Scholarlink

Purpose of Review <p>Ganaxolone (GNX) is a synthetic analog that acts as a positive allosteric modulator and potentiates the signal of gamma-aminobutyric acid inhibitory system. GNX was first discovered by Edward Monaghan in the 1990s, and its development soared in the early 2000s when Marinus Pharmaceuticals acquired CoSensys from Purdue Pharmaceuticals. GNX received its first approval in the US for the treatment of cyclin-dependent kinase-like 5 deficiency disorder (CDD) related seizure in 2022. Since then, GNX is under development for multiple conditions. Our review aims to identify the GNX trials on ClinicalTrials.gov and analyze its current indications, efficacy and safety profile.</p> Recent Findings <p>We queried the GNX trials from 2015 to 2024 using search engine ClinicalTrails.gov without a literature review. We then analyzed the indications, intervention, study type, phase of the trial, funding, sponsorship, outcomes and study period. We found 25 clinical trials through our search. In one trial, NCT 03572933, patients with CDD associated seizure in the GNX group experienced 23.8% more in median seizure reduction than the placebo group (<i>p</i> = 0.0036). Other trials, including Fragile X Syndrome and Protocadherin 19 (PCDH19) related epilepsy, showed mixed results. Tuberous sclerosis complex Phase 3 trial, NCT 05323734, did not meet primary endpoint of seizure frequency at 28-day (<i>p</i> = 0.09). Lennox-Gastaut syndrome trial with second generation GNX is as well in the horizon.</p> Summary <p>Current studies suggest that GNX is both safe and efficacious in the treatment of CDD-associated epilepsy. Other trials showed mixed results. Further research is necessary to expand its clinical applications.</p>

错误：搜索内容不能为空，请输入英文关键词
错误：关键词超出字数限制，请精简
高级检索

Ganaxolone for the Treatment of CDKL5 Disorder and Other Neurological and Psychiatric Conditions: A Review of the Clinical Trials

  • 期刊论文

  • 发表日期：

    2025年9月23日

查看全文
  • Sammie Lai,
  • Douglas R. Nordli III,
  • Fernando N. Galan

摘要

Purpose of Review

Ganaxolone (GNX) is a synthetic analog that acts as a positive allosteric modulator and potentiates the signal of gamma-aminobutyric acid inhibitory system. GNX was first discovered by Edward Monaghan in the 1990s, and its development soared in the early 2000s when Marinus Pharmaceuticals acquired CoSensys from Purdue Pharmaceuticals. GNX received its first approval in the US for the treatment of cyclin-dependent kinase-like 5 deficiency disorder (CDD) related seizure in 2022. Since then, GNX is under development for multiple conditions. Our review aims to identify the GNX trials on ClinicalTrials.gov and analyze its current indications, efficacy and safety profile.

Recent Findings

We queried the GNX trials from 2015 to 2024 using search engine ClinicalTrails.gov without a literature review. We then analyzed the indications, intervention, study type, phase of the trial, funding, sponsorship, outcomes and study period. We found 25 clinical trials through our search. In one trial, NCT 03572933, patients with CDD associated seizure in the GNX group experienced 23.8% more in median seizure reduction than the placebo group (p = 0.0036). Other trials, including Fragile X Syndrome and Protocadherin 19 (PCDH19) related epilepsy, showed mixed results. Tuberous sclerosis complex Phase 3 trial, NCT 05323734, did not meet primary endpoint of seizure frequency at 28-day (p = 0.09). Lennox-Gastaut syndrome trial with second generation GNX is as well in the horizon.

Summary

Current studies suggest that GNX is both safe and efficacious in the treatment of CDD-associated epilepsy. Other trials showed mixed results. Further research is necessary to expand its clinical applications.