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Open Label Trial of Triheptanoin (UX007) in Treatment of Rett Syndrome. (UX007)

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ClinicalTrials.gov Identifier: NCT03059160
Recruitment Status : Unknown
Verified February 2017 by Prof. Bruria Ben-Zeev MD, Sheba Medical Center.
Recruitment status was:  Not yet recruiting
First Posted : February 23, 2017
Last Update Posted : February 23, 2017
Sponsor:
Collaborator:
Ultragenyx Pharmaceutical Inc
Information provided by (Responsible Party):
Prof. Bruria Ben-Zeev MD, Sheba Medical Center

Brief Summary:
This is a single-center, exploratory, open-label study in 10 girls diagnosed with Rett Syndrome. The study will consist of the following 4 parts: Screening/Baseline run-in, Titration/Dose-Setting, Treatment, and Washout/Follow-up.

Condition or disease Intervention/treatment Phase
Rett Syndrome Drug: Tridecanoic Acid Phase 2

  Show Detailed Description

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 10 participants
Intervention Model: Single Group Assignment
Intervention Model Description: open label study with one doze
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Open Label Trial of Triheptanoin (UX007) in Treatment of Rett Syndrome.
Estimated Study Start Date : April 1, 2017
Estimated Primary Completion Date : April 1, 2018
Estimated Study Completion Date : August 1, 2018

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Rett Syndrome

Arm Intervention/treatment
Experimental: open label Drug: Tridecanoic Acid
3 times daily oral doze of the drug for 20 weeks with 4 weeks of baseline and 4 weeks of washout
Other Name: triheptanoin




Primary Outcome Measures :
  1. The number of patients with adverse events. [ Time Frame: 30 weeks ]
    The number of patients which will show adverse events during the screening, treatment and washout periods.

  2. The number of patients with ECG changes [ Time Frame: 30 weeks ]
    The number of patients that show ECG changes and the type of change during the screening, treatment and washout periods.

  3. The number of patients with changes in vital signs. [ Time Frame: 30 weeks ]
    The number of patients which will show change in vital signs during the screening, treatment and washout periods including bloodpressure, heart rate, respiration rate and body temperature.

  4. The number of patients with changes in physical examination. [ Time Frame: 30 weeks ]
    The number of patients who show a change in their physical examination during the screening, treatment and washout periods including height, neurological findings, change in size of liver and spleen , skin changes.

  5. The number of patients with changes in BMI [ Time Frame: 30 weeks ]
    The number of patients who will show change in BMI and its direction during the screening, treatment and washout periods.

  6. The number of patients with changes in laboratory examination including hematology, biochemistry and endocrinological measurements [ Time Frame: 30 weeks ]
    The number of patients who will show changes in laboratory examination including hematology, biochemistry and endocrinological measurements during the screening, treatment and washout periods.


Secondary Outcome Measures :
  1. Change in seizure frequency during treatment with triheptanoin in Rett syndrome [ Time Frame: 30 weeks ]
    The change in number of seizures in each patient from the base line period to the treatment period and washout period



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Ages Eligible for Study:   5 Years to 18 Years   (Child, Adult)
Sexes Eligible for Study:   Female
Gender Based Eligibility:   Yes
Accepts Healthy Volunteers:   No
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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03059160


Contacts
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Contact: Bruria Ben-Zeev, MD 97235302687 bruria.benzeev@sheba.health.gov.il
Contact: Andreea Nissenkorn, MD 97235302687 andreea.nissenkorn@sheba.health.gov.il

Sponsors and Collaborators
Sheba Medical Center
Ultragenyx Pharmaceutical Inc
Investigators
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Principal Investigator: Bruria Ben-Zeev, MD Head of pediatric neurology unit in Sheba medical center

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Responsible Party: Prof. Bruria Ben-Zeev MD, Head of pediatric neurology unit, Sheba Medical Center
ClinicalTrials.gov Identifier: NCT03059160     History of Changes
Other Study ID Numbers: 3027-16-SMC
First Posted: February 23, 2017    Key Record Dates
Last Update Posted: February 23, 2017
Last Verified: February 2017
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Yes
Plan Description: the final results of the study will be shared with ultragenix pharmaceutical and with other RETT syndrome researchers

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No

Keywords provided by Prof. Bruria Ben-Zeev MD, Sheba Medical Center:
Rett syndrome, Triheptanoic acid, Walking, EEG

Additional relevant MeSH terms:
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Syndrome
Rett Syndrome
Disease
Pathologic Processes
Mental Retardation, X-Linked
Intellectual Disability
Neurobehavioral Manifestations
Neurologic Manifestations
Nervous System Diseases
Genetic Diseases, X-Linked
Genetic Diseases, Inborn
Heredodegenerative Disorders, Nervous System