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
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Condition or disease | Intervention/treatment | Phase |
---|---|---|
Rett Syndrome | Drug: Tridecanoic Acid | Phase 2 |
Study Type : | Interventional (Clinical Trial) |
Estimated Enrollment : | 10 participants |
Allocation: | N/A |
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 |
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 |
- 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.
- 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.
- 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.
- 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.
- 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.
- 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.
- 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 |
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.
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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
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
Contact: Bruria Ben-Zeev, MD | 97235302687 | bruria.benzeev@sheba.health.gov.il | |
Contact: Andreea Nissenkorn, MD | 97235302687 | andreea.nissenkorn@sheba.health.gov.il |
Principal Investigator: | Bruria Ben-Zeev, MD | Head of pediatric neurology unit in Sheba medical center |
Responsible Party: | Prof. Bruria Ben-Zeev MD, Head of pediatric neurology unit, Sheba Medical Center |
ClinicalTrials.gov Identifier: | NCT03059160 |
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 |
Studies a U.S. FDA-regulated Drug Product: | No |
Studies a U.S. FDA-regulated Device Product: | No |
Rett syndrome, Triheptanoic acid, Walking, EEG |
Rett Syndrome 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 |