Large-scale testing (Phase 3)Study completedNCT02526160What this trial is testingStudy of KRN23 in Adults With X-linked Hypophosphatemia (XLH)Who this might be right forX-linked Hypophosphatemia Kyowa Kirin, Inc. 134
Large-scale testing (Phase 3)Study completedNCT00930176What this trial is testingA Study Investigating Treatment Factor X in People With Factor X DeficiencyWho this might be right forFactor X Deficiency Bio Products Laboratory 16
Not applicableStudy completedNCT02190149What this trial is testingStudy of Prophylaxis, ACtivity and Effectiveness (SPACE) in Hemophilia Patients Currently Treated With ADVATE or RIXUBISWho this might be right forHemophilia AHemophilia B Baxalta now part of Shire 64
Not applicableEnded earlyNCT02402829What this trial is testingA Study Comparing Factor Level and Inhibitor Titer Testing Results Drawn From Central Venous Lines and VenipunctureWho this might be right forHemophilia AFactor VIII DeficiencyHemophilia+2 more Children's Mercy Hospital Kansas City 3
Large-scale testing (Phase 3)Looking for participantsNCT06963216What this trial is testingA Study Investigating Coagadex in the Treatment AFXD Associated With AL AmyloidosisWho this might be right forAcquired Factor X Deficiency Kedrion S.p.A. 15
Early research (Phase 1)Looking for participantsNCT06349473What this trial is testingSafety, Tolerability, Pharmacokinetics, and Pharmacodynamics of SR604 in Two Participants Groups (Part A: Healthy Participants, and Part B: Participants With Hemophilia A or Hemophilia B or Factor VII Deficiency)Who this might be right forHealthy ParticipantsHemophilia AHemophilia B+1 more Equilibra Bioscience LLC 36
Large-scale testing (Phase 3)Study completedNCT02537431What this trial is testingOpen Label Study of KRN23 on Osteomalacia in Adults With X-linked Hypophosphatemia (XLH)Who this might be right forX-linked Hypophosphatemia Kyowa Kirin, Inc. 14
Not applicableStudy completedNCT04429568What this trial is testingTHC Crossover StudyWho this might be right forTHCCannabisCannabis Smoking+6 more University of California, San Francisco 14
Post-approval studies (Phase 4)UnknownNCT04419363What this trial is testingBurosumab in Children and Adolescents With X-linked HypophosphatemiaWho this might be right forRare DiseasesX-linked Hypophosphatemia Bicetre Hospital 57
Testing effectiveness (Phase 2)Study completedNCT03734588What this trial is testingDose-finding Study of SPK-8016 Gene Therapy in Patients With Hemophilia A to Support Evaluation in Individuals With FVIII InhibitorsWho this might be right forAdeno-Associated Virus (AAV)Blood Coagulation DisorderBlood Coagulation Disorders, Inherited+14 more Spark Therapeutics, Inc. 4
Testing effectiveness (Phase 2)Study completedNCT02163577What this trial is testingStudy of KRN23 (Burosumab), a Recombinant Fully Human Monoclonal Antibody Against Fibroblast Growth Factor 23 (FGF23), in Pediatric Subjects With X-linked Hypophosphatemia (XLH)Who this might be right forX-linked Hypophosphatemia Kyowa Kirin, Inc. 52
Post-approval studies (Phase 4)Study completedNCT01988662What this trial is testingUNcovering the Difference Between Ranibizumab and Aflibercept, Focusing on Systemic Anti-vascular Endothelial Growth Factor (VEGF) Effects in Patients With neovascuLar Age-related Macular Degeneration (AMD)Who this might be right forNeovacular Age-related Macular Degeneration Novartis Pharmaceuticals 205
Post-approval studies (Phase 4)Study completedNCT00002386What this trial is testingEffect of Indinavir Plus Two Other Anti-HIV Drugs on Blood Clotting in HIV-Positive Males With HemophiliaWho this might be right forHIV InfectionsHemophilia A Merck Sharp & Dohme LLC 55
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
Testing effectiveness (Phase 2)Active Not RecruitingNCT01687608What this trial is testingOpen-Label Single Ascending Dose of Adeno-associated Virus Serotype 8 Factor IX Gene Therapy in Adults With Hemophilia BWho this might be right forHemophilia B Baxalta now part of Shire 30
Testing effectiveness (Phase 2)Study completedNCT03003533What this trial is testingA Gene Transfer Study for Hemophilia AWho this might be right forHemophilia A Spark Therapeutics, Inc. 25
Testing effectiveness (Phase 2)Ended earlyNCT00490100What this trial is testingTreatment for Growth Failure in Patients With X-Linked Severe Combined Immunodeficiency: Phase 2 Study of Insulin-Like Growth Factor-1Who this might be right forGrowth FailureX-linked Severe Combined Immunodeficiency (XSCID)Growth Hormone Resistence National Institute of Allergy and Infectious Diseases (NIAID) 6
Testing effectiveness (Phase 2)Study completedNCT00004801What this trial is testingPhase I/II Study of Monoclonal Factor IX Concentrate for Factor IX DeficiencyWho this might be right forHemophilia BFactor IX Deficiency National Center for Research Resources (NCRR) 3
Not applicableUnknownNCT06014320What this trial is testingAlterations in Coagulation Factor Levels in Patients With End Stage Liver DiseaseWho this might be right forLiver CirrhosisHemophiliaHemophilia A+4 more Stanford University 25
Testing effectiveness (Phase 2)Study completedNCT00004787What this trial is testingPhase II Pilot Study of Granulocyte Colony-Stimulating Factor for Inherited Bone Marrow Failure SyndromesWho this might be right forShwachman SyndromeFanconi's AnemiaDyskeratosis Congenita+1 more National Center for Research Resources (NCRR) 20