Overview
A Safety Study of NNZ-2566 in Pediatric Rett Syndrome
Status:
Completed
Completed
Trial end date:
2017-01-05
2017-01-05
Target enrollment:
0
0
Participant gender:
Female
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 2Accepts Healthy Volunteers?
NoDetails
Lead Sponsor:
Neuren Pharmaceuticals LimitedCollaborator:
rettsyndrome.orgTreatments:
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.