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Clinical Trial Details — Status: Completed

Administrative data

NCT number NCT02715115
Other study ID # Neu-2566-RETT-002
Secondary ID
Status Completed
Phase Phase 2
First received
Last updated
Start date March 2016
Est. completion date January 5, 2017

Study information

Verified date August 2020
Source Neuren Pharmaceuticals Limited
Contact n/a
Is FDA regulated No
Health authority
Study type Interventional

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.


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.


Recruitment information / eligibility

Status Completed
Enrollment 82
Est. completion date January 5, 2017
Est. primary completion date January 5, 2017
Accepts healthy volunteers No
Gender Female
Age group 5 Years to 15 Years
Eligibility 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.

Study Design


Related Conditions & MeSH terms


Intervention

Drug:
NNZ-2566
Glycyl-L-2-Methylpropyl-L-Glutamic Acid (NNZ-2566) supplied as a lyophilized powder for reconstitution with strawberry flavored solution 0.5% v/v in Water for Injection.
Placebo
Strawberry flavored solution and Water for Injection

Locations

Country Name City State
United States Children's Hosptial Colorado Aurora Colorado
United States University of Alabama Birmingham Alabama
United States Boston Children's Hospital Boston Massachusetts
United States Rush University Medical Center Chicago Illinois
United States Cincinnati Children's Hospital Medical Center Cincinnati Ohio
United States Greenwood Genetic Center Greenwood South Carolina
United States Baylor College of Medicine Houston Texas
United States Vanderbilt University Nashville Tennessee
United States UCSF Benioff Children's Hospital Oakland Oakland California
United States Children's Hospital of Philadelphia Philadelphia Pennsylvania
United States Gillette Children's Specialty Healthcare Saint Paul Minnesota
United States University of California, San Diego San Diego California

Sponsors (2)

Lead Sponsor Collaborator
Neuren Pharmaceuticals Limited rettsyndrome.org

Country where clinical trial is conducted

United States, 

Outcome

Type Measure Description Time frame Safety issue
Primary Adverse events Incidence of adverse events (AEs), including serious adverse events (SAEs), will be compared across the three NNZ-2566 doses and placebo. SAEs and AEs will be examined throughout the study. Through study completion, an average of 11 weeks
Secondary Motor Behaviour Assessment Scale (MBA) Through study completion, an average of 11 weeks
Secondary Clinical Global Impression of Improvement (CGI-I) Through study completion, an average of 11 weeks
Secondary Caregiver Top 3 Concerns via a Visual Analogue Scale (VAS) Through study completion, an average of 11 weeks
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