Working...
ClinicalTrials.gov
ClinicalTrials.gov Menu

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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
ClinicalTrials.gov Identifier: NCT03572933
Recruitment Status : Recruiting
First Posted : June 28, 2018
Last Update Posted : June 12, 2019
Sponsor:
Information provided by (Responsible Party):
Marinus Pharmaceuticals

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.

Layout table for study information
Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 70 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
Estimated 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

Arm Intervention/treatment
Experimental: Ganaxolone
ganaxolone suspension (50 mg/ml) 3x's /day or ganaxolone 225 mg capsules 2x's/day for 17 weeks
Drug: ganaxolone
active drug

Placebo Comparator: Placebo
placebo suspension 3x's /day or placebo capsules 2x's/day for 17 weeks
Drug: Placebo
inactive
Other Name: Placebo (for ganaxolone)




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



Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


Layout table for eligibility information
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
  • 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.
  • Exposure to any other investigational drug within 30 days or fewer than 5 half-lives prior to screening

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


Contacts
Layout table for location contacts
Contact: Julia Tsai, PhD 203-215-7084 jtsai@marinuspharma.com
Contact: Rebekah Fries, BS 717-673-6231 rfries@marinuspharma.com

  Show 31 Study Locations
Sponsors and Collaborators
Marinus Pharmaceuticals
Investigators
Layout table for investigator information
Study Director: Lorianne Masuoka, MD Marinus Pharmaceuticals, Inc.

Layout table for additonal information
Responsible Party: Marinus Pharmaceuticals
ClinicalTrials.gov Identifier: NCT03572933     History of Changes
Other Study ID Numbers: 1042-CDD-3001
2018-001180-23 ( EudraCT Number )
First Posted: June 28, 2018    Key Record Dates
Last Update Posted: June 12, 2019
Last Verified: June 2019

Layout table for additional information
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:
Layout table for MeSH terms
Disease
Pathologic Processes
Pregnanolone
Anesthetics
Central Nervous System Depressants
Physiological Effects of Drugs