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Bocidelpar for Mitochondrial Myopathy (MOUNTAINSIDE Trial)

Phase 2 & 3
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
Participant has been on stable dose regimen of coenzyme Q10 (CoQ10), carnitine, creatine or other mitochondrial disease-focused vitamins or supplemental therapies for 3 months prior to randomization and intends to stay on a stable dose for duration of study period.
Diagnosed with primary mitochondrial myopathy (PMM) with specific genetic abnormality and reported symptoms or physical examination findings of myopathy.
Timeline
Screening 3 weeks
Treatment Varies
Follow Up through week 80
Awards & highlights

MOUNTAINSIDE Trial Summary

This trial is testing a new drug, ASP0367, to see if it is safe and effective at treating people with a certain disease. The trial is divided into two phases: phase 2 and phase 3. In phase 2, the researchers will test the drug to see what dose is biologically active and how it affects the body. In phase 3, the researchers will test the drug to see if it improves the symptoms of the disease.

Who is the study for?
This trial is for individuals with primary mitochondrial myopathy who have been on a stable regimen of certain supplements and exercise. They must be able to perform specific physical tests, use digital apps for the study, and agree to contraception if applicable. People with recent cancers, other medical conditions that could affect test performance, or those on investigational drugs within 28 days are excluded.Check my eligibility
What is being tested?
The trial is testing ASP0367's effectiveness in improving function compared to a placebo in two phases: Phase 2 determines an active dose by evaluating how the body processes it; Phase 3 measures functional improvement and fatigue reduction against a placebo.See study design
What are the potential side effects?
While not explicitly listed here, potential side effects may include reactions related to drug tolerability such as gastrointestinal issues, skin reactions, muscle or joint pain based on its pharmacological class. Specific side effects will be monitored throughout the trial.

MOUNTAINSIDE Trial Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
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I've been taking the same dose of CoQ10 or similar supplements for 3 months and plan to continue.
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I have been diagnosed with PMM and show symptoms or signs of muscle disease.
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I am not pregnant and follow the required birth control guidelines.
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I agree to use contraception with my partner who can become pregnant.
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I agree to follow the study's requirements, including tests and using apps.

MOUNTAINSIDE Trial Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~through week 80
This trial's timeline: 3 weeks for screening, Varies for treatment, and through week 80 for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.
Primary outcome measures
Phase 2 and Phase 3 portions: Number of participants with 12-lead electrocardiogram (ECG) abnormalities and/or AEs
Phase 2 and Phase 3 portions: Number of participants with body weight change abnormalities and/or AEs
Phase 2 and Phase 3 portions: Number of participants with laboratory value abnormalities and/or AEs
+13 more
Secondary outcome measures
Phase 3 portion: Change from baseline in Modified Fatigue Impact Scale (MFIS)
Phase 3 portion: Change from baseline in Neuro-QoL Short Form Lower Extremity Function (Mobility) score
Phase 3 portion: Change from baseline in Patient Global Impression of Severity (PGIS
+3 more

MOUNTAINSIDE Trial Design

6Treatment groups
Experimental Treatment
Placebo Group
Group I: Phase 3: ASP0367Experimental Treatment1 Intervention
Participants will receive ASP0367 once daily in the morning for up to 52 weeks.
Group II: Phase 2: Low dose ASP0367Experimental Treatment1 Intervention
Participants will receive ASP0367 once daily in the morning for 2 weeks.
Group III: Phase 2: High dose ASP0367Experimental Treatment1 Intervention
Participants will receive ASP0367 once daily in the morning for 2 weeks.
Group IV: Open Label Extension: ASP0367Experimental Treatment1 Intervention
Participants will receive ASP0367 once daily in the morning for 24 weeks.
Group V: Phase 2: PlaceboPlacebo Group1 Intervention
Participants will receive placebo once daily in the morning for 2 weeks.
Group VI: Phase 3: PlaceboPlacebo Group1 Intervention
Participants will receive placebo once daily in the morning for up to 52 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
689 Previous Clinical Trials
219,844 Total Patients Enrolled
Senior Medical DirectorStudy DirectorAstellas Pharma Inc
54 Previous Clinical Trials
12,882 Total Patients Enrolled

Media Library

ASP0367 (Other) Clinical Trial Eligibility Overview. Trial Name: NCT04641962 — Phase 2 & 3
Mitochondrial Myopathy Research Study Groups: Phase 2: Low dose ASP0367, Phase 2: High dose ASP0367, Phase 2: Placebo, Phase 3: ASP0367, Phase 3: Placebo, Open Label Extension: ASP0367
Mitochondrial Myopathy Clinical Trial 2023: ASP0367 Highlights & Side Effects. Trial Name: NCT04641962 — Phase 2 & 3
ASP0367 (Other) 2023 Treatment Timeline for Medical Study. Trial Name: NCT04641962 — Phase 2 & 3

Frequently Asked Questions

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

Are there any specific medical requirements to become a test subject in this experiment?

"As this is a study for luft disease, only individuals aged 18-80 can be accepted as participants. Up to 195 people will be able to join the trial."

Answered by AI

Does this experiment require participants to be over 30 years old?

"This clinical trial is specifically for patients aged 18-80. However, there are 9 other trials for minors and 30 studies involving seniors above the age of 65."

Answered by AI

Can new patients still join this clinical trial?

"The trial is currently recruiting participants, with the most recent update on November 11th, 2022. The original posting was on May 17th, 2021. Across 14 locations, the study is looking for a total of 195 patients."

Answered by AI

Is this clinical trial widely available in the United States?

"In addition to Baylor College of Medicine in Houston, Texas, Akron Children's Hospital in Akron, Ohio, and Mayo Clinic Arizona in Phoenix, Arizona; this study is also taking place at 14 other research facilities."

Answered by AI

How many people can sign up for this experiment at most?

"One hundred and ninety-five patients that meet the inclusion criteria are needed for this clinical trial. Children's hospitals like Akron Children's Hospital in Ohio and Baylor College of Medicine in Texas are two examples of where patients can participate from."

Answered by AI

What are the goals that this research study is aiming to achieve?

"According to the study sponsor, Astellas Pharma Inc, the primary objective of this study is to assess the number of participants with body weight change abnormalities and/or AEs over a Through Week 80 time frame. Additionally, this trial will also measure secondary outcomes including change from baseline in quality of life in neurological disorders (Neuro-QoL) Short Form Fatigue score, Patient Global Impression of Change (PGIC) score, and Neuro-QoL Short Form Lower Extremity Function (Mobility) score."

Answered by AI
~57 spots leftby May 2025