NCT02715115

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.

Trial Health

87
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
82

participants targeted

Target at P50-P75 for phase_2

Timeline
Completed

Started Mar 2016

Shorter than P25 for phase_2

Geographic Reach
1 country

12 active sites

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

February 21, 2016

Completed
9 days until next milestone

Study Start

First participant enrolled

March 1, 2016

Completed
21 days until next milestone

First Posted

Study publicly available on registry

March 22, 2016

Completed
10 months until next milestone

Primary Completion

Last participant's last visit for primary outcome

January 5, 2017

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

January 5, 2017

Completed
Last Updated

August 14, 2020

Status Verified

August 1, 2020

Enrollment Period

10 months

First QC Date

February 21, 2016

Last Update Submit

August 6, 2020

Conditions

Rett Syndrome

Keywords

AutismRett's syndromeRett disorderRett's disorderAtaxia

Outcome Measures

Primary Outcomes (1)

  • 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 Outcomes (3)

  • Motor Behaviour Assessment Scale (MBA)

    Through study completion, an average of 11 weeks

  • Clinical Global Impression of Improvement (CGI-I)

    Through study completion, an average of 11 weeks

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

    Through study completion, an average of 11 weeks

Study Arms (2)

NNZ-2566

EXPERIMENTAL

Glycyl-L-2-Methylpropyl-L-Glutamic Acid

Drug: NNZ-2566

Placebo (strawberry flavored solution)

PLACEBO COMPARATOR

Strawberry flavored solution and Water for Injection

Drug: Placebo

Interventions

NNZ-2566DRUG

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.

Also known as: trofinetide
NNZ-2566
PlaceboDRUG

Strawberry flavored solution and Water for Injection

Also known as: Strawberry flavoring
Placebo (strawberry flavored solution)

Eligibility Criteria

Age5 Years - 15 Years
Sexfemale
Healthy VolunteersNo
Age GroupsChild (0-17)

You may qualify if:

  • 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.

You may not qualify if:

  • 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.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (12)

University of Alabama

Birmingham, Alabama, 35233, United States

Location

UCSF Benioff Children's Hospital Oakland

Oakland, California, 94609, United States

Location

University of California, San Diego

San Diego, California, 92093, United States

Location

Children's Hosptial Colorado

Aurora, Colorado, 80045, United States

Location

Rush University Medical Center

Chicago, Illinois, 60612, United States

Location

Boston Children's Hospital

Boston, Massachusetts, 02115, United States

Location

Gillette Children's Specialty Healthcare

Saint Paul, Minnesota, 55101, United States

Location

Cincinnati Children's Hospital Medical Center

Cincinnati, Ohio, 45229, United States

Location

Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, 19104, United States

Location

Greenwood Genetic Center

Greenwood, South Carolina, 29646, United States

Location

Vanderbilt University

Nashville, Tennessee, 37235, United States

Location

Baylor College of Medicine

Houston, Texas, 77030, United States

Location

Related Publications (2)

  • Darwish M, Passarell J, Youakim JM, Bradley H, Bishop KM. Exposure-Response Efficacy Modeling to Support Trofinetide Dosing in Individuals with Rett Syndrome. Adv Ther. 2024 Apr;41(4):1462-1480. doi: 10.1007/s12325-024-02796-y. Epub 2024 Feb 16.

    PMID: 38363467DERIVED

  • Parent H, Ferranti A, Niswender C. Trofinetide: a pioneering treatment for Rett syndrome. Trends Pharmacol Sci. 2023 Oct;44(10):740-741. doi: 10.1016/j.tips.2023.06.008. Epub 2023 Jul 16.

    PMID: 37460385DERIVED

MeSH Terms

Conditions

Rett SyndromeAutistic DisorderAtaxia

Interventions

trofinetide

Condition Hierarchy (Ancestors)

X-Linked Intellectual DisabilityIntellectual DisabilityNeurobehavioral ManifestationsNeurologic ManifestationsNervous System DiseasesGenetic Diseases, X-LinkedGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesHeredodegenerative Disorders, Nervous SystemAutism Spectrum DisorderChild Development Disorders, PervasiveNeurodevelopmental DisordersMental DisordersDyskinesiasSigns and SymptomsPathological Conditions, Signs and Symptoms

Study Officials

  • 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

    PRINCIPAL INVESTIGATOR

Study Design

Study Type
interventional
Phase
phase 2
Allocation
RANDOMIZED
Masking
QUADRUPLE
Who Masked
PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
Purpose
TREATMENT
Intervention Model
PARALLEL
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

February 21, 2016

First Posted

March 22, 2016

Study Start

March 1, 2016

Primary Completion

January 5, 2017

Study Completion

January 5, 2017

Last Updated

August 14, 2020

Record last verified: 2020-08

Locations

US(12)