3 completed, 2 withdrawn, 1 recruiting, 1 unknown. 7 Phase 2, 1 Phase 1, 1 Phase 3.
| Phase | Trials | Status |
|---|---|---|
| Phase 2 | 7 | Completed 3, Withdrawn 2, Recruiting 1, Unknown 1 |
| Phase 1 | 1 | Recruiting 1 |
| Phase 3 | 1 | Unknown 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 |
| Trial of Erythrocyte Encapsulated Thymidine Phosphorylase In Mitochondrial Neurogastrointestinal Encephalomyopathy EE-TP - Mitochondrial Diseases, Metabolic Disease | Phase 2 | Withdrawn | St George's, University of London | 2024-11-01 | 2023-10-11 | |
| 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 |
| Increasing Ureagenesis in Inborn Errors of Metabolism With N-Carbamylglutamate N-carbamylglutamate - Urea Cycle Disorders, Inborn, Inborn Errors of Metabolism | Phase 2 | Withdrawn | Children's Hospital of Philadelphia | 2010-12-01 | 2014-05-29 | |
| N-Carbamylglutamate (Carbaglu) In The Treatment Of Hyperammonemia N-carbamylglutamate - Inborn Errors of Metabolism | Phase 2 / Phase 3 | Unknown | Mendel Tuchman | 52 | 2008-08-01 | 2020-02-17 |
| 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 (7) · Bone Marrow Failure Disorders (3) · Hemoglobinopathies (2) · Immunologic Deficiency Syndromes (2) · Leukemia (2) · Albinism (1) · Albinism, Oculocutaneous (1) · Anemia, Refractory, with Excess of Blasts (1) · Anemia, Sickle Cell (1) · Carbamoyl-Phosphate Synthase I Deficiency Disease (1) · Congenital Bone Marrow Failure Syndromes (1) · Gastrointestinal Diseases (1) · Hermanski-Pudlak Syndrome (1) · Histiocytosis (1) · Hyperammonemia (1)
Congenital, Hereditary, and Neonatal Diseases and Abnormalities (7) · Genetic Diseases, Inborn (7) · Nutritional and Metabolic Diseases (7) · Metabolic Diseases (6) · Hematologic Diseases (4) · Hemic and Lymphatic Diseases (4) · Bone Marrow Diseases (3) · Amino Acid Metabolism, Inborn Errors (2) · Anemia (2) · Immune System Diseases (2) · Immunoproliferative Disorders (2) · Lymphatic Diseases (2) · Lymphoproliferative Disorders (2) · Neoplasms (2) · Neoplasms by Histologic Type (2)
Busulfan (2) · N-carbamylglutamate (2) · carglumic acid (2) · Alemtuzumab (1) · Antilymphocyte Serum (1) · Clinical Trials, Phase I as Topic (1) · Cyclophosphamide (1) · Melphalan (1) · Thiotepa (1) · Whole-Body Irradiation (1) · fludarabine phosphate (1) · pirfenidone (1) · treosulfan (1)
Children's Hospital of Philadelphia (2) · Mendel Tuchman (1) · New York Medical College (1) · St George's, University of London (1) · William Gahl, M.D. (1) · medac GmbH (1)
United States (4) · Czechia (1) · Germany (1) · Italy (1) · Poland (1)
Karl-Walter Sykora, MD and Prof (1) · Marc Yudkoff, MD (1) · Mendel Tuchman, MD (1) · Mitchell S Cairo, MD (1) · Niranjanan Nirmalananthan, MBBS (1) · Timothy Olson, MD, PhD (1)
PMID 18414145, PMID 18725595, PMID 19660971, PMID 20338795, PMID 20566609, PMID 23966250, PMID 26929599, PMID 30627136
R01HD058567 (2), 2018 (1)