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Results for “Primary Mitochondrial Myopathy”

29 trials

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Showing 20 of 29 results

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
Testing effectiveness (Phase 2)Looking for participantsNCT06754098
What this trial is testing

Doxecitin and Doxribthymine in Adult Subjects With Thymidine Kinase 2 (TK2) Deficiency

Who this might be right for
Thymidine Kinase 2 (TK2 ) DeficiencyMitochondrial Myopathies
Cristina Domínguez González 15
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
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
Early research (Phase 1)Looking for participantsNCT05569122
What this trial is testing

Applying pGz in Mitochondrial Disease

Who this might be right for
Mitochondrial MyopathiesMitochondrial Diseases
Children's Hospital of Philadelphia 90
Testing effectiveness (Phase 2)Study completedNCT06051448
What this trial is testing

Promoting Resilience in Stress Management (PRISM) and Clinical-focused Narrative (CFN) Pilot in Adults With Primary Mitochondrial Disease (PMD).

Who this might be right for
Mitochondrial DiseasesMitochondrial MyopathiesMitochondrial Encephalomyopathies+2 more
Children's Hospital of Philadelphia 16
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)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
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
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
Not applicableStudy completedNCT06180421
What this trial is testing

Mitochondrial Video Assessment Source Material Study

Who this might be right for
PolG, Primary Mitochondrial Myopathy
The Emmes Company, LLC 28
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 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
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
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
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 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)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
Large-scale testing (Phase 3)Ended earlyNCT05267574
What this trial is testing

An Open Label, Long Term Safety Study of REN001 in Primary Mitochondrial Myopathy Patients (Stride Ahead)

Who this might be right for
Primary Mitochondrial Myopathy
Reneo Pharma Ltd 155
Early research (Phase 1)Ended earlyNCT03862846
What this trial is testing

The Safety of REN001 in Patients With Primary Mitochondrial Myopathy

Who this might be right for
Primary Mitochondrial Myopathy
Reneo Pharma Ltd 23
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