Not applicableAvailableNCT05930223What this trial is testingIntravenous VAL-1221 Lafora Expanded Access ProtocolWho this might be right forLafora Disease Parasail, LLC
Not applicableStudy completedNCT00007124What this trial is testingKetogenic Diet in Lafora DiseaseWho this might be right forLafora Disease National Institute of Neurological Disorders and Stroke (NINDS) 15
Testing effectiveness (Phase 2)Study completedNCT06051448What this trial is testingPromoting Resilience in Stress Management (PRISM) and Clinical-focused Narrative (CFN) Pilot in Adults With Primary Mitochondrial Disease (PMD).Who this might be right forMitochondrial DiseasesMitochondrial MyopathiesMitochondrial Encephalomyopathies+2 more Children's Hospital of Philadelphia 16
Not applicableLooking for participantsNCT06593951What this trial is testingRegistry and Natural History Study for Progressive Myoclonus Epilepsy Type 1 (EPM1)Who this might be right forProgressive Myoclonus Epilepsy Type 1EPM1CSTB-related Disease+5 more Boston Children's Hospital 200
Not applicableLooking for participantsNCT05554835What this trial is testingGlobal Registry and Natural History Study for Mitochondrial DisordersWho this might be right forMitochondrial DiseasesKearns-Sayre SyndromeMIDD+15 more LMU Klinikum 6,000
Testing effectiveness (Phase 2)UnknownNCT00639119What this trial is testingEffect of Ropinirole Hydrochloride in Progressive Myoclonic Epilepsy of Unverricht-Lundborg TypeWho this might be right forUnverricht-Lundborg Syndrome University of Turku 16
Testing effectiveness (Phase 2)Active Not RecruitingNCT07084311What this trial is testingPersonalized Antisense Oligonucleotide for A Single Participant With ATN1 Gene MutationWho this might be right forDentatorubral-Pallidoluysian Atrophy n-Lorem Foundation 1
Not applicableStudy completedNCT00006176What this trial is testingClinical and Genetic Studies of Familial Presenile Dementia With Neuronal Inclusion BodiesWho this might be right forFamilial Dementia With Neuroserpin Inclusion BodiesNervous System Heredodegenerative Disorder National Human Genome Research Institute (NHGRI) 100
Testing effectiveness (Phase 2)Study completedNCT02023866What this trial is testingOpen-Label, Dose-Escalating Study Assessing Safety, Tolerability, Efficacy, of RP103 in Mitochondrial DiseaseWho this might be right forInherited Mitochondrial Disease, Including Leigh Syndrome Amgen 36
Large-scale testing (Phase 3)Study completedNCT05218655What this trial is testingA Safety Study for Previously Treated Vatiquinone (PTC743) Participants With Inherited Mitochondrial DiseaseWho this might be right forInherited Mitochondrial Disease PTC Therapeutics 101
Testing effectiveness (Phase 2)Ended earlyNCT02473445What this trial is testingA Long-term Extension of Study RP103-MITO-001 (NCT02023866) to Assess Cysteamine Bitartrate Delayed-release Capsules (RP103) in Children With Inherited Mitochondrial DiseaseWho this might be right forMitochondrial Diseases Amgen 22
Testing effectiveness (Phase 2)Study completedNCT05972954What this trial is testingOMT-28 in Patients With Primary Mitochondrial Disease (PMD) (PMD-OPTION)Who this might be right forPrimary Mitochondrial Disease Omeicos Therapeutics GmbH 28
Testing effectiveness (Phase 2)Looking for participantsNCT06609889What this trial is testingA Safety and Efficacy of Intrathecally Administered ION283 in Patients With Lafora DiseaseWho this might be right forLafora Disease Berge Minassian 10
Large-scale testing (Phase 3)Ended earlyNCT04378075What this trial is testingEvaluate Efficacy and Safety of Vatiquinone for Treating Mitochondrial Disease in Participants With Refractory EpilepsyWho this might be right forMitochondrial DiseasesDrug Resistant EpilepsyLeigh Disease+5 more PTC Therapeutics 68
Large-scale testing (Phase 3)UnknownNCT03351569What this trial is testingIntravenous Immunoglobulin for Unverricht-Lundborg Disease.Who this might be right forUnverricht-Lundborg Disease Azienda Socio Sanitaria Territoriale di Mantova 1
Not applicableLooking for participantsNCT01694940What this trial is testingNorth American Mitochondrial Disease Consortium Patient Registry and Biorepository (NAMDC)Who this might be right forMitochondrial DisordersMitochondrial Genetic DisordersMitochondrial Diseases+2 more Columbia University 1,000
Large-scale testing (Phase 3)Study completedNCT00357669What this trial is testingBrivaracetam as add-on Treatment of Unverricht-Lundborg Disease in Adolescents and AdultsWho this might be right forUnverricht-Lundborg Disease UCB Pharma SA 50
Not applicableStudy completedNCT03876522What this trial is testingNatural History and Functional Status Study of Patients With Lafora DiseaseWho this might be right forLafora Disease Ionis Pharmaceuticals, Inc. 33
Testing effectiveness (Phase 2)Active Not RecruitingNCT07221760What this trial is testingPersonalized Antisense Oligonucleotide for A Single Participant (nL62541) With ATN1 Gene MutationWho this might be right forDentatorubral-Pallidoluysian Atrophy n-Lorem Foundation 1
Testing effectiveness (Phase 2)Active Not RecruitingNCT06706388What this trial is testingPersonalized Antisense Oligonucleotide Therapy for A Single Participant With ATN1 Gene MutationWho this might be right forDentatorubral-Pallidoluysian Atrophy n-Lorem Foundation 1