Rett Syndrome Clinical Trial
Official title:
A Phase II Randomized, Double-Blind, Placebo-Controlled, Parallel-Group, Dose-Escalation Study of NNZ-2566 in Rett Syndrome
The purpose of this study is to determine whether NNZ-2566 is safe and well tolerated in the treatment of Rett Syndrome in adolescent and adult females.
Rett Syndrome is a developmental disorder primarily if not exclusively 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 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 and tolerability of treatment with oral administration
of NNZ-2566 at 35 mg/kg or 70 mg/kg BID in adolescent or adult females with Rett Syndrome.
The study also will also investigate measures of efficacy during treatment.
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