Clinical Trial: A Safety Study of NNZ-2566 in Pediatric Rett Syndrome

Study Status: Completed
Recruit Status: Completed
Study Type: Interventional

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

Brief 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 Summary:

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.


Sponsor: Neuren Pharmaceuticals Limited

Current Primary Outcome: Adverse events [ Time Frame: Through study completion, an average of 11 weeks ]

Incidence of adverse events (AEs), including serious adverse events (SAEs), will be compared across the three NNZ-2566 doses and placebo. SAEs and AEs will be examined throughout the study.


Original Primary Outcome: Same as current

Current Secondary Outcome:

  • Motor Behaviour Assessment Scale (MBA) [ Time Frame: Through study completion, an average of 11 weeks ]
  • Clinical Global Impression of Improvement (CGI-I) [ Time Frame: Through study completion, an average of 11 weeks ]
  • Caregiver Top 3 Concerns via a Visual Analogue Scale (VAS) [ Time Frame: Through study completion, an average of 11 weeks ]


Original Secondary Outcome: Same as current

Information By: Neuren Pharmaceuticals Limited

Dates:
Date Received: February 21, 2016
Date Started: March 2016
Date Completion:
Last Updated: February 7, 2017
Last Verified: February 2017