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

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ClinicalTrials.gov Identifier: NCT03572933
Recruitment Status : Active, not recruiting
First Posted : June 28, 2018
Last Update Posted : August 18, 2020
Sponsor:
Information provided by (Responsible Party):
Marinus Pharmaceuticals

Study Description
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Brief 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.

Condition or disease Intervention/treatment Phase
CDKL5 Deficiency Disorder Drug: ganaxolone Drug: Placebo Phase 3

Detailed Description:
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.
Study Design
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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 102 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Intervention Model Description: The double-blind phase will randomize subjects to adjunctive ganaxolone or placebo at a 1:1 ratio to standard of care
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Official Title: A Double-blind, Randomized, Placebo-controlled Trial of Adjunctive Ganaxolone Treatment in Children and Young Adults With Cyclin-dependent Kinase-like 5 (CDKL5) Deficiency Disorder (CDD) Followed by Long-term Open-label Treatment
Actual Study Start Date : June 30, 2018
Actual Primary Completion Date : July 31, 2020
Estimated Study Completion Date : October 31, 2021

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Seizures

Arms and Interventions
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Arm Intervention/treatment
Experimental: Ganaxolone
ganaxolone suspension (50 mg/ml) 3x's /day for 17 weeks
 Drug: ganaxolone
active drug
Placebo Comparator: Placebo
placebo suspension 3x's /day for 17 weeks
 Drug: Placebo
inactive
Other Name: Placebo (for ganaxolone)


Outcome Measures
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Primary Outcome Measures :
  1. Percent change in 28-day seizure frequency [ Time Frame: End of the double-blind 17 week treatment period ]
    Percent change in 28-day seizure frequency during the double-blind treatment period relative to the 6-week prospective baseline period


Secondary Outcome Measures :
  1. Change in caregiver global impression of change in attention [ Time Frame: End of the double-blind 17 week treatment period ]
    Change in caregiver global impression of change in attention during the double-blind treatment period of ganaxolone compared to placebo

  2. Change in caregiver global impression of change in target behavior [ Time Frame: End of the double-blind 17 week treatment period ]
    Change in caregiver global impression of change in target behavior during the double-blind treatment period of ganaxolone compared to placebo

  3. Clinical Global Impression of Improvement [ Time Frame: End of the double-blind 17 week treatment period ]
    Clinical global impression of improvement during the double-blind treatment period of ganaxolone compared to placebo

  4. Arithmetic change in percent of seizure-free days of primary seizure types [ Time Frame: End of the double-blind 17 week treatment period ]
    Arithmetic change in percent of seizure-free days of primary seizure types during the double-blind treatment period of ganaxolone compared to placebo

  5. Arithmetic change in longest seizure free interval, based on primary seizure types [ Time Frame: End of the double-blind 17 week treatment period ]
    Arithmetic change in longest seizure free interval, based on primary seizure types during the double-blind treatment period of ganaxolone compared to placebo

  6. Caregiver global impression of change in seizure intensity and duration [ Time Frame: End of the double-blind 17 week treatment period ]
    Caregiver global impression of change in seizure intensity and duration during the double-blind treatment period of ganaxolone compared to placebo


Eligibility Criteria
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Information from the National Library of Medicine

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Ages Eligible for Study:   2 Years to 21 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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
Contacts and Locations
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Information from the National Library of Medicine

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): NCT03572933


Locations
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United States, Arizona
Marinus Research Site
Phoenix, Arizona, United States, 85016
United States, California
Marinus Research Site
Los Angeles, California, United States, 90095-1742
United States, Colorado
Marinus Research Site
Aurora, Colorado, United States, 80045
United States, Florida
Marinus Research Site
Gulf Breeze, Florida, United States, 32561
Marinus Research Site
Orlando, Florida, United States, 32819
United States, Georgia
Marinus Research Site
Norcross, Georgia, United States, 30093
United States, Illinois
Marinus Research Site
Chicago, Illinois, United States, 60612-3852
United States, Iowa
Marinus Research Site
Iowa City, Iowa, United States, 52242
United States, Massachusetts
Marinus Research Site
Boston, Massachusetts, United States, 02115
United States, Minnesota
Marinus Research Site
Rochester, Minnesota, United States, 55905
United States, Missouri
Marinus Research Site
Saint Louis, Missouri, United States, 63130
United States, New Jersey
Marinus Research Site
Livingston, New Jersey, United States, 07039
United States, Ohio
Marinus Research Site
Cleveland, Ohio, United States, 44195
United States, Pennsylvania
Marinus Research Site
Philadelphia, Pennsylvania, United States, 19104-4318
Marinus Research Site
Pittsburgh, Pennsylvania, United States, 15224
United States, Texas
Marinus Research Site
Fort Worth, Texas, United States, 76104
Marinus Research Site
Houston, Texas, United States, 77030
Australia, Queensland
Marinus Research Site
Brisbane, Queensland, Australia, 4101
Australia, Victoria
Marinus Research Site
Heidelberg, Victoria, Australia, 3084
Marinus Research Site
Melbourne, Victoria, Australia, 3168
France
Marinus Research Site
Paris, France
Israel
Marinus Research Site
Ramat Gan, Israel
Italy
Marinus Research Site
Firenze, Italy
Marinus Research Site
Milano, Italy
Marinus Research Site
Pavia, Italy
Marinus Research Site
Roma, Italy
Marinus Research Site
Verona, Italy
Poland
Marinus Research Site
Bydgoszcz, Poland
Marinus Research Site
Kraków, Poland
Russian Federation
Marinus Research Site
Moscow, Russian Federation
Marinus Research Site
Nizhniy Novgorod, Russian Federation
Marinus Research Site
Novosibirsk, Russian Federation
United Kingdom
Marinus Research Site
Birmingham, United Kingdom
Marinus Research Site
Bristol, United Kingdom
Marinus Research Facility
Glasgow, United Kingdom
Marinus Research Site
London, United Kingdom
Sponsors and Collaborators
Marinus Pharmaceuticals
Investigators
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Study Director: Joseph Hulihan, MD Marinus Pharmaceuticals, Inc.
More Information
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Responsible Party: Marinus Pharmaceuticals
ClinicalTrials.gov Identifier: NCT03572933    
Other Study ID Numbers: 1042-CDD-3001
2018-001180-23 ( EudraCT Number )
First Posted: June 28, 2018    Key Record Dates
Last Update Posted: August 18, 2020
Last Verified: August 2020

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Marinus Pharmaceuticals:
refractory seizures
genetic pediatric encephalopathies
epilepsy in children
seizure disorder
Additional relevant MeSH terms:
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Disease
Pathologic Processes
Pregnanolone
 Anesthetics
Central Nervous System Depressants
Physiological Effects of Drugs