2 completed, 1 recruiting, 1 withdrawn. 4 Phase 1, 1 Phase 2.
| Phase | Trials | Status |
|---|---|---|
| Phase 1 | 4 | Completed 2, Recruiting 1, Withdrawn 1 |
| Phase 2 | 1 | Recruiting 1 |
Follow Inborn Errors of Metabolism: new trials by email Literature Review of Inborn Errors of Metabolism trials Research Report on Inborn Errors of Metabolism
Tick up to 5 trials to compare them side by side (eligibility, arms, endpoints, adverse events) in a Literature Matrix.
| Trial | Phase | Status | Sponsor | Enrollment | Start | Updated |
|---|---|---|---|---|---|---|
| Phase 1/2: CD45RA Depleted Stem Cell Addback to Prevent Viral or Fungal Infections Post TCRab/CD19 Depleted HSCT Phase 1 Dose Level 1, Phase 1 Dose Level 2, Phase 1 Dose Level 3 - Leukemia, High Risk Acute Lymphoblastic Leukemia | Phase 1 / Phase 2 | Recruiting | Children's Hospital of Philadelphia | 100 | 2025-03-21 | 2026-04-15 |
| Fetal Umbilical Cord Blood (UCB) Transplant for Lysosomal Storage Diseases ALD-601 - Lysosomal Storage Diseases, Inborn Errors of Metabolism | Phase 1 | Withdrawn | Joanne Kurtzberg, MD | 2009-10-01 | 2017-06-29 | |
| Reduced Intensity Conditioning for Umbilical Cord Blood Transplant in Pediatric Patients With Non-Malignant Disorders Unrelated Umbilical Cord Blood Transplant, Reduced Intensity Conditioning - Non Malignant Disorders, Immunodeficiencies | Phase 1 | Completed | Duke University | 22 | 2008-10-01 | 2014-08-13 |
| Unrelated Umbilical Cord Blood Transplantation Augmented With ALDHbr Umbilical Cord Blood Cells ALDHbr Umbilical Cord Blood Cells - MDS, Anemia, Aplastic | Phase 1 | Completed | Joanne Kurtzberg, MD | 37 | 2005-10-01 | 2017-06-29 |
Metabolism, Inborn Errors (4) · Hemoglobinopathies (2) · Immunologic Deficiency Syndromes (2) · Lysosomal Storage Diseases (2) · Mucopolysaccharidosis I (2) · Mucopolysaccharidosis II (2) · Anemia, Aplastic (1) · Anemia, Diamond-Blackfan (1) · Anemia, Refractory, with Excess of Blasts (1) · Bone Marrow Failure Disorders (1) · Congenital Bone Marrow Failure Syndromes (1) · Gangliosidoses, GM2 (1) · Hematologic Neoplasms (1) · Leukemia (1) · Lymphoma, Non-Hodgkin (1)
Congenital, Hereditary, and Neonatal Diseases and Abnormalities (4) · Genetic Diseases, Inborn (4) · Metabolic Diseases (4) · Nutritional and Metabolic Diseases (4) · Anemia (3) · Bone Marrow Diseases (3) · Genetic Diseases, X-Linked (3) · Hematologic Diseases (3) · Hemic and Lymphatic Diseases (3) · Immune System Diseases (3) · Nervous System Diseases (3) · Bone Marrow Failure Disorders (2) · Carbohydrate Metabolism, Inborn Errors (2) · Connective Tissue Diseases (2) · Heredodegenerative Disorders, Nervous System (2)
Alemtuzumab (1) · Clinical Trials, Phase I as Topic (1) · Hydroxyurea (1) · Melphalan (1) · Thiotepa (1) · fludarabine (1)
Joanne Kurtzberg, MD (2) · Children's Hospital of Philadelphia (1) · Duke University (1)
United States (4)
Joanne Kurtzberg, MD (2) · Suhag Parikh, MD (1) · Timothy Olson, MD, PhD (1)