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
Primary Outcome
Adverse events
Secondary Outcome
Motor Behaviour Assessment Scale (MBA)
Condition
Rett Syndrome
Intervention
NNZ-2566
Study Arms / Comparison Groups
NNZ-2566
Description: Glycyl-L-2-Methylpropyl-L-Glutamic Acid
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 5, 2017
Primary Completion Date
January 5, 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
Daniel Glaze, MD, ,
Location Countries
United States
Location Countries
United States
Administrative Informations
NCT ID
NCT02715115
Organization ID
Neu-2566-RETT-002
Responsible Party
Sponsor
Study Sponsor
Neuren Pharmaceuticals Limited
Collaborators
rettsyndrome.org
Study Sponsor
Daniel Glaze, MD, Principal Investigator, Baylor College of Medicine
Verification Date
August 2020