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A Safety Study of NNZ-2566 in Patients With Rett Syndrome

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Disease Information

Descriptive Information
Brief Title † A Safety Study of NNZ-2566 in Patients With Rett Syndrome
Official Title † A Phase II Randomized, Double-Blind, Placebo-Controlled, Parallel-Group, Dose-Escalation Study of NNZ-2566 in 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 adolescent and adult females.
Detailed Description 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.
Study Phase Phase 2
Study Type † Interventional
Study Design †
Primary Outcome Measure † Adverse events
Secondary Outcome Measure † Change in EEG activity
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 † 67
Start Date † March 2013
Completion Date September 2014
Primary Completion Date September 2014
Eligibility Criteria † Inclusion Criteria: - Diagnosis of Rett Syndrome with proven mutation of the MeCP2 gene - Age 16 to 45 years - Severity rating of between 10 and 36 (Rett Syndrome Natural History/Clinical Severity Scale) - Concomitant medications must be stable for >4 weeks prior to enrollment. The following concomitant medications are permitted: anticonvulsants which do not have liver inducing effects; beta-blockers; medications for the treatment of gastroesophageal reflux disease (GERD); medications for the treatment of chronic respiratory conditions such as asthma; medications for the treatment of anxiety, of depression and of psychosis, hormonal contraceptives. Melatonin for difficulties with sleep onset. - Ability to swallow study medication provided as a liquid solution, or via gastrostomy tube Exclusion Criteria: - No detectable abnormality of the EEG during screening period - Actively undergoing regression - QTcF exclusions (any of the following): baseline/screening QT/QTcF interval of 450 msec; history of risk factors for torsade de pointes (e.g. heart failure, hypokalemia (serum potassium at screening
Gender Female
Ages 16 Years - 45 Years
Accepts Healthy Volunteers No
Contacts ††
Location Countries † United States
Administrative Information
NCT ID † NCT01703533
Organization ID Neu-2566-RETT-001
Secondary IDs ††
Responsible Party Sponsor
Study Sponsor † Neuren Pharmaceuticals Limited
Collaborators †† Baylor College of Medicine
Investigators † Principal Investigator: Daniel G Glaze, M.D., Baylor College of Medicine
Information Provided By
Verification Date March 2017
First Received Date † October 4, 2012
Last Updated Date March 27, 2017
† Required WHO trial registration data element.
†† WHO trial registration data element that is required only if it exists.
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