Medical Information Only. Consult your healthcare provider before considering clinical trial enrollment.
Pitt Hopkins Syndrome: early-phase trial registry
2 of 2 indexed Pitt Hopkins Syndrome trials are currently recruiting, with more weight in Phase 1-2.
Corpus placement: #7,188 of 28,707 conditions by registered trial count.
2 US clinical trials · 2 currently recruiting · #7,188 of 28,707 conditions by registered trial count
Pitt Hopkins Syndrome: 2 US clinical trials tracked, 2 recruiting.
Pitt Hopkins Syndrome is the subject of 2 registered US clinical trials on ClinicalTrials.gov, 2 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 Gut-Brain-Axis Therapeutics, running 1 of these trials.
Enrollment posture brief
Registry condition pitt-hopkins-syndrome
OPEN 2 | RECR 100% | LATE 0 | EARLY 2 | RANK #7188 | SPON 1
Volume-matched condition peers
Nearest open-share peer: Aortic Arch Aneurysm (100% recruiting · 5 trials)
Pitt Hopkins Syndrome lists 2 open slots (100% of 2 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 Pitt Hopkins Syndrome 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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Pitt Hopkins Syndrome is recruiting above its size-band peers.
2 of 2 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
2 total, page 1 of 1
Microbiota Transfer Therapy for Children and Adults With Both Pitt Hopkins Syndrome and Gastrointestinal Disorders
Gut-Brain-Axis Therapeutics
NCT06321796
Phase 1/2 Study of MZ-1866, an AAV-9 Gene Therapy Delivered by Intracerebroventricular Injection to Participants With Pitt Hopkins Syndrome
Mahzi Therapeutics
NCT07135050
Phase Distribution
| Phase | Trial count |
|---|---|
| Phase 1 | 1 |
| Phase 2 | 1 |
Top Sponsors
Named interventions
Most-linked intervention names on Pitt Hopkins Syndrome records (sponsor-reported on ClinicalTrials.gov). Counts are trial links, not unique products.
US sites by state
Facility locations filed on Pitt Hopkins Syndrome 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 Pitt Hopkins Syndrome page
2 registered trials, 2 currently recruiting, is a starting point for a conversation, not a diagnosis or a recommendation.
- 2 Pitt Hopkins Syndrome trials are currently recruiting, check eligibility criteria before contacting a site. Browse recruiting trials
- Gut-Brain-Axis Therapeutics sponsors the most Pitt Hopkins Syndrome trials on record, review their full trial history before evaluating a specific study. See Gut-Brain-Axis Therapeutics'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 Pitt Hopkins Syndrome condition card totals 2 registered trials; most-linked intervention Placebo MTP-101P; top US site state Illinois. See our editorial standards & corrections policy, the methodology behind these numbers, the data changelog, or report a data error.