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Results for “Leigh Syndrome”

20 trials

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Showing 20 of 20 results

Not applicableNo Longer AvailableNCT01370447
What this trial is testing

EPI-743 for Mitochondrial Respiratory Chain Diseases

Who this might be right for
Mitochondrial Diseases
PTC Therapeutics
Not applicableLooking for participantsNCT05554835
What this trial is testing

Global Registry and Natural History Study for Mitochondrial Disorders

Who this might be right for
Mitochondrial DiseasesKearns-Sayre SyndromeMIDD+15 more
LMU Klinikum 6,000
Testing effectiveness (Phase 2)Study completedNCT02023866
What this trial is testing

Open-Label, Dose-Escalating Study Assessing Safety, Tolerability, Efficacy, of RP103 in Mitochondrial Disease

Who this might be right for
Inherited Mitochondrial Disease, Including Leigh Syndrome
Amgen 36
Large-scale testing (Phase 3)Study completedNCT05218655
What this trial is testing

A Safety Study for Previously Treated Vatiquinone (PTC743) Participants With Inherited Mitochondrial Disease

Who this might be right for
Inherited Mitochondrial Disease
PTC Therapeutics 101
Testing effectiveness (Phase 2)Ended earlyNCT02473445
What this trial is testing

A Long-term Extension of Study RP103-MITO-001 (NCT02023866) to Assess Cysteamine Bitartrate Delayed-release Capsules (RP103) in Children With Inherited Mitochondrial Disease

Who this might be right for
Mitochondrial Diseases
Amgen 22
Early research (Phase 1)Study completedNCT02544217
What this trial is testing

A Dose-escalating Clinical Trial With KH176

Who this might be right for
MELASLHONLeigh Syndrome+2 more
Khondrion BV 32
Testing effectiveness (Phase 2)Temporarily pausedNCT04846036
What this trial is testing

The KHENERGYC Study

Who this might be right for
Mitochondrial DiseasesMitochondrial DNA tRNALeu(UUR) m.3243A<G MutationMELAS+1 more
Khondrion BV 24
Large-scale testing (Phase 3)Ended earlyNCT04378075
What this trial is testing

Evaluate Efficacy and Safety of Vatiquinone for Treating Mitochondrial Disease in Participants With Refractory Epilepsy

Who this might be right for
Mitochondrial DiseasesDrug Resistant EpilepsyLeigh Disease+5 more
PTC Therapeutics 68
Not applicableLooking for participantsNCT06967831
What this trial is testing

Drug Repurposing for Mitochondrial Disorders Using iPSCs Derived Neural Cells

Who this might be right for
Leigh Syndrome (Maternally Inherited, MILS)Leigh Syndrome (AR, AD, XR)
Charite University, Berlin, Germany 80
Not applicableLooking for participantsNCT01694940
What this trial is testing

North American Mitochondrial Disease Consortium Patient Registry and Biorepository (NAMDC)

Who this might be right for
Mitochondrial DisordersMitochondrial Genetic DisordersMitochondrial Diseases+2 more
Columbia University 1,000
Testing effectiveness (Phase 2)Not Yet RecruitingNCT06990984
What this trial is testing

A Dose-ranging Study of TTI-0102 in Adults and Children With Leigh Syndrome Spectrum (LSS)

Who this might be right for
Leigh Syndrome
Thiogenesis Therapeutics, Inc. 18
Not applicableLooking for participantsNCT01780168
What this trial is testing

The NIH MINI Study: Metabolism, Infection, and Immunity in Inborn Errors of Metabolism

Who this might be right for
Oxidative Phosphorylation DeficienciesElectron Transport Chain Disorders, MitochondrialMitochondrial Disorders+1 more
National Human Genome Research Institute (NHGRI) 500
Not applicableLooking for participantsNCT03137355
What this trial is testing

The International Registry for Leigh Syndrome

Who this might be right for
Leigh SyndromeLeigh DiseaseLeigh's Necrotizing Encephalopathy+2 more
The University of Texas Health Science Center, Houston 200
Testing effectiveness (Phase 2)WithdrawnNCT03747328
What this trial is testing

ABI-009 (Nab-sirolimus) in Patients With Genetically-confirmed Leigh or Leigh-like Syndrome

Who this might be right for
Leigh Syndrome
Aadi Bioscience, Inc.
Not applicableWithdrawnNCT05277363
What this trial is testing

The Natural Course of SURF1 Deficiency

Who this might be right for
Leigh Syndrome
Taysha Gene Therapies, Inc.
Not applicableEnrolling By InvitationNCT01803906
What this trial is testing

Tissue Sample Study for Mitochondrial Disorders

Who this might be right for
Mitochondrial DisordersMitochondrial DiseaseMelas+6 more
Columbia University 6,900
Testing effectiveness (Phase 2)Study completedNCT02352896
What this trial is testing

Long-Term Safety and Efficacy Evaluation of EPI-743 in Children With Leigh Syndrome

Who this might be right for
Leigh Syndrome
PTC Therapeutics 30
Not applicableLooking for participantsNCT01793168
What this trial is testing

Rare Disease Patient Registry & Natural History Study - Coordination of Rare Diseases at Sanford

Who this might be right for
Rare DisordersUndiagnosed DisordersDisorders of Unknown Prevalence+340 more
Sanford Health 20,000
Testing effectiveness (Phase 2)Study completedNCT01721733
What this trial is testing

Safety and Efficacy Study of EPI-743 in Children With Leigh Syndrome

Who this might be right for
Leigh Syndrome
PTC Therapeutics 35
Testing effectiveness (Phase 2)Enrolling By InvitationNCT06843811
What this trial is testing

Sirolimus for Leigh Syndrome

Who this might be right for
Leigh Syndrome
Matthew Demczko 15
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