Phelan-McDermid Syndrome
16
3
5
8
Key Insights
Highlights
Success Rate
100% trial completion (above average)
Clinical Risk Assessment
Based on trial outcomes
Low Risk
Score: 27/100
0.0%
0 terminated out of 16 trials
100.0%
+13.4% vs benchmark
13%
2 trials in Phase 3/4
50%
4 of 8 completed with results
Key Signals
Data Visualizations
Phase Distribution
Trial Status
Trial Success Rate
Benchmark: 86.6%
Based on 8 completed trials
Clinical Trials (16)
Mapping the Genotype, Phenotype, and Natural History of Phelan-McDermid Syndrome
RB001 Gene Therapy Study in Children With SHANK3-related Phelan-McDermid Syndrome (PMS)
An Open-label Study of NNZ-2591 in Pediatric Participants With Phelan-McDermid Syndrome
Long-Term Follow-Up Study for RB001 Gene Therapy Study in Children With SHANK3-related Phelan McDermid Syndrome (PMS)
Parent and Infant Inter(X)Action Intervention (PIXI)
A Study of NNZ-2591 in Pediatric Participants With Phelan-McDermid Syndrome
JAG201 Gene Therapy Study in Children & Adults With SHANK3 Haploinsufficiency
AMO-01 to Treat Adolescents and Adults With Phelan-McDermid Syndrome (PMS) and Co-morbid Epilepsy
An Open-Label Study of Oral NNZ-2591 in Phelan-McDermid Syndrome (PMS-001)
A Controlled Trial of Growth Hormone in Phelan-McDermid Syndrome and Idiopathic Autism
Mapping the Phenotype in Adults With Phelan-McDermid Syndrome
Effectiveness of Recombinant Human Growth Hormone Therapy for Children With PMS
Clinical Trial in 22q13 Deletion Syndrome(Phelan-McDermid Syndrome)
Piloting Treatment With Intranasal Oxytocin in Phelan-McDermid Syndrome
Mitochondrial Dysfunction in Phelan-McDermid Syndrome
Q10 Ubiquinol in Autism Spectrum Disorder and in Phelan-McDermid Syndrome.