Not applicableStudy completedNCT00004760What this trial is testingStudy of Axial Mobility Exercises in Parkinson DiseaseWho this might be right forParkinson Disease Duke University 80
Not applicableStudy completedNCT00004274What this trial is testingEffect of Estrogen on Mental and Social Functioning in Girls With Turner's SyndromeWho this might be right forTurner's Syndrome National Institute of Neurological Disorders and Stroke (NINDS) 260
Early research (Phase 1)Active Not RecruitingNCT03478462What this trial is testingDose Escalation Study of CLR 131 in Pediatric Relapsed/Refractory Malignant Tumors Including Neuroblastoma and SarcomasWho this might be right forPediatric Solid TumorPediatric LymphomaPediatric Brain Tumor+5 more Cellectar Biosciences, Inc. 30
Not applicableEnrolling By InvitationNCT04703179What this trial is testingRare and Undiagnosed Disease Research BiorepositoryWho this might be right forUndiagnosed DiseaseRare Diseases Mayo Clinic 5,000
Not applicableStudy completedNCT00004479What this trial is testingRandomized Study of Midodrine, an Alpha Adrenergic Agonist, in Patients With Neurally Mediated SyncopeWho this might be right forSyncope Icahn School of Medicine at Mount Sinai
Testing effectiveness (Phase 2)Study completedNCT02923934What this trial is testingA Phase II Trial of Ipilimumab and Nivolumab for the Treatment of Rare CancersWho this might be right forGastrointestinal CancerNeuroendocrine TumoursMalignant Female Reproductive System Neoplasm Olivia Newton-John Cancer Research Institute 120
Not applicableLooking for participantsNCT06615206What this trial is testingA First-in-Human Clinical Trial to Evaluate the Safety, Tolerability, and Efficacy of a Novel CRISPR RNA-editing Therapy in Patients with Mecp2 Duplication Syndrome, a Rare Orphan Disease (HERO)Who this might be right forMECP2 Duplication Syndrome HuidaGene Therapeutics Co., Ltd. 6
Not applicableStudy completedNCT00004329What this trial is testingStudy of Alpha-2 Adrenergic Receptor Dysfunction in Regional LipoatrophyWho this might be right forLipodystrophy National Center for Research Resources (NCRR) 12
Testing effectiveness (Phase 2)Ended earlyNCT04171700What this trial is testingEvaluate Rucaparib in Participants With Solid Tumors and With Deleterious Mutations in HRR GenesWho this might be right forSolid Tumor pharmaand GmbH 83
Not applicableStudy completedNCT04725292What this trial is testingRare Benign Lesion of PeinsWho this might be right forSkin DiseasesPenile Diseases Assiut University 1
Not applicableStudy completedNCT00004495What this trial is testingRandomized Study of Folic Acid Therapy for Hyperhomocysteinemia in Patients With End Stage Renal Disease Receiving HemodialysisWho this might be right forEnd Stage Renal DiseaseHyperhomocysteinemia Georgetown University 84
Not applicableLooking for participantsNCT07786142What this trial is testingTransition of Care in Pediatric Rare Endocrine DisordersWho this might be right forEndocrine DisorderRare Disorders Seoul National University Hospital 150
Not applicableStudy completedNCT01914172What this trial is testingHealth Needs of Patients With Kallmann SyndromeWho this might be right forKallmann SyndromeCongenital Hypogonadotropic HypogonadismIdiopathic Hypogonadotropic Hypogonadism Centre Hospitalier Universitaire Vaudois 249
Testing effectiveness (Phase 2)Study completedNCT00004488What this trial is testingPhase II Randomized Study of Alendronate Sodium for Osteopenia in Patients With Gaucher's DiseaseWho this might be right forGaucher's DiseaseOsteopenia Children's Hospital Medical Center, Cincinnati 82
Not applicableUnknownNCT00004343What this trial is testingStudy of Hypercortisolism in Cushing's Syndrome and Stress-Induced Pseudo-Cushing's SyndromeWho this might be right forCushing's Syndrome National Center for Research Resources (NCRR) 40
Not applicableLooking for participantsNCT06213402What this trial is testingRADeep Multicenter European Epidemiological Platform for Patients Diagnosed With Rare Anemia Disorders (RADs)Who this might be right forSickle Cell DiseaseThalassemiaHemolytic; Anemia, Hereditary, Due to Enzyme Disorder+6 more Hospital Universitari Vall d'Hebron Research Institute 32,564
Not applicableStudy completedNCT04916977What this trial is testingGlobal Burden and Treatment Trajectiories in Italian Patients With Fabry DiseaseWho this might be right forFabry Disease Chiesi Italia 200
Not applicableLooking for participantsNCT06595940What this trial is testingGenetic Analysis of Uncommon Disease Presentations in Non-US PopulationsWho this might be right forUndiagnosed DiseasesRare Diseases National Human Genome Research Institute (NHGRI) 400
Large-scale testing (Phase 3)Study completedNCT00004650What this trial is testingPhase III Randomized Study of the Effect of Postmenopausal Estrogen Replacement Therapy on Alveolar Bone LossWho this might be right forOsteoporosis National Institute of Dental and Craniofacial Research (NIDCR) 150
Not applicableLooking for participantsNCT06676046What this trial is testingNatural History of Uncommon Dyslipidemias, Rare Lipid Disorders and Unusual Atherosclerotic ConditionsWho this might be right forDyslipidemiaAtherosclerosis National Heart, Lung, and Blood Institute (NHLBI) 1,500