Clinical Trials

Listed below are clinical trials which have been added or updated in the last 90 days. If you would like to see the full list of clinical trials please click here.

  • Familial Investigations of Childhood Cancer Predisposition
    Conditions:   Acute Leukemia;   Adenomatous Polyposis;   Adrenocortical Carcinoma;   AML;   BAP1 Tumor Predisposition Syndrome;   Carney Complex;   Choroid Plexus Carcinoma;   Constitutional Mismatch Repair Deficiency Syndrome;   Diamond-Blackfan Anemia;   DICER1 Syndrome;   Dyskeratosis Congenita;   Emberger Syndrome;   Familial Acute Myeloid Leukemia;   Familial Adenomatous Polyposis;   Fanconi Anemia;   Familial Cancer;   Familial Wilms Tumor;   Familial Neuroblastoma;   GIST;   Hereditary Breast and Ovarian Cancer;   Hereditary Paraganglioma-Pheochromocytoma Syndrome;   Hodgkin Lymphoma;   Juvenile Polyposis;   Li-Fraumeni Syndrome;   Lynch Syndrome;   MDS;   Melanoma Syndrome;   Multiple Endocrine Neoplasia Type 1;   Multiple Endocrine Neoplasia Type 2;   Neuroblastoma;   Neurofibromatosis Type 1;   Neurofibromatosis Type II;   Nevoid Basal Cell Carcinoma Syndrome;   Non Hodgkin Lymphoma;   Noonan Syndrome and Other Rasopathy;   Overgrowth Syndromes;   Pancreatic Cancer;   Peutz-Jeghers Syndrome;   Pheochromocytoma/Paraganglioma;   PTEN Hamartoma Tumor Syndrome;   Retinoblastoma;   Rhabdoid Tumor Predisposition Syndrome;   Rhabdomyosarcoma;   Rothmund-Thomson Syndrome;   Tuberous Sclerosis;   Von Hippel-Lindau Disease
    Sponsor:   St. Jude Children’s Research Hospital
  • Safety Study of Gene Modified Donor T Cell Infusion After Stem Cell Transplant for Non-Malignant Diseases
    Conditions:   Primary Immune Deficiency Disorders;   Hemophagocytic Lymphohistiocytosis;   Inherited Bone Marrow Failure Syndrome;   Hemoglobinopathies;   Metabolic Disorders
    Intervention:   Biological: BPX-501 and AP1903
    Sponsor:   Bellicum Pharmaceuticals
  • Human Placental-Derived Stem Cell Transplantation
    Conditions:   Mucopolysaccharidosis I;   Mucopolysaccharidosis VI;   Adrenoleukodystrophy;   Niemann-Pick Disease;   Metachromatic Leukodystrophy;   Wolman Disease;   Krabbe’s Disease;   Gaucher’s Disease;   Fucosidosis;   Batten Disease;   Severe Aplastic Anemia;   Diamond-Blackfan Anemia;   Amegakaryocytic Thrombocytopenia;   Myelodysplastic Syndrome;   Acute Myelogenous Leukemia;   Acute Lymphocytic Leukemia
    Intervention:   Drug: Human Placental Derived Stem Cell
    Sponsor:   New York Medical College
    Active, not recruiting
  • Safety and Efficacy Study of Sotatercept in Adults With Transfusion Dependent Diamond Blackfan Anemia
    Condition:   Diamond Blackfan Anemia
    Interventions:   Drug: Sotatercept;   Drug: Sotatercept with prednisone boost
    Sponsor:   Northwell Health
  • Pilot Phase I/II Study of Amino Acid Leucine in Treatment of Patients With Transfusion-Dependent Diamond Blackfan Anemia
    Conditions:   Diamond Blackfan Anemia;   Blackfan Diamond Syndrome;   DBA;   Congenital Hypoplastic Anemia;   Pure Red Cell Aplasia
    Intervention:   Drug: leucine
    Sponsor:   Northwell Health
    Active, not recruiting
  • Stem Cell Transplant for Bone Marrow Failure Syndromes
    Conditions:   Diamond-Blackfan Anemia;   Kostmann’s Neutropenia;   Shwachman-Diamond Syndrome
    Interventions:   Procedure: Stem cell transplant;   Drug: Fludarabine monophosphate;   Procedure: Total lymphoid irradiation;   Drug: Busulfan;   Biological: anti-thymocyte globulin
    Sponsor:   Masonic Cancer Center, University of Minnesota
  • Diamond Blackfan Anemia Registry (DBAR)
    Conditions:   Anemia;   Blood Disease
    Sponsors:   Northwell Health;   National Heart, Lung, and Blood Institute (NHLBI)
  • Improving the Results of Bone Marrow Transplantation for Patients With Severe Congenital Anemias
    Conditions:   Congenital Hemolytic Anemia;   Diamond-Blackfan Anemia
    Interventions:   Procedure: Radiotherapy;   Drug: Alemtuzumab (Campath );   Drug: Sirolimus (Rapamune )
    Sponsor:   National Heart, Lung, and Blood Institute (NHLBI)
  • Cancer in Inherited Bone Marrow Failure Syndromes
    Conditions:   Aplastic Anemia;   Head and Neck Neoplasms;   Bone Marrow Failure
    Sponsor:   National Cancer Institute (NCI)