Condition
Phelan-McDermid Syndrome clinical trials and research
As of November 12, 2026, Find a Trial lists 3 Phelan-McDermid Syndrome clinical trials that are recruiting, at sites in 2 countries (most in United States (3), Canada (1)). By phase: Phase 3 (2), Phase 1/2 (1). Every study is explained in plain English. Data from ClinicalTrials.gov and NIH RePORTER, updated November 12, 2026.
- 3Clinical trials
- 2Countries
Newest Phelan-McDermid Syndrome clinical trials
| Study | Phase | Where | Status | Registered |
|---|---|---|---|---|
| NNZ-2591 in kids with Phelan-McDermid syndrome | Phase 3 | Birmingham, Alabama and 11 other locations | Recruiting | May 18, 2026 |
| NNZ-2591 for children with Phelan-McDermid syndrome | Phase 3 | Birmingham, Alabama and 17 other locations | Recruiting | Dec 15, 2025 |
| Gene therapy for children and adults with SHANK3 haploinsufficiency | Phase 1/2 | Chicago, Illinois and 2 other locations | Recruiting | Oct 28, 2024 |
All recruiting trials (3)
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NNZ-2591 in kids with Phelan-McDermid syndrome
This study tests whether NNZ-2591 is safe and helpful for children with Phelan-McDermid syndrome over a year.
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NNZ-2591 for children with Phelan-McDermid syndrome
This randomized, placebo-controlled trial will test whether NNZ-2591 helps children ages 3–12 with Phelan-McDermid syndrome over a 13-week treatment period.
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Gene therapy for children and adults with SHANK3 haploinsufficiency
This study is testing a new gene therapy for children and possibly adults with SHANK3 haploinsufficiency to see if it is safe and helps improve their condition.
Quick answers
How many Phelan-McDermid Syndrome clinical trials are recruiting right now?
3, as of November 12, 2026. We update this list each time we refresh our data from ClinicalTrials.gov.
Where are these trials running?
In 2 countries. The most sites are in United States (3 trials), Canada (1 trial). Each study page lists its sites and which ones are recruiting.
What phases are they in?
Phase 3: 2, Phase 1/2: 1. Trials with no phase listed are usually device, behavioural or observational studies.
How do I join one?
Open a study, check the "Is this trial a fit for you?" section, then contact the study team listed on the page. They will ask a few screening questions. Asking is free and does not commit you to anything. Talk it over with your doctor first.
Find the studies that fit you
A list is a starting point. Tell us your situation in your own words and we'll rank the studies that match and explain why. It's free and needs no account.