A Safety Study of NNZ-2566 in Pediatric 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 children and adolescents.

Key facts

Study ID

NCT02715115

Run by

Neuren Pharmaceuticals Limited

People needed

82

Starts

2016-03-01

Expected to finish

2017-01-05

Last updated by the study team

2020-08-14

Who can join

Age: 5 and older, up to 15. Sex: female. Healthy volunteers: not accepted.

You may qualify if…

• 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.

You may not qualify if…

• 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.

Where it is running

• University of Alabama — Birmingham, Alabama, United States
• UCSF Benioff Children's Hospital Oakland — Oakland, California, United States
• University of California, San Diego — San Diego, California, United States
• Children's Hosptial Colorado — Aurora, Colorado, United States
• Rush University Medical Center — Chicago, Illinois, United States
• Boston Children's Hospital — Boston, Massachusetts, United States
• Gillette Children's Specialty Healthcare — Saint Paul, Minnesota, United States
• Cincinnati Children's Hospital Medical Center — Cincinnati, Ohio, United States
• Children's Hospital of Philadelphia — Philadelphia, Pennsylvania, United States
• Greenwood Genetic Center — Greenwood, South Carolina, United States
• Vanderbilt University — Nashville, Tennessee, United States
• Baylor College of Medicine — Houston, Texas, United States

Full record on ClinicalTrials.gov

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