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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.
Register: ClinicalTrials.gov
Last refreshed on: 16 December 2017
Main ID:  NCT03059160
Date of registration: 02/02/2017
Prospective Registration: Yes
Primary sponsor: Sheba Medical Center
Public title: Open Label Trial of Triheptanoin (UX007) in Treatment of Rett Syndrome. UX007
Scientific title: Open Label Trial of Triheptanoin (UX007) in Treatment of Rett Syndrome.
Date of first enrolment: April 1, 2017
Target sample size: 10
Recruitment status: Not yet recruiting
URL:  https://clinicaltrials.gov/show/NCT03059160
Study type:  Interventional
Study design:  Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).  
Phase:  Phase 2
Countries of recruitment
Contacts
Name:     Bruria Ben-Zeev, MD
Address: 
Telephone: 97235302687
Email: bruria.benzeev@sheba.health.gov.il
Affiliation: 
Name:     Bruria Ben-Zeev, MD
Address: 
Telephone:
Email:
Affiliation:  Head of pediatric neurology unit in Sheba medical center
Key inclusion & exclusion criteria

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
Health Condition(s) or Problem(s) studied
Rett Syndrome
Intervention(s)
Drug: Tridecanoic Acid
Primary Outcome(s)
The number of patients with adverse events. [Time Frame: 30 weeks]
The number of patients with changes in BMI [Time Frame: 30 weeks]
The number of patients with changes in laboratory examination including hematology, biochemistry and endocrinological measurements [Time Frame: 30 weeks]
The number of patients with changes in physical examination. [Time Frame: 30 weeks]
The number of patients with changes in vital signs. [Time Frame: 30 weeks]
The number of patients with ECG changes [Time Frame: 30 weeks]
Secondary Outcome(s)
Change in seizure frequency during treatment with triheptanoin in Rett syndrome [Time Frame: 30 weeks]
Secondary ID(s)
3027-16-SMC
Source(s) of Monetary Support
Please refer to primary and secondary sponsors
Secondary Sponsor(s)
Ultragenyx Pharmaceutical Inc
Ethics review
Results
Results available:
Date Posted:
Date Completed:
URL:
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