NCT02715115

A Safety Study of NNZ-2566 in Pediatric Rett Syndrome

A Randomized, Double-blind, Placebo-controlled, Dose-ranging Study of the Safety and Pharmacokinetics of Oral NNZ-2566 in Pediatric Rett Syndrome

• Status: Completed
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 82 (actual)
• Sponsor: Neuren Pharmaceuticals Limited · Industry
• Sex: Female
• Age: 5 Years – 15 Years
• Healthy volunteers: Not accepted

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.

Detailed 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.

Conditions

• Rett Syndrome

Interventions

Type	Name	Description
DRUG	NNZ-2566	Glycyl-L-2-Methylpropyl-L-Glutamic Acid (NNZ-2566) supplied as a lyophilized powder for reconstitution with strawberry flavored solution 0.5% v/v in Water for Injection.
DRUG	Placebo	Strawberry flavored solution and Water for Injection

Timeline

• Start date: 2016-03-01
• Primary completion: 2017-01-05
• Completion: 2017-01-05
• First posted: 2016-03-22
• Last updated: 2020-08-14

Locations

12 sites across 1 country: United States