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
Status	Clinical Trial	Phase
Completed	`NCT04988867` - An Open-Label Study of Trofinetide for the Treatment of Girls Two to Five Years of Age Who Have Rett Syndrome	Phase 2/Phase 3
Recruiting	`NCT00069550` - Independent Studies of Dextromethorphan and of Donepezil Hydrochloride for Rett Syndrome	Phase 3
Enrolling by invitation	`NCT06139172` - Promoting Prosocial Behavior in Syndromic Intellectual and Developmental Disabilities	N/A
Not yet recruiting	`NCT04041713` - A Pilot Study of an Antioxidant Cocktail vs. Placebo in the Treatment of Children and Adolescents With Rett Syndrome	Phase 2
Not yet recruiting	`NCT04014985` - Patients With RETT Syndrome	N/A
Completed	`NCT02705677` - Biobanking of Rett Syndrome and Related Disorders
Terminated	`NCT02790034` - Evaluation of the Efficacy, Safety, and Tolerability of Sarizotan in Rett Syndrome With Respiratory Symptoms	Phase 2/Phase 3
Enrolling by invitation	`NCT03655223` - Early Check: Expanded Screening in Newborns
Recruiting	`NCT05932589` - Neurophysiologic Biomarkers in Rett Syndrome
Recruiting	`NCT04463316` - GROWing Up With Rare GENEtic Syndromes
Completed	`NCT04776746` - Open-Label Extension Study of Trofinetide for Rett Syndrome	Phase 3
Completed	`NCT04181723` - Study of Trofinetide for the Treatment of Girls and Women With Rett Syndrome (LAVENDER™)	Phase 3
Enrolling by invitation	`NCT03836300` - Parent and Infant Inter(X)Action Intervention (PIXI)	N/A
Completed	`NCT04514549` - ASSESSING EMERALD AND MC10 BIOSTAMP nPOINT BIOSENSORS FOR RETT SYNDROME
Completed	`NCT02738281` - Natural History of Rett Syndrome & Related Disorders
Terminated	`NCT02562820` - An Exploratory Trial of Ketamine for the Treatment of Rett Syndrome	Phase 1
Completed	`NCT05687214` - Osteopathic Manipulative Treatment for Constipation in People With Rett Syndrome	N/A
Recruiting	`NCT06199700` - Esketamine for the Treatment of Rett Syndrome	Early Phase 1
Completed	`NCT03941444` - ANAVEX2-73 Study in Patients With Rett Syndrome	Phase 3
Recruiting	`NCT06346106` - The Diagnostic Experience of Male Rett Syndrome