Mavodelpar | 1/108 (0.9%) | 8/108 (7.4%) | 85/108 (78.7%) |
| Matched Placebo | 0/104 (0%) | 7/104 (6.7%) | 81/104 (77.9%) |
| Event | Mavodelpar | Matched Placebo |
|---|---|---|
| Cerebrovascular AccidentNervous system disorders | 2/108 | 0/104 |
| COVID-19Infections and infestations | 0/108 | 1/104 |
| Pneumonia InfluenzaInfections and infestations | 0/108 | 1/104 |
| Atrioventricular BlockCardiac disorders | 0/108 | 1/104 |
| Coronary Artery DiseaseCardiac disorders | 0/108 | 1/104 |
| Nodal RhythmCardiac disorders | 0/108 | 1/104 |
| HaematemesisGastrointestinal disorders | 0/108 | 1/104 |
| Cervical Vertebral FractureInjury, poisoning and procedural complications | 0/108 | 1/104 |
| Limb InjuryInjury, poisoning and procedural complications | 0/108 | 1/104 |
| Rib FractureInjury, poisoning and procedural complications | 0/108 | 1/104 |
| Event | Mavodelpar | Matched Placebo |
|---|---|---|
| COVID-19Infections and infestations | 20/108 | 13/104 |
| Refraction DisorderEye disorders | 20/108 | 15/104 |
| HeadacheNervous system disorders | 10/108 | 13/104 |
| Blood Creatine Phosphokinase IncreasedInvestigations | 11/108 | 3/104 |
| DiarrheaGastrointestinal disorders | 10/108 | 3/104 |
| NauseaGastrointestinal disorders | 10/108 | 4/104 |
| FatigueGeneral disorders | 7/108 | 9/104 |
| VomitingGastrointestinal disorders | 9/108 | 5/104 |
| Back PainMusculoskeletal and connective tissue disorders | 9/108 | 4/104 |
| MyalgiaMusculoskeletal and connective tissue disorders | 9/108 | 6/104 |
| Age, Customized(Participants) | Mavodelpar | Matched Placebo | Total |
|---|---|---|---|
| 18-25 years | 12 | 6 | 18 |
| 26-45 years | 34 | 44 | 78 |
| 46-64 years | 51 | 49 | 100 |
| =>65 years | 11 | 5 | 16 |
| Sex: Female, Male(Participants) | Mavodelpar | Matched Placebo | Total |
|---|---|---|---|
| Female | 75 | 76 | 151 |
| Male | 33 | 28 | 61 |
| Ethnicity (NIH/OMB)(Participants) | Mavodelpar | Matched Placebo | Total |
|---|---|---|---|
| Hispanic or Latino | 1 | 7 | 8 |
| Not Hispanic or Latino | 101 | 93 | 194 |
| Unknown or Not Reported | 6 | 4 | 10 |
| Race/Ethnicity, Customized(Participants) | Mavodelpar | Matched Placebo | Total |
|---|---|---|---|
| Asian | 1 | 3 | 4 |
| White | 99 | 95 | 194 |
| Not Reported | 4 | 3 | 7 |
| Other | 4 | 3 | 7 |
| Region of Enrollment(participants) | Mavodelpar | Matched Placebo | Total |
|---|---|---|---|
| North America | 17 | 15 | 32 |
| Australia | 7 | 10 | 17 |
| Europe | 84 | 79 | 163 |
PDFs hosted by the registry, ClinicalTrials.gov.
Plan to share: No
Ran from May 2021 to primary completion in Sep 2023, 27 months.
Results were first posted in Apr 2024, 6 months after primary completion.
How these figures are worked out, and which studies are left out of them.
This study is completed, as verified in Apr 2024. You cannot join it, but the record below documents what was studied.
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Studies of the drug tested here: Mavodelpar
Reneo Pharma Ltd