Clinical trials: Phelan-McDermid Syndrome
Phelan-McDermid Syndrome, Epilepsy
AMO-01 to Treat Adolescents and Adults With Phelan-McDermid Syndrome (PMS) and Co-morbid Epilepsy
The purpose of this study is to investigate the safety, tolerability and efficacy of a single 6-hour intravenous infusion of AMO-01 to treat adolescents and adults with PMS and co-morbid epilepsy.
- Trial name
- NCT03493607
- Testing
- AMO-01
- Where
- 2 sites in United States
- Volunteers
- 6 people
- Sponsor
- Alexander Kolevzon
Phelan-McDermid Syndrome
An Open-Label Study of Oral NNZ-2591 in Phelan-McDermid Syndrome (PMS-001)
A study of the safety, tolerability and pharmacokinetics of NNZ-2591 and measures of efficacy in children and adolescents with Phelan-McDermid Syndrome.
- Trial name
- PMS-001 (NCT05025241)
- Testing
- NNZ-2591
- Where
- 4 sites in United States
- Volunteers
- 18 people
- Sponsor
- Neuren Pharmaceuticals Limited
Phelan McDermid Syndrome
Growth Hormone Treatment in Children With Phelan McDermid Syndrome
Phelan McDermid syndrome (PMS) is a rare genetic form of autism spectrum disorder (ASD) due to deletions or mutations in the SHANK3 gene.
- Trial name
- NCT04003207
- Testing
- Recombinant human Growth hormone
- Where
- United States
- Volunteers
- 6 people
- Sponsor
- Swathi Sethuram
22q13 Deletion Syndrome, Phelan-McDermid Syndrome
Clinical Trial in 22q13 Deletion Syndrome(Phelan-McDermid Syndrome)
The purpose of this study is to pilot the use of Insulin-Like Growth Factor-1 (IGF-1) treatment in 22q13 Deletion Syndrome (Phelan-McDermid Syndrome) caused by SHANK3 gene deficiency in order to evaluate safety, tolerability, and…
- Trial name
- NCT01525901
- Testing
- Insulin-Like Growth Factor-1 (IGF-1), Normal saline
- Where
- United States
- Volunteers
- 19 people
- Sponsor
- Icahn School of Medicine at Mount Sinai
Phelan-McDermid Syndrome
Piloting Treatment With Intranasal Oxytocin in Phelan-McDermid Syndrome
This is a pilot study examining the efficacy, safety and tolerability of intranasal oxytocin as a novel treatment in Phelan-McDermid syndrome (PMS).
- Trial name
- NCT02710084
- Testing
- Oxytocin, Saline
- Where
- United States
- Volunteers
- 18 people
- Sponsor
- Alexander Kolevzon