Comment Period Extended to 3/23/2015 for Notice of Proposed Rulemaking (NPRM) for FDAAA 801 and NIH Draft Reporting Policy for NIH-Funded Trials
2 studies found for:    "immune dysregulation, polyendocrinopathy, enteropathy, X-linked syndrome"
Show Display Options
Rank Status Study
1 Recruiting Treosulfan and Fludarabine Phosphate Before Donor Stem Cell Transplant in Treating Patients With Nonmalignant Inherited Disorders
Condition: Nonmalignant Neoplasm
Interventions: Drug: treosulfan;   Drug: fludarabine phosphate;   Biological: anti-thymocyte globulin;   Radiation: total-body irradiation;   Procedure: allogeneic bone marrow transplantation;   Procedure: peripheral blood stem cell transplantation;   Procedure: umbilical cord blood transplantation;   Drug: tacrolimus;   Drug: methotrexate;   Drug: cyclosporine;   Drug: mycophenolate mofetil;   Other: laboratory biomarker analysis
2 Recruiting Reduced Intensity Conditioning in Patients Aged ≤35 With Non-Malignant Disorders Undergoing UCBT, BMT, or PBSCT
Conditions: Primary Immunodeficiency Syndromes;   Congenital Bone Marrow Failure Syndromes;   Inherited Metabolic Disorders (IMD);   Hereditary Anemias;   Patients With Sickle Disease Presenting Specific Symptoms
Interventions: Drug: Hydroxyurea;   Drug: Alemtuzumab;   Drug: Fludarabine;   Drug: Melphalan;   Drug: Thiotepa

Indicates status has not been verified in more than two years