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Methylmalonic Acidemia: early-phase trial registry
3 of 7 indexed Methylmalonic Acidemia 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 · 3 currently recruiting · #2,497 of 28,707 conditions by registered trial count
Methylmalonic Acidemia: 7 US clinical trials tracked, 3 recruiting.
Methylmalonic Acidemia is the subject of 7 registered US clinical trials on ClinicalTrials.gov, 3 of them currently open to new participants. 0 are in Phase 3-4 (later-stage) and 3 in Phase 1-2 (earlier-stage). The most active sponsor is ModernaTX, running 3 of these trials.
Enrollment posture brief
Registry condition methylmalonic-acidemia
OPEN 3 | RECR 43% | LATE 0 | EARLY 3 | RANK #2497 | SPON 3
Volume-matched condition peers
Nearest open-share peer: Adrenoleukodystrophy (43% recruiting · 7 trials)
Methylmalonic Acidemia lists 3 open slots (43% 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 Methylmalonic Acidemia 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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Methylmalonic Acidemia is recruiting below its size-band peers.
3 of 7 indexed trials (42.9%) 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 Methylmalonic Acidemia research.
ModernaTX accounts for 3 of 7 trials (42.9%). 5 distinct sponsors appear on at least one study for this condition label.
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Most Methylmalonic Acidemia trials plan to enrol fewer than 100 people.
Median target enrolment is 56 among the 7 trials that report a genuine target (sentinel values excluded). 6 of those (85.7%) plan for fewer than 100 participants.
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Methylmalonic Acidemia splits between interventional and observational designs.
3 interventional and 4 observational studies make up this index (42.9% 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
An Extension Study to Evaluate the Long-Term Safety and Clinical Activity of mRNA-3705 in Participants Previously Enrolled in Other Clinical Studies of mRNA-3705
ModernaTX
NCT05295433
An Observational Study of Carbaglu® for the Treatment of MMA and PA in Adults and Pediatrics
Recordati Group
NCT05040178
A Study to Assess the Safety, Pharmacokinetics, and Pharmacodynamics of mRNA-3705 in Participants With Isolated Methylmalonic Acidemia
ModernaTX
NCT04899310
Long-term Follow-up Study of Patients Who Received hLB-001 Gene Therapy
Alexion Pharmaceuticals
NCT05506254
"The MaP Study": Mapping the Patient Journey in MMA and PA
ModernaTX
NCT03484767
Short-Term Outcome of N-Carbamylglutamate in the Treatment of Acute Hyperammonemia
Mendel Tuchman
NCT01599286
Phase Distribution
| Phase | Trial count |
|---|---|
| Phase 1 | 2 |
| Phase 2 | 1 |
Top Sponsors
Named interventions
Most-linked intervention names on Methylmalonic Acidemia records (sponsor-reported on ClinicalTrials.gov). Counts are trial links, not unique products.
US sites by state
Facility locations filed on Methylmalonic Acidemia 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 Methylmalonic Acidemia page
7 registered trials, 3 currently recruiting, is a starting point for a conversation, not a diagnosis or a recommendation.
- 3 Methylmalonic Acidemia trials are currently recruiting, check eligibility criteria before contacting a site. Browse recruiting trials
- ModernaTX sponsors the most Methylmalonic Acidemia trials on record, review their full trial history before evaluating a specific study. See ModernaTX'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 Methylmalonic Acidemia condition card totals 7 registered trials; most-linked intervention Carbaglu; top US site state Pennsylvania. See our editorial standards & corrections policy, the methodology behind these numbers, the data changelog, or report a data error.