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Can I read Phase 2, placebo-controlled clinical study of oral ganaxolone in PCDH19-clustering epilepsy on EtoBox?
Phase 2, placebo-controlled clinical study of oral ganaxolone in PCDH19-clustering epilepsy by Joseph Sullivan; Boudewijn Gunning; Muhammad Zafar; Renzo Guerrini; Jozef Gecz; Kristy L. Kolc; Yufan Zhao; Maciej Gasior; Alex A. Aimetti; Debopam Samanta is a Medicine article available to read on EtoBox.
What is Phase 2, placebo-controlled clinical study of oral ganaxolone in PCDH19-clustering epilepsy about?
Introduction Protocadherin-19 (PCDH19)-clustering epilepsy is a distinct developmental and epileptic encephalopathy characterized by early-onset seizures that are often treatment refractory. Caused by a mutation of the PCDH19 gene on the X chromosome, this rare epilepsy syndrome primarily affects females with seizure onset commonly in the first year of life. A global, randomized, double-blind, placebo-controlled, phase 2 trial was conducted to evaluate the efficacy, safety, and tolerability of ganaxolone compared with placebo as adjunctive therapy to a standard antiseizure medication regimen in patients with PCDH19-clustering epilepsy (VIOLET; NCT03865732). Methods Females aged 1–17 years with a molecularly confirmed pathogenic or likely pathogenic PCDH19 variant who were experiencing ≥12 seizures during a 12-week screening period were stratified by baseline allopregnanolone sulfate (Allo-S) levels (low: ≤2.5 ng/mL; high: >2.5 ng/mL) at screening and randomized 1:1 within each strata to receive ganaxolone (maximum daily dose of 63 mg/kg/day if ≤28 kg or 1800 mg/day if >28 kg) or matching placebo in addition to their standard antiseizure treatment for the 17-week double-blind phase.
Who reads Phase 2, placebo-controlled clinical study of oral ganaxolone in PCDH19-clustering epilepsy?
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- Author
- Joseph Sullivan; Boudewijn Gunning; Muhammad Zafar; Renzo Guerrini; Jozef Gecz; Kristy L. Kolc; Yufan Zhao; Maciej Gasior; Alex A. Aimetti; Debopam Samanta
- Publisher
- Elsevier BV
- Published
- 2023
- Language
- EN
- Field
- Medicine (Health Sciences)