Follistatin 315
Activin-Binding Protein, FSH-Suppressing Protein, FST-315, FS-315
Terminated
PHASE1
INTERVENTIONAL
NCT04184882
A Study to Assess the Safety, Tolerability and Preliminary Efficacy of ASP0367 (MA-0211) in Pediatric Male Participants With Duchenne Muscular Dystrophy (DMD)
View on ClinicalTrials.gov
Updated Dec 15, 2025
Brief Summary
The primary purpose of this study is to evaluate the safety and tolerability of ASP0367. This study will also evaluate the pharmacokinetics, pharmacodynamics and efficacy on muscle function of ASP0367.
Detailed Description
This study is comprised of a 4-week pre-treatment screening period, 24-week treatment period and 4-week post-treatment follow-up period. The 24-week treatment period consists of a 12-week double-blind (DB) part and 12-week open-label extension (OLE) part and each part includes a 2 week Low dose Period and a 10-week High-dose Period.
Interventions
Name:
Bocidelpar
Type:
DRUG
Description:
Oral
Name:
Placebo
Type:
DRUG
Description:
Oral
Primary Outcomes
Measure:
Number of participants with Treatment Emergent Adverse Events (TEAEs)
TimeFrame:
Up to Week 28
Description:
An AE is any untoward medical occurrence in a subject administered an investigational product (IP), and which does not necessarily have to have a causal relationship with this treatment.
A TEAE is defined as an AE observed after starting administration of the investigational product (IP) to 28 days after the last dose of IP for the double blind part or moving to the open-label extension part, whichever comes first.
An IP-related TEAE is defined as any TEAE with a causal relationship of "yes" by the investigator.
Measure:
Number of participants with vital sign abnormalities and/or AEs
TimeFrame:
Up to Week 28
Description:
Number of participants with potentially clinically significant vital sign values.
Measure:
Number of participants with body weight change abnormalities and/or AEs
TimeFrame:
Up to Week 28
Description:
Number of participants with potentially clinically significant body weight.
Measure:
Number of participants with electrocardiogram (ECG) abnormalities
TimeFrame:
Up to Week 28
Description:
Number of participants with potentially clinically significant 12-ECG values.
Measure:
Number of participants with echocardiography abnormalities and/or AEs
TimeFrame:
Up to Week 28
Description:
Number of participants with potentially clinically significant echocardiography values.
Measure:
Number of participants with laboratory value abnormalities and/or AEs
TimeFrame:
Up to Week 28
Description:
Number of participants with potentially clinically significant laboratory values.
Measure:
Number of participants with suicidal ideation and/or behavior as assessed by Columbia-Suicide Severity Rating Scale (C-SSRS) Change from baseline in Columbia-Suicide Severity Rating Scale (C-SSRS)
TimeFrame:
Baseline and up to Week 28
Description:
The Columbia-Suicide Severity Rating Scale (C-SSRS) is a clinician administered assessment tool that evaluates suicidal ideation and behavior. Number of participants that have an affirmative response provided to the 5 items 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/or to the 6 items for suicidal behavior (1. Actual attempt, 2. Interrupted attempt, 3. Aborted attempt, 4. Preparatory acts or behavior, 5. Suicidal Behavior 6. Completed suicide,) will be reported.
Measure:
Change from baseline in digit span test
TimeFrame:
Baseline and up to Week 24
Description:
The Digit span test is a subtest of Wechsler Intelligence Scale for children (WISC). This test comprises 3 parts on the fifth edition (WISC-V). Digit Span Forward requires the subject to repeat numbers in the same order as presented by the interviewer. Digit Span Backward requires the subject to repeat the numbers in the reverse order of that presented by the interviewer. Digit Span Sequencing requires the subject to sequentially order the numbers presented by the interviewer. Scores of this test are based on each raw score and total raw score.
Trial Information
NCT ID
NCT04184882
Status
Terminated
Study Type
INTERVENTIONAL
Phases
PHASE1
Sponsor
Astellas Pharma Inc
Last Updated
December 15, 2025