Study of Adjunctive Ganaxolone Treatment in Children and Young Adults With CDKL5 Deficiency Disorder

Study Results

Results available

Outcome measurements, participant flow, baseline characteristics, and adverse events have been published for this study.

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Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE3

Total Enrollment

101 participants

Study Classification

INTERVENTIONAL

Study Start Date

2018-06-30

Study Completion Date

2021-05-28

Brief Summary

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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.

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Detailed Description

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The Marigold Study is a global, double-blind, placebo-controlled, Phase 3 clinical trial that will enroll approximately 70 patients between the ages of 2 and 21 with a confirmed disease-related CDKL5 gene variant. Patients will undergo a baseline period before being randomized to receive, in addition to their existing anti-seizure treatment, either ganaxolone or placebo for 17 weeks. Following the treatment period, all patients that meet certain eligibility requirements will have the opportunity to receive ganaxolone in the open label phase of the study. The study's primary efficacy endpoint is percent reduction in seizures. Secondary outcome measures will include non-seizure-related endpoints to capture certain behavioral and sleep disturbances that have been seen in previous clinical studies with ganaxolone.

Conditions

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CDKL5 Deficiency Disorder

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

The double-blind phase will randomize subjects to adjunctive ganaxolone or placebo at a 1:1 ratio to standard of care

Primary Study Purpose

TREATMENT

Blinding Strategy

QUADRUPLE

Participants Caregivers Investigators Outcome Assessors

Study Groups

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Ganaxolone

ganaxolone suspension (50 mg/ml) 3x's /day for 17 weeks

Group Type EXPERIMENTAL

ganaxolone

Intervention Type DRUG

active drug

Placebo

placebo suspension 3x's /day for 17 weeks

Group Type PLACEBO_COMPARATOR

Placebo

Intervention Type DRUG

inactive

Interventions

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ganaxolone

active drug

Intervention Type DRUG

Placebo

inactive

Intervention Type DRUG

Other Intervention Names

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Placebo (for ganaxolone)

Eligibility Criteria

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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)

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

Minimum Eligible Age

2 Years

Maximum Eligible Age

21 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No