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A Safety Study of NNZ-2566 in Pediatric Rett Syndrome

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
ClinicalTrials.gov Identifier: NCT02715115
Recruitment Status : Completed
First Posted : March 22, 2016
Last Update Posted : August 14, 2020
Information provided by (Responsible Party):
Neuren Pharmaceuticals Limited

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.

Condition or disease Intervention/treatment Phase
Rett Syndrome Drug: NNZ-2566 Drug: Placebo Phase 2

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.

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 82 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Official Title: A Randomized, Double-blind, Placebo-controlled, Dose-ranging Study of the Safety and Pharmacokinetics of Oral NNZ-2566 in Pediatric Rett Syndrome
Actual Study Start Date : March 2016
Actual Primary Completion Date : January 5, 2017
Actual Study Completion Date : January 5, 2017

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Rett Syndrome

Arm Intervention/treatment
Experimental: NNZ-2566
Glycyl-L-2-Methylpropyl-L-Glutamic Acid
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.
Other Name: trofinetide

Placebo Comparator: Placebo (strawberry flavored solution)
Strawberry flavored solution and Water for Injection
Drug: Placebo
Strawberry flavored solution and Water for Injection
Other Name: Strawberry flavoring

Primary Outcome Measures :
  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 Outcome Measures :
  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 ]

Information from the National Library of Medicine

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Ages Eligible for Study:   5 Years to 15 Years   (Child)
Sexes Eligible for Study:   Female
Accepts Healthy Volunteers:   No

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.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02715115

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United States, Alabama
University of Alabama
Birmingham, Alabama, United States, 35233
United States, California
UCSF Benioff Children's Hospital Oakland
Oakland, California, United States, 94609
University of California, San Diego
San Diego, California, United States, 92093
United States, Colorado
Children's Hosptial Colorado
Aurora, Colorado, United States, 80045
United States, Illinois
Rush University Medical Center
Chicago, Illinois, United States, 60612
United States, Massachusetts
Boston Children's Hospital
Boston, Massachusetts, United States, 02115
United States, Minnesota
Gillette Children's Specialty Healthcare
Saint Paul, Minnesota, United States, 55101
United States, Ohio
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, United States, 45229
United States, Pennsylvania
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States, 19104
United States, South Carolina
Greenwood Genetic Center
Greenwood, South Carolina, United States, 29646
United States, Tennessee
Vanderbilt University
Nashville, Tennessee, United States, 37235
United States, Texas
Baylor College of Medicine
Houston, Texas, United States, 77030
Sponsors and Collaborators
Neuren Pharmaceuticals Limited
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Principal Investigator: Daniel Glaze, MD Baylor College of Medicine
Principal Investigator: Alan Percy, MD University of Alabama at Birmingham
Principal Investigator: Timothy Feyma, MD Gillette Children's Specialty Healthcare
Principal Investigator: Peter Heydemann, MD Rush University Medical Center
Principal Investigator: Jeff Neul, MD University of California, San Diego
Principal Investigator: Tim Benke, MD Children's Hospital Colorado
Principal Investigator: Mary Jones, MD UCSF Benioff Children's Hospital Oakland
Principal Investigator: Steve Skinner, MD Greenwood Genetic Center
Principal Investigator: Mustafa Sahin, MD Boston Children's Hospital
Principal Investigator: Sarika Peters, PhD Vanderbilt University
Principal Investigator: Shannon Standridge Children's Hospital Medical Center, Cincinnati
Principal Investigator: Eric Marsh, MD Children's Hospital of Philadelphia
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Responsible Party: Neuren Pharmaceuticals Limited
ClinicalTrials.gov Identifier: NCT02715115    
Other Study ID Numbers: Neu-2566-RETT-002
First Posted: March 22, 2016    Key Record Dates
Last Update Posted: August 14, 2020
Last Verified: August 2020
Keywords provided by Neuren Pharmaceuticals Limited:
Rett's syndrome
Rett disorder
Rett's disorder
Additional relevant MeSH terms:
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Rett Syndrome
Pathologic Processes
Mental Retardation, X-Linked
Intellectual Disability
Neurobehavioral Manifestations
Neurologic Manifestations
Nervous System Diseases
Genetic Diseases, X-Linked
Genetic Diseases, Inborn
Heredodegenerative Disorders, Nervous System