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Results for “Primary Mitochondrial Myopathies”

30 trials

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Showing 20 of 30 results

Large-scale testing (Phase 3)Ended earlyNCT03323749
What this trial is testing

A Trial to Evaluate Safety and Efficacy of Elamipretide Primary Mitochondrial Myopathy Followed by Open-Label Extension

Who this might be right for
Primary Mitochondrial Myopathy
Stealth BioTherapeutics Inc. 218
Not applicableEnrolling By InvitationNCT03655223
What this trial is testing

Early Check: Expanded Screening in Newborns

Who this might be right for
Spinal Muscular AtrophyFragile X SyndromeFragile X - Premutation+182 more
RTI International 30,000
Testing effectiveness (Phase 2)Ended earlyNCT02976038
What this trial is testing

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

Who this might be right for
Primary Mitochondrial Disease
Stealth BioTherapeutics Inc. 28
Not applicableLooking for participantsNCT05653544
What this trial is testing

Natural History in Primary Mitochondrial Myopathies

Who this might be right for
Primary Mitochondrial Myopathies
Cristina Domínguez González 150
Not applicableLooking for participantsNCT05250375
What this trial is testing

Natural History Study of Mitochondrial Myopathy

Who this might be right for
Primary Mitochondrial Disease
Children's Hospital of Philadelphia 1,300
Not applicableStudy completedNCT03048617
What this trial is testing

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

Who this might be right for
Primary Mitochondrial Disease
Stealth BioTherapeutics Inc. 215
Not applicableUnknownNCT05910151
What this trial is testing

Selective Screening of Children for Hereditary Metabolic Diseases by Tandem Mass Spectrometry in Kazakhstan

Who this might be right for
Propionic/Methylmalonic AcidemiasMaple Syrup Urine DiseaseCitrullinemia+26 more
West Kazakhstan Medical University 2,250
Testing effectiveness (Phase 2)Study completedNCT05972954
What this trial is testing

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

Who this might be right for
Primary Mitochondrial Disease
Omeicos Therapeutics GmbH 28
Testing effectiveness (Phase 2)Not Yet RecruitingNCT06792500
What this trial is testing

A Basket Clinical Study to Assess Glycerol Tributyrate in Patients With Mitochondrial Encephalopathy, Lactic Acidosis, Stroke-like Episodes (MELAS) or Leber's Hereditary Optic Neuropathy-Plus (LHON-Plus)

Who this might be right for
MELAS SyndromeLebers Hereditory Optic Neuropathy With Extra Ocular Symptoms (LHON-Plus)
George Washington University 24
Not applicableLooking for participantsNCT07478172
What this trial is testing

Effects of Whole-body Electrical Muscle Stimulation Exercise on Adults With Neuromuscular Disease

Who this might be right for
Neuromuscular Diseases (NMD)Amyotrophic Lateral SclerosisMyasthenia Gravis+15 more
University of Missouri-Columbia 50
Testing effectiveness (Phase 2)Study completedNCT04535609
What this trial is testing

An Efficacy and Safety Study of 24 Week Treatment With Mavodelpar (REN001) in Primary Mitochondrial Myopathy Patients

Who this might be right for
Primary Mitochondrial Myopathy
Reneo Pharma Ltd 213
Testing effectiveness (Phase 2)Active Not RecruitingNCT03845712
What this trial is testing

An Open-Label Study of Continuation Treatment With Combination Pyrimidine Nucleosides in Patients With TK2 Deficiency

Who this might be right for
Thymidine Kinase 2 Deficiency
UCB BIOSCIENCES, Inc. 47
Large-scale testing (Phase 3)Study completedNCT05162768
What this trial is testing

Study to Evaluate Efficacy and Safety of Elamipretide in Subjects With Primary Mitochondrial Disease From Nuclear DNA Mutations (nPMD)

Who this might be right for
Mitochondrial MyopathiesMitochondrial PathologyMitochondrial DNA Mutation+5 more
Stealth BioTherapeutics Inc. 102
Early research (Phase 1)Study completedNCT03888716
What this trial is testing

A Phase Ia/Ib, SAD and MAD Study of of KL1333 in Healthy Subjects and Patients With Primary Mitochondrial Disease

Who this might be right for
Mitochondrial DiseasesMitochondrial Respiratory Chain DeficienciesMELAS Syndrome+1 more
Abliva AB 72
Testing effectiveness (Phase 2)Ended earlyNCT04641962
What this trial is testing

Evaluate ASP0367 in Participants With Primary Mitochondrial Myopathy

Who this might be right for
Primary Mitochondrial Myopathy
Astellas Pharma Inc 34
Not applicableLooking for participantsNCT04602325
What this trial is testing

Systemic Biomarkers of Brain Injury From Hyperammonemia

Who this might be right for
Urea Cycle DisorderOrganic AcidemiaMaple Syrup Urine Disease+3 more
Children's National Research Institute 24
Testing effectiveness (Phase 2)Looking for participantsNCT05650229
What this trial is testing

Efficacy of KL1333 in Adult Patients With Primary Mitochondrial Disease

Who this might be right for
Primary Mitochondrial Disease
Abliva AB 180
Large-scale testing (Phase 3)Looking for participantsNCT06451757
What this trial is testing

KHENERFIN Study: A Trial to Evaluate the Efficacy and Safety of Sonlicromanol in Primary Mitochondrial Diseases

Who this might be right for
Mitochondrial DiseasesMaternally Inherited Diabetes and Deafness (MIDD)Mitochondrial Encephalomyopathy, Lactic Acidosis and Stroke-like Episodes (MELAS)+1 more
Khondrion BV 220
Testing effectiveness (Phase 2)Study completedNCT02805790
What this trial is testing

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

Who this might be right for
Primary Mitochondrial Disease
Stealth BioTherapeutics Inc. 30
Not applicableStudy completedNCT05687474
What this trial is testing

Baby Detect : Genomic Newborn Screening

Who this might be right for
Congenital Adrenal HyperplasiaFamilial Hyperinsulinemic Hypoglycemia 1Phosphoglucomutase 1 Deficiency+121 more
Centre Hospitalier Universitaire de Liege 6,824
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