Voronistat in Pediatric Patients With Drug Resistant Epilepsy
NCT ID: NCT03894826
Last Updated: 2019-03-29
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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UNKNOWN
PHASE2
12 participants
INTERVENTIONAL
2018-12-10
2020-10-31
Brief Summary
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Detailed Description
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It is it is anticipated that Vorinostat in a population of pediatric patients with epilepsy will be well-tolerated, similar to that seen with valproic acid and other normally prescribed anti-epileptic drugs.
Participants consenting to participate will enter a 4-week screening period to ensure eligibility. Eligible participants will then enter the 6-week treatment phase. Participants will be seen for a final Safety Follow-up visit 6 weeks after end of treatment. Participants who are enrolled and complete all study procedures will be in the study for a total of 16 weeks.
Conditions
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Keywords
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Study Design
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NA
SINGLE_GROUP
TREATMENT
NONE
Study Groups
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TREATMENT
Participants will be administered 230 mg/m2/day of oral Vorinostat \[100 mg tablets\] in addition to standard of care anti-seizure medication for a duration of 6 weeks.
Vorinostat 100 MG
Vorinostat administered by mouth, once daily at a dose of 230 mg/m2/day for a total of 6 weeks
Interventions
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Vorinostat 100 MG
Vorinostat administered by mouth, once daily at a dose of 230 mg/m2/day for a total of 6 weeks
Other Intervention Names
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Eligibility Criteria
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Inclusion Criteria
2. Medically intractable epilepsy, defined as having failed at least 2 standard anti-seizures therapies and experiencing at least 3 motor seizures per week, separated by at least 24 hours that are quantifiable by observation (e.g. discrete episodes of motor activity). Participants experiencing other seizure types in addition to motor seizures may also be enrolled but must meet the minimal requirement for motor seizures.
3. Ability and willingness of family and/or caregiver (when appropriate) to give written informed consent and to comply with requirement of the study
4. Adequate bone marrow function (defined as an absolute neutrophil count (ANC) of \> 2 x 109/L; platelet count of \> 150 x 109/L; hemoglobin of \> 110 g/L \[3-11 years\], \> 120 g/L \[females 12 years or over\], \> 125 g/L \[males 12-14 years\], \> 137 g/L \[males 15 years or older\])
5. Adequate renal function (defined as serum creatinine \< 1.5X age-adjusted upper limit of normal \[ULN\], or glomerular filtration rate ≥ 70 mL/min/1.73 m2)
6. Adequate hepatic function (defined as total bilirubin \<1.5 times ULN, and alanine aminotransferase \[ALT\] and aspartate transaminase \[AST\] \< 3 times ULN, and albumin \>33 g/L)
7. Corrected QT (QTc) interval of \< 450 msec
8. Prothrombin time (PTT) \< 1.5 ULN/International Normalized Ratio (INR) \< 1.5 ULN
9. Participants on corticosteroids must be taking a stable or decreasing dose for at least 7 days prior to enrollment
Exclusion Criteria
2. Enzyme-inducing AEDs (including oxcarbazepine (Trileptal), phenobarbital, phenytoin (Dilantin), topiramate (Topamax)
3. Coumarin-derivative anti-coagulants
4. Participants being considered for surgery for management of seizures during screening or who will be receiving surgery during for management of seizures during study period (includes all neurosurgery for the management of seizures or device implantation for the management of seizures)
5. Neurosurgery within the past 12 months
6. Use of Vagus Nerve Stimulator (VNS) where settings have not been stable for at least 6 months
7. Planned surgery or other invasive medical treatment during screening of during treatment period
8. Hypokalemia or hypomagnesemia
9. Participants starting or currently on any neurometabolic diet (including but not limited to ketogenic diet; medium-chain triglyceride diet; modified Atkins diet; low glycemic index diet) during study
10. History of non-catheter related deep venous thrombosis
11. Pleural effusion
12. Malignancy within the past 5 years.
13. Any serious medical condition that according to the investigator could interfere with the conduct of the study
14. Serious comorbid disease in which the life expectancy of the patient is shorter than the duration of the trial
15. Unwillingness or inability to comply with study requirements
16. Positive pregnancy test, lactating females or heterosexually active participants not willing to use highly effective methods of contraception
17. Participation in any clinical trial with an investigational drug, or therapy not approved by Health Canada, within one month prior to screening
2 Years
17 Years
ALL
No
Sponsors
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University of Calgary
OTHER
Responsible Party
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Principal Investigators
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Jong Rho, MD
Role: PRINCIPAL_INVESTIGATOR
University of Calgary
Locations
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Alberta Children's Hospital
Calgary, Alberta, Canada
Countries
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Central Contacts
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Facility Contacts
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Jong Rho, MD
Role: primary
Sabrina D'Alfonso, MSc
Role: backup
References
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Ibhazehiebo K, Gavrilovici C, de la Hoz CL, Ma SC, Rehak R, Kaushik G, Meza Santoscoy PL, Scott L, Nath N, Kim DY, Rho JM, Kurrasch DM. A novel metabolism-based phenotypic drug discovery platform in zebrafish uncovers HDACs 1 and 3 as a potential combined anti-seizure drug target. Brain. 2018 Mar 1;141(3):744-761. doi: 10.1093/brain/awx364.
Garcia AA, Koperniku A, Ferreira JCB, Mochly-Rosen D. Treatment strategies for glucose-6-phosphate dehydrogenase deficiency: past and future perspectives. Trends Pharmacol Sci. 2021 Oct;42(10):829-844. doi: 10.1016/j.tips.2021.07.002. Epub 2021 Aug 10.
Provided Documents
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Document Type: Study Protocol and Statistical Analysis Plan
Other Identifiers
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VOR-17-2471
Identifier Type: -
Identifier Source: org_study_id