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Results for “Primary Mitochondrial Myopathies”

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Showing 11 of 0 results

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 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 participantsNCT07775872
What this trial is testing

Prospective Natural History Study of POLG Disease

Who this might be right for
PolG, Primary Mitochondrial Myopathy
The POLG Foundation 300
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
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
Pharming Technologies B.V. 180
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
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
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
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
Large-scale testing (Phase 3)WithdrawnNCT04581733
What this trial is testing

The Efficacy and Safety of MT1621 in Thymidine Kinase 2 (TK2) Deficiency (Treatment naïve)

Who this might be right for
Thymidine Kinase 2 Deficiency
Zogenix MDS, Inc.
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