Not applicableLooking for participantsNCT06619197What this trial is testingExploratory Study on Global Reflexology in Sickle Cell DiseaseWho this might be right forSickle Cell Disease (SCD) Hospices Civils de Lyon 30
Not applicableEnrolling By InvitationNCT03655223What this trial is testingEarly Check: Expanded Screening in NewbornsWho this might be right forSpinal Muscular AtrophyFragile X SyndromeFragile X - Premutation+182 more RTI International 30,000
Not applicableLooking for participantsNCT02399527What this trial is testingLymphatic Anomalies Registry for the Assessment of Outcome DataWho this might be right forLymphatic MalformationGeneralized Lymphatic Anomaly (GLA)Central Conducting Lymphatic Anomaly+7 more Boston Children's Hospital 1,000
Not applicableEnded earlyNCT04964323What this trial is testingPyruvate Kinase (PK) Deficiency Global Longitudinal Registry: Patient-Reported Outcomes (PRO)Who this might be right forPyruvate Kinase DeficiencyPyruvate Kinase Deficiency AnemiaAnemia+1 more Agios Pharmaceuticals, Inc. 15
Not applicableLooking for participantsNCT06065852What this trial is testingNational Registry of Rare Kidney DiseasesWho this might be right forAdenine Phosphoribosyltransferase DeficiencyAH AmyloidosisAHL Amyloidosis+81 more UK Kidney Association 35,000
Testing effectiveness (Phase 2)UnknownNCT05920512What this trial is testingCombination Regimen With Sodium Valproate for Severe Hemophilia: a Single-arm, Phase 1, Pilot Trial.Who this might be right forHemophilia Xue-chun Lu 10
Not applicableStudy completedNCT02787486What this trial is testingExpanded Noninvasive Genomic Medical Assessment: The Enigma StudyWho this might be right forDown SyndromeEdwards SyndromePatau Syndrome+5 more Progenity, Inc. 760
Testing effectiveness (Phase 2)Study completedNCT03370172What this trial is testingBAX 888 in Male Adults With Severe Hemophilia AWho this might be right forHemophilia A Baxalta now part of Shire 4
Not applicableLooking for participantsNCT06755775What this trial is testingPET Imaging Targeting Granzyme B Predicts Immunotherapy Efficacy in Diffuse Large B-cell LymphomaWho this might be right forDiffuse Large B-Cell Lymphoma Ruijin Hospital 150
Not applicableLooking for participantsNCT04806620What this trial is testingUnhide® Project: A Digital Health Platform to Collect Lifestyle Data for Brain Inflammation ResearchWho this might be right forPost-Acute COVID-19 SyndromeME/CFSRheumatic Arthritis+41 more Brain Inflammation Collaborative 10,000
Not applicableUnknownNCT03758534What this trial is testingNatural History of GACI With or Without ARHR2 or PXEWho this might be right forGeneralized Arterial Calcification in InfancyAutosomal Recessive Hypophosphatemic RicketsPseudoxanthoma Elasticum Universität Münster 80
Not applicableUnknownNCT02389205What this trial is testingThe Effect of Functional Activities and Balance Ability With Taping in Subjects of HemophiliaWho this might be right forHemophilia Tri-Service General Hospital 30
Testing effectiveness (Phase 2)Ended earlyNCT05789537What this trial is testingSerpinPC in Participants with Hemophilia B (HemB) with InhibitorsWho this might be right forHemophilia B with InhibitorHemophilia B ApcinteX Ltd 3
Post-approval studies (Phase 4)Active Not RecruitingNCT02386800What this trial is testingCINC424A2X01B Rollover ProtocolWho this might be right forPrimary MyelofibrosisPolycythemia VeraGraft Versus Host Disease+2 more Novartis Pharmaceuticals 279
Not applicableStudy completedNCT01949220What this trial is testingWillebrand International Non-interventional Global SurveillanceWho this might be right forVon Willebrand Disease Laboratoire français de Fractionnement et de Biotechnologies 80
Testing effectiveness (Phase 2)Looking for participantsNCT06462547What this trial is testingADAPT Study: Long-term Safety Study of INZ-701 in Patients With ENPP1 Deficiency and ABCC6 DeficiencyWho this might be right forGene MutationsPseudoxanthoma ElasticumArterial Calcification+2 more Inozyme Pharma 200
Not applicableStudy completedNCT02744027What this trial is testingImaging of Lymphatic AnomaliesWho this might be right forGorham DiseaseLymphatic DiseasesLymphangiomatosis Children's Hospital of Philadelphia 10
Not applicableLooking for participantsNCT05361980What this trial is testingPediatric Orthopaedic Implant Safety & EfficacyWho this might be right forFractureLengthening; LegOrthopedic Disorder+8 more University of British Columbia 4,000
Not applicableStudy completedNCT01931644What this trial is testingAt-Home Research Study for Patients With Autoimmune, Inflammatory, Genetic, Hematological, Infectious, Neurological, CNS, Oncological, Respiratory, Metabolic ConditionsWho this might be right forAll Diagnosed Health ConditionsADD/ADHDAlopecia Areata+58 more Sanguine Biosciences 17,667
Not applicableLooking for participantsNCT06999954What this trial is testingShwachman-Diamond Syndrome Global Patient Survey and Partnering PlatformWho this might be right forShwachman-Diamond SyndromeSDSIBMF+24 more Shwachman-Diamond Syndrome Alliance Inc 8,000