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Trial · Interventional

CompletedPhase 2Registry data

A Safety Study of NNZ-2566 in Pediatric Rett Syndrome

NCTNCT02715115 (opens ClinicalTrials.gov in a new tab)Other idNeu-2566-RETT-002
Open at ClinicalTrials.govAsk about this trial

Overview

Status
Completed
Phase
Phase 2
Enrollment
82 participants (actual)
Sponsor
Neuren Pharmaceuticals Limited · Industry
Design
Randomized · Quadruple masking
Countries
1 country

United States

Start
Mar 2016
Primary completion
5 Jan 2017
Completion
5 Jan 2017
First posted
22 Mar 2016 (estimated)
Last update posted
14 Aug 2020
Conditions
Rett Syndrome

Timeline

1 trial · 1 completed

  • Completed
  • Ongoing
  • Not yet recruiting
  • Stopped
  • Estimated dates (est.)
  • Results posted
201620172018
  1. Ph 2

    A Safety Study of NNZ-2566 in Pediatric Rett Syndrome

    NCT02715115

    Mar 2016 to 5 Jan 2017

Registry summary

Text and fields below are the sponsor's entries at ClinicalTrials.gov, shown as posted.

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.

Interventions

  • NNZ-2566Drug
  • PlaceboDrug

Arms2

  • Experimental

    NNZ-2566

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

  • Placebo comparator

    Placebo (strawberry flavored solution)

    Strawberry flavored solution and Water for Injection

Primary outcomes1

Outcomes the sponsor listed as primary.

  1. Adverse events

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

    Registry description

    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(3)

  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

Posted results

Values are shown as posted at ClinicalTrials.gov, with their units. PepBank has not converted, pooled or compared them.

No results are posted for this trial.

Completed trials do not always post results.

Registry data comes from ClinicalTrials.gov. Study results come from studies, not from the registry. How PepBank works

Studies1

Studies that cite this trial.

Studies with results

1 study on this page

Indexing hint: Clinical trial · 2024

4 results

Exposure-Response Efficacy Modeling to Support Trofinetide Dosing in Individuals with Rett Syndrome.

Darwish et al. 2024Advances in therapycited by 3

PMID 38363467PMC10960884DOI 10.1007/s12325-024-02796-yPUB-38363467

Free to read · publisher termsCC BY-NC4 results
Read at sourceSee results (4)

Other registry references

References listed at the registry that are not in PepBank. They link to PubMed.

  • DerivedParent 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 37460385

Eligibility

Who could join, as posted.

Sex
Female
Age
from 5 Years up to 15 Years
Healthy volunteers
Not accepted
Inclusion and exclusion criteria, as posted
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.

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