Phase 2/3 and Open-Label Extension Study of ASP0367 in Individuals with Primary Mitochondrial Myopathy [MOUNTAINSIDE]

Trial ID: NCT04641962 (ISN: 0367-CL-1201)

A study to assess the Efficacy, Safety and Tolerability of ASP0367 (Bocidelpar) in Participants with Primary Mitochondrial Myopathy (PMM). [MOUNTAINSIDE]

Trial Details

We are testing to find out if the study drug, ASP0367 is effective and safe as a potential treatment for people with primary mitochondrial myopathy (PMM). We will look at how ASP0367, an investigational treatment, can act in the body. ASP0367 is an orally administered PPARδ modulator that is being investigated regarding whether it can increase the number and function of the mitochondria in cells, thereby increasing energy production. This has the potential to improve muscle health and function, reduce inflammation, and increase endurance in people living with PMM. The primary purpose of this study is to assess the effect of ASP0367 on functional improvement compared to placebo and also to assess the safety and tolerability of ASP0367. This study has four parts and takes up to 1 year and 8 months to finish in total.

Screening period for 4 weeks (where you will be assessed to see if the study is a match)
Double-blind period for 52 weeks (where neither you nor the study doctor would know whether you were taking ASP0367 tablets or placebo tablets [inactive tablets])
Open-label period for 24 weeks (where everyone takes ASP0367)
Follow up period for 4 weeks (where you would not take the study medicine).

Estimated Enrollment

175 Participants

Phase

2/3

Eligibility Criteria

Participants must:

Have a diagnosis of PMM confirmed by genetic testing that is causing physical symptoms
Be able to follow study requirements such as performing 6-minute walk test (6MWT), using a mobile phone app to provide video recordings of walking and sitting activities
Be regularly taking coenzyme Q10 (CoQ10), carnitine, creatine or other mitochondrial disease-focused vitamins or supplemental therapies for more than 3 months and intends to stay on a stable dose for duration of study period (for participants who take any above-mentioned medications or supplements)

Participants must not:

Have a BMI of below 18.5 kg/m² or above 35 kg/m²
Have other physical symptoms or limitations that are not related to PMM that may make it difficult to complete walking or sit to stand tests for the study
Have an acute illness or have a mental health condition, or behavioral problem, or is not healthy enough for the study
Have an active cancer (malignancy) except for curative treated localized non-melanoma skin cancer (e.g., basal cell or squamous cell carcinoma)
Have clinically significant and unstable respiratory/heart disease
Have severe sideways or forward rounding curvature of your back (scoliosis or kyphoscoliosis) that interferes with your breathing
Have kidney or liver problems
Have any ventilator support
Have recently used an investigational drug or treatment, or participated in an investigational study

What Will Happen in This Study

Sites are located in the cities/states identified with the red pin:

The study takes up to 1 year and 8 months to finish in total. Participants will be evaluated for various outcome measures at about 11-12 hospital visits and 14-15 phone calls with your study team. On the same day you receive some of these phone calls, a qualified home healthcare provider will visit your home and collect your blood sample. The home healthcare provider will visit your home 11-12 times during the study.

Evaluations may include examinations of general health (e.g., - blood and urine tests, electrocardiogram (ECG)) and functional exams (e.g., 6-minute walk test (6MWT), 5 times sit to stand, muscle function tests with imaging markers). At home tests, using video recordings and a mobile app, and remote interviews and surveys may also be required.

You may be reimbursed for reasonable travel expenses. Additional site locations will become available on a rolling basis. Please check back if you do not see an enrolling site near you.

Frequently Asked Questions (FAQs)

Is there a patient advocacy group in the United States that supports patients with mitochondrial disease?

Yes, you can find more information about the United Mitochondrial Disease Foundation (UMDF) on its website: [https://www.umdf.org/].

Will I get paid to be a part of this clinical trial?

No, you will not be paid for taking part in this study. You may be reimbursed for: reasonable travel expenses including airfare, hotel accommodations, and ground transportation for all required study visits. You do not have to pay anything to be in the study. All study related services are provided at no cost.

What is a placebo?

A placebo is a substance that looks like a drug or treatment but does not have an active product or treatment in it. You may have heard the phrase “a placebo effect.” This refers to when people who think they are receiving a treatment may show signs of improvement even if the “treatment” is a placebo. This happens because they believe that they are receiving treatment which affects how they feel about their condition. Usually when a placebo is used, the clinical trial participants do not know who is receiving the placebo. This type of clinical trial is called a “single-blind study.”

Sometimes, neither the participants nor the doctors know who is receiving the placebo. This type of clinical trial is called a “double-blind study.” Researchers use a placebo to make sure that any effects of a clinical trial treatment they find in the trial are actually caused by the treatment being studied. However, use of placebo as the only comparison to an experimental treatment may not be appropriate in some conditions or diseases that require active treatment.

How can I check to see if I’m eligible for this trial?

You can check if you are potentially eligible for this trial by completing a set of screening questions. You can find these on this web page by clicking the ‘Get Started’ button.

Can I quit the trial at any time?

Yes. A participant can leave a clinical trial, at any time. When withdrawing from the trial, the participant should let the research team know about it, and the reasons for leaving the clinical trial.

What personal information is collected about you for the study?

The study doctor and other study staff will collect your personal information. This may include:

Your name, address, and phone number,
Your ethnic origin,
Medical information, including information from other hospitals/institutions, such as your physical health, mental health, and past treatments,
Audio files and information from interviews and questionnaires,
Movement videos,
Test results and information from your samples, and
Other information you give to the study team, which may include limited financial information such as bank account routing information.

Who has access to your personal information?

All of your personal information collected for this study will be securely stored in the study medical records at the study site. Sponsor staff, review boards and ethics committees (that approve and monitor studies), and others who have a need to know such as our data hosting provider, and government regulators may check the study records. This is done to make sure that the study is being run properly. Regulatory agencies, such as the US Food and Drug Administration (FDA), European Medicine Agency (EMA) or others, review and approve new medicines. These agencies will be granted direct access to your information. This is so they can verify clinical trial procedures and/or data. If you share your personal data to contact a site, that data will be used by TrialScope to contact you about clinical trials and related information.

Can we participate in the study from countries other than the United States?

Unfortunately, this study is open in the United States only and we are unable to accept participants from other countries for this clinical trial.

This trial is not actively recruiting.