Voronistat in Pediatric Patients With Drug Resistant Epilepsy
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|ClinicalTrials.gov Identifier: NCT03894826|
Recruitment Status : Recruiting
First Posted : March 29, 2019
Last Update Posted : March 29, 2019
|Condition or disease||Intervention/treatment||Phase|
|Refractory Epilepsy||Drug: Vorinostat 100 MG||Phase 2|
Vorinostat is a potent inhibitor of histone deacetylases (HDAC). HDACs catalyze the removal of acetyl groups from the lysine residues of proteins, including histones and transcription factors. Valproic acid, a broad anti-epileptic drug, commonly used as first line treatment in epilepsy, has also been shown to inhibit HDAC activity .
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.
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||12 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||A Phase 2 Clinical Trial Testing the Safety and Efficacy of Voronistat in Pediatric Patients With Drug Resistant Epilepsy|
|Actual Study Start Date :||December 10, 2018|
|Estimated Primary Completion Date :||April 2020|
|Estimated Study Completion Date :||October 2020|
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.
Drug: Vorinostat 100 MG
Vorinostat administered by mouth, once daily at a dose of 230 mg/m2/day for a total of 6 weeks
Other Name: ZOLINZA
- Incidence of Treatment-Emergent Adverse Events [ Time Frame: 14 days post drug initiation; 30 days post drug initiation; 42 days post drug initiation; 42 days following drug discontinuation ]The number of treatment emergent adverse events will be tabulated at each time point: adverse events, serious adverse events, and discontinuations due to adverse events.
- Incidence of Drug Discontinuations due to Adverse Drug Reaction [ Time Frame: 14 days post drug initiation; 30 days post drug initiation; 42 days post drug initiation; 42 days following drug discontinuation ]The number of participants with drug discontinuations due to Adverse Drug Reaction will be tabulated at each time point: adverse events, serious adverse events, and discontinuations due to adverse events.
- Proportion of participants who have at least a 50% reduction in seizures from baseline [ Time Frame: 42 days post drug initiation; change from baseline ]Treatment response will be evaluated by calculating the proportion of response of participants who demonstrate a 50% reduction rate or greater at 6 weeks as compared to baseline.
- Change in seizure status at each time point. [ Time Frame: 14 days post drug initiation; 30 days post drug initiation; 42 days post drug initiation; 42 days following drug discontinuation ]Seizure status will be tabulated 14 days following drug initiation; 30 days following drug initiation; 42 days following drug initiation; and 42 days following drug discontinuation. Seizure status will be defined as: significant response (at least 50% reduction in seizure frequency as compared to baseline, or no seizures while on medication; partial response (25% - 49% reduction in seizure frequency as compared to baseline); no change, (0 - 24% reduction in seizure frequency as compared to baseline); and worsening (increase in seizure frequency as compared to baseline).
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): NCT03894826
|Contact: Sabrina D'Alfonso, MScfirstname.lastname@example.org|
|Contact: Jong Rho, MDemail@example.com|
|Alberta Children's Hospital||Recruiting|
|Calgary, Alberta, Canada, T3B6A8|
|Contact: Jong Rho, MD 403-955-2296 firstname.lastname@example.org|
|Contact: Sabrina D'Alfonso, MSc 403-955-2745 email@example.com|
|Principal Investigator: Jong Rho, MD|
|Sub-Investigator: Luis Bello-Espinosa, MD|
|Sub-Investigator: Juan-Pablo Appendino, MD|
|Sub-Investigator: Deborah Kurrasch, PhD|
|Principal Investigator:||Jong Rho, MD||University of Calgary|