Inborn Errors of Metabolism Clinical Trials

41 clinical trials for Inborn Errors of Metabolism across international registries, including 6 currently recruiting participants. Compare status, phase, eligibility criteria and locations.

• Clinical and Laboratory Study of Methylmalonic Acidemia — RECRUITING
• Phase 1/2: CD45RA Depleted Stem Cell Addback to Prevent Viral or Fungal Infections Post TCRab/CD19 Depleted HSCT — RECRUITING · PHASE1
• Expanded Access Protocol Using CD3+/CD19+ Depleted PBSC — RECRUITING · PHASE1
• Institutional Registry of Rare Diseases — RECRUITING
• Targeted Interventions for Successful Transition and Transfer of Adolescents With Inborn Errors of Metabolism to Adult Services — RECRUITING · NA
• Blood Spot and Urine Metabolomic Screening Applied to Rare Diseases — RECRUITING · NA
• Metabolites in Blood and Breath in Metabolic Conditions — NOT_YET_RECRUITING
• Maternal Inborn Errors of Metabolism in Pregnancy: A Pregnancy Registry Protocol — COMPLETED
• EasiVits Acceptability & Tolerance Study — ACTIVE_NOT_RECRUITING · NA
• Identification and Characterization of Novel Non-Coding Variants That Contribute to Genetic Disorders — COMPLETED
• Treosulfan-based Versus Busulfan-based Conditioning in Paediatric Patients With Non-malignant Diseases — COMPLETED · PHASE2
• North Carolina Genomic Evaluation by Next-generation Exome Sequencing, 2 — COMPLETED · NA
• ORCHARD- Optimising Home Assessment of Rural Patients — COMPLETED · NA
• Orphan Drugs for Inherited Metabolic Diseases — COMPLETED
• Growth and Development of Children With Inborn Errors of Metabolism in Assiut Governorate — NOT_YET_RECRUITING
• Characterization of Intestinal Microbiota in Children With Inborn Errors of Metabolism (IEM) — COMPLETED
• Evaluation of PKU Explore — COMPLETED · NA
• Evaluation of PKU Start — COMPLETED · NA
• Trial of Erythrocyte Encapsulated Thymidine Phosphorylase In Mitochondrial Neurogastrointestinal Encephalomyopathy — WITHDRAWN · PHASE2
• Induced Pluripotent Stem Cells for the Development of Novel Drug Therapies for Inborn Errors of Metabolism (iPSC-IEM) — OTHER
• Utilizing Augmented Artificial Intelligence for Aminoacidopathies — COMPLETED
• Biomarkers for Inborn Errors of Metabolism — COMPLETED
• A Collection of Case Studies in Infants With UCD to Evaluate Infant Growth and the Safety of a New Medical Food for UCD — COMPLETED · NA
• Retrospective Study of Adult Patients With Inborn Errors of Metabolism in Switzerland — COMPLETED
• Evaluating Face-Recognition Technology in Syndrome Diagnosis — COMPLETED · NA
• Protecting Brains and Saving Futures - the PBSF Protocol — OTHER
• Patient and Observer Reported Outcome Measurements in Inborn Errors of Metabolism — COMPLETED
• BPX-501 T Cells Infused Post Stem Cell Transplant in Pediatrics With Non-Malignant Disorders Ineligible for BPU004 Study — TERMINATED
• Market Research - Acceptability Study for a New PKU Protein Substitute — COMPLETED · NA
• Market Research - Acceptability Trial for a New PKU Amino Acid Based Protein Substitute — COMPLETED · NA
• N-Carbamylglutamate (Carbaglu) In The Treatment Of Hyperammonemia — OTHER · PHASE2
• CD34+Selection for Partially Matched Family or Matched Unrelated Adult Donor Transplant — COMPLETED · PHASE2
• Oral Pirfenidone for the Pulmonary Fibrosis of Hermansky-Pudlak Syndrome — COMPLETED · PHASE2
• Unrelated Umbilical Cord Blood Transplantation Augmented With ALDHbr Umbilical Cord Blood Cells — COMPLETED · PHASE1
• Fetal Umbilical Cord Blood (UCB) Transplant for Lysosomal Storage Diseases — WITHDRAWN · PHASE1
• The Early History of Universal Screening for Metabolic Disorders — COMPLETED
• Reduced Intensity Conditioning for Umbilical Cord Blood Transplant in Pediatric Patients With Non-Malignant Disorders — COMPLETED · PHASE1
• ALD-101 Adjuvant Therapy of Unrelated Umbilical Cord Blood Transfusion (UCBT) in Patients With Inherited Metabolic Diseases — TERMINATED · PHASE3
• Increasing Ureagenesis in Inborn Errors of Metabolism With N-Carbamylglutamate — WITHDRAWN · PHASE2
• Nutritional Therapy of the Deficits of Oxidation Mitochondrial of the Fatty Acids — COMPLETED · NA
• Stem Cell Transplantation (SCT) for Genetic Diseases — COMPLETED · NA

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