Not applicableUnknownNCT05750693What this trial is testingPIK3CA in HER2+ BC and pCR TrialWho this might be right forHER2-positive Breast Cancer Fondazione Policlinico Universitario Agostino Gemelli IRCCS 58
Testing effectiveness (Phase 2)Study completedNCT01502046What this trial is testingNeuroprotection by Cannabinoids in Huntington's DiseaseWho this might be right forHuntington's Disease Fundacion para la Investigacion Biomedica del Hospital Universitario Ramon y Cajal 25
Large-scale testing (Phase 3)Study completedNCT02471339What this trial is testingAcceptance and Commitment Training for Adolescents and Young Adults With Neurofibromatosis Type 1, Plexiform Neurofibromas, and Chronic PainWho this might be right forNeurofibromatosis Type 1Plexiform Neurofibromas National Cancer Institute (NCI) 66
Testing effectiveness (Phase 2)Study completedNCT04114539What this trial is testingEcopipam Tablets to Study Tourette Syndrome in Children and Adolescents - Open Label ExtensionWho this might be right forTourette Syndrome in ChildrenTourette Syndrome in Adolescence Emalex Biosciences Inc. 124
Testing effectiveness (Phase 2)Study completedNCT01965327What this trial is testingInterferon Gamma-1b in Friedreich Ataxia (FRDA)Who this might be right forFriedreich Ataxia Children's Hospital of Philadelphia 12
Testing effectiveness (Phase 2)Study completedNCT02595983What this trial is testingThe Study of an Investigational Drug, Revusiran (ALN-TTRSC), for the Treatment of Transthyretin (TTR)-Mediated Amyloidosis in Patients Whose Disease Has Continued to Worsen Following Liver TransplantWho this might be right forTransthyretin (TTR)-Mediated AmyloidosisFamilial Amyloidotic Polyneuropathy (FAP)ATTR Amyloidosis+1 more Alnylam Pharmaceuticals 12
Testing effectiveness (Phase 2)Study completedNCT02147886What this trial is testingStudy To Assess Safety, Tolerability and Efficacy of Intravenous Cabaletta in Patients With Machado-Joseph DiseaseWho this might be right forMachado-Joseph Disease / Spinocerebellar Ataxia 3 Bioblast Pharma Ltd. 15
Not applicableLooking for participantsNCT07154394What this trial is testingCardiac Amyloid Deposits and Heart Dysfunction in Alzheimer's DiseaseWho this might be right forDementia, Alzheimer TypeMild Cognitive ImpairmentCardiac Amyloidosis University Hospital, Essen 15
Not applicableUnknownNCT04370470What this trial is testingDevelopment of Assessments for Later Stage HDWho this might be right forHuntington Disease CHDI Foundation, Inc. 340
Not applicableStudy completedNCT03709173What this trial is testingFuRST 2.0 Cognitive Pre-testing - Round 2Who this might be right forHuntington Disease CHDI Foundation, Inc. 52
Not applicableLooking for participantsNCT03475173What this trial is testingNew Non-invasive Modalities for Assessing Retinal Structure and FunctionWho this might be right forIschemic Optic NeuropathyBranch Retinal Artery OcclusionHemianopia+2 more Randy Kardon 500
Early research (Phase 1)UnknownNCT00675077What this trial is testingFar Infrared Irradiation for Managing, Control and Treatment of Huntington's Disease (HD)Who this might be right forHuntington Disease GAAD Medical Research Institute Inc. 5
Early research (Phase 1)UnknownNCT04432857What this trial is testingAN0025 and Pembrolizumab Combination in Advanced Solid TumorsWho this might be right forTriple-negative Breast CancerNSCLC, Squamous or Non-SquamousUrothelial Carcinoma of the Bladder+2 more Adlai Nortye Biopharma Co., Ltd. 63
Large-scale testing (Phase 3)Study completedNCT04298372What this trial is testingFrontline Lenalidomide for AL Amyloidosis Involving MyocardiumWho this might be right forAmyloidosis Cardiac Seoul National University Hospital 2
Testing effectiveness (Phase 2)Study completedNCT07010575What this trial is testingPatient Preference Study: Standard of Care Versus Once-daily Trientine TetrahydrochlorideWho this might be right forWilson's Disease Orphalan 10
Not applicableEnded earlyNCT03056144What this trial is testingWhole Body Vibration Therapy in Children With Spinal Muscular AtrophyWho this might be right forSpinal Muscular Atrophy Type 3 The Hong Kong Polytechnic University 1
Testing effectiveness (Phase 2)Study completedNCT02934256What this trial is testingIcotinib Study for Patients With Neurofibromatosis Type 2 (NF2) and NF2-Related TumorsWho this might be right forVestibular SchwannomaNeurofibromatosis Type 2 Li Peng 10
Not applicableUnknownNCT04698551What this trial is testingNIPD on cffDNA for Triplet Repeat DiseasesWho this might be right forMyotonic Dystrophy 1Huntington DiseaseFragile X Syndrome University Hospital, Montpellier 36
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
Not applicableLooking for participantsNCT07362875What this trial is testingDevelopment of Quantitative Muscle Imaging as a Biomarker of Disease Endpoints in Myotonic DystrophyWho this might be right forMyotonic Dystrophy Wake Forest University Health Sciences 75