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Inborn Errors of Metabolism: early-phase trial registry
2 of 7 indexed Inborn Errors of Metabolism trials are currently recruiting, with more weight in Phase 1-2.
Corpus placement: #2,497 of 28,707 conditions by registered trial count.
7 US clinical trials · 2 currently recruiting · #2,497 of 28,707 conditions by registered trial count
Inborn Errors of Metabolism: 7 US clinical trials tracked, 2 recruiting.
Inborn Errors of Metabolism is the subject of 7 registered US clinical trials on ClinicalTrials.gov, 2 of them currently open to new participants. 0 are in Phase 3-4 (later-stage) and 4 in Phase 1-2 (earlier-stage). The most active sponsor is National Human Genome Research Institute (NHGRI), running 2 of these trials.
Enrollment posture brief
Registry condition inborn-errors-of-metabolism
OPEN 2 | RECR 29% | LATE 0 | EARLY 4 | RANK #2497 | SPON 2
Volume-matched condition peers
Nearest open-share peer: Acromegaly (29% recruiting · 14 trials)
Inborn Errors of Metabolism lists 2 open slots (29% of 7 indexed trials). Peers are matched on registered volume, not therapeutic-area browse lists. How peer matching works →
Key findings
What ClinicalTrials.gov does not surface for Inborn Errors of Metabolism on its own pages , computed from the registry mirror as of 2026-08-08. Each line carries its own denominator so it can be quoted as it stands.
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Inborn Errors of Metabolism is recruiting below its size-band peers.
2 of 7 indexed trials (28.6%) are open to enrollment, against a 46.5% average across 27,806 conditions with fewer than 20 trials. The peer set uses the same ClinicalTrials.gov-derived counts as this page.
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One sponsor concentrates Inborn Errors of Metabolism research.
National Human Genome Research Institute (NHGRI) accounts for 2 of 7 trials (28.6%). 6 distinct sponsors appear on at least one study for this condition label.
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Most Inborn Errors of Metabolism trials plan to enrol fewer than 100 people.
Median target enrolment is 37 among the 6 trials that report a genuine target (sentinel values excluded). 4 of those (66.7%) plan for fewer than 100 participants.
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Inborn Errors of Metabolism research is mostly interventional.
5 interventional and 2 observational studies make up this index (71.4% interventional). Study type is sponsor-reported on ClinicalTrials.gov.
Open and recent trials
7 total, page 1 of 1
Clinical and Laboratory Study of Methylmalonic Acidemia
National Human Genome Research Institute (NHGRI)
NCT00078078
Expanded Access Protocol Using CD3+/CD19+ Depleted PBSC
Children's Hospital of Philadelphia
NCT02356653
Unrelated Umbilical Cord Blood Transplantation Augmented With ALDHbr Umbilical Cord Blood Cells
Joanne Kurtzberg, MD
NCT00692926
Reduced Intensity Conditioning for Umbilical Cord Blood Transplant in Pediatric Patients With Non-Malignant Disorders
Duke University
NCT00744692
CD34+Selection for Partially Matched Family or Matched Unrelated Adult Donor Transplant
New York Medical College
NCT01049854
Maternal Inborn Errors of Metabolism in Pregnancy: A Pregnancy Registry Protocol
National Human Genome Research Institute (NHGRI)
NCT02322177
Stem Cell Transplantation (SCT) for Genetic Diseases
National Center for Research Resources (NCRR)
NCT00004378
Phase Distribution
| Phase | Trial count |
|---|---|
| Phase 1 | 3 |
| Phase 2 | 1 |
Top Sponsors
Named interventions
Most-linked intervention names on Inborn Errors of Metabolism records (sponsor-reported on ClinicalTrials.gov). Counts are trial links, not unique products.
US sites by state
Facility locations filed on Inborn Errors of Metabolism trials. A trial with sites in several states counts in each.
Source: ClinicalTrials.gov, National Library of Medicine. Data is informational only.
Related
What to do with this Inborn Errors of Metabolism page
7 registered trials, 2 currently recruiting, is a starting point for a conversation, not a diagnosis or a recommendation.
- 2 Inborn Errors of Metabolism trials are currently recruiting, check eligibility criteria before contacting a site. Browse recruiting trials
- National Human Genome Research Institute (NHGRI) sponsors the most Inborn Errors of Metabolism trials on record, review their full trial history before evaluating a specific study. See National Human Genome Research Institute (NHGRI)'s trials
- Bring the specific trial ID (NCT number) to your own doctor before enrolling, this page is a directory, not medical guidance. Read the trial-finding guide
Trial and recruiting counts reflect ClinicalTrials.gov registry status as of the data vintage above; individual trial eligibility and enrollment status can change.
Every figure on PlainTrial is rendered directly from the ClinicalTrials.gov registry, no number is typed in by an editor. This Inborn Errors of Metabolism condition card totals 7 registered trials; most-linked intervention ALDHbr Umbilical Cord Blood Cells; top US site state Pennsylvania. See our editorial standards & corrections policy, the methodology behind these numbers, the data changelog, or report a data error.