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A Safety Study of NNZ-2566 in Pediatric Rett Syndrome

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Disease Information

Descriptive Information
Brief Title † A Safety Study of NNZ-2566 in Pediatric Rett Syndrome
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 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 Phase Phase 2
Study Type † Interventional
Study Design †
Primary Outcome Measure † Adverse events
Secondary Outcome Measure † Motor Behaviour Assessment Scale (MBA)
Condition † Rett Syndrome
Intervention † DrugNNZ-2566
Study Arms / Comparison Groups NNZ-2566 Glycyl-L-2-Methylpropyl-L-Glutamic Acid Placebo (strawberry flavored solution) Strawberry flavored solution and Water for Injection
Publications *

* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.

Recruitment Information
Recruitment Status † Drug
Estimated Enrollment † 82
Start Date † March 2016
Completion Date January 2017
Primary Completion Date January 2017
Eligibility Criteria † Inclusion Criteria: - Diagnosis of classic/typical Rett syndrome with a documented mutation of the MeCP2 gene. - Age 5 - 15 years. - Weight at Screening and Baseline between 15.0 kg-100.0 kg (at least 15.0 kg and no greater than 100.0 kg). - Each subject must be able to swallow the study medication provided as a liquid solution, or via gastrostomy tube. Exclusion Criteria: - Actively undergoing neurological regression - Abnormal QT interval, prolongation or significant cardiovascular history. - Current treatment with insulin. - Anti-convulsants with liver enzyme inducing effects. - Unstable seizure profile. - Excluded concomitant medications. - Current clinically significant (as determined by the investigator). cardiovascular, renal, hepatic, or respiratory disease. - Gastrointestinal disease which may interfere with the absorption, distribution, metabolism or excretion of the study medication. - History of, or current cerebrovascular disease or brain trauma. - History of, or current clinically significant endocrine disorder, e.g. hypo- or hyperthyroidism, or diabetes mellitus. - History of, or current, malignancy. - Significant hearing and/or visual impairments that may affect ability to complete the test procedures. - Allergy to strawberry.
Gender Female
Ages 5 Years - 15 Years
Accepts Healthy Volunteers No
Contacts ††
Location Countries † United States
Administrative Information
NCT ID † NCT02715115
Organization ID Neu-2566-RETT-002
Secondary IDs ††
Responsible Party Sponsor
Study Sponsor † Neuren Pharmaceuticals Limited
Collaborators †† rettsyndrome.org
Investigators † Principal Investigator: Daniel Glaze, MD, Baylor College of Medicine
Information Provided By
Verification Date February 2017
First Received Date † February 21, 2016
Last Updated Date February 7, 2017
† Required WHO trial registration data element.
†† WHO trial registration data element that is required only if it exists.
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