Phase 3
N=101
Study of Adjunctive Ganaxolone Treatment in Children and Young Adults With CDKL5 Deficiency Disorder
CDKL5 Deficiency Disorder
Bottom Line
View on ClinicalTrials.gov: NCT03572933 ↗Enrolled (actual)
101
Serious AEs
10.9%
Results posted
Jul 2022
Primary outcome: Primary: Summary of 28-day Seizure Frequency for Major Motor Seizure Types — 49.17; 54.00; 55.50; 45.03 Seizures per day
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 3
- Interventions
- ganaxolone (Drug); Placebo (Drug)
- Age
- Pediatric, Adult · 2+ yrs
- Sex
- All
- Sponsor
- Marinus Pharmaceuticals
- Primary completion
- Jul 2020
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Summary of 28-day Seizure Frequency for Major Motor Seizure Types |
49.17; 54.00; 55.50; 45.03 | — |
| SECONDARY Caregiver Global Impression of Change in Attention |
1; 1; 7; 2; 14; 21 | — |
| SECONDARY Caregiver Global Impression of Change in Target Behavior |
0; 0; 6; 4; 14; 20 | — |
| SECONDARY Clinical Global Impression of Improvement - Parent/Caregiver |
1; 0; 7; 13; 13; 17 | — |
| SECONDARY Clinical Global Impression of Improvement - Clinician |
0; 0; 7; 7; 13; 19 | — |
| SECONDARY Percentage of Seizure-free Days for Major Motor Seizure Types |
30.32; 22.57; 36.17; 32.29; 5.86; 9.62 | — |
| SECONDARY Arithmetic Change in Longest Seizure Free Interval, Based on Primary Seizure Types |
-4.63; -0.02 | — |
| SECONDARY Caregiver Global Impression of Change in Seizure Intensity and Duration |
1; 2; 5; 15; 11; 11 | — |
Summary
A clinical study to evaluate the efficacy, safety, and tolerability of adjunctive ganaxolone therapy compared to placebo for the treatment of seizures in children and young adults with genetically confirmed CDKL5 gene mutation.
Eligibility Criteria
Inclusion Criteria
- Genetically confirmed CDKL5 gene mutation, seizure onset by 1 year of age and lack of independent ambulation by 2 years of age
- Failure to control seizures despite 2 or more anti-seizure medications
- At least 16 seizures per 28 days of primary seizure types
- On a stable regimen of 0-4 anti-seizure medications (Vagus nerve stimulator, ketogenic diet, and modified Atkins diet do not count towards this limit)
- Additional Inclusion Criteria apply and can be discussed with study team
Exclusion Criteria
- Previous exposure to ganaxolone
- West Syndrome with hypsarrhythmia pattern on EEG or seizures predominantly of Infantile Spasms type
- Use of adrenocorticotropic hormone (ACTH), prednisone or other glucocorticoid or use of moderate or strong inducers or inhibitors of CYP3A4/5/7 are prohibited
- Use of tetrahydrocannabinol (THC) or cannabidiol (CBD) is prohibited during the double-blind phase, unless patient has a prescription of Epidiolex®
- Exposure to any other investigational drug within 30 days or fewer than 5 half-lives prior to screening
- Plasma allopregnanolone-sulfate (Allo-S) levels greater than or equal to 6.0 ng/ml at screening visit
- Additional Exclusion Criteria apply and can be discussed with study team
Data sourced from ClinicalTrials.gov (NCT03572933). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.