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.

Efanesoctocog Alfa Prophylaxis in Patients With Hemophilia A With Synovial Hypertrophy Conditions: Factor VIII Deficiency Interventions: Drug: Efanesoctocog alfa Sponsors: Sanofi Not yet recruiting

Long-term Study Evaluating Joint Health in People With Haemophilia A Receiving Real-world Prophylactic Treatment With Efanesoctocog Alfa Conditions: Hemophilia A Interventions: Drug: Efanesoctocog alfa Sponsors: Swedish Orphan Biovitrum; PSI CRO Not yet recruiting

Palliative Care Needs of Children With Rare Diseases and Their Families Conditions: Trisomy 13 Syndrome; Arthrogryposis Congenita Multiplex With Intestinal Atresia; Asparagine Synthetase Deficiency; CHARGE Syndrome; Early Infantile Epileptic Encephalopathy; FOXG1 Syndrome; KBG Syndrome; Noonan Syndrome; Severe Hemophilia A; Short Bowel Syndrome; Beta-Propeller Protein-Associated Neurodegeneration; Brain Injury of Prematurity With Periventricular Leukomalacia; Chromosome 17p13.3 Microdeletion Syndrome; Chromosome 1q43-1q44 Deletion; Cockayne Syndrome; Congenital Diaphragmatic Hernia; End-Stage Renal Disease With Cloacal Anomaly; Mitochondrial Depletion Disorder; Severe Factor VII Deficiency Interventions: Behavioral: Family Centered pediatric palliative care for family caregivers of children with rare diseases. Sponsors: Children's National Research Institute; Akron Children's Hospital; Stanford University; Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) Not yet recruiting

Low Dose Emicizumab vs Low Dose Factor VIII in Prophylaxis in Hemophilia A Patients Conditions: Hemophilia A Interventions: Drug: Emicizumab Injection; Drug: Factor VIII (FVIII) Sponsors: Dhaka Medical College Not yet recruiting