A Safety Study of NNZ-2566 in Pediatric Rett Syndrome

Status Completed

Clinical Trial Summary

The purpose of this study is to determine whether NNZ-2566 is safe and well tolerated in the treatment of Rett syndrome in children and adolescents.

Clinical Trial Description

Rett syndrome is a neurodevelopmental disorder primarily affecting females. The disorder is characterized by apparent normal development in early infancy (6-18 months), followed by a period of regression with onset of systemic and neurological signs. The CNS symptoms of Rett syndrome include learning disability, autism symptomatology and epilepsy and these can be severe and highly debilitating. Affected individuals also show signs of autonomic dysfunction, reflected in cardiovascular and respiratory abnormalities. There is no currently effective treatment for Rett syndrome.

This study will investigate the safety, tolerability and blood pharmacokinetics of treatment with oral administration of NNZ-2566 at 50 mg/kg, 100 mg/kg, 200 mg/kg BID, or placebo BID, in children and adolescent females with Rett syndrome. The study also will also investigate measures of efficacy and biomarkers during treatment. ;

Study Design

Related Conditions & MeSH terms

Clinical Trial Details

NCT number	NCT02715115
Study type	Interventional
Source	Neuren Pharmaceuticals Limited
Contact
Status	Completed
Phase	Phase 2
Start date	March 2016
Completion date	January 5, 2017

View Details

See also
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