Trial · NCT04535609

An Efficacy and Safety Study of 24 Week Treatment With Mavodelpar (REN001) in Primary Mitochondrial Myopathy Patients

NCT04535609, Phase 2, Completed, Reneo Pharma Ltd; results first posted 2024-04-03

3 sourcestrials-2026-11-12-0.5.0

Cite this record

ConductScience Library. An Efficacy and Safety Study of 24 Week Treatment With Mavodelpar (REN001) in Primary Mitochondrial Myopathy Patients. Trials, record NCT04535609. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT04535609

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Trial

As registered on ClinicalTrials.gov, in the graph's capture of the registry.

Title: An Efficacy and Safety Study of 24 Week Treatment With Mavodelpar (REN001) in Primary Mitochondrial Myopathy Patients
NCT number: NCT04535609
Status: Completed
Phases: Phase 2
Enrollment: 213 participants, actual
Lead sponsor: Reneo Pharma Ltd
Conditions: Primary Mitochondrial Myopathy
Interventions: Mavodelpar, Placebo
First posted: 2020-09-02
Last updated: 2024-05-08
Results first posted: 2024-04-03

Source ClinicalTrials.gov

Registered outcomes2

Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.

List	Outcome	Time frame	Description
Primary	Change in Distance Walked During a 12 Minute Walk Test	Baseline to Week 24	Distance walked in meters
Secondary	Change in PROMIS Short Form - Fatigue 13a (FACIT-fatigue) Scores	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

Source ClinicalTrials.gov

Outcomes2

Each outcome the trial posted results for, in ClinicalTrials.gov's words.

Type	Outcome	Time frame	Population	Units	Measured as
PRIMARY	Change in Distance Walked During a 12 Minute Walk Test	Baseline to Week 24	The full analysis set includes all subjects in the randomized set who received at least one dose of study drug and were not subsequently discontinued from the study for failing eligibility criteria. Subjects were analysed according to the treatment they were assigned at randomization. The FAS was used for baseline analyses and was the primary analysis set for efficacy.	meters	MEAN
SECONDARY	Change in PROMIS Short Form - Fatigue 13a (FACIT-fatigue) Scores	Baseline to Week 24	The full analysis set includes all subjects in the randomized set who received at least one dose of study drug and were not subsequently discontinued from the study for failing eligibility criteria. Subjects were analysed according to the treatment they were assigned at randomization. The FAS was used for baseline analyses and was the primary analysis set for efficacy.	score on a scale	MEAN

Source ClinicalTrials.gov

Results by group4

Each measurement the trial posted, group by group, in the words and numbers it posted.

Outcome	Group	Value	Spread	Units
Change in Distance Walked During a 12 Minute Walk Test	Mavodelpar	26.75	95% Confidence Interval 12.97 to 40.53	meters
Change in Distance Walked During a 12 Minute Walk Test	Matched Placebo	30.89	95% Confidence Interval 15.03 to 46.74	meters
Change in PROMIS Short Form - Fatigue 13a (FACIT-fatigue) Scores	Mavodelpar	-0.98	95% Confidence Interval -2.03 to 0.08	score on a scale
Change in PROMIS Short Form - Fatigue 13a (FACIT-fatigue) Scores	Matched Placebo	-2.60	95% Confidence Interval -3.80 to -1.40	score on a scale

Source ClinicalTrials.gov

Participant flow6

How many started, completed or left each group, period by period.

Period	Milestone	Group	Participants
Overall Study	STARTED	Mavodelpar	108
Overall Study	STARTED	Matched Placebo	104
Overall Study	COMPLETED	Mavodelpar	100
Overall Study	COMPLETED	Matched Placebo	98
Overall Study	NOT COMPLETED	Mavodelpar	8
Overall Study	NOT COMPLETED	Matched Placebo	6

Source ClinicalTrials.gov

Adverse events72

Each adverse event the trial posted, group by group: the participants affected out of those at risk.

Type	Event	Organ system	Group	Affected	At risk
other	COVID-19	Infections and infestations	Mavodelpar	20	108
other	COVID-19	Infections and infestations	Matched Placebo	13	104
other	Diarrhea	Gastrointestinal disorders	Mavodelpar	10	108
other	Diarrhea	Gastrointestinal disorders	Matched Placebo	3	104
other	Nausea	Gastrointestinal disorders	Mavodelpar	10	108
other	Nausea	Gastrointestinal disorders	Matched Placebo	4	104
other	Vomiting	Gastrointestinal disorders	Mavodelpar	9	108
other	Vomiting	Gastrointestinal disorders	Matched Placebo	5	104
other	Refraction Disorder	Eye disorders	Mavodelpar	20	108
other	Refraction Disorder	Eye disorders	Matched Placebo	15	104

1 to 10 of 72Source ClinicalTrials.gov

See all 72 adverse events

Adverse event totals6

The totals the trial posted for each group.

Total	Group	Affected	At risk
Total, all-cause mortality	Mavodelpar	1	108
Total, all-cause mortality	Matched Placebo	0	104
Total, other adverse events	Mavodelpar	85	108
Total, other adverse events	Matched Placebo	81	104
Total, serious adverse events	Mavodelpar	8	108
Total, serious adverse events	Matched Placebo	7	104

Source ClinicalTrials.gov

Papers0

The papers the trial lists on ClinicalTrials.gov.

None in this release.

Grants0

The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.

None in this release.

Trial comparisons2

Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.

Comparison	Source
Mavodelpar vs Matched Placebo: deaths from any cause	Trial comparisons
Mavodelpar vs Matched Placebo: serious adverse events	Trial comparisons

Tests0

The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.

None in this release.

Data dates

The day each source was copied for this release.

ClinicalTrials.gov: 2026-11-12; ClinicalTrials.gov
Results, from AACT: 2026-03-23; AACT
NIH grant links: 2026-11-13; NIH RePORTER

Sources 3

ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
ClinicalTrials.govNo license stated
NIH RePORTER's clinical studies and project filesPublic domain

Each source's terms

ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
ClinicalTrials.govnone stated
NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)

ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.

For software

Read this trial from software.

The record and each of its lists are JSON, twenty rows a page. No key.

GET https://conductscience.org/api/trials/v1/NCT04535609

GET https://conductscience.org/api/trials/v1/NCT04535609/events?page=2

API reference For AI assistants