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Medical Condition

pitt hopkins syndrome

Total Trials
3
Recruiting Now
3
Trial Phases
Phase 1, Phase 1, Phase 2

ClinicalMetric tracks all active clinical trials for pitt hopkins syndrome sourced from ClinicalTrials.gov. Research in this area spans early-phase safety investigations through large Phase 3 confirmatory trials, conducted by NIH-funded academic centers, independent investigators, and pharmaceutical sponsors worldwide. Each listing is updated daily as new studies open enrollment and existing ones reach capacity or complete follow-up.

Trial listings include the NCT identifier, current recruitment status, phase classification, primary and secondary endpoints, estimated enrollment size, participating countries, and direct links to the full protocol on ClinicalTrials.gov. Eligibility criteria — including age range, disease stage, and prior treatment requirements — are documented for every study to help patients and clinicians assess suitability before contacting a trial site.

Frequently Asked Questions — pitt hopkins syndrome Clinical Trials

How many clinical trials are currently recruiting for pitt hopkins syndrome?
ClinicalMetric currently tracks 3 actively recruiting clinical trials for pitt hopkins syndrome, sourced in real time from ClinicalTrials.gov. The total number of registered studies—including those not yet enrolling or in active follow-up—is 3. Trial availability changes daily as new studies open enrollment and existing ones reach capacity.
What trial phases are available for pitt hopkins syndrome?
pitt hopkins syndrome research spans Phase 1 (2 trials), Phase 2 (1 trial). Phase 1 studies evaluate safety and dosing in small groups, Phase 2 studies assess preliminary efficacy in 100–300 participants, and Phase 3 trials compare the new treatment against the standard of care in 300–3,000+ patients. Phase 4 post-approval studies monitor long-term outcomes in real-world populations.
How do I find out if I qualify for a pitt hopkins syndrome clinical trial?
Eligibility criteria for pitt hopkins syndrome trials vary by study and typically specify age range, disease stage or severity, prior treatment history, and specific diagnostic or laboratory parameters. Each listing on ClinicalMetric links to the full protocol on ClinicalTrials.gov, where inclusion and exclusion criteria are documented. Contact the sponsoring site's research coordinator directly to confirm your eligibility—your treating physician or specialist can also help identify the most appropriate trial based on your medical history and current treatment status.
Trial Phases
Phase 1
2
Phase 2
1
Top Sponsors
Unravel Biosciences, Inc. 1 trial
Sanford Health 1 trial
Mahzi Therapeutics 1 trial

Recruiting Clinical Trials

NCT07150026 Phase 1
Recruiting
An Exploratory Evaluation of the Safety and Efficacy of Vorinostat in Pitt Hopkins Syndrome
Enrollment
5 pts
Location
Colombia
Sponsor
Unravel Biosciences, Inc.
View Trial →
NCT01793168
Recruiting
Rare Disease Patient Registry & Natural History Study - Coordination of Rare Diseases at Sanford
Enrollment
20,000 pts
Location
United States
Sponsor
Sanford Health
View Trial →
NCT07135050 Phase 1, Phase 2
Recruiting
Phase 1/2 Study of MZ-1866, an AAV-9 Gene Therapy Delivered by Intracerebroventricular Injection to Participants With Pitt Hopkins Syndrome
Enrollment
12 pts
Location
United States, Israe...
Sponsor
Mahzi Therapeutics
View Trial →

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ClinicalMetric — Independent clinical trial intelligence platform. Not affiliated with NIH, ClinicalTrials.gov, the U.S. FDA, or any pharmaceutical company, hospital, or clinical research organization. Trial data is sourced from ClinicalTrials.gov for informational purposes only and does not constitute medical advice. Do not make any treatment, enrollment, or health decisions based solely on information found here — always consult a qualified healthcare professional. Full Disclaimer  ·  Last Reviewed: November 2026  ·  Data Methodology