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Trial · Interventional

CompletedPhase 2Registry data

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

NCTNCT01703533 (opens ClinicalTrials.gov in a new tab)Other idNeu-2566-RETT-001
Open at ClinicalTrials.govAsk about this trial

Overview

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

United States

Start
Mar 2013
Primary completion
Sept 2014
Completion
Sept 2014
First posted
10 Oct 2012 (estimated)
Last update posted
5 Feb 2018
Conditions
Rett Syndrome

Timeline

1 trial · 1 completed

  • Completed
  • Ongoing
  • Not yet recruiting
  • Stopped
  • Estimated dates (est.)
  • Results posted
201320142015
  1. Ph 2

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

    NCT01703533

    Mar 2013 to Sept 2014

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 adolescent and adult females.

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 to Day 40

    Registry description

    Incidence of adverse events (AE), including Serious adverse events (SAE), will be evaluated between the two NNZ-2566 doses and placebo. SAEs will be examined from randomization through to Day 40. AEs will be examined from dosing through to Day 40.

Secondary outcomes(4)

  1. Change in EEG activity

    Time frame · Baseline through to Day 40

    Registry description

    Absolute change in the number of spikes in the EEG per hour during the awake state will be calculated for each subject between baseline (pre-treatment), during treatment (Days 1 thru 4, 14 and 26) and after treatment (Day 40). Absolute change in the power of frequency bands in the EEG over an hour in the awake state as determined by the Fast Fourier method will be calculated for each subject between baseline (pre-treatment), during treatment (Days 1 thru 4, 14 and 26) and after treatment (Day 40). Changes in the frequency of the characteristic repetitive stereotypic hand movements during wakefulness will be calculated for each subject between baseline (pre-treatment), during treatment (Days 1 thru 4, 14 and 26) and after treatment (Day 40).

  2. Behavior

    Time frame · Baseline through to Day 40

    Registry description

    The following measures will be assessed at baseline and Day 26 and the changes compared between active and placebo groups: Symptom severity according to the Rett Syndrome Natural History Motor Behavior Assessment (MBA), Clinical Severity Scale (CSS), Aberrant Behavior Checklist (ABC), Vineland Adaptive Behavior Scale (VABS), and Clinical Global Impression of Severity (CGI-S). The following assessments will be undertaken at the additional time points specified: CGI-S (screening, baseline, Days 5, 14, 26, and 40), CGI-I (Days 5, 14, 26, and 40), MBA (Baseline, Days 26 and 40), CSS (screening, baseline, Days 26, and 40).

  3. Physiological changes

    Time frame · Baseline through to Day 40

    Registry description

    Changes in autonomic function, i.e. respiratory rhythm, hyperventilation, apneas, oxygen desaturation, and heart rate variation will be calculated between baseline (pre-treatment), during treatment (Days 1 thru 4, 14 and 26) and after treatment (Day 40).

  4. Global and Functional outcome Measures

    Time frame · Baseline through to Day 40

    Registry description

    Global outcome as measured by the change in scores on the Rett Syndrome Clinical Severity Score (CSS), The Rett Syndrome Motor-Behavior Assessment Scale (MBA), and the Clinical Global Impression - Severity and Improvement scales (CGI-S and -I) from baseline, during treatment, and post treatment. Changes in caregiver assessment of the top three causes for concern as assessed via a Visual Analogue Scale (VAS) will be evaluated for each subject between baseline (pre-treatment), during treatment (Day 26) and after treatment (Day 40). Changes in the Aberrant Behavior Checklist (ABC) and Vineland Adaptive Behavior Scales (VABS) will be calculated for each subject between baseline (pre-treatment), and during treatment (Day 26).

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 16 Years up to 45 Years
Healthy volunteers
Not accepted
Inclusion and exclusion criteria, as posted
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

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