Not applicableEnded earlyNCT01330277What this trial is testingBiomarkers for Hunter SyndromeWho this might be right forHunter SyndromeMucopolysaccharidosis IIHunter's Syndrome, Mild Form+1 more CENTOGENE GmbH Rostock 11
Not applicableStudy completedNCT03396042What this trial is testingNatural History Study of CEP290-Related Retinal DegenerationWho this might be right forBlindnessLeber Congenital Amaurosis 10Vision Disorders+5 more Editas Medicine, Inc. 26
Large-scale testing (Phase 3)Ended earlyNCT02291237What this trial is testingEffect of Eleclazine (GS-6615) on Exercise Capacity in Subjects With Symptomatic Hypertrophic CardiomyopathyWho this might be right forHypertrophic Cardiomyopathy Gilead Sciences 172
Testing effectiveness (Phase 2)UnknownNCT02191839What this trial is testingSingle Dose Administration of Alpha-1 Anti-Trypsin for the Amelioration of Organ Injury in Patients Undergoing Cardiac SurgeryWho this might be right forPost Cardiac Surgery Systemic Inflammatory Response Soroka University Medical Center 20
Not applicableUnknownNCT05534139What this trial is testingClinicopathological MRI and CSF Correlates in Huntington's Disease.Who this might be right forHuntington Disease Leiden University Medical Center 90
Large-scale testing (Phase 3)Study completedNCT05253781What this trial is testingLow Dose Aspirin for Preventing Intrauterine Growth Restriction and Preeclampsia in Sickle Cell Pregnancy (PIPSICKLE)Who this might be right forIntrauterine Growth RestrictionPreeclampsiaSickle Cell Disease+1 more University of Lagos, Nigeria 476
Not applicableStudy completedNCT00497575What this trial is testingDiagnosis and Follow-up of Patients With Subclinical HypothyroidismWho this might be right forCongenital HypothyroidismL-ThyroxineFollow-Up Zhejiang University
Testing effectiveness (Phase 2)Ended earlyNCT04414631What this trial is testingConestat Alfa in the Prevention of Severe SARS-CoV-2 Infection in Hospitalized Patients With COVID-19Who this might be right forCoronavirus Infections University Hospital, Basel, Switzerland 80
Large-scale testing (Phase 3)Enrolling By InvitationNCT02796937What this trial is testingLong Term Safety of Alpha1-Proteinase Inhibitor in Subjects With Alpha1 Antitrypsin DeficiencyWho this might be right forPulmonary Emphysema in Alpha-1 Antitrypsin Deficiency Grifols Therapeutics LLC 290
Large-scale testing (Phase 3)Study completedNCT00880100What this trial is testingUse of Ultrase® MT12 in Young Cystic Fibrosis Children (CF)Who this might be right forCystic FibrosisPancreatic Insufficiency Forest Laboratories 49
Early research (Phase 1)Study completedNCT00003107What this trial is testingInterleukin-12 in Treating Patients With Hematologic Cancers or Solid TumorsWho this might be right forBreast CancerChronic Myeloproliferative DisordersGestational Trophoblastic Tumor+8 more Indiana University
Not applicableStudy completedNCT01920464What this trial is testingPlace of Flixovate® in the Treatment Pathway and Its Conditions of Use in Infants Aged From 3 to 12 Months Between 2010 and 2012 by French GPs and Private PaediatriciansWho this might be right forDermatitis, Atopic GlaxoSmithKline 160
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
Not applicableNot Yet RecruitingNCT07799792What this trial is testingImplementation-effectiveness Trial of Mainstreaming of Clinical Genomic Sequencing for Rare Disease in Ontario, CanadaWho this might be right forEvaluation of Mainstreamed Models of Genetic Service DeliveryGenetic DiseaseNeurodevelomental Disorders The Hospital for Sick Children 100
Early research (Phase 1)Study completedNCT03718234What this trial is testingSubcutaneous Hydrocortisone Children With Congenital Adrenal HyperplasiaWho this might be right forCongenital Adrenal HyperplasiaHyperplasiaAdrenal Hyperplasia+8 more University of Minnesota 11
Not applicableStudy completedNCT01322165What this trial is testingNational Registry of Genetically Triggered Thoracic Aortic Aneurysms and Cardiovascular ConditionsWho this might be right forMarfan SyndromeTurner SyndromeEhlers-Danlos Syndrome+9 more National Heart, Lung, and Blood Institute (NHLBI) 3,706
Not applicableStudy completedNCT00960128What this trial is testingObservational Prospective Study on Patients Treated With Norditropin®Who this might be right forGrowth Hormone DisorderGrowth Hormone Deficiency in ChildrenAdult Growth Hormone Deficiency+7 more Novo Nordisk A/S 21,249
Not applicableStudy completedNCT05123339What this trial is testingClinical Signs and Activity Limitations Associated With Dural Ectasia in Patients With Marfan DiseaseWho this might be right forMarfan Syndrome Assistance Publique - Hôpitaux de Paris 90
Early research (Phase 1)Study completedNCT00020267What this trial is testingVaccine Therapy in Treating Patients With Metastatic CancerWho this might be right forLung CancerAdult Soft Tissue SarcomaColorectal Cancer+8 more National Cancer Institute (NCI)
Testing effectiveness (Phase 2)Study completedNCT00799071What this trial is testingPharmacokinetics of Posaconazole in Children With Chronic Granulomatous Disease (CGD)Who this might be right forChronic Granulomatous Disease Radboud University Medical Center 12