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Main
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Note: This record shows only 22 elements of the WHO Trial Registration Data Set. To view changes that have been made to the source record, or for additional information about this trial, click on the URL below to go to the source record in the primary register. |
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Register:
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ClinicalTrials.gov |
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Last refreshed on:
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12 December 2020 |
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Main ID: |
NCT01703533 |
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Date of registration:
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04/10/2012 |
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Prospective Registration:
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Yes |
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Primary sponsor: |
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Public title:
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A Safety Study of NNZ-2566 in Patients With Rett Syndrome
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Scientific title:
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A Phase II Randomized, Double-Blind, Placebo-Controlled, Parallel-Group, Dose-Escalation Study of NNZ-2566 in Rett Syndrome |
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Date of first enrolment:
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March 2013 |
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Target sample size:
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67 |
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Recruitment status: |
Completed |
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URL:
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https://clinicaltrials.gov/show/NCT01703533 |
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Study type:
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Interventional |
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Study design:
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Allocation: Randomized. Intervention model: Parallel Assignment. Primary purpose: Treatment. Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor).
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Phase:
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Phase 2
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Countries of recruitment
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United States
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Contacts
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Name:
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Alan Percy, MD |
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Address:
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Telephone:
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Email:
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Affiliation:
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University of Alabama at Birmingham |
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Name:
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Jeffrey L Neul, M.D., Ph.D. |
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Address:
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Telephone:
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Email:
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Affiliation:
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Baylor College of Medicine |
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Name:
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Arthur Beisang, MD |
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Address:
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Telephone:
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Email:
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Affiliation:
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Gillette Children's Specialty Healthcare |
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Name:
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Daniel G Glaze, M.D. |
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Address:
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Telephone:
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Email:
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Affiliation:
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Baylor College of Medicine |
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Name:
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Timothy Feyma, MD |
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Address:
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Telephone:
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Email:
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Affiliation:
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Gillette Children's Specialty Healthcare |
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Key inclusion & exclusion criteria
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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
Age minimum:
16 Years
Age maximum:
45 Years
Gender:
Female
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Health Condition(s) or Problem(s) studied
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Rett Syndrome
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Intervention(s)
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Drug: Placebo
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Drug: NNZ-2566
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Primary Outcome(s)
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Adverse events
[Time Frame: Through to Day 40]
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Secondary Outcome(s)
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Behavior
[Time Frame: Baseline through to Day 40]
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Physiological changes
[Time Frame: Baseline through to Day 40]
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Change in EEG activity
[Time Frame: Baseline through to Day 40]
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Global and Functional outcome Measures
[Time Frame: Baseline through to Day 40]
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Secondary ID(s)
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Neu-2566-RETT-001
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Source(s) of Monetary Support
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Please refer to primary and secondary sponsors
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Results
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Results available:
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Date Posted:
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Date Completed:
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URL:
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