Testing effectiveness (Phase 2)Study completedNCT03126019What this trial is testingINCB050465 in Relapsed or Refractory Follicular LymphomaWho this might be right forLymphoma Incyte Corporation 126
Not applicableStudy completedNCT04256681What this trial is testingSNAP: Measurement of the Subjective Perception of the Symptom in Hereditary Spastic Paraparesis (HSP)Who this might be right forHereditary Spastic Paraparesis IRCCS Eugenio Medea 80
Testing effectiveness (Phase 2)Study completedNCT01733407What this trial is testingL-Serine Supplementation in Hereditary Sensory Neuropathy Type 1Who this might be right forHereditary Sensory and Autonomic Neuropathy Type I Massachusetts General Hospital 18
Not applicableLooking for participantsNCT05848271What this trial is testingNatural History Study of Patients with HPDL MutationsWho this might be right forMitochondrial EncephalomyopathiesHereditary Spastic ParaplegiaSpastic Paraplegia+4 more University of California, San Diego 50
Large-scale testing (Phase 3)UnknownNCT01289704What this trial is testingTreadmill, Stretching and Proprioceptive Exercise (TreSPE) Rehabilitation Program for Charcot-Marie-Tooth Neuropathy Type 1A (CMT1A)Who this might be right forCharcot-Marie-Tooth DiseaseCharcot-Marie-Tooth Disease Type 1A University of Genova 92
Not applicableStudy completedNCT02876939What this trial is testingProprioception and Sensorimotor Control in Hereditary Sensory and Autonomic NeuropathyWho this might be right forAtunomic Neuropathy NYU Langone Health 16
Not applicableLooking for participantsNCT01193075What this trial is testingNatural History Evaluation of Charcot Marie Tooth Disease (CMT) Types CMT1B, CMT2A, CMT4A, CMT4C, and OthersWho this might be right forCharcot Marie Tooth Disease Michael Shy 5,000
Large-scale testing (Phase 3)UnknownNCT06293716What this trial is testingCVL237 Tablets for APDS/PASLIWho this might be right forPI3K and P110delta Hyperactivation Syndrome Convalife (Shanghai) Co., Ltd. 35
Post-approval studies (Phase 4)Study completedNCT04828993What this trial is testingThe Effect Of Tafamidis Meglumine In Transthyretin Amyloid Polyneuropathy PatientsWho this might be right forTransthyretin Amyloid Polyneuropathy (ATTR-PN) Pfizer 15
Testing effectiveness (Phase 2)Study completedNCT02693119What this trial is testingA Study Investigating the Safety, Tolerability, and Efficacy of Elamipretide Topical Ophthalmic Solution for Treatment of Leber's Hereditary Optic NeuropathyWho this might be right forLeber's Hereditary Optic Neuropathy Stealth BioTherapeutics Inc. 12
Not applicableUnknownNCT03984877What this trial is testingImpact of Amyloidosis on TAVI PatientsWho this might be right forValve Stenoses, AorticAmyloidosis Cardiac Javier López Pais 320
Not applicableLooking for participantsNCT06813443What this trial is testingCharacterization of Patients With Cardiomyopathy to Identify Critical Patients Candidates for Cardiac TransplantationWho this might be right forCardiomyopathiesAmyloidosis CardiacFabry Disease+14 more IRCCS Azienda Ospedaliero-Universitaria di Bologna 700
Large-scale testing (Phase 3)Study completedNCT03860935What this trial is testingEfficacy and Safety of AG10 in Subjects With Transthyretin Amyloid CardiomyopathyWho this might be right forAmyloidosisAmyloid CardiomyopathyTransthyretin Amyloidosis+2 more Eidos Therapeutics, a BridgeBio company 632
Not applicableLooking for participantsNCT07188415What this trial is testingCMT Gait, Mobility, Balance - AOFAS GrantWho this might be right forCharcot Marie Tooth Disease (CMT) Bopha Chrea 66
Not applicableUnknownNCT04967716What this trial is testingGenetics of Charcot-Marie-Tooth Dystrophy and Related DiseasesWho this might be right forPeroneal Muscular Atrophy Peking University Third Hospital 200
Early research (Phase 1)Study completedNCT01744704What this trial is testingStudy to Evaluate Safety, Tolerability and Pharmacokinetics of rhNGF Eye Drops in Healthy VolunteersWho this might be right forHealthy Dompé Farmaceutici S.p.A 74
Not applicableStudy completedNCT00023075What this trial is testingNuclear Magnetic Spectroscopy Imaging to Evaluate Primary Lateral Sclerosis, Hereditary Spastic Paraplegia and Amyotrophic Lateral SclerosisWho this might be right forPrimary Lateral SclerosisHereditary Spastic ParaplegiaAmyotrophic Lateral Sclerosis National Institute of Neurological Disorders and Stroke (NINDS) 55
Testing effectiveness (Phase 2)Active Not RecruitingNCT03536767What this trial is testingOpen-Label Study of AG10 in Patients with CardiomyopathyWho this might be right forAmyloid Cardiomyopathy Eidos Therapeutics, a BridgeBio company 55
Not applicableLooking for participantsNCT07476365What this trial is testingA Multi-omic Approach to the Identification of Novel Biomarkers in Early Charcot-Marie-Tooth 1A Disease (CMT1A)Who this might be right forCMTCMT (Charcot Marie Tooth Disease)CMT - Charcot-Marie-Tooth Disease+2 more University Medical Center Goettingen 70
Large-scale testing (Phase 3)Looking for participantsNCT06672237What this trial is testingNTLA-2001 in ATTRv-PNWho this might be right forNeuromuscular DiseaseNeuromuscular Diseases (NMD)Neurodegenerative Disease+17 more Intellia Therapeutics 60