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.

CD19-B Cell Depletion in PAIS-ME/CFS Patients Conditions: Post-acute Infectious Syndrome (PAIS); Post-COVID 19 Condition (PCC or Long COVID); Myalgic Encephalomyelitis/Chronic Fatigue Syndrome (ME/CFS) Interventions: Drug: Inebilizumab; Drug: Placebo Sponsors: Charite University, Berlin, Germany Not yet recruiting

Study of Surgical Practices in Hemophilia A Patients Treated With Efanesoctocog Alfa (Altuvoct®) Conditions: Haemophilia A; Factor VIII Interventions: Other: Clinical Data Sponsors: Hospices Civils de Lyon Recruiting

Ultrasound Assessment of Joint Health in Patients With Mild Hemophilia (Factor Levels 5-40%) Conditions: Mild Hemophilia Interventions: Other: Joint Ultrasound (HEAD-US); Other: Blood Sample for Coagulation Factor Measurement Sponsors: Assistance Publique - Hôpitaux de Paris; URC-CIC Paris Descartes Necker Cochin; Swedish Orphan Biovitrum Not yet recruiting

Safety, Tolerability and Efficacy of NPI-001 in Patients With Hereditary Cystatin C Amyloid Angiopathy (HCCAA) Conditions: Hereditary Cystatin C Amyloid Angiopathy (HCCAA); Hemorrhage Brain; Hemorrhage Cerebral Interventions: Drug: NACA Sponsors: Arctic Therapeutics Active, not recruiting

Mercaptopurine for the Treatment of Hereditary Leiomyomatosis and Renal Cell Carcinoma (HLRCC), Uterine/Cutaneous Leiomyomas, and Kidney Cancer Conditions: Advanced Kidney Carcinoma; Advanced Renal Cell Carcinoma; Hereditary Leiomyomatosis and Renal Cell Carcinoma; Metastatic Kidney Carcinoma; Metastatic Renal Cell Carcinoma; Skin Leiomyoma; Stage III Renal Cell Cancer AJCC v8; Stage IV Renal Cell Cancer AJCC v8; Uterine Corpus Leiomyoma Interventions: Procedure: Biopsy Procedure; Procedure: Biospecimen Collection; Procedure: Computed Tomography; Procedure: Magnetic Resonance Imaging; Drug: Mercaptopurine; Other: Questionnaire Administration; Procedure: Skin Biopsy Sponsors: Jonsson Comprehensive Cancer Center; The V Foundation; Phase One Foundation Not yet recruiting

Post-acute Infectious Syndrome - Aripiprazole Symptom Evaluation (PAIS-AriSE) Conditions: Post-acute Infectious Syndrome (PAIS); Post-COVID-19 Condition (PCC or Long COVID) Interventions: Drug: Aripiprazole (low-dose, 1mg); Drug: Placebo Sponsors: Christiana Franke Not yet recruiting

A Phase II Exploratory Study of SR604 Injection Evaluating the Safety and Efficacy in the Treatment of Congenital Coagulation Factor VII Deficiency Conditions: Factor VII Deficiency Interventions: Drug: SR604 Sponsors: Shanghai RAAS Blood Products Co., Ltd. Not yet recruiting

Pediatric Von Hippel-Lindau Disease: Natural History, Predictive Factors, and Long-Term Functional Outcomes of Central Nervous System Hemangioblastomas Conditions: Von Hippel-Lindau Disease; Central Nervous System Hemangioblastoma Sponsors: Assistance Publique - Hôpitaux de Paris Not yet recruiting

Myo-Inositol and D-Chiro-Inositol for Menstrual Irregularities and Hormone Changes in Women With Polycystic Ovarian Syndrome Conditions: Polycystic Ovary Syndrome (PCOS); Myo-inositol; D-chiro-inositol Interventions: Drug: Myo-Inositol and D-Chiro-Inositol Combination Therapy; Drug: placebo (folic acid) Sponsors: Nishtar Medical University Completed

Dexamethasone, Intravenous Injection of Human Immunoglobulin, and Increased Infusion of Mononuclear Cells to Reduce Donor Specific Antibodies in Haploidentical Hematopoietic Stem Cell Transplantation Conditions: Leukemia; Lymphoma; Thalassemia; Aplastic Anemia; Myelodysplastic Syndromes Interventions: Biological: Intravenous Immunoglobulin; Drug: Dexamethasone; Biological: Mononuclear Cells Sponsors: Hematology department of the 920th hospital Not yet recruiting

Delayed Versus Immediate Cord Clamping in Preterm Birth Conditions: Preterm Birth Complication; Neonatal Morbidity and Mortality; Cord Clamping; Delayed Cord Clamping; Anemia; NEC - Necrotizing Enterocolitis; Intraventricular Hemorrhage Neonatal; Respiratory Distress Neonatal Interventions: Procedure: Delayed Umbilical Cord Clamping; Procedure: Immediate Umbilical Cord Clamping Sponsors: Faculty of Medicine of Tunis Recruiting