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

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

Primary Outcome

Adverse events

Secondary Outcome

 Change in EEG activity

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 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 < 3.0 mmol/L) or family history of long QT syndrome;
             QT/QTcF prolongation previously or currently controlled with medication

          -  Current treatment with insulin

          -  Hgb A1C values outside the normal reference range at screening

          -  Current or past treatment with IGF-1

          -  Current or past treatment with growth hormone

          -  Current treatment with N-methyl-D-aspartate (NMDA) antagonists

          -  Current or planned use of non-medication based interventional therapy during the
             period of the study (defined as 4-6 week screening period followed by 4 week dosing
             and 2 week follow-up period)

          -  Current clinically significant cardiovascular, renal, hepatic or respiratory disease

          -  Gastrointestinal disease which may interfere with the absorption, distribution,
             metabolism or excretion of the the study medication

          -  History of, or current cerebrovascular disease or brain trauma

          -  History of, or current significant endocrine disorder e.g. hypo or hyperthyroidism or
             diabetes mellitus

          -  History of, or current malignancy

          -  Clinically significant abnormalities in safety laboratory tests, vital signs or ECG,
             as measured at screening or baseline

          -  Confirmed pregnancy

          -  Significant hearing and/or visual impairment that may affect ability to complete the
             test procedures

          -  Enrollment in another clinical trial within the previous 30 days

          -  Previously randomized in this clinical trial

          -  Allergy to strawberries
      

Gender

Female

Ages

16 Years - 45 Years

Accepts Healthy Volunteers

No

Contacts

Daniel G Glaze, M.D., , 

Location Countries

United States

Location Countries

United States

NCT ID

NCT01703533

Organization ID

Neu-2566-RETT-001

Responsible Party

Sponsor

Study Sponsor

Neuren Pharmaceuticals Limited

Collaborators

 Baylor College of Medicine

Study Sponsor

Daniel G Glaze, M.D., Principal Investigator, Baylor College of Medicine

Verification Date

January 2018