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Condition: Congenital Hematological Disorder Synced from ClinicalTrials.gov · July 29, 2026

Recruiting studies: Congenital Hematological Disorder

Studies currently listed as recruiting on the public registry for this search term. Each page explains the study in plain language, with locations and the study team's contact details. Clinicians: generate a printable list for your patient →

Afatinib in Patients With Fanconi Anemia (FA) and Advanced Head and Neck Squamous Cell Carcinoma (HNSCC)
Phase 1/Phase 2 · 2 location(s) · Fundació Institut de Recerca de l'Hospital de la Santa Creu · NCT06648096Ruxolitinib-Enhanced Haplo HCT for Children and Young Adults With Sickle Cell Disease
Phase 1/Phase 2 · 4 location(s) · Arkansas Children's Hospital Research Institute · NCT07252050Cerebral Oxygen Metabolism in Children
NA · 1 location(s) · Washington University School of Medicine · NCT04157179Assessing Different FVIII Doses and Frequencies in Immune Tolerance Induction (ITI) with ADVATE Among Hemophilia a Boys
Phase 4 · 1 location(s) · Runhui WU · NCT06864975A Study Evaluating the Safety and Efficacy of LentiRed Drug Product in Transfusion-dependent β-Thalassemia [TDT]
EARLY_Phase 1 · 1 location(s) · First Affiliated Hospital of Guangxi Medical University · NCT05762510Oral Ketamine for Treatment of Vaso-Occlusive Pain
Phase 3 · 1 location(s) · Boston Children's Hospital · NCT05378555A Point-of-care Electrochemical-based Device for Rapid Detection of Fibrinogen on Type A Aortic Dissection Surgery
Phase N/A · 1 location(s) · Second Affiliated Hospital, School of Medicine, Zhejiang Uni · NCT06434389The HOPE Biobank Resource (BMT CTN 2402 HOPE)
Phase N/A · 6 location(s) · Medical College of Wisconsin · NCT07227155Case Notes Review on Patients With Hereditary Haemorrhagic Telangiectasia
Phase N/A · 1 location(s) · Imperial College London · NCT00230685A Prospective Study on the Role of Karl Storz Curved and Straight Fetoscopes (11508AAK and 11506AAK) for Fetoscopic Intr
NA · 1 location(s) · Boston Children's Hospital · NCT06056635Phase 3 Study of Fibrinogen Concentrate (CSL511) in Subjects With Pseudomyxoma Peritonei Undergoing Cytoreductive Surger
Phase 3 · 1 location(s) · CSL Behring · NCT06617897A Natural History Study Seeks to Understand the Clinical, Genomic, Pharmacological, Laboratory, and Dietary Determinates
Phase N/A · 1 location(s) · National Human Genome Research Institute (NHGRI) · NCT06092346A Study of Nipocalimab in Reducing the Risk of Fetal and Neonatal Alloimmune Thrombocytopenia (FNAIT)
Phase 3 · 21 location(s) · Janssen Research & Development, LLC · NCT06449651PET Imaging of Vaso-Occlusive Crisis (VOC) in SCD
Phase 1 · 1 location(s) · Enrico Novelli · NCT04925492Phase 1/2: CD45RA Depleted Stem Cell Addback to Prevent Viral or Fungal Infections Post TCRab/CD19 Depleted HSCT
Phase 1/Phase 2 · 1 location(s) · Children's Hospital of Philadelphia · NCT06839456Gene Therapy for Hemophilia B Patients Aged 12-18 Years Old
Phase 1 · 1 location(s) · Institute of Hematology & Blood Diseases Hospital, China · NCT05709288ALS20-101 Lentiviral Gene Therapy for Beta Thalassemia
Phase 1/Phase 2 · 1 location(s) · Children's Hospital of Philadelphia · NCT06364774A Study of Participants with Β-Thalassemia Treated with Betibeglogene Autotemcel
Phase N/A · 6 location(s) · Genetix Biotherapeutics Inc. · NCT06271512Delivering Patient-Facing Evidence-Based Guidelines Through mHealth to Adults With Sickle Cell Disease
NA · 4 location(s) · Ohio State University · NCT06919224The Severe Von Willebrand Disease (sVWD) Patient Registry
Phase N/A · 1 location(s) · VWD Connect Foundation · NCT05437536Empowering Adolescents and Young Adults With Sickle Cell Disease as Partners in Treatment Decision Making (EMPOWER-AYA)
NA · 1 location(s) · Nemours Children's Clinic · NCT05383911Prevalence of Synovitis in Patients With Haemophilia A
Phase N/A · 1 location(s) · University Hospital, Bonn · NCT06352216Progression Assessment of PXE-associated Alterations
Phase N/A · 1 location(s) · University Hospital, Bonn · NCT07048106Hematopoietic Stem Cell BCL11A Enhancer Gene Editing for Severe β-Hemoglobinopathies
Phase 1 · 1 location(s) · Daniel Bauer · NCT06647979Thal-Fabs: Reduced Toxicity Conditioning for High Risk Thalassemia
Phase 1/Phase 2 · 1 location(s) · The Hospital for Sick Children · NCT05426252Determination of Red Cell Survival in Sickle Cell Disease and Other Hemoglobinopathies Using Biotin Labeling
EARLY_Phase 1 · 1 location(s) · National Heart, Lung, and Blood Institute (NHLBI) · NCT06313398Phase 3 Study on the Efficacy and Safety of Human Plasma Derived Antithrombin (Atenativ) in Heparin-Resistant Patients S
Phase 3 · 27 location(s) · Octapharma · NCT06096116Zoledronic Acid Treatment in Patients With Congenital Dyserythropoietic Anemia
Phase 1/Phase 2 · 1 location(s) · Institute of Hematology & Blood Diseases Hospital, China · NCT07471516Gene Therapy Communication: Use of a Needs Assessment to Drive Decision-AIDS for Gene Therapy for Rare Diseases (GENETX)
Phase N/A · 1 location(s) · St. Jude Children's Research Hospital · NCT05810181HPC Offered for PRESERVE Expansion
Phase N/A · 4 location(s) · Ossium Health, Inc. · NCT07145411
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This page is independently generated by Eichor from the public ClinicalTrials.gov record and re-synced daily. It is not the sponsor's official website unless claimed. Nothing here is medical advice; eligibility is always determined by the study team — talk to your own doctor first.