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 : November 13, 2020
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Condition or disease | Intervention/treatment | Phase |
---|---|---|
CDKL5 Deficiency Disorder | Drug: ganaxolone Drug: Placebo | Phase 3 |
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 |

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) |
- 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
- 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
- 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
- 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
- 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
- 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
- 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

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Ages Eligible for Study: | 2 Years to 21 Years (Child, Adult) |
Sexes Eligible for Study: | All |
Accepts Healthy Volunteers: | No |
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

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
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 |
Study Director: | Joseph Hulihan, MD | Marinus Pharmaceuticals, Inc. |
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: | November 13, 2020 |
Last Verified: | November 2020 |
Studies a U.S. FDA-regulated Drug Product: | Yes |
Studies a U.S. FDA-regulated Device Product: | No |
refractory seizures genetic pediatric encephalopathies epilepsy in children seizure disorder |
Disease Pathologic Processes Pregnanolone |
Anesthetics Central Nervous System Depressants Physiological Effects of Drugs |