What side effects are associated with this type of intervention?

The most common treatments for Mitochondrial DNA Syndrome, particularly those aimed at enhancing mitochondrial function like Elamipretide, often have side effects that include injection site reactions, gastrointestinal disturbances (such as nausea and diarrhea), and potential fatigue. Other mitochondrial function enhancers may also cause headaches, elevated liver enzymes, and muscle pain. It is important for patients to discuss these potential side effects with their healthcare provider to manage them effectively.

What prior FDA approvals exist?

As of now, there are no FDA-approved treatments specifically for Mitochondrial DNA Syndrome that function similarly to Elamipretide, which aims to enhance mitochondrial function. Elamipretide is currently being studied for its potential benefits in improving mitochondrial function in patients with primary mitochondrial myopathy associated with nuclear DNA mutations. Other treatments for mitochondrial disorders generally focus on managing symptoms and may include vitamins, supplements, and supportive therapies, but they do not specifically target mitochondrial function enhancement in the same way as Elamipretide.

What other types of research exist?

Promising alternatives to Elamipretide for mitochondrial DNA syndrome patients include: 1) Gene therapy approaches targeting specific mitochondrial DNA mutations, 2) Antioxidant therapies such as EPI-743 (Vincerinone), which has shown potential in clinical trials, and 3) NAD+ precursors like Nicotinamide Riboside (NR) and Nicotinamide Mononucleotide (NMN), which aim to enhance mitochondrial function and biogenesis.

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Mitochondrial Peptide

Elamipretide for Mitochondrial Myopathy (NuPower Trial)

Phase 3

Waitlist Available

Research Sponsored by Stealth BioTherapeutics Inc.

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Diagnosed with nPMD with a predominant clinical manifestation of myopathy, which must include progressive external ophthalmoplegia (PEO) and exercise intolerance and/or skeletal muscle weakness, with genetic confirmation of either nuclear DNA mutation of the mitochondrial replisome (replisome-related mutations) or other pathogenic mutations specific to nuclear DNA

MPV17 or

Must not have

Has been previously diagnosed with human immunodeficiency virus (HIV), hepatitis B, or hepatitis C infection

Has received elamipretide (MTP-131) within the past one year of the Screening Visit

Timeline

Screening 3 weeks

Treatment Varies

Follow Up baseline, weeks 12, 24, 36, 48, 52 (end of trial visit)

Awards & highlights

Pivotal Trial

Summary

This trial tests if regular injections of elamipretide can help patients with muscle weakness due to specific genetic mutations by improving their mitochondria's function. Elamipretide has shown promise in improving mitochondrial function and exercise tolerance in various studies.

Who is the study for?

Adults aged 18-70 with primary mitochondrial myopathy from nuclear DNA mutations, including progressive external ophthalmoplegia and muscle weakness. Participants must agree to trial requirements and contraception use. Excluded are those with recent major medical events, severe neurological issues, substance abuse history, certain infections or transplants, participation in other trials within 30 days, significant kidney impairment, inability to perform specific functional tests, pregnancy/breastfeeding status or certain walking limitations.

What is being tested?

The study is testing the effectiveness and safety of Elamipretide given daily through a subcutaneous injection compared to a placebo over a period of 48 weeks. The focus is on individuals with mitochondrial myopathy due to nuclear DNA mutations.

What are the potential side effects?

While not specified here, potential side effects may include reactions at the injection site such as redness or pain; general symptoms like fatigue or headache; and possibly more serious conditions related to organs affected by mitochondrial disease.

Eligibility Criteria

Inclusion Criteria

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

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I have nPMD with symptoms like eye movement issues and muscle weakness, confirmed by genetic tests.

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My condition involves the MPV17 gene.

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I am between 18 and 70 years old.

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I meet the specific study's eligibility criteria.

Exclusion Criteria

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

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I have been diagnosed with HIV, hepatitis B, or hepatitis C.

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I have received elamipretide treatment within the last year.

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I have received a solid organ transplant.

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I have a history of high eosinophil levels or a related illness.

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My kidney function, measured by eGFR, is less than 30 mL/min.

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I can walk less than 150 meters or more than 450 meters in six minutes.

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I am not pregnant, planning to become pregnant, or breastfeeding.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ baseline, weeks 12, 24, 36, 48, 52 (end of trial visit)

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~baseline, weeks 12, 24, 36, 48, 52 (end of trial visit)

This trial's timeline: 3 weeks for screening, Varies for treatment, and baseline, weeks 12, 24, 36, 48, 52 (end of trial visit) for reporting.

Treatment Details

Study Objectives

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

Primary study objectives

Six-minute walk test (6MWT)

Secondary study objectives

5 times sit-to-stand test (5XSST)

Patient Global Impression of Severity (PGI-S) Scale

Triple Timed up-and-go test (3TUG)

Side effects data

From 2021 Phase 2 & 3 trial • 12 Patients • NCT03098797

100%

Dizziness

60%

Injection site pruritus

60%

Injection site erythema

60%

Injection site pain

40%

Headache

40%

Injection site urticaria

40%

Injection site induration

40%

Nausea

40%

Drug eruption

20%

Gastroenteritis

20%

Viral upper respiratory tract infection

20%

Pain in extremity

20%

Aphthous ulcer

20%

Eczema

20%

Leukopenia

20%

Palpitations

20%

Feeling cold

20%

Vessel puncture site bruise

20%

Injection site cellulitis

20%

Procedural vomiting

20%

Echocardiogram abnormal

20%

Myalgia

20%

Anxiety

20%

Initial insomnia

20%

Urticaria

20%

Vaccination site rash

20%

Eosinophil count increased

100%

80%

60%

40%

20%

Study treatment Arm

Part 2 (OLE): Elamipretide (Sequence AB)

Part 2 (OLE): Elamipretide (Sequence BA)

Part 1: Elamipretide

Part 1: Placebo

Awards & Highlights

Pivotal Trial

The final step before approval, pivotal trials feature drugs that have already shown basic safety & efficacy.

Trial Design

2Treatment groups

Experimental Treatment

Placebo Group

Group I: ElamipretideExperimental Treatment1 Intervention

0.75 mL of 80mg/mL solution of elamipretide for a single daily SC dose of 60mg elamipretide

Group II: PlaceboPlacebo Group1 Intervention

0.75 mL of 80mg/mL solution of matching placebo for a single daily SC dose of 60mg

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

Elamipretide

2016

Completed Phase 3

~150

0 / 11Eligibility 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

The most common treatments for Mitochondrial DNA Syndrome, such as Elamipretide, work by enhancing mitochondrial function. Elamipretide stabilizes the inner mitochondrial membrane, improves mitochondrial respiration, and reduces the production of reactive oxygen species. This is important for patients with Mitochondrial DNA Syndrome because their condition typically involves impaired mitochondrial function, leading to reduced energy production and increased oxidative stress. By improving mitochondrial function, these treatments can help increase cellular energy levels and alleviate symptoms associated with mitochondrial dysfunction.

A comparison of genetic mitochondrial disease and nucleoside analogue toxicity. Does fetal nucleoside toxicity underlie reports of mitochondrial disease in infants born to women treated for HIV infection?