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

Completed · Phase 2 · Has a placebo group

Conditions studied: Rett Syndrome

In brief

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.

Key facts

Study ID

NCT01703533

Run by

Neuren Pharmaceuticals Limited

People needed

67

Starts

2013-03-01

Expected to finish

2014-09-01

Last updated by the study team

2018-02-05

Who can join

Age: 16 and older, up to 45. Sex: female. Healthy volunteers: not accepted.

You may qualify if…

• 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

You may not qualify if…

• 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

Where it is running

• University of Alabama — Birmingham, Alabama, United States
• Gillette Children's Specialty Healthcare — Saint Paul, Minnesota, United States
• Baylor School of Medicine — Houston, Texas, United States

Full record on ClinicalTrials.gov

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