A Safety Study of NNZ-2566 in Pediatric Rett Syndrome

• Status: Completed
• Trial end date: 2017-01-05
• 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 children and adolescents.

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

Details

• Lead Sponsor: Neuren Pharmaceuticals Limited
• Collaborator: rettsyndrome.org
• Treatments: Pharmaceutical Solutions

Criteria

Inclusion Criteria:

• Diagnosis of classic/typical Rett syndrome with a documented mutation of the MeCP2 gene.

• Age 5 - 15 years.

• Weight at Screening and Baseline between 15.0 kg-100.0 kg (at least 15.0 kg and no greater than 100.0 kg).

• Each subject must be able to swallow the study medication provided as a liquid solution, or via gastrostomy tube.

Exclusion Criteria:

• Actively undergoing neurological regression

• Abnormal QT interval, prolongation or significant cardiovascular history.

• Current treatment with insulin.

• Anti-convulsants with liver enzyme inducing effects.

• Unstable seizure profile.

• Excluded concomitant medications.

• Current clinically significant (as determined by the investigator). cardiovascular, renal, hepatic, or respiratory disease.

• Gastrointestinal disease which may interfere with the absorption, distribution, metabolism or excretion of the study medication.

• History of, or current cerebrovascular disease or brain trauma.

• History of, or current clinically significant endocrine disorder, e.g. hypo- or hyperthyroidism, or diabetes mellitus.

• History of, or current, malignancy.

• Significant hearing and/or visual impairments that may affect ability to complete the test procedures.

• Allergy to strawberry.