Eligibility Module
The Eligibility Module contains detailed information about who can participate in the clinical trial. This includes eligibility criteria, age restrictions, gender requirements, healthy volunteer status, and study population descriptions, helping researchers understand who is eligible to participate in the study.
Eligibility Module path is as follows:
Study -> Protocol Section -> Eligibility Module
Eligibility Module
Ignite Creation Date:
2025-12-25 @ 3:03 AM
Ignite Modification Date:
2025-12-25 @ 3:03 AM
NCT ID:
NCT01703533
Eligibility Criteria:
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
Healthy Volunteers:
False
Sex:
FEMALE
Minimum Age:
16 Years
Maximum Age:
45 Years
Study:
NCT01703533
Study Brief:
Protocol Section:
NCT01703533