3 completed. 3 Phase 2.
| Phase | Trials | Status |
|---|---|---|
| Phase 2 | 3 | Completed 3 |
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 |
|---|---|---|---|---|---|---|
| Treosulfan-based Versus Busulfan-based Conditioning in Paediatric Patients With Non-malignant Diseases Treosulfan, Busilvex - Primary Immunodeficiencies, Inborn Errors of Metabolism | Phase 2 | Completed | medac GmbH | 106 | 2015-04-01 | 2025-12-08 |
| CD34+Selection for Partially Matched Family or Matched Unrelated Adult Donor Transplant Full Intensity with TBI, Full Intensity, Reduced Intensity - Leukemia, Lymphoma | Phase 2 | Completed | New York Medical College | 20 | 2011-09-01 | 2018-08-21 |
| Oral Pirfenidone for the Pulmonary Fibrosis of Hermansky-Pudlak Syndrome Pirfenidone, Placebo - Albinism, Inborn Errors of Metabolism | Phase 2 | Completed | William Gahl, M.D. | 35 | 2005-09-01 | 2017-10-16 |
Metabolism, Inborn Errors (3) · Bone Marrow Failure Disorders (2) · Albinism (1) · Albinism, Oculocutaneous (1) · Anemia, Sickle Cell (1) · Hemoglobinopathies (1) · Hermanski-Pudlak Syndrome (1) · Histiocytosis (1) · Immunologic Deficiency Syndromes (1) · Leukemia (1) · Lymphoma (1) · Platelet Storage Pool Deficiency (1) · Pulmonary Fibrosis (1) · beta-Thalassemia (1)
Congenital, Hereditary, and Neonatal Diseases and Abnormalities (3) · Genetic Diseases, Inborn (3) · Hematologic Diseases (3) · Hemic and Lymphatic Diseases (3) · Metabolic Diseases (3) · Nutritional and Metabolic Diseases (3) · Bone Marrow Diseases (2) · Amino Acid Metabolism, Inborn Errors (1) · Anemia (1) · Anemia, Hemolytic (1) · Anemia, Hemolytic, Congenital (1) · Blood Coagulation Disorders (1) · Blood Coagulation Disorders, Inherited (1) · Blood Platelet Disorders (1) · Eye Diseases (1)
Busulfan (2) · Alemtuzumab (1) · Antilymphocyte Serum (1) · Cyclophosphamide (1) · Melphalan (1) · Thiotepa (1) · Whole-Body Irradiation (1) · fludarabine phosphate (1) · pirfenidone (1) · treosulfan (1)
New York Medical College (1) · William Gahl, M.D. (1) · medac GmbH (1)
United States (2) · Czechia (1) · Germany (1) · Italy (1) · Poland (1)
Karl-Walter Sykora, MD and Prof (1) · Mitchell S Cairo, MD (1)