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Results for “Pitt Hopkins Syndrome”

8 trials

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Showing 8 of 8 results

Testing effectiveness (Phase 2)Looking for participantsNCT07135050
What this trial is testing

Phase 1/2 Study of MZ-1866, an AAV-9 Gene Therapy Delivered by Intracerebroventricular Injection to Participants With Pitt Hopkins Syndrome

Who this might be right for
Pitt Hopkins Syndrome
Mahzi Therapeutics 12
Testing effectiveness (Phase 2)UnknownNCT05165017
What this trial is testing

Safety & Efficacy of AlloRx SC® in PTHS Patients

Who this might be right for
Pitt Hopkins Syndrome
Vitro Biopharma Inc. 26
Testing effectiveness (Phase 2)Study completedNCT05025332
What this trial is testing

An Open-Label Study of Oral NNZ-2591 in Pitt Hopkins Syndrome (PTHS-001)

Who this might be right for
Pitt Hopkins Syndrome
Neuren Pharmaceuticals Limited 28
Testing effectiveness (Phase 2)Study completedNCT04132427
What this trial is testing

MTT for Children With Both Pitt Hopkins Syndrome and Gastrointestinal Disorders

Who this might be right for
Pitt Hopkins Syndrome
Arizona State University 6
Not applicableActive Not RecruitingNCT03655223
What this trial is testing

Early Check: Expanded Screening in Newborns

Who this might be right for
Spinal Muscular AtrophyFragile X SyndromeFragile X - Premutation+182 more
RTI International 30,000
Early research (Phase 1)Looking for participantsNCT07150026
What this trial is testing

An Exploratory Evaluation of the Safety and Efficacy of Vorinostat in Pitt Hopkins Syndrome

Who this might be right for
Pitt Hopkins Syndrome
Unravel Biosciences, Inc. 5
Not applicableLooking for participantsNCT01793168
What this trial is testing

Rare Disease Patient Registry & Natural History Study - Coordination of Rare Diseases at Sanford

Who this might be right for
Rare DisordersUndiagnosed DisordersDisorders of Unknown Prevalence+340 more
Sanford Health 20,000
Testing effectiveness (Phase 2)UnknownNCT06321796
What this trial is testing

Microbiota Transfer Therapy for Children and Adults With Both Pitt Hopkins Syndrome and Gastrointestinal Disorders

Who this might be right for
Pitt Hopkins Syndrome
Gut-Brain-Axis Therapeutics Inc. 20