Trial Outcomes & Findings for An Efficacy and Safety Study of 24 Week Treatment With Mavodelpar (REN001) in Primary Mitochondrial Myopathy Patients (NCT NCT04535609)
NCT ID: NCT04535609
Last Updated: 2024-05-08
Results Overview
Distance walked in meters
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
213 participants
Primary outcome timeframe
Baseline to Week 24
Results posted on
2024-05-08
Participant Flow
One subject completed all screening procedures, was randomized in error and was removed from the study before any study procedures were completed.
Participant milestones
| Measure |
Mavodelpar
100 mg Once Daily
|
Matched Placebo
Once Daily
|
|---|---|---|
|
Overall Study
STARTED
|
108
|
104
|
|
Overall Study
COMPLETED
|
100
|
98
|
|
Overall Study
NOT COMPLETED
|
8
|
6
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
An Efficacy and Safety Study of 24 Week Treatment With Mavodelpar (REN001) in Primary Mitochondrial Myopathy Patients
Baseline characteristics by cohort
| Measure |
Mavodelpar
n=108 Participants
100 mg Once Daily
|
Matched Placebo
n=104 Participants
Once Daily
|
Total
n=212 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Customized
18-25 years
|
12 Participants
n=39 Participants
|
6 Participants
n=41 Participants
|
18 Participants
n=35 Participants
|
|
Age, Customized
26-45 years
|
34 Participants
n=39 Participants
|
44 Participants
n=41 Participants
|
78 Participants
n=35 Participants
|
|
Age, Customized
46-64 years
|
51 Participants
n=39 Participants
|
49 Participants
n=41 Participants
|
100 Participants
n=35 Participants
|
|
Age, Customized
=>65 years
|
11 Participants
n=39 Participants
|
5 Participants
n=41 Participants
|
16 Participants
n=35 Participants
|
|
Sex: Female, Male
Female
|
75 Participants
n=39 Participants
|
76 Participants
n=41 Participants
|
151 Participants
n=35 Participants
|
|
Sex: Female, Male
Male
|
33 Participants
n=39 Participants
|
28 Participants
n=41 Participants
|
61 Participants
n=35 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
1 Participants
n=39 Participants
|
7 Participants
n=41 Participants
|
8 Participants
n=35 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
101 Participants
n=39 Participants
|
93 Participants
n=41 Participants
|
194 Participants
n=35 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
6 Participants
n=39 Participants
|
4 Participants
n=41 Participants
|
10 Participants
n=35 Participants
|
|
Race/Ethnicity, Customized
Asian
|
1 Participants
n=39 Participants
|
3 Participants
n=41 Participants
|
4 Participants
n=35 Participants
|
|
Race/Ethnicity, Customized
White
|
99 Participants
n=39 Participants
|
95 Participants
n=41 Participants
|
194 Participants
n=35 Participants
|
|
Race/Ethnicity, Customized
Not Reported
|
4 Participants
n=39 Participants
|
3 Participants
n=41 Participants
|
7 Participants
n=35 Participants
|
|
Race/Ethnicity, Customized
Other
|
4 Participants
n=39 Participants
|
3 Participants
n=41 Participants
|
7 Participants
n=35 Participants
|
|
Region of Enrollment
North America
|
17 participants
n=39 Participants
|
15 participants
n=41 Participants
|
32 participants
n=35 Participants
|
|
Region of Enrollment
Australia
|
7 participants
n=39 Participants
|
10 participants
n=41 Participants
|
17 participants
n=35 Participants
|
|
Region of Enrollment
Europe
|
84 participants
n=39 Participants
|
79 participants
n=41 Participants
|
163 participants
n=35 Participants
|
PRIMARY outcome
Timeframe: Baseline to Week 24Population: 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.
Distance walked in meters
Outcome measures
| Measure |
Mavodelpar
n=106 Participants
100 mg Once Daily
|
Matched Placebo
n=104 Participants
Once Daily
|
|---|---|---|
|
Change in Distance Walked During a 12 Minute Walk Test
|
26.75 meters
Interval 12.97 to 40.53
|
30.89 meters
Interval 15.03 to 46.74
|
SECONDARY outcome
Timeframe: Baseline to Week 24Population: 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.
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
Outcome measures
| Measure |
Mavodelpar
n=106 Participants
100 mg Once Daily
|
Matched Placebo
n=104 Participants
Once Daily
|
|---|---|---|
|
Change in PROMIS Short Form - Fatigue 13a (FACIT-fatigue) Scores
|
-0.98 score on a scale
Interval -2.03 to 0.08
|
-2.60 score on a scale
Interval -3.8 to -1.4
|
Adverse Events
Mavodelpar
Serious events: 8 serious events
Other events: 85 other events
Deaths: 1 deaths
Matched Placebo
Serious events: 7 serious events
Other events: 81 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
Mavodelpar
n=108 participants at risk
100 mg Once Daily
|
Matched Placebo
n=104 participants at risk
Once Daily
|
|---|---|---|
|
Nervous system disorders
Cerebrovascular Accident
|
1.9%
2/108 • 24 weeks
Regular Investigator assessment
|
0.00%
0/104 • 24 weeks
Regular Investigator assessment
|
|
Nervous system disorders
Epilepsy
|
0.93%
1/108 • 24 weeks
Regular Investigator assessment
|
0.00%
0/104 • 24 weeks
Regular Investigator assessment
|
|
Nervous system disorders
Seizure
|
0.93%
1/108 • 24 weeks
Regular Investigator assessment
|
0.00%
0/104 • 24 weeks
Regular Investigator assessment
|
|
Infections and infestations
Pneumonia
|
0.93%
1/108 • 24 weeks
Regular Investigator assessment
|
0.00%
0/104 • 24 weeks
Regular Investigator assessment
|
|
Infections and infestations
Pyelonephritis Acute
|
0.93%
1/108 • 24 weeks
Regular Investigator assessment
|
0.00%
0/104 • 24 weeks
Regular Investigator assessment
|
|
Infections and infestations
COVID-19
|
0.00%
0/108 • 24 weeks
Regular Investigator assessment
|
0.96%
1/104 • 24 weeks
Regular Investigator assessment
|
|
Infections and infestations
Pneumonia Influenza
|
0.00%
0/108 • 24 weeks
Regular Investigator assessment
|
0.96%
1/104 • 24 weeks
Regular Investigator assessment
|
|
Cardiac disorders
Angina Pectoris
|
0.93%
1/108 • 24 weeks
Regular Investigator assessment
|
0.00%
0/104 • 24 weeks
Regular Investigator assessment
|
|
Cardiac disorders
Atrioventricular Block
|
0.00%
0/108 • 24 weeks
Regular Investigator assessment
|
0.96%
1/104 • 24 weeks
Regular Investigator assessment
|
|
Cardiac disorders
Coronary Artery Disease
|
0.00%
0/108 • 24 weeks
Regular Investigator assessment
|
0.96%
1/104 • 24 weeks
Regular Investigator assessment
|
|
Cardiac disorders
Nodal Rhythm
|
0.00%
0/108 • 24 weeks
Regular Investigator assessment
|
0.96%
1/104 • 24 weeks
Regular Investigator assessment
|
|
Gastrointestinal disorders
Pancreatitis
|
0.93%
1/108 • 24 weeks
Regular Investigator assessment
|
0.00%
0/104 • 24 weeks
Regular Investigator assessment
|
|
Gastrointestinal disorders
Haematemesis
|
0.00%
0/108 • 24 weeks
Regular Investigator assessment
|
0.96%
1/104 • 24 weeks
Regular Investigator assessment
|
|
General disorders
Death
|
0.93%
1/108 • 24 weeks
Regular Investigator assessment
|
0.00%
0/104 • 24 weeks
Regular Investigator assessment
|
|
Injury, poisoning and procedural complications
Concussion
|
0.93%
1/108 • 24 weeks
Regular Investigator assessment
|
0.00%
0/104 • 24 weeks
Regular Investigator assessment
|
|
Injury, poisoning and procedural complications
Humerus Fracture
|
0.93%
1/108 • 24 weeks
Regular Investigator assessment
|
0.00%
0/104 • 24 weeks
Regular Investigator assessment
|
|
Injury, poisoning and procedural complications
Cervical Vertebral Fracture
|
0.00%
0/108 • 24 weeks
Regular Investigator assessment
|
0.96%
1/104 • 24 weeks
Regular Investigator assessment
|
|
Injury, poisoning and procedural complications
Limb Injury
|
0.00%
0/108 • 24 weeks
Regular Investigator assessment
|
0.96%
1/104 • 24 weeks
Regular Investigator assessment
|
|
Injury, poisoning and procedural complications
Rib Fracture
|
0.00%
0/108 • 24 weeks
Regular Investigator assessment
|
0.96%
1/104 • 24 weeks
Regular Investigator assessment
|
|
Metabolism and nutrition disorders
Diabetic Ketoacidosis
|
0.93%
1/108 • 24 weeks
Regular Investigator assessment
|
0.00%
0/104 • 24 weeks
Regular Investigator assessment
|
|
Surgical and medical procedures
Hospitalization
|
0.93%
1/108 • 24 weeks
Regular Investigator assessment
|
0.00%
0/104 • 24 weeks
Regular Investigator assessment
|
|
Surgical and medical procedures
Cranial Operation
|
0.00%
0/108 • 24 weeks
Regular Investigator assessment
|
0.96%
1/104 • 24 weeks
Regular Investigator assessment
|
Other adverse events
| Measure |
Mavodelpar
n=108 participants at risk
100 mg Once Daily
|
Matched Placebo
n=104 participants at risk
Once Daily
|
|---|---|---|
|
Infections and infestations
COVID-19
|
18.5%
20/108 • 24 weeks
Regular Investigator assessment
|
12.5%
13/104 • 24 weeks
Regular Investigator assessment
|
|
Gastrointestinal disorders
Diarrhea
|
9.3%
10/108 • 24 weeks
Regular Investigator assessment
|
2.9%
3/104 • 24 weeks
Regular Investigator assessment
|
|
Gastrointestinal disorders
Nausea
|
9.3%
10/108 • 24 weeks
Regular Investigator assessment
|
3.8%
4/104 • 24 weeks
Regular Investigator assessment
|
|
Gastrointestinal disorders
Vomiting
|
8.3%
9/108 • 24 weeks
Regular Investigator assessment
|
4.8%
5/104 • 24 weeks
Regular Investigator assessment
|
|
Eye disorders
Refraction Disorder
|
18.5%
20/108 • 24 weeks
Regular Investigator assessment
|
14.4%
15/104 • 24 weeks
Regular Investigator assessment
|
|
General disorders
Fatigue
|
6.5%
7/108 • 24 weeks
Regular Investigator assessment
|
8.7%
9/104 • 24 weeks
Regular Investigator assessment
|
|
Nervous system disorders
Headache
|
9.3%
10/108 • 24 weeks
Regular Investigator assessment
|
12.5%
13/104 • 24 weeks
Regular Investigator assessment
|
|
Nervous system disorders
Dizziness
|
6.5%
7/108 • 24 weeks
Regular Investigator assessment
|
2.9%
3/104 • 24 weeks
Regular Investigator assessment
|
|
Musculoskeletal and connective tissue disorders
Back Pain
|
8.3%
9/108 • 24 weeks
Regular Investigator assessment
|
3.8%
4/104 • 24 weeks
Regular Investigator assessment
|
|
Musculoskeletal and connective tissue disorders
Myalgia
|
8.3%
9/108 • 24 weeks
Regular Investigator assessment
|
5.8%
6/104 • 24 weeks
Regular Investigator assessment
|
|
Skin and subcutaneous tissue disorders
Rash
|
7.4%
8/108 • 24 weeks
Regular Investigator assessment
|
0.00%
0/104 • 24 weeks
Regular Investigator assessment
|
|
Investigations
Blood Creatine Phosphokinase Increased
|
10.2%
11/108 • 24 weeks
Regular Investigator assessment
|
2.9%
3/104 • 24 weeks
Regular Investigator assessment
|
|
Injury, poisoning and procedural complications
Fall
|
5.6%
6/108 • 24 weeks
Regular Investigator assessment
|
0.96%
1/104 • 24 weeks
Regular Investigator assessment
|
|
Metabolism and nutrition disorders
Vitamin D Deficiency
|
5.6%
6/108 • 24 weeks
Regular Investigator assessment
|
2.9%
3/104 • 24 weeks
Regular Investigator assessment
|
Additional Information
Alex Dorenbaum, MD, Chief Medical Officer
Reneo Pharmaceuticals, Inc.
Phone: (415) 328-9115
Email: [email protected]
Results disclosure agreements
- Principal investigator is a sponsor employee Material for public dissemination will be submitted to the Sponsor for review at least 60 days prior to submission for publication, public dissemination, or review by a publication committee. During this period, the Sponsor shall be entitled to make a reasoned request that publication be delayed for a period of up to 6 months from the date of first submission to the Sponsor in order to enable the protection of proprietary information and/or Intellectual Property Rights.
- Publication restrictions are in place
Restriction type: OTHER