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Main
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Note: This record shows only 22 elements of the WHO Trial Registration Data Set. To view changes that have been made to the source record, or for additional information about this trial, click on the URL below to go to the source record in the primary register. |
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Register:
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ClinicalTrials.gov |
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Last refreshed on:
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12 December 2020 |
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Main ID: |
NCT02715115 |
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Date of registration:
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21/02/2016 |
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Prospective Registration:
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Yes |
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Primary sponsor: |
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Public title:
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A Safety Study of NNZ-2566 in Pediatric Rett Syndrome
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Scientific title:
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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 |
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Date of first enrolment:
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March 2016 |
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Target sample size:
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82 |
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Recruitment status: |
Completed |
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URL:
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https://clinicaltrials.gov/show/NCT02715115 |
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Study type:
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Interventional |
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Study design:
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Allocation: Randomized. Intervention model: Parallel Assignment. Primary purpose: Treatment. Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor).
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Phase:
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Phase 2
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Countries of recruitment
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United States
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Contacts
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Name:
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Shannon Standridge |
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Address:
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Telephone:
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Email:
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Affiliation:
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Children's Hospital Medical Center, Cincinnati |
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Name:
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Peter Heydemann, MD |
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Address:
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Affiliation:
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Rush University Medical Center |
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Name:
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Sarika Peters, PhD |
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Address:
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Email:
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Affiliation:
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Vanderbilt University |
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Name:
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Steve Skinner, MD |
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Address:
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Telephone:
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Email:
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Affiliation:
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Greenwood Genetic Center |
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Name:
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Mary Jones, MD |
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Address:
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Email:
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Affiliation:
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UCSF Benioff Children's Hospital Oakland |
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Name:
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Alan Percy, MD |
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Address:
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Affiliation:
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University of Alabama at Birmingham |
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Name:
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Timothy Feyma, MD |
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Address:
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Affiliation:
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Gillette Children's Specialty Healthcare |
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Name:
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Daniel Glaze, MD |
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Affiliation:
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Baylor College of Medicine |
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Name:
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Eric Marsh, MD |
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Address:
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Affiliation:
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Children's Hospital of Philadelphia |
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Name:
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Jeff Neul, MD |
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Address:
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Affiliation:
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University of California, San Diego |
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Name:
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Mustafa Sahin, MD |
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Address:
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Affiliation:
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Boston Children's Hospital |
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Name:
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Tim Benke, MD |
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Affiliation:
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Children's Hospital Colorado |
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Key inclusion & exclusion criteria
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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.
Age minimum:
5 Years
Age maximum:
15 Years
Gender:
Female
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Health Condition(s) or Problem(s) studied
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Rett Syndrome
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Intervention(s)
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Drug: Placebo
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Drug: NNZ-2566
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Primary Outcome(s)
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Adverse events
[Time Frame: Through study completion, an average of 11 weeks]
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Secondary Outcome(s)
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Motor Behaviour Assessment Scale (MBA)
[Time Frame: Through study completion, an average of 11 weeks]
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Caregiver Top 3 Concerns via a Visual Analogue Scale (VAS)
[Time Frame: Through study completion, an average of 11 weeks]
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Clinical Global Impression of Improvement (CGI-I)
[Time Frame: Through study completion, an average of 11 weeks]
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Secondary ID(s)
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Neu-2566-RETT-002
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Source(s) of Monetary Support
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Please refer to primary and secondary sponsors
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Results
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Results available:
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Date Posted:
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Date Completed:
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URL:
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