Trials Sponsors Conditions Drugs Pricing

Conditions

Primary Mitochondrial Myopathy

Outcome Measures

Primary Outcomes (1)

Change in Distance Walked During a 12 Minute Walk Test
Distance walked in meters
Baseline to Week 24

Secondary Outcomes (1)

Change in PROMIS Short Form - Fatigue 13a (FACIT-fatigue) Scores
Baseline to Week 24

Study Arms (2)

Mavodelpar

EXPERIMENTAL

Once daily

Drug: Mavodelpar

Matched placebo

PLACEBO COMPARATOR

Once daily

Drug: Placebo

Interventions

MavodelparDrug

Once daily

Also known as: REN001

Mavodelpar

PlaceboDrug

Once daily

Matched placebo

Registry-reported names are shown in full. Names without a link have not yet been matched to a Syfrah profile.

Eligibility Criteria

Age18 Years+

Sexall

Healthy VolunteersNo

Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

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.

Sponsors & Collaborators

Reneo Pharma Ltd
Industrylead

Study Sites (41)

University of California, San Diego

La Jolla, California, 92093, United States

Myology Institute, University of Florida

Gainesville, Florida, 32608, United States

Massachusetts General Hospital

Boston, Massachusetts, 02114, United States

Columbia University Irving Medical Center

New York, New York, 10032, United States

Akron Children's Hospital

Akron, Ohio, 44308, United States

The Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, 19104, United States

UPMC Children's Hospital of Pittsburgh

Results Point of Contact

Title: Alex Dorenbaum, MD, Chief Medical Officer
Organization: Reneo Pharmaceuticals, Inc.

Study Officials

Amel Karaa, MD
Massachusetts General Hospital (MGH)
PRINCIPAL INVESTIGATOR

Publication Agreements

PI is Sponsor Employee: No
Restriction Type: OTHER
Restrictive Agreement: Yes

Enrollment

213 participants (actual enrollment reported by the registry).

Study Design

Study Type: interventional
Phase: phase 2
Allocation: RANDOMIZED
Masking: QUADRUPLE
Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
Purpose: TREATMENT
Intervention Model: PARALLEL
Sponsor Type: INDUSTRY
Responsible Party: SPONSOR

Study Record Dates

First Submitted

August 27, 2020

First Posted

September 2, 2020

Study Start

May 21, 2021

Actual (reported)

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

Brief Summary

Registry Overview

Trial Relationships

Study Timeline

First Submitted

First Posted

Study Start

Primary Completion

Study Completion

Results Posted

Conditions

Outcome Measures

Primary Outcomes (1)

Secondary Outcomes (1)

Study Arms (2)

Mavodelpar

Matched placebo

Interventions

Eligibility Criteria

You may qualify if:

Sponsors & Collaborators

Study Sites (41)

Results Point of Contact

Study Officials

Publication Agreements

Enrollment

Study Design

Study Record Dates

You may not qualify if:

Data Sharing

Source record

Documents

Locations

Brief Summary

Registry Overview

Trial Relationships

Related Scientific Literature

Study Timeline

First Submitted

First Posted

Study Start

Primary Completion

Study Completion

Results Posted

Conditions

Outcome Measures

Primary Outcomes (1)

Secondary Outcomes (1)

Study Arms (2)

Mavodelpar

Matched placebo

Interventions

Eligibility Criteria

You may qualify if:

Sponsors & Collaborators

Study Sites (41)

Results Point of Contact

Study Officials

Publication Agreements

Enrollment

Study Design

Study Record Dates

You may not qualify if:

Data Sharing

Source record

Documents

Locations