What is the mechanism of action of this treatment?

Common treatments for Mitochondrial Myopathy focus on enhancing mitochondrial function and compensating for energy deficits. ASP0367, for instance, is being studied for its ability to target specific biological pathways to improve mitochondrial function. Other treatments include Coenzyme Q10, which supports the electron transport chain, and antioxidants that reduce oxidative stress. These treatments are essential as they aim to improve cellular energy production, alleviate symptoms, and enhance the quality of life for patients with Mitochondrial Myopathy.

What side effects are associated with this type of intervention?

Common treatments for mitochondrial myopathy, particularly those targeting specific biological pathways like ASP0367, often include drugs aimed at improving mitochondrial function. Known side effects of such treatments can include gastrointestinal issues, fatigue, headache, and potential liver enzyme abnormalities. Additionally, some patients may experience muscle pain or weakness, and there is always a risk of allergic reactions. It is important to monitor these side effects closely and discuss them with a healthcare provider.

What prior FDA approvals exist?

There is no specific information provided about prior FDA approvals for the treatment of Mitochondrial Myopathy, particularly with drugs targeting specific biological pathways similar to ASP0367. Generally, treatments for mitochondrial diseases are limited and often focus on managing symptoms rather than targeting the underlying biological mechanisms.

What other types of research exist?

Currently, there are no specific alternatives to ASP0367 mentioned in the provided context for mitochondrial myopathy. However, patients should consult with their healthcare provider about ongoing clinical trials and emerging treatments targeting mitochondrial function and energy metabolism.

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Other

Bocidelpar for Mitochondrial Myopathy (MOUNTAINSIDE Trial)

Phase 2

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

Diagnosed with primary mitochondrial myopathy (PMM) with specific genetic abnormality and reported symptoms or physical examination findings of myopathy.

Be older than 18 years old

Must not have

Participant has known or suspected hypersensitivity to study drug, symptomatic COVID-19 infection within a certain timeframe, or specific BMI range.

Participant has a history of active malignancy or any other cancer within a certain timeframe.

Timeline

Screening 3 weeks

Treatment Varies

Follow Up through week 28 (end of study)

Summary

This trial is testing a new drug called ASP0367 to see if it can help people with a muscle disease called Primary Mitochondrial Myopathy. The drug aims to improve muscle function, making it easier for patients to walk and do other activities.

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.

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.

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.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

Select...

I have been diagnosed with PMM and show symptoms or signs of muscle disease.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

Select...

I am not allergic to the study drug, don't have COVID-19 symptoms, and my weight is within the required range.

Select...

I have had cancer or another type of malignancy in the past.

Select...

I have weakness in my face or throat muscles due to nerve or muscle disease.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ through week 28 (end of study)

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~through week 28 (end of study)

This trial's timeline: 3 weeks for screening, Varies for treatment, and through week 28 (end of study) for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

Change from baseline in distance walked in 6 minutes assessed in meters

Number of participants with 12-lead electrocardiogram (ECG) abnormalities and/or AEs

Number of participants with body weight change abnormalities and/or AEs

+6 more

Secondary study objectives

Change from baseline in Modified Fatigue Impact Scale (MFIS)

Change from baseline in Patient Global Impression of Severity (PGIS)

Change from baseline in quality of life in neurological disorders (Neuro-QoL) Short Form Fatigue and Lower Extremity Function (Mobility) scores

+2 more

Trial Design

3Treatment groups

Experimental Treatment

Placebo Group

Group I: Low dose ASP0367Experimental Treatment1 Intervention

Participants will receive ASP0367 once daily in the morning for 24 weeks.

Group II: High dose ASP0367Experimental Treatment1 Intervention

Participants will receive ASP0367 once daily in the morning for 24 weeks.

Group III: PlaceboPlacebo Group1 Intervention

Participants will receive placebo once daily in the morning for 24 weeks.

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

Bocidelpar

2021

Completed Phase 1

~40

0 / 4Eligibility criteria met

Research Highlights

Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.

Mechanism Of Action

Side Effect Profile

Prior Approvals

Other Research

Common treatments for Mitochondrial Myopathy focus on enhancing mitochondrial function and compensating for energy deficits. ASP0367, for instance, is being studied for its ability to target specific biological pathways to improve mitochondrial function. Other treatments include Coenzyme Q10, which supports the electron transport chain, and antioxidants that reduce oxidative stress. These treatments are essential as they aim to improve cellular energy production, alleviate symptoms, and enhance the quality of life for patients with Mitochondrial Myopathy.

Reversible Valproate-Induced Subacute Encephalopathy Associated With a MT-ATP8 Variant in the Mitochondrial Genome.