A Study to Evaluate ASP0367 in Participants With Primary Mitochondrial Myopathy

NCT04641962 | Terminated Phase 2 Results DMC FDA Drug

• 1 other identifier: 0367-CL-1201
• Study Type: interventional
• Target: 34
• Locations: 1 country
• Sites: 11

Brief Summary

The purpose of this study was to evaluate the dose response of Bocidelpar on functional improvement relative to placebo, safety, and tolerability in participants with Primary Mitochondrial Myopathy.

Conditions

Primary Mitochondrial Myopathy

Keywords

ASP0367; fatigue; exercise intolerance; muscle weakness; Primary Mitochondrial Myopathy

Outcome Measures

Primary Outcomes (3)

• Change From Baseline in Distanced Walked During a 6 Minute Walk Test (6MWT).

  6MWT assessed functional capacity and endurance by measuring the distance walked in 6 minutes. This test helped to assess exercise tolerance, physical fitness, disease progression, and treatment effectiveness in individuals with myopathy. The total distance walked by a participant, as well as distance per minute was calculated by rounding to the nearest meter.

  Baseline, week 24

• Number of Participants With Treatment Emergent Adverse Events

  An Adverse event (AE) was any untoward medical occurrence in a participant administered a study drug, and which dint have to have a causal relationship with this treatment. An AE could therefore be any unfavorable and unintended sign (including an abnormal laboratory finding), symptom or disease (new or exacerbated) temporally associated with the use of a medicinal product, whether or not considered related to the medicinal product. TEAE was defined as an AE observed after starting administration of the study drug through 28 days after the last dose.

  From first dose up to week 52

• Number of Participants With Suicidal Ideation and/ or Behavior as Assessed by Columbia-Suicide Severity Rating Scale (C-SSRS)

  The C-SSRS was a questionnaire used for suicide risk assessment. Affirmative or negative responses were provided to items 1 to 5 for suicidal ideation (1. Wish to be dead, 2. Non-specific active suicidal thoughts, 3. Active suicidal ideation with any methods \[not plan\] without intent to act, 4. Active suicidal ideation with some intent to act, without specific plan, 5. Active suicidal ideation with specific plan and intent) and items 6 to 10 for suicide behavior (6. Preparatory acts or behavior, 7. Aborted attempt, 8. Interrupted attempt, 9. Actual attempt, 10. Completed suicide). The overall data was reported.

  From the first dose up to week 52

Secondary Outcomes (6)

• Change From Baseline in Quality of Life in Neurological Disorders (Neuro-QoL) Short Form Fatigue Score

  Baseline, week 24

• Change From Baseline in Neuro-QoL Short Form Lower Extremity Function (Mobility) Scores

  Baseline, week 24

• Change From Baseline in Time to Perform the 5 Times Sit to Stand (5XSTS) Test

  Baseline, week 24

• Number of Participants With Patient's Global Impression of Change (PGIC) Score at Week 24

  Week 24

• Number of Participants With Change From Baseline in Patient Global Impression of Severity (PGIS) at Week 24

  Baseline, week 24

Study Arms (4)

DBT Bocidelpar 30 mgs

EXPERIMENTAL

Participants received Bocidelpar 30 milligrams (mgs) orally once daily during the double blind treatment period. The Participants were followed up for another 4 weeks after the completion of treatment period.

Drug: Bocidelpar

DBT Bocidelpar 75mgs

EXPERIMENTAL

Participants received Bocidelpar 75 mg orally once daily during the DB treatment period.. The Participants were followed up for another 4 weeks after the completion of treatment period.

Drug: Bocidelpar

DBT: Placebo to OLE Period: Bocidelpar 30 mg

PLACEBO COMPARATOR

Participants received Bocidelpar matching placebo orally once daily during the DB treatment period and received 30 mg ASP0367 in the OLE period.

Drug: Bocidelpar; Drug: Placebo

DBT Bocidelpar 30 mg or 75mg to Open label Extension Period: Bocidelpar 30 mg

EXPERIMENTAL

Participants received Bocidelpar 30 mg or 75 mg orally once daily during the DB treatment period and received 30 mg Bocidelpar in the OLE period.

Drug: Bocidelpar

Interventions

Bocidelpar DRUG

Oral

Also known as: MA-0211, ASP0367

DBT Bocidelpar 30 mg or 75mg to Open label Extension Period: Bocidelpar 30 mg; DBT Bocidelpar 30 mgs; DBT Bocidelpar 75mgs; DBT: Placebo to OLE Period: Bocidelpar 30 mg

Placebo DRUG

Oral

DBT: Placebo to OLE Period: Bocidelpar 30 mg

Eligibility Criteria

• Age: 18 Years - 65 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Participant agrees and is able to adhere to the study requirements for the length of the study, including performing 6MWT.
• Diagnosed with primary mitochondrial myopathy (PMM), consisting of the following:
• Molecular genetic abnormality (i.e., nuclear or mitochondrial) known to be associated with mitochondrial dysfunction (such as, but not limited to, mitochondrial DNA (mtDNA) single deletions in chronic progressive external ophthalmoplegia (CPEO) and Kearns-Sayre syndrome (KSS); mtDNA m.3243 A \> G pathogenic nuclear or mitochondrial genome variants demonstrated to cause primary mitochondrial disease), and
• Participant reported symptoms (i.e., muscle weakness, fatigue and exercise intolerance) or physical examination findings of myopathy that are the predominant symptoms of the participant's mitochondrial disorder.
• Participant has been on stable dose regimen of coenzyme Q10 (CoQ10), carnitine, creatine, or other mitochondrial disease- focused vitamins or supplemental therapies for the treatment of symptoms of the mitochondrial disease for at least 3 months prior to randomization and intends to stay on a stable dose for duration of study period.
• Participant has been on stable exercise regimen within 4 weeks prior to randomization and intends to stay on a stable regimen for duration of study period (for participants who participate in a regular exercise regimen).
• Female participant is not pregnant and at least one of the following conditions apply:
• Not a woman of childbearing potential (WOCBP).
• WOCBP who agrees to follow the contraceptive guidance from the time of informed consent through at least 30 days after final study treatment administration.
• Female participant must agree not to breastfeed starting at screening and throughout the study period and for 30 days after final study treatment administration.
• Female participant must not donate ova starting at first dose of IP and throughout the study period and for 30 days after final study treatment administration.
• Male participant with female partner(s) of childbearing potential (including breastfeeding partner) must agree to use contraception throughout the treatment period and for 30 days after final study treatment administration.
• Male participant must not donate sperm during the treatment period and for 30 days after final study treatment administration.
• Male participant with pregnant partner(s) must agree to remain abstinent or use a condom for the duration of the pregnancy throughout the study period and for 30 days after final study treatment administration.
• Participant agrees not to participate in another interventional study while participating in the present study.

You may not qualify if:

• Participant has additional signs and/or symptoms due to non-myopathic process (e.g., cerebellar dysfunctions, movement disorder, peripheral neuropathy, stroke or other) or a gait problem not attributed to the myopathy that would interfere with the participant's performance during 6MWT or 5 times sit to stand (5XSTS).
• Participant has received any investigational therapy within 28 days or 5 half-lives, whichever is longer, prior to screening.
• Participant has any condition, which makes the participant unsuitable for study participation.
• Participant has cardiac troponin I (cTnI) \> upper limit of normal (ULN) at screening.
• Participant has estimated glomerular filtration rate (eGFR) calculated by the chronic kidney disease epidemiology collaboration equation \< 60 milliliter per minute per 1.73-meter square at screening or a history of chronic kidney disease stage 3 or greater.
• Participant has at screening\*: total bilirubin (TBL) \> ULN or transaminase(s) (aspartate aminotransferase \[AST\] or alanine aminotransferase \[ALT\]) \> ULN in the absence of elevations in CK. Participants who have a slightly elevated TBL and/or ALT and/or AST and are suitable candidates for the study may be enrolled after discussion of the case with the medical monitor and completion of further evaluation as warranted.
• Participant has psychiatric conditions such as schizophrenia, bipolar disorder or major depressive disorder that has not been under control within 3 months prior to screening.
• Participant has a history of suicide attempt, suicidal behavior or has any suicidal ideation within 1 year prior to screening that meets criteria at a level of 4 or 5 by using the Columbia- Suicide Severity Rating Scale (C-SSRS) or who is at significant risk to commit suicide.
• Participant has severe behavioral or cognitive problems that preclude participation in the study.
• Participant has undergone an in-patient hospitalization that precludes participation in the study, within the 30 days prior to the randomization.
• Participant has a planned hospitalization or a surgical procedure during the study, which may affect the study assessments.
• Participant has clinically significant and unstable respiratory disease and/or cardiac disease (medical history or current clinical findings), or prior interventional cardiac procedure (e.g. cardiac catheterization, angioplasty/percutaneous coronary intervention, balloon valvuloplasty, etc.) within 3 months prior to randomization. Participants with pacemakers are allowed in the study per investigator's discretion and after discussion with the medical monitor, and as long as it is used for prevention and there is no underlying cardiac dysfunction.
• Participant has a corrected mean QT interval using Fridericia's correction (QTcF) \> 450 msec for male participants and \> 480 msec for female participants at screening or randomization. If QTcF exceeds these limits, one additional triplicate ECG can be repeated on the same day in order to determine the participant's eligibility.
• ECG evidence of acute ischemia, atrial fibrillation or active conduction system abnormalities. The following conduction system abnormalities may be permitted per the investigator's discretion, only after discussing the case with the medical monitor:
• First degree atrioventricular (AV)- block

Sponsors & Collaborators

• Astellas Pharma Inc: lead

Study Sites (11)

University of California, San Diego

La Jolla, California, 92093, United States

Location

Stanford University Medical Center

Stanford, California, 94305, United States

Location

Children's Hospital Colorado

Aurora, Colorado, 80045, United States

Location

Massachusetts General Hospital

Boston, Massachusetts, 02114, United States

Location

Columbia University Irving Medical Center

New York, New York, 10032, United States

Location

Akron Children's Hospital

Akron, Ohio, 44308, United States

Location

Cleveland Clinic

Cleveland, Ohio, 44195, United States

Location

Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, 19104, United States

Location

Baylor College of Medicine

Houston, Texas, 77030, United States

Location

University of Texas Health Science Center at Hosuton

Houston, Texas, 77030, United States

Location

Seattle Children's Hospital

Seattle, Washington, 98105, United States

Location

Related Links

• Link to plain language summary of the study on the Trial Results Summaries website.
• Link to results and other applicable study documents on the Astellas Clinical Trials website.

MeSH Terms

Conditions

Fatigue; Muscle Weakness

Condition Hierarchy (Ancestors)

Signs and Symptoms; Pathological Conditions, Signs and Symptoms; Muscular Diseases; Musculoskeletal Diseases; Neuromuscular Manifestations; Neurologic Manifestations; Nervous System Diseases; Pathologic Processes

Limitations and Caveats

The study was terminated due to futility after protocol version 9.0 was introduced. Some participants received treatment for up to 52 weeks, resulting in efficacy reported for up to 24 weeks and safety for up to 52 weeks.

Results Point of Contact

• Title: Clinical Transparency
• Organization: Astellas Pharma Global Development, Inc

astellas.resultsdisclosure@astellas.com

8008887704

Study Officials

• Senior Medical Director

  Astellas Pharma Inc

  STUDY DIRECTOR

Publication Agreements

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

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: TRIPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR
• Masking Details: Double blind treatment period: Participant, Caregiver, Investigator
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: November 19, 2020
• First Posted: November 24, 2020
• Study Start: May 24, 2021
• Primary Completion: May 8, 2024
• Study Completion: May 8, 2024
• Last Updated: July 3, 2025
• Results First Posted: June 17, 2025

Record last verified: 2025-06

Data Sharing

• IPD Sharing: Will not share

Locations

US (11)