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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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16 December 2017 |
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Main ID: |
NCT03059160 |
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Date of registration:
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02/02/2017 |
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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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Open Label Trial of Triheptanoin (UX007) in Treatment of Rett Syndrome.
UX007 |
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Scientific title:
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Open Label Trial of Triheptanoin (UX007) in Treatment of Rett Syndrome. |
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Date of first enrolment:
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April 1, 2017 |
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Target sample size:
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10 |
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Recruitment status: |
Not yet recruiting |
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URL:
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https://clinicaltrials.gov/show/NCT03059160 |
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Study type:
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Interventional |
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Study design:
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Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
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Phase:
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Phase 2
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Contacts
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Name:
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Bruria Ben-Zeev, MD |
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Address:
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Telephone:
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97235302687 |
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Email:
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bruria.benzeev@sheba.health.gov.il |
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Affiliation:
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Name:
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Bruria Ben-Zeev, 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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Head of pediatric neurology unit in Sheba medical center |
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Key inclusion & exclusion criteria
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Inclusion Criteria:
- Female patients aged 5 to18 years (inclusive).
- A classical diagnosis of RTT, defined according to the internationally agreed 2010
Rett Search criteria, and with MECP2 pathogenic mutation.
- Patients with one or both of the following:
- At least 2 seizures per month as per history during the four-week baseline period
according to parent diary or per 3 hours video EEG recording
- Walking abilities, independent or with support
- Patients with breathing abnormalities as recorded by baseline NOX recording.
Exclusion Criteria:
- Patients with significant metabolic, liver, cardiac, or respiratory morbidity not
related to RTT
- Patients with significant liver, cardiac or respiratory morbidity related to RTT
Age minimum:
5 Years
Age maximum:
18 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: Tridecanoic Acid
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Primary Outcome(s)
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The number of patients with adverse events.
[Time Frame: 30 weeks]
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The number of patients with ECG changes
[Time Frame: 30 weeks]
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The number of patients with changes in physical examination.
[Time Frame: 30 weeks]
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The number of patients with changes in BMI
[Time Frame: 30 weeks]
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The number of patients with changes in laboratory examination including hematology, biochemistry and endocrinological measurements
[Time Frame: 30 weeks]
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The number of patients with changes in vital signs.
[Time Frame: 30 weeks]
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Secondary Outcome(s)
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Change in seizure frequency during treatment with triheptanoin in Rett syndrome
[Time Frame: 30 weeks]
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Secondary ID(s)
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3027-16-SMC
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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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