Status:
COMPLETED
Adjunctive Ganaxolone Treatment (Part A) in TSC Followed by Long-term Treatment (Part B)
Lead Sponsor:
Marinus Pharmaceuticals
Conditions:
Tuberous Sclerosis
Eligibility:
All Genders
2-65 years
Phase:
PHASE2
Brief Summary
To assess preliminary safety and efficacy of ganaxolone as adjunctive therapy for the treatment of primary seizure types in patients with genetically- or clinically-confirmed TSC-related epilepsy thro...
Detailed Description
This is an OL proof of concept study of adjunctive GNX treatment in patients with a confirmed clinical diagnosis of TSC and/or a mutation in either the TSC1 or TSC2 gene. The trial consists of two par...
Eligibility Criteria
Inclusion
- Inclusion Criteria (Part A):
- Clinical or mutational diagnosis of TSC
- Failure to control seizures despite appropriate trial of 2 or more ASMs at therapeutic doses.
- Have at least 8 countable/witnessed primary seizures during the 4-week baseline period with at least 1 primary seizure occurring in at least 3 of the 4 weeks of baseline.
- Inclusion Criteria (Part B)
- • Patients have experienced ≥ 35% reduction in primary seizure frequency during the Part A treatment period compared to the 4-week Baseline Period.
- Exclusion Criteria (Part A):
- Previous exposure to GNX
- Pregnant or breastfeeding
- Concurrent use of strong inducers or inhibitors of cytochrome P450 (CYP)3A4/5/7. Any strong inhibitor or inducer of CYP3A4/5/7 must be discontinued at least 28 days before Visit 2, study drug initiation. This does not include approved ASMs.
- Patients who have been taking felbamate for less than 1 year prior to screening
- Patients who test positive for tetrahydrocannabinol (THC) or non-approved cannabidiol (CBD) via plasma drug screen
- Chronic use of oral steroid medications, ketoconazole (except for topical formulations), St. John's Wort, or other IPs is not permitted
- Have an active CNS infection, demyelinating disease, degenerative neurological disease, or CNS disease deemed progressive. This includes tumor growth which in the opinion of the investigator could affect primary seizure control
- Patients with significant renal insufficiency, estimated glomerular filtration rate (eGFR) \< 30 mL/min (calculated using the Cockcroft-Gault formula or Pediatric GFR calculator or Bedside Schwartz), will be excluded from study entry or will be discontinued if the criterion is met post baseline
- Have been exposed to any other investigational drug within 30 days or fewer than 5 half lives (whichever is shorter) prior to the screening visit
Exclusion
Key Trial Info
Start Date :
April 8 2020
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
August 30 2022
Estimated Enrollment :
23 Patients enrolled
Trial Details
Trial ID
NCT04285346
Start Date
April 8 2020
End Date
August 30 2022
Last Update
April 4 2023
Active Locations (7)
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1
Marinus Research Site
Los Angeles, California, United States, 90095
2
Marinus Research Site
Palo Alto, California, United States, 94304
3
Marinus Research Site
Boston, Massachusetts, United States, 02115
4
Marinus Research Site
Livingston, New Jersey, United States, 07039