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Results for “Inherited Mitochondrial Disease”

338 trials

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Showing 20 of 338 results

Early research (Phase 1)Ended earlyNCT00060515
What this trial is testing

RG2133 (2',3',5'-Tri-O-Acetyluridine) in Mitochondrial Disease

Who this might be right for
Mitochondrial Diseases
Repligen Corporation 12
Not applicableNo Longer AvailableNCT04381091
What this trial is testing

Expanded Access Program for Idebenone in Patients With Leber's Hereditary Optic Neuropathy Who Completed the LEROS Study

Who this might be right for
Leber's Hereditary Optic Neuropathy
Santhera Pharmaceuticals
Not applicableLooking for participantsNCT05522374
What this trial is testing

TIRCON International NBIA Registry

Who this might be right for
Neurodegeneration With Brain Iron Accumulation (NBIA)Pantothenate Kinase-associated Neurodegeneration (PKAN)Beta-Propeller Protein-Associated Neurodegeneration (BPAN)+8 more
LMU Klinikum 2,000
Not applicableStudy completedNCT01776918
What this trial is testing

Energy Requirements in Mitochondrial Disease

Who this might be right for
Mitochondrial DiseaseChronic Metabolic Disorder
University of British Columbia 23
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 applicableStudy completedNCT01625663
What this trial is testing

Heart and Muscle Metabolism in Barth Syndrome

Who this might be right for
Barth Syndrome
Duke University 64
Testing effectiveness (Phase 2)Study completedNCT02445794
What this trial is testing

A First in Human Study of RT001 in Patients With Friedreich's Ataxia

Who this might be right for
Friedreich's Ataxia
Biojiva LLC 19
Early research (Phase 1)Study completedNCT02161380
What this trial is testing

Safety Study of an Adeno-associated Virus Vector for Gene Therapy of Leber's Hereditary Optic Neuropathy

Who this might be right for
Leber's Hereditary Optic Neuropathy
Byron Lam 28
Testing effectiveness (Phase 2)Study completedNCT00824512
What this trial is testing

Efficacy of EGb761 in Patients Suffering From Friedreich Ataxia

Who this might be right for
Friedreich Ataxia
Ipsen 22
Not applicableStudy completedNCT00000665
What this trial is testing

Studies of the Ocular Complications of AIDS (SOCA) CMV Retinitis Trial: Foscarnet-Ganciclovir Component

Who this might be right for
Cytomegalovirus RetinitisHIV Infections
National Institute of Allergy and Infectious Diseases (NIAID) 240
Large-scale testing (Phase 3)Active Not RecruitingNCT05515536
What this trial is testing

Assess the Safety and Efficacy of Vatiquinone in Participants With Friedreich Ataxia

Who this might be right for
Friedreich Ataxia
PTC Therapeutics 130
Not applicableStudy completedNCT03295071
What this trial is testing

REALITY LHON Registry

Who this might be right for
Leber Hereditary Optic Neuropathy
GenSight Biologics 44
Testing effectiveness (Phase 2)Study completedNCT00031434
What this trial is testing

Valganciclovir in Congenital CMV Infants

Who this might be right for
Cytomegalovirus Infections
National Institute of Allergy and Infectious Diseases (NIAID) 24
Large-scale testing (Phase 3)Study completedNCT02593773
What this trial is testing

Safety, Tolerability and Efficacy of ACTIMMUNE® Dose Escalation in Friedreich's Ataxia Study

Who this might be right for
Friedreich's Ataxia
Amgen 86
Not applicableStudy completedNCT03418740
What this trial is testing

Neurology Measures in FA Children

Who this might be right for
Friedreich Ataxia
Children's Hospital of Philadelphia 108
Testing effectiveness (Phase 2)WithdrawnNCT03866954
What this trial is testing

Trial of Erythrocyte Encapsulated Thymidine Phosphorylase In Mitochondrial Neurogastrointestinal Encephalomyopathy

Who this might be right for
Mitochondrial DiseasesMetabolic DiseaseInborn Errors of Metabolism
St George's, University of London
Testing effectiveness (Phase 2)Study completedNCT01016366
What this trial is testing

Safety Study of Carbamylated Erythropoietin to Treat Patients With the Neurodegenerative Disorder Friedreich's Ataxia

Who this might be right for
Friedreich's Ataxia
H. Lundbeck A/S 36
Not applicableLooking for participantsNCT05929209
What this trial is testing

Exploring Biomarkers in Hereditary Transthyretin Amyloidosis

Who this might be right for
Hereditary Transthyretin Amyloidosis
Fondazione Policlinico Universitario Agostino Gemelli IRCCS 80
Post-approval studies (Phase 4)Study completedNCT01831934
What this trial is testing

Responses to Influenza Vaccine in Patients With Mitochondrial Disorders (MELAS)

Who this might be right for
MELAS Syndrome
Stanford University 22
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
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