Testing effectiveness (Phase 2)Study completedNCT03861871What this trial is testingFenfluramine in CDKL5 Deficiency Disorder (CDD)Who this might be right forCDD NYU Langone Health 7
Testing effectiveness (Phase 2)Study completedNCT02358538What this trial is testingOpen-Label PoC Trial of Ganaxolone in Children With PCDH19 Female Pediatric Epilepsy and Other Rare Genetic EpilepsiesWho this might be right forEpilepsy Marinus Pharmaceuticals 30
Testing effectiveness (Phase 2)Study completedNCT02758626What this trial is testingAtaluren for Nonsense Mutation in CDKL5 and Dravet SyndromeWho this might be right forEpilepsy NYU Langone Health 15
Testing effectiveness (Phase 2)Ended earlyNCT03635073What this trial is testingSoticlestat in Adults and Children With Rare EpilepsiesWho this might be right forEpilepsyDravet Syndrome (DS)Lennox-Gastaut Syndrome (LGS) Takeda 156
Not applicableStudy completedNCT02738281What this trial is testingNatural History of Rett Syndrome & Related DisordersWho this might be right forRett SyndromeMECP2 Duplication dIsorderCDKL5 Disorder+1 more University of Alabama at Birmingham 1,044
Not applicableLooking for participantsNCT06585605What this trial is testingA Retrospective Survey-based Multicenter Study to Delineate the Molecular and Phenotypic Spectrum of Epilepsy-dyskinesia SyndromesWho this might be right forEpilepsy in ChildrenDyskinesiasMovement Disorders in Children+7 more Boston Children's Hospital 500
Not applicableTemporarily pausedNCT04486768What this trial is testingInternational CDKL5 RegistryWho this might be right forCDKL5 Deficiency Disorder (CDD) University of Pennsylvania 500
Not applicableLooking for participantsNCT06967727What this trial is testingRegistry and Natural History of Epilepsy-Dyskinesia SyndromesWho this might be right forEpilepsy-DyskinesiaEpilepsyDyskinesia+11 more Boston Children's Hospital 700
Testing effectiveness (Phase 2)Study completedNCT03694275What this trial is testingA Multicenter, Open-label, Pilot Study of Soticlestat (TAK-935/OV935) in Participants With 15Q Duplication Syndrome (Dup 15q) or Cyclin-Dependent Kinase-Like 5 (CDKL5) Deficiency Disorder (ARCADE STUDY)Who this might be right for15q Duplication SyndromeCDKL5 Deficiency Disease Takeda 20
Not applicableNo Longer AvailableNCT04678479What this trial is testingGanaxolone Expanded Access Program Compassionate UseWho this might be right forCDKL5 Disorder Marinus Pharmaceuticals
Not applicableActive Not RecruitingNCT03655223What this trial is testingEarly Check: Expanded Screening in NewbornsWho this might be right forSpinal Muscular AtrophyFragile X SyndromeFragile X - Premutation+182 more RTI International 30,000
Not applicableStudy completedNCT06663163What this trial is testingThe Association Between Fever and CDKL5 Deficiency Disorder: a Nationwide SurveyWho this might be right forCDKL5 Deficiency Disorder Xuanwu Hospital, Beijing 131
Not applicableStudy completedNCT06447675What this trial is testingEfficacy and Safety Evaluation of Hyperthermic Baths in the Treatment of Seizures in CDKL5 Deficiency DisorderWho this might be right forCDKL5 Deficiency Disorder Xuanwu Hospital, Beijing 8
Testing effectiveness (Phase 2)Study completedNCT05626634What this trial is testingOpen-label, Long-term Safety Study of LP352 in Subjects With Developmental and Epileptic EncephalopathyWho this might be right forDevelopmental and Epileptic EncephalopathyDravet SyndromeLennox-Gastaut Syndrome Longboard Pharmaceuticals 41
Not applicableActive Not RecruitingNCT05373719What this trial is testingObservational Study in Patients With Cyclin-dependent Kinase-like 5 Deficiency DisorderWho this might be right forCyclin-Dependent Kinase-Like 5 Deficiency Disorder Loulou Foundation 112
Testing effectiveness (Phase 2)Study completedNCT05364021What this trial is testingStudy to Investigate LP352 in Subjects With Developmental and Epileptic EncephalopathiesWho this might be right forDevelopmental and Epileptic EncephalopathyDravet SyndromeLennox Gastaut Syndrome Longboard Pharmaceuticals 52
Not applicableUnknownNCT06448663What this trial is testingTargeting the Gut to Improve Seizure Control in CDKL5 Deficiency Disorder (CDD)Who this might be right forCDKL5 University of Milan 20
Testing effectiveness (Phase 2)Study completedNCT03650452What this trial is testingA Phase 2, Multicenter, Randomized, Double-blind, Placebo-controlled Study to Evaluate the Efficacy, Safety, and Tolerability of TAK-935 (OV935) as an Adjunctive Therapy in Pediatric Participants With Developmental and/or Epileptic EncephalopathiesWho this might be right forEpilepsyDravet SyndromeLennox-Gastaut Syndrome Takeda 141
Large-scale testing (Phase 3)Not Yet RecruitingNCT05249556What this trial is testingDouble-blind, Randomized, Placebo-controlled Trial of Ganaxolone in CDKL5 Deficiency Patients 6 Months to Less Than 2 Years OldWho this might be right forCDKL5 Deficiency Disorder Immedica Pharma AB 20
Not applicableStudy completedNCT00299312What this trial is testingGenetic and Physical Characteristics of Rett SyndromeWho this might be right forRett SyndromeMECP2 Duplication DisorderRett-related Disorder University of Alabama at Birmingham 10