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

• Status: Completed
• Trial end date: 2014-09-01
• Target enrollment: 0
• Participant gender: Female

Summary

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.

• Phase: Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Neuren Pharmaceuticals Limited
• Collaborators: Baylor College of Medicine; International Rett Syndrome Foundation; Texas Children's Hospital
• Treatments: Pharmaceutical Solutions

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