A Safety Study of NNZ-2566 in Pediatric Rett Syndrome

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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