Inborn Errors of Metabolism Clinical Trials

7 recruitingLast updated: June 17, 2026

There are 7 actively recruiting inborn errors of metabolism clinical trials across 5 countries. Studies span Not Applicable, Early Phase 1, Phase 1, Phase 2. Top locations include Vienna, Austria, Philadelphia, Pennsylvania, United States, Bethesda, Maryland, United States. Updated daily from ClinicalTrials.gov.


Inborn Errors of Metabolism Trials at a Glance

7 actively recruiting trials for inborn errors of metabolism are listed on ClinicalTrialsFinder across 6 cities in 5 countries. The largest study group is Not Applicable with 3 trials, with the heaviest enrollment activity in Vienna, Philadelphia, and Bethesda. Lead sponsors running inborn errors of metabolism studies include Children's Hospital of Philadelphia, Cliniques universitaires Saint-Luc- Université Catholique de Louvain, and Hospital Italiano de Buenos Aires.

Browse inborn errors of metabolism trials by phase

Treatments under study

About Inborn Errors of Metabolism Clinical Trials

Looking for clinical trials for Inborn Errors of Metabolism? There are currently 6 studies actively recruiting participants. Clinical trials offer access to new treatments before they are widely available, and every approved therapy in use today was first tested through a clinical trial.

Below you can browse trials, sign up for alerts when new Inborn Errors of Metabolism trials open, and view eligibility criteria for each study. Each listing includes the study phase, locations, and enrollment details.

Frequently Asked Questions

Common questions about Inborn Errors of Metabolism clinical trials

A clinical trial is a carefully designed research study that tests new medical treatments, drugs, devices, or approaches in human volunteers. Every approved medication and treatment available today was proven safe and effective through clinical trials.

All clinical trials are reviewed and approved by Institutional Review Boards (IRBs) — independent committees that evaluate patient safety. Trials follow strict protocols, and your health is monitored closely throughout. You can withdraw at any time.

Not necessarily. Many trials compare the new treatment against the current standard of care, meaning all participants receive active treatment. When placebos are used, they are typically combined with standard treatment, not given alone. The trial description will always specify the design.

Under the Affordable Care Act, most private insurers are required to cover routine patient care costs during a clinical trial. The sponsor typically covers the investigational treatment itself. Medicare also covers routine costs for qualifying trials.

Yes. Participation is completely voluntary. You can withdraw at any time, for any reason, without it affecting your access to standard medical care.

Each trial has specific eligibility criteria — including age, diagnosis, disease stage, prior treatments, and general health. Browse the trials listed above and check their eligibility sections. You can also contact the trial site directly to discuss your situation.

Showing 17 of 7 trials

Recruiting

Clinical and Laboratory Study of Methylmalonic Acidemia

Inborn Errors of MetabolismOrganic AcidemiaMethylmalonic Acidemia
National Human Genome Research Institute (NHGRI)2,275 enrolled3 locationsNCT00078078
Recruiting
Phase 1Phase 2

Phase 1/2: CD45RA Depleted Stem Cell Addback to Prevent Viral or Fungal Infections Post TCRab/CD19 Depleted HSCT

LeukemiaInborn Errors of MetabolismHLH+11 more
Children's Hospital of Philadelphia100 enrolled1 locationNCT06839456
Recruiting
Early Phase 1

Expanded Access Protocol Using CD3+/CD19+ Depleted PBSC

LeukemiaInborn Errors of MetabolismBone Marrow Failure Syndromes+2 more
Children's Hospital of Philadelphia100 enrolled1 locationNCT02356653
Recruiting

Institutional Registry of Rare Diseases

ParagangliomaPheochromocytomaAmyloidosis+23 more
Hospital Italiano de Buenos Aires380 enrolled1 locationNCT06573723
Recruiting
Not Applicable

Education & Care in RARE: Efficacy of Targeted Psychoeducational Intervention Among Pediatric Rare Disease Patients

Rare DisordersOrphan DiseasesPediatric Diseases+1 more
Medical University of Vienna100 enrolled7 locationsNCT06729554
Recruiting
Not Applicable

Targeted Interventions for Successful Transition and Transfer of Adolescents With Inborn Errors of Metabolism to Adult Services

Inborn Errors of MetabolismTransition
University Children's Hospital, Zurich20 enrolled8 locationsNCT05413278
Recruiting
Not Applicable

Blood Spot and Urine Metabolomic Screening Applied to Rare Diseases

Rare DiseasesInborn Errors of Metabolism
Cliniques universitaires Saint-Luc- Université Catholique de Louvain2,286 enrolled4 locationsNCT06360913