Phase 1 Pitt Hopkins Syndrome Clinical Trials
2 recruitingPhase 1
What is a Phase 1 trial?
Phase 1 trials test a new treatment in a small group of people for the first time. The primary goal is to evaluate safety, determine a safe dosage range, and identify side effects. These studies typically enroll 20 to 80 participants.
Showing 1–2 of 2 trials
Recruiting
Phase 1
An Exploratory Evaluation of the Safety and Efficacy of Vorinostat in Pitt Hopkins Syndrome
Pitt Hopkins Syndrome
Unravel Biosciences, Inc.5 enrolled1 locationNCT07150026
Recruiting
Phase 1Phase 2
Phase 1/2 Study of MZ-1866, an AAV-9 Gene Therapy Delivered by Intracerebroventricular Injection to Participants With Pitt Hopkins Syndrome
Pitt Hopkins Syndrome
Mahzi Therapeutics12 enrolled3 locationsNCT07135050