Pitt Hopkins Syndrome
8
4
4
2
Key Insights
Highlights
Success Rate
100% trial completion (above average)
Clinical Risk Assessment
Based on trial outcomes
Low Risk
Score: 25/100
0.0%
0 terminated out of 8 trials
100.0%
+13.5% vs benchmark
0%
0 trials in Phase 3/4
100%
2 of 2 completed with results
Key Signals
Data Visualizations
Phase Distribution
Trial Status
Trial Success Rate
Benchmark: 86.5%
Based on 2 completed trials
Clinical Trials (8)
An Exploratory Evaluation of the Safety and Efficacy of Vorinostat in Pitt Hopkins Syndrome
Phase 1/2 Study of MZ-1866, an AAV-9 Gene Therapy Delivered by Intracerebroventricular Injection to Participants With Pitt Hopkins Syndrome
An Open-Label Study of Oral NNZ-2591 in Pitt Hopkins Syndrome (PTHS-001)
Rare Disease Patient Registry & Natural History Study - Coordination of Rare Diseases at Sanford
Early Check: Expanded Screening in Newborns
MTT for Children With Both Pitt Hopkins Syndrome and Gastrointestinal Disorders
Microbiota Transfer Therapy for Children and Adults With Both Pitt Hopkins Syndrome and Gastrointestinal Disorders
Safety & Efficacy of AlloRx SC® in PTHS Patients