Pitt Hopkins Syndrome Study


The purpose of the study is to look at the safety, tolerability, and efficacy of NNZ-2591 in the treatment of children with Pitt Hopkins syndrome…

Info For Caregivers

You will be responsible for ensuring that the participant attends the study visits to meet with your study doctor and other study staff…


This clinical study is sponsored by Neuren Pharmaceuticals, a biopharmaceutical company headquartered in Melbourne, Australia… 

Does someone you know have Pitt Hopkins syndrome?

They may be eligible to participate in a research study on a new medication for children and adolescents aged 3-17 years.

Pitt Hopkins syndrome is a genetically determined neurological disorder, characterized by developmental delays (including motor delays), profound intellectual disability, absent or limited speech, distinctive facial dysmorphia, breathing problems, repetitive behaviors, social communication impairments, sleep disturbances, seizures, symptoms of Autism Spectrum Disorder (ASD).

This is a spectrum disorder which means not all people display the same behaviors and symptoms.

People with Pitt Hopkins syndrome need continuous care and are unable to live independently.

There is currently no treatment for Pitt Hopkins syndrome.