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Primary Mitochondrial Disease clinical trials

View clinical trials related to Primary Mitochondrial Disease.

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NCT ID: NCT05972954 Recruiting - Clinical trials for Primary Mitochondrial Disease

OMT-28 in Patients With Primary Mitochondrial Disease (PMD) (PMD-OPTION)

PMD-OPTION
Start date: May 22, 2023
Phase: Phase 2
Study type: Interventional

The goal of this clinical trial is to learn about the treatment effects of the investigational new drug OMT-28 in patients with Primary Mitochondrial Disease. The main question[s] it aims to answer are: - Is OMT-28 safe and well tolerated in this patient population? - Does OMT-28 reduce Growth Differentiation Factor 15 (GDF-15) and other relevant blood markers of mitochondrial dysfunction and inflammation? - Does OMT-28 improve symptoms of the disease, e.g. fatigue or exercise intolerance? Participants will be asked to participate in 6 study visits at an experienced clinical center, including physical examinations and exercise tests, and take the study medication regularly once per day according to the protocol. Researchers will compare for every participant the results after 3 months and 6 months of treatment with a preceding 3 month period of standard care treatment to investigate the effects of OMT-28 on clinical parameters and a number of blood parameters.

NCT ID: NCT05650229 Active, not recruiting - Clinical trials for Primary Mitochondrial Disease

Efficacy of KL1333 in Adult Patients With Primary Mitochondrial Disease

FALCON
Start date: December 13, 2022
Phase: Phase 2
Study type: Interventional

The primary objective of the FALCON study is to evaluate the efficacy of KL1333 on selected disease manifestations of primary mitochondrial disease (PMD) following 48 weeks of treatment. This objective involves evaluating the efficacy of KL1333 versus placebo on fatigue symptoms and impacts on daily living as well as on functional lower extremity strength and endurance. Additionally, the study evaluates the safety and tolerability of KL1333.

NCT ID: NCT05250375 Recruiting - Clinical trials for Primary Mitochondrial Disease

Mitochondrial Myopathy Rating Scale

Start date: March 24, 2017
Phase:
Study type: Observational [Patient Registry]

Investigators have assembled an existing infrastructure of physical therapists, clinical coordinators and Bioinformatics; as well as expertise in developing and validating tools to measure disease course in a longitudinal study, to support completion of the proposed studies. Aim 1 serves to validate the Mitochondrial Myopathy Objective Assessment Tool (MM-COAST) and Mitochondrial Myopathy Functional Scale (MMFS) in nucleotide-binding protein-like (NUBPL)-subjects. Aim 2 aims to devise a Primary Mitochondrial Diseases (PMD)-specific cerebellar ataxia outcome measure for future clinical trials. Nucleotide-binding protein-like (NUBPL)-Natural history data will be used to inform future interventional clinical trial design, while the validated MM-COAST, Mitochondrial Myopathy Rating Scale (MMRS) and newly devised PMD-ataxia scale would be utilized as meaningful quantitative outcome measures in future NUBPL-multicenter natural history and clinical trials.

NCT ID: NCT03048617 Completed - Clinical trials for Primary Mitochondrial Disease

An Observational Study of Patients With Primary Mitochondrial Disease (SPIMM-300)

RePOWER
Start date: February 13, 2017
Phase:
Study type: Observational

This is an observational study of patients with Primary Mitochondrial Disease with either signs or symptoms suggestive of myopathy. The Investigator will identify potential patients through existing medical records and one on-site visit.

NCT ID: NCT02976038 Terminated - Clinical trials for Primary Mitochondrial Disease

Open-Label Extension Trial to Characterize the Long-term Safety and Tolerability of Elamipretide in Subjects With Genetically Confirmed Primary Mitochondrial Myopathy (PMM)

Start date: December 2016
Phase: Phase 2
Study type: Interventional

This is a Phase 2 Open-Label extension study to evaluate the long term safety and tolerability of daily elamipretide injections in patients with genetically confirmed Primary Mitochondrial Disease who previously participated in the SPIMM-202 Clinical Trial

NCT ID: NCT02805790 Completed - Clinical trials for Primary Mitochondrial Disease

Safety, Tolerability, Efficacy of MTP-131 for Treatment of Mitochondrial Disease in Subjects From the MMPOWER Study

MMPOWER-2
Start date: August 22, 2016
Phase: Phase 2
Study type: Interventional

This randomized, double-blind, placebo-controlled, crossover study screened 32 subjects with primary mitochondrial myopathy (PMM) to evaluate the safety, tolerability, pharmacokinetics (PK), and efficacy of subcutaneous elamipretide in this patient population.