Not applicableUnknownNCT03347591What this trial is testingRare Bleeding Disorders in the NetherlandsWho this might be right forRare Bleeding Disorders Radboud University Medical Center 300
Not applicableStudy completedNCT00007033What this trial is testingStudy of Magnesium Sulfate in Children With Reduced Bone Density Secondary to Chronic Cholestatic Liver DiseaseWho this might be right forAlagille SyndromeCholestasisBiliary Atresia National Center for Research Resources (NCRR) 25
Not applicableStudy completedNCT00004761What this trial is testingEstablishment of the National Epidermolysis Bullosa RegistryWho this might be right forEpidermolysis Bullosa National Center for Research Resources (NCRR)
Not applicableUnknownNCT03683966What this trial is testingMigALastat Therapy Adherence Among FABRY Patients: A Prospective Multicentral Observational StudyWho this might be right forRare DiseasesFabry DiseaseAdherence, Medication+1 more Wuerzburg University Hospital 30
Not applicableEnded earlyNCT00004442What this trial is testingStudy of Bile Acids in Patients With Peroxisomal DisordersWho this might be right forInfantile Refsum's DiseaseZellweger SyndromeBifunctional Enzyme Deficiency+1 more University of Cincinnati 25
Not applicableStudy completedNCT00004788What this trial is testingStudy of Nutritional Factors in PorphyriaWho this might be right forPorphyria National Center for Research Resources (NCRR) 30
Not applicableLooking for participantsNCT06795152What this trial is testingRare Glycogen Storage Diseases Natural History StudyWho this might be right forGlycogen Storage DiseaseGSD Type 0AGSD Type 0B+12 more Duke University 200
Testing effectiveness (Phase 2)Active Not RecruitingNCT02978625What this trial is testingTalimogene Laherparepvec and Nivolumab in Treating Patients With Refractory Lymphomas or Advanced or Refractory Non-melanoma Skin CancersWho this might be right forAnaplastic Large Cell Lymphoma, ALK-NegativeAnaplastic Large Cell Lymphoma, ALK-PositiveApocrine Carcinoma+36 more National Cancer Institute (NCI) 68
Not applicableLooking for participantsNCT07575347What this trial is testingPeriodontal Disease in Rare Renal Disorders (PERIO-RA-RE)Who this might be right forPeriodontal DiseasePeriodontitisCKD+6 more Stefan Lujinschi 100
Testing effectiveness (Phase 2)Study completedNCT02056665What this trial is testingStudy to Evaluate the Efficacy and Safety of Minocycline in Angelman SyndromeWho this might be right forAngelman Syndrome Puerta de Hierro University Hospital 32
Not applicableStudy completedNCT00006130What this trial is testingDecompression Coring Versus Conservative Therapy in Patients With Avascular Necrosis of the Hip Related to Sickle Cell DiseaseWho this might be right forBone Avascular NecrosisSickle Cell Anemia UCSF Benioff Children's Hospital Oakland 50
Not applicableLooking for participantsNCT06573723What this trial is testingInstitutional Registry of Rare DiseasesWho this might be right forRare DiseasesAmyloidosisSarcoidosis+23 more Hospital Italiano de Buenos Aires 380
Not applicableLooking for participantsNCT06353620What this trial is testingStructural-functional Connectome in Drug-resistant Epilepsies and Neurodevelopmental Syndromes With EpilepsyWho this might be right forEpilepsyAngelman SyndromeDup15q Syndrome IRCCS Eugenio Medea 120
Not applicableStudy completedNCT05712564What this trial is testingFatigue, Depressive Disorders and Insomnia in Adult Patients with Marfan Syndrome and Ehlers-Danlos Syndrome: SurveyWho this might be right forRare DiseasesFatigueInsomnia+1 more IRCCS Policlinico S. Donato 282
Not applicableLooking for participantsNCT06360913What this trial is testingBlood Spot and Urine Metabolomic Screening Applied to Rare DiseasesWho this might be right forInborn Errors of MetabolismRare Diseases Cliniques universitaires Saint-Luc- Université Catholique de Louvain 2,286
Not applicableStudy completedNCT00005893What this trial is testingStudy of Allogeneic Bone Marrow Transplantation Using Matched, Related Donors in Patients With Nonmalignant Hematologic DisordersWho this might be right forNeutropeniaSickle Cell AnemiaThalassemia Major+1 more Fairview University Medical Center
Not applicableStudy completedNCT00006142What this trial is testingStudy of a Phenylalanine Restricted Diet During Pregnancy to Prevent Symptoms in Offspring of Patients With PhenylketonuriaWho this might be right forPhenylketonuria National Center for Research Resources (NCRR)
Not applicableLooking for participantsNCT01087320What this trial is testingGenome Medical Sequencing for Gene DiscoveryWho this might be right forIntellectual DisabilitiesCongenital AnomalyRare Disorders National Human Genome Research Institute (NHGRI) 2,000
Large-scale testing (Phase 3)Study completedNCT00004402What this trial is testingPhase III Randomized Study of Interferon Gamma in Children With Severe, Congenital OsteopetrosisWho this might be right forOsteopetrosis FDA Office of Orphan Products Development 30
Not applicableLooking for participantsNCT05868629What this trial is testingClinical Study To Further Evaluate The Efficacy Of Dabrafenib Plus Trametinib In Patients With Rare BRAF V600E Mutation-Positive Unresectable or Metastatic Solid TumorsWho this might be right forRare Unresectable or Metastatic BRAF V600E Mutation-positive Solid Tumors Novartis Pharmaceuticals 40