Clinical Trials

Below is a list of active clinical trials worldwide which involve patients with inherited bleeding disorders. The link from each study title will take you to ClinicalTrials.gov which gives you more information about the trial.
To see a list of trials for a particular bleeding disorder use the drop-down list below to select the condition you're interested in. A new window will open up with a list of trials for this condition only.

Neonatal Catheter Lock for Infection Prevention Conditions: CLABSI - Central Line Associated Bloodstream Infection; CRBSI - Catheter Related Bloodstream Infection; Neonatal Sepsis, Late-Onset Interventions: Device: Taurolidine 2% lock; Other: Normal saline lock Sponsors: University of Turin, Italy Not yet recruiting

A Study to Observe the Long-term Safety of Odevixibat in Patients With Alagille Syndrome (ALGS) Who Are Receiving Ongoing Treatment Conditions: Alagille Syndrome Sponsors: Ipsen Not yet recruiting

A Multi-institution Prospective Assessment of Bone Health in Patients With Severe Hemophilia A on Factor VIII vs Factor Mimetic Prophylaxis (Efa Emi Bone Health Study) Conditions: Hemophilia a Sponsors: Arkansas Children's Hospital Research Institute; Genzyme, a Sanofi Company Not yet recruiting

Effects of Photobiomodulation in Hemophilia Patients. Conditions: Hemarthrosis, Knee; Hemophilia Interventions: Radiation: photobiomodulation Sponsors: Riphah International University Recruiting

Safety and Efficacy of Fetoscopic Endoluminal Tracheal Occlusion (FETO) in Congenital Diaphragmatic Hernia (CDH) Conditions: Congenital Diaphragmatic Hernia Interventions: Device: FETO with Goldballoon Detachable Balloon (GOLDBAL2) along with the Delivery Microcatheter (BALTACCI-BDPE100) Sponsors: Dr Erin Perrone Not yet recruiting

Periodontal Disease in Rare Renal Disorders (PERIO-RA-RE) Conditions: Periodontal Disease; Periodontitis; CKD; Chronic Kidney Disease; Alport Syndrome; Fabry Disease; Lupus or SLE; Tuberous Sclerosis Complex (TSC); Systemic Lupus Erythematosus (SLE) Interventions: Other: Observational assessment Sponsors: Stefan Lujinschi; Institutul Clinic Fundeni Not yet recruiting

HMBeacon: A Phase 2 Study to Evaluate ALN-6400 in Adult and Adolescent Female Patients With VWD and HMB Conditions: Von Willebrand Disease (VWD); Heavy Menstrual Bleeding (HMB) Interventions: Drug: ALN-6400 Sponsors: Alnylam Pharmaceuticals Not yet recruiting

North American Fetal Therapy Network Prospective Registry for Long-term Outcome Following Fetoscopic Endoluminal Tracheal Occlusion in Severe Left and Right Congenital Diaphragmatic Hernia Conditions: Congenital Diaphragmatic Hernia Interventions: Device: Fetoscopic Endoluminal Tracheal Occlusion; Other: Standard of Care Sponsors: Mayo Clinic Not yet recruiting