| Status | Study |
| Recruiting |
Study Name: SCD-PROMIS: A Software Platform to Enhance Self-efficacy and Patient-provider Engagement for Patients With Sickle Cell Pain Condition: Sickle Cell Disease Anemia Anemia, Hemolytic Date: 2016-12-22 Interventions: Other: PROMIS for Pain Management App At hospital discharge, the investigators will collect baseline sur |
| Recruiting |
Study Name: Fetal Hemoglobin Induction Treatment Metformin Condition: Sickle Cell Anemia Sickle Cell Disease Hemoglobin Disorder Date: 2016-12-01 Interventions: Drug: Metformin Subjects will be started at a dose of 500 |
| Recruiting |
Study Name: The Congenital Dyserythropoietic Anemia Registry (CDAR) Condition: Congenital Dyserythropoietic Anemia (CDA) Date: 2016-10-04 |
| Recruiting |
Study Name: Fat and Sugar Metabolism During Exercise in Patients With Metabolic Myopathy Condition: Metabolism, Inborn Errors Lipid Metabolism, Inborn Errors Date: 2015-12-09 Interventions: Other: Sugar 10 minutes before exercising, the subjects are given a bolus of glucose solution 0.2 g/kg i |
| Enrolling by invitation |
Study Name: Longterm Follow-up of Subjects With Hemoglobinopathies Treated With Ex Vivo Gene Therapy Condition: Beta-Thalassemia Sickle Cell Disease Date: 2015-11-30 Interventions: Other: Safety and efficacy assessments Vector copy number (VCN) measurement, safety evaluations, disease |
| Active, not recruiting |
Study Name: A Study of AG-348 in Adult Patients With Pyruvate Kinase Deficiency Condition: Pyruvate Kinase Deficiency Date: 2015-06-10 Interventions: Drug: AG-348 A range of doses of AG-348 will be tested based on the assessment of safety, tolerability a |
| Recruiting |
Study Name: HSCT For Patients With High Risk Hemoglobinopathies Using Reduced Intensity Condition: Sickle Cell Disease Beta Thalassemia-Major Date: 2015-04-24 Interventions: Drug: alemtuzumab (Campath IH) |
| Recruiting |
Study Name: Evaluation of the AMICUS RBCx System in Sickle Cell Patients Condition: Sickle Cell Anemia Date: 2015-02-06 Interventions: Device: Amicus Red Cell Exchange in SCD patients Each patient will be treated with one RBCx procedure us |
| Recruiting |
Study Name: Allogeneic SCT of CordIn™, in Patients With Hemoglobinopathies Condition: Sickle Cell Disease Thalassemia Date: 2015-01-21 Interventions: Biological: CordIn CordIn™ is a cryopreserved stem/progenitor cell-based product of purified CD133 |
| Recruiting |
Study Name: Sickle Cell Hemoglobinopathies and Bone Health Condition: Sickle Cell Disease Sickle Cell Trait Date: 2014-12-01 Interventions: Other: Healthy volunteers without SCT |