Trials / Conditions / Pitt Hopkins Syndrome

Pitt Hopkins Syndrome

8 registered clinical trials studyying Pitt Hopkins Syndrome — 4 currently recruiting.

Status	Trial	Sponsor	Phase
Recruiting	Phase 1/2 Study of MZ-1866, an AAV-9 Gene Therapy Delivered by Intracerebroventricular Injection to Participan NCT07135050	Mahzi Therapeutics	Phase 1 / Phase 2
Not Yet Recruiting	An Exploratory Evaluation of the Safety and Efficacy of Vorinostat in Pitt Hopkins Syndrome NCT07150026	Unravel Biosciences, Inc.	Phase 1
Unknown	Microbiota Transfer Therapy for Children and Adults With Both Pitt Hopkins Syndrome and Gastrointestinal Disor NCT06321796	Gut-Brain-Axis Therapeutics Inc.	Phase 2
Completed	An Open-Label Study of Oral NNZ-2591 in Pitt Hopkins Syndrome (PTHS-001) NCT05025332	Neuren Pharmaceuticals Limited	Phase 2
Unknown	Safety & Efficacy of AlloRx SC® in PTHS Patients NCT05165017	Vitro Biopharma Inc.	Phase 1 / Phase 2
Completed	MTT for Children With Both Pitt Hopkins Syndrome and Gastrointestinal Disorders NCT04132427	Arizona State University	Phase 2
Enrolling By Invitation	Early Check: Expanded Screening in Newborns NCT03655223	RTI International	—
Recruiting	Rare Disease Patient Registry & Natural History Study - Coordination of Rare Diseases at Sanford NCT01793168	Sanford Health	—