← Back to Search

Unknown

Bocidelpar for Duchenne Muscular Dystrophy

Phase 1
Waitlist Available
Research Sponsored by Astellas Pharma Inc
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Be younger than 18 years old
Timeline
Screening 3 weeks
Treatment Varies
Follow Up baseline, week 12 and week 24
Awards & highlights

Study Summary

This trial is testing a new drug, ASP0367, to see if it is safe and effective. The study will also look at how the body processes the drug and how it affects muscle function.

Eligible Conditions
  • Duchenne Muscular Dystrophy

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~baseline, week 12 and week 24
This trial's timeline: 3 weeks for screening, Varies for treatment, and baseline, week 12 and week 24 for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.
Primary outcome measures
Change from baseline in digit span test
Number of participants with Treatment Emergent Adverse Events (TEAEs)
Number of participants with body weight change abnormalities and/or AEs
+5 more
Secondary outcome measures
Change from baseline in Performance of Upper Limb Module (PUL) (v2.0) assessment score
Change from baseline in distance walked in 2 minutes assessed in meters
Change from baseline in fat fraction by magnetic resonance spectroscopy (MRS)
+7 more

Trial Design

2Treatment groups
Experimental Treatment
Placebo Group
Group I: ASP0367 groupExperimental Treatment1 Intervention
Participants will be dosed investigational product (IP) at the low dose for 2 weeks and then at the high dose for 10 weeks with the total duration of 12 weeks in the DB part and OLE part, respectively.
Group II: Placebo to ASP0367 groupPlacebo Group2 Interventions
Participants will be dosed matching placebo in the DB part. In OLE part, participants will dosed IP at the low dose for 2 weeks and then at the high dose for 10 weeks with the total duration of 12 weeks.
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Bocidelpar
2021
Completed Phase 1
~40

Find a Location

Who is running the clinical trial?

Astellas Pharma IncLead Sponsor
691 Previous Clinical Trials
232,206 Total Patients Enrolled
Associate DirectorStudy DirectorAstellas Pharma Inc

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

Who might be the best suited participants for this clinical trial?

"This clinical trial is looking for 8 people with muscular dystrophy, duchenne between the ages of 8 and 16. Candidates are required to meet the following criteria:Subject and subject's parent(s) or legal guardian agrees not to participate in another interventional study while participating in the present study., Abstain from sexual intercourse, OR If having heterosexual intercourse, must use a condom and their female partners who are of childbearing potential must use a highly effective contraception method., Subject has been on a stable regimen of corticosteroids for 6 months prior to the time of enrollment (at baseline)., Subject has been on"

Answered by AI

How many different medical clinics are conducting this study?

"9 hospitals are enrolling patients for this trial, they are: Children's Hospital of The King's Daughters in Norfolk, University of Minnesota in Minneapolis, Children's Hospital of Richmond at VCU in Richmond, and 6 other locations."

Answered by AI

What are the benefits that participants in this study can expect?

"The primary outcome being measured in this clinical trial is the number of participants with vital sign abnormalities and/or adverse events, over a period of up to 28 weeks. Other outcomes being evaluated include maximum concentration (Cmax) of ASP0367 in plasma, percent change from baseline in serum myostatin/follistatin ratio, and change from baseline score in Performance of Upper Limb Module (PUL) assessment."

Answered by AI

Are patients being enrolled in this research project at present?

"Data on clinicaltrials.gov indicates that this trial is not seeking candidates at the present moment. The listing for this trial was first posted on February 24th, 2021 and updated as recently as October 24th, 2022. There are other 88 trials currently recruiting patients."

Answered by AI

Can young adults participate in this research project?

"Children that are 8 years old or older but have not yet reached their 16th birthday may participate in this clinical trial."

Answered by AI

What are the short and long term risks of Bocidelpar?

"Bocidelpar, which is still in Phase 1 clinical trials, has received a score of 1 for safety."

Answered by AI
Recent research and studies
clinicaltrials.govStudy
A Study to Assess the Safety, Tolerability and Preliminary Efficacy of ASP0367 (MA-0211) in Pediatric Male Participants With Duchenne Muscular Dystrophy (DMD)
2021. "A Study to Assess the Safety, Tolerability and Preliminary Efficacy of ASP0367 (MA-0211) in Pediatric Male Participants With Duchenne Muscular Dystrophy (DMD)". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT04184882.
~2 spots leftby Apr 2025
Unbiased ResultsWe believe in providing patients with all the options.
Your Data Stays Your DataWe only share your information with the clinical trials you're trying to access.
Verified Trials OnlyAll of our trials are run by licensed doctors, researchers, and healthcare companies.
Back to top