Overview

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.