Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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COMPLETED
PHASE2
82 participants
INTERVENTIONAL
2016-03-31
2017-01-05
Brief Summary
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Detailed Description
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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.
Conditions
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Study Design
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RANDOMIZED
PARALLEL
TREATMENT
QUADRUPLE
Study Groups
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NNZ-2566
Glycyl-L-2-Methylpropyl-L-Glutamic Acid
NNZ-2566
Glycyl-L-2-Methylpropyl-L-Glutamic Acid (NNZ-2566) supplied as a lyophilized powder for reconstitution with strawberry flavored solution 0.5% v/v in Water for Injection.
Placebo (strawberry flavored solution)
Strawberry flavored solution and Water for Injection
Placebo
Strawberry flavored solution and Water for Injection
Interventions
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NNZ-2566
Glycyl-L-2-Methylpropyl-L-Glutamic Acid (NNZ-2566) supplied as a lyophilized powder for reconstitution with strawberry flavored solution 0.5% v/v in Water for Injection.
Placebo
Strawberry flavored solution and Water for Injection
Other Intervention Names
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Eligibility Criteria
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Inclusion Criteria
* 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
* 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.
5 Years
15 Years
FEMALE
No
Sponsors
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rettsyndrome.org
UNKNOWN
Neuren Pharmaceuticals Limited
INDUSTRY
Responsible Party
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Principal Investigators
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Daniel Glaze, MD
Role: PRINCIPAL_INVESTIGATOR
Baylor College of Medicine
Alan Percy, MD
Role: PRINCIPAL_INVESTIGATOR
University of Alabama at Birmingham
Timothy Feyma, MD
Role: PRINCIPAL_INVESTIGATOR
Gillette Children's Specialty Healthcare
Peter Heydemann, MD
Role: PRINCIPAL_INVESTIGATOR
Rush University Medical Center
Jeff Neul, MD
Role: PRINCIPAL_INVESTIGATOR
University of California, San Diego
Tim Benke, MD
Role: PRINCIPAL_INVESTIGATOR
Children's Hospital Colorado
Mary Jones, MD
Role: PRINCIPAL_INVESTIGATOR
UCSF Benioff Children's Hospital Oakland
Steve Skinner, MD
Role: PRINCIPAL_INVESTIGATOR
Greenwood Genetic Center
Mustafa Sahin, MD
Role: PRINCIPAL_INVESTIGATOR
Boston Children's Hospital
Sarika Peters, PhD
Role: PRINCIPAL_INVESTIGATOR
Vanderbilt University
Shannon Standridge
Role: PRINCIPAL_INVESTIGATOR
Children's Hospital Medical Center, Cincinnati
Eric Marsh, MD
Role: PRINCIPAL_INVESTIGATOR
Children's Hospital of Philadelphia
Locations
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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
Countries
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References
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Darwish M, Passarell J, Youakim JM, Bradley H, Bishop KM. Exposure-Response Efficacy Modeling to Support Trofinetide Dosing in Individuals with Rett Syndrome. Adv Ther. 2024 Apr;41(4):1462-1480. doi: 10.1007/s12325-024-02796-y. Epub 2024 Feb 16.
Parent H, Ferranti A, Niswender C. Trofinetide: a pioneering treatment for Rett syndrome. Trends Pharmacol Sci. 2023 Oct;44(10):740-741. doi: 10.1016/j.tips.2023.06.008. Epub 2023 Jul 16.
Other Identifiers
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Neu-2566-RETT-002
Identifier Type: -
Identifier Source: org_study_id
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