Medical Information Only. Consult your healthcare provider before considering clinical trial enrollment.
Pelizaeus-Merzbacher Disease: early-phase trial registry
3 of 3 indexed Pelizaeus-Merzbacher Disease trials are currently recruiting, with more weight in Phase 1-2.
Corpus placement: #5,209 of 28,707 conditions by registered trial count.
3 US clinical trials · 3 currently recruiting · #5,209 of 28,707 conditions by registered trial count
Pelizaeus-Merzbacher Disease: 3 US clinical trials tracked, 3 recruiting.
Pelizaeus-Merzbacher Disease is the subject of 3 registered US clinical trials on ClinicalTrials.gov, 3 of them currently open to new participants. 0 are in Phase 3-4 (later-stage) and 2 in Phase 1-2 (earlier-stage). The most active sponsor is Ionis Pharmaceuticals, running 2 of these trials.
Enrollment posture brief
Registry condition pelizaeus-merzbacher-disease
OPEN 3 | RECR 100% | LATE 0 | EARLY 2 | RANK #5209 | SPON 2
Volume-matched condition peers
Nearest open-share peer: Aortic Arch Aneurysm (100% recruiting · 5 trials)
Pelizaeus-Merzbacher Disease lists 3 open slots (100% of 3 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 Pelizaeus-Merzbacher Disease 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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Pelizaeus-Merzbacher Disease is recruiting above its size-band peers.
3 of 3 indexed trials (100%) 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.
Open and recent trials
3 total, page 1 of 1
UCB Transplant of Inherited Metabolic Diseases With Administration of Intrathecal UCB Derived Oligodendrocyte-Like Cells
Joanne Kurtzberg, MD
NCT02254863
Rocket Study: A Study to Characterize Biomarkers and Disease Progression in Participants With Pelizaeus-Merzbacher Disease
Ionis Pharmaceuticals
NCT05659901
Orbit Study: A Study to Evaluate the Safety, Pharmacokinetics, and Pharmacodynamics of Intrathecally Administered ION356 in Participants With Pelizaeus Merzbacher Disease (PMD)
Ionis Pharmaceuticals
NCT06150716
Phase Distribution
| Phase | Trial count |
|---|---|
| Phase 1 | 2 |
Top Sponsors
Named interventions
Most-linked intervention names on Pelizaeus-Merzbacher Disease records (sponsor-reported on ClinicalTrials.gov). Counts are trial links, not unique products.
US sites by state
Facility locations filed on Pelizaeus-Merzbacher Disease 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 Pelizaeus-Merzbacher Disease page
3 registered trials, 3 currently recruiting, is a starting point for a conversation, not a diagnosis or a recommendation.
- 3 Pelizaeus-Merzbacher Disease trials are currently recruiting, check eligibility criteria before contacting a site. Browse recruiting trials
- Ionis Pharmaceuticals sponsors the most Pelizaeus-Merzbacher Disease trials on record, review their full trial history before evaluating a specific study. See Ionis Pharmaceuticals'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 Pelizaeus-Merzbacher Disease condition card totals 3 registered trials; most-linked intervention DUOC-01; top US site state Pennsylvania. See our editorial standards & corrections policy, the methodology behind these numbers, the data changelog, or report a data error.