A Safety Study of NNZ-2566 in Pediatric Rett Syndrome



Status:Completed
Conditions:Other Indications, Neurology
Therapuetic Areas:Neurology, Other
Healthy:No
Age Range:5 - 15
Updated:2/2/2018
Start Date:March 2016
End Date:January 2017

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A Randomized, Double-blind, Placebo-controlled, Dose-ranging Study of the Safety and Pharmacokinetics of Oral NNZ-2566 in Pediatric Rett Syndrome

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.

Rett syndrome is a neurodevelopmental disorder primarily affecting females. The disorder is
characterized by apparent normal development in early infancy (6-18 months), followed by a
period of regression with onset of systemic and neurological signs. The CNS symptoms of Rett
syndrome include learning disability, autism symptomatology and epilepsy and these can be
severe and highly debilitating. Affected individuals also show signs of autonomic
dysfunction, reflected in cardiovascular and respiratory abnormalities. There is no currently
effective treatment for Rett syndrome.

This study will investigate the safety, tolerability and blood pharmacokinetics of treatment
with oral administration of NNZ-2566 at 50 mg/kg, 100 mg/kg, 200 mg/kg BID, or placebo BID,
in children and adolescent females with Rett syndrome. The study also will also investigate
measures of efficacy and biomarkers during treatment.

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.
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San Diego, California 92093
Principal Investigator: Jeffrey Neul, MD
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3333 Burnet Avenue # Mlc3008
Cincinnati, Ohio 45229
 1-513-636-4200 
Cincinnati Children's Hospital Medical Center Patients and families from across the region and around the...
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1200 Moursund Street
Houston, Texas 77030
(713) 798-4951
Principal Investigator: Daniel Glaze, MD
Phone: 832-822-3751
Baylor College of Medicine Baylor College of Medicine in Houston, the only private medical school...
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South 34th Street
Philadelphia, Pennsylvania 19104
 215-590-1000
Principal Investigator: Eric Marsh, MD
Phone: 215-590-3057
Children's Hospital of Philadelphia Since its start in 1855 as the nation's first hospital devoted...
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Aurora, Colorado 80045
Principal Investigator: Tim Benke, MD
Phone: 720-777-8008
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Birmingham, Alabama 35294
Principal Investigator: Alan Percy, MD
Phone: 205-934-1130
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300 Longwood Ave
Boston, Massachusetts 02115
(617) 355-6000
Principal Investigator: Mustafa Sahin, MD
Phone: 617-355-5230
Boston Children's Hospital Boston Children's Hospital is a 395-bed comprehensive center for pediatric health care....
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1653 W. Congress Parkway
Chicago, Illinois 60612
(312) 942-5000
Principal Investigator: Peter Heydemann, MD
Phone: 312-563-6636
Rush University Medical Center Rush University Medical Center encompasses a 664-bed hospital serving adults and...
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Greenwood, South Carolina 29646
Principal Investigator: Steve Skinner, MD
Phone: 864-941-8100
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2201 West End Ave
Nashville, Tennessee 37232
(615) 322-7311
Principal Investigator: Sarika Peters, PhD
Phone: 615-875-9311
Vanderbilt University Vanderbilt offers undergraduate programs in the liberal arts and sciences, engineering, music, education...
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Oakland, California 94609
Principal Investigator: Mary Jones, MD
Phone: 925-979-4055
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Saint Paul, Minnesota 55101
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