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Completed

NCT Number: NCT02715115

A Safety Study of NNZ-2566 in Pediatric Rett Syndrome

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.

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Key information

Conditions

Rett Syndrome Ataxia Autism Spectrum Disorder Autistic Disorder Child Development Disorders, Pervasive Congenital, Hereditary, and Neonatal Diseases and Abnormalities Dyskinesias Genetic Diseases, Inborn Genetic Diseases, X-Linked Heredodegenerative Disorders, Nervous System Intellectual Disability Mental Disorders Nervous System Diseases Neurobehavioral Manifestations Neurodevelopmental Disorders Neurologic Manifestations Pathological Conditions, Signs and Symptoms Signs and Symptoms X-Linked Intellectual Disability

Age range

5 year–15 year

Sex eligibility

Female

Study type

Interventional

Phase

Phase 2

Primary location

University of Alabama, Birmingham, Alabama, United States

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About this study

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.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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.

Treatment and study plan

NNZ-2566

Drug

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.

Other names: trofinetide

Placebo

Drug

Strawberry flavored solution and Water for Injection

Other names: Strawberry flavoring

Primary outcomes

  1. Adverse events

    Time frame: Through study completion, an average of 11 weeks

    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.

Secondary outcomes

  1. Motor Behaviour Assessment Scale (MBA)

    Time frame: Through study completion, an average of 11 weeks

  2. Clinical Global Impression of Improvement (CGI-I)

    Time frame: Through study completion, an average of 11 weeks

  3. Caregiver Top 3 Concerns via a Visual Analogue Scale (VAS)

    Time frame: Through study completion, an average of 11 weeks

Sponsors and collaborators

Lead sponsor

Neuren Pharmaceuticals Limited

Industry

Collaborators

  • rettsyndrome.org

Registry information

Official study title

A Randomized, Double-blind, Placebo-controlled, Dose-ranging Study of the Safety and Pharmacokinetics of Oral NNZ-2566 in Pediatric Rett Syndrome

Important dates

Study start
2016
Primary completion
2017
Study completion
2017
First posted
Mar 22, 2016
Registry last updated
Aug 14, 2020

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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