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.

Analyzing Retinal Microanatomy in Retinopathy of Prematurity to Improve Care Conditions: Retinopathy of Prematurity (ROP) Interventions: Device: Ultra-widefield Optical Coherence Tomography (UWF-OCT); Device: Wide-field ophthalmic imaging system Sponsors: Duke University; University of Pennsylvania; National Eye Institute (NEI) Not yet recruiting

Comparison of 30-Second Sit-to-Stand Test Performance Between Individuals With Hemophilia and Healthy Peers Conditions: Hemophilia A and B Sponsors: Bahçeşehir University Not yet recruiting

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

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

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

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