Adjunctive Ganaxolone Treatment (Part A) in TSC Followed by Long-term Treatment (Part B)

NCT04285346 | Completed Phase 2 Results FDA Drug

• 1 other identifier: 1042-TSC-2001
• Study Type: interventional
• Target: 23
• Locations: 1 country
• Sites: 7

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 through the end of the 12 week treatment period.

Conditions

Tuberous Sclerosis

Outcome Measures

Primary Outcomes (1)

• Percent Change From Baseline in 28-day Seizure Frequency Through the End of 12-Week Treatment Period

  Primary seizures include atonic/drop, bilateral clonic, bilateral tonic, focal motor without impairment of consciousness or awareness, focal (motor or non-motor) with impairment of consciousness or awareness, focal to bilateral tonic-clonic, generalized tonic-clonic. Baseline 28-day seizure frequency was calculated as the total number of primary seizures in the Baseline period divided by the number of days with non-missing seizure data in the Baseline period, multiplied by 28. The Baseline Visit was defined as Week 0. Percent change from Baseline in 28-day seizure frequent was calculated as the difference in post-Baseline 28-day seizure frequency and Baseline 28-day seizure frequency, divided by Baseline 28-day seizure frequency, multiplied by 100.

  Baseline and Up to Week 12

Secondary Outcomes (1)

• Percentage of Participants Experiencing a >=50 Percent Reduction in 28-day Primary Seizure Frequency Through the End of the 12-week Treatment Period Compared to the Baseline Period

  Baseline and up to 12 Weeks

Study Arms (1)

Open-label

OTHER

ganaxolone suspension (50 mg/ml) TID for 12 weeks with 24 week extension

Drug: Ganaxolone

Interventions

Ganaxolone DRUG

titration followed by maintenance and extension period

Open-label

Eligibility Criteria

• Age: 2 Years - 65 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.
• Patients have experienced ≥ 35% reduction in primary seizure frequency during the Part A treatment period compared to the 4-week Baseline Period.

You may not qualify if:

• 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

Sponsors & Collaborators

• Marinus Pharmaceuticals: lead

Study Sites (7)

Marinus Research Site

Los Angeles, California, 90095, United States

Location

Marinus Research Site

Palo Alto, California, 94304, United States

Location

Marinus Research Site

Boston, Massachusetts, 02115, United States

Location

Marinus Research Site

Livingston, New Jersey, 07039, United States

Location

Marinus Research Site

Durham, North Carolina, 27710, United States

Location

Marinus Research Site

Cincinnati, Ohio, 45229, United States

Location

Marinus Research Site

Houston, Texas, 77030, United States

Location

MeSH Terms

Conditions

Tuberous Sclerosis

Interventions

ganaxolone

Condition Hierarchy (Ancestors)

Hamartoma; Neoplasms; Neoplasms, Multiple Primary; Neoplastic Syndromes, Hereditary; Malformations of Cortical Development, Group I; Malformations of Cortical Development; Nervous System Malformations; Nervous System Diseases; Neurocutaneous Syndromes; Heredodegenerative Disorders, Nervous System; Neurodegenerative Diseases; Congenital Abnormalities; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Genetic Diseases, Inborn

Results Point of Contact

• Title: Marinus Clinical Trials Submission Manager
• Organization: Marinus Pharmaceuticals, Inc.

clinicaltrials@marinuspharma.com

484-801-4670

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: No

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: February 18, 2020
• First Posted: February 26, 2020
• Study Start: April 8, 2020
• Primary Completion: June 25, 2021
• Study Completion: August 30, 2022
• Last Updated: April 4, 2023
• Results First Posted: April 4, 2023

Record last verified: 2023-03

Data Sharing

• IPD Sharing: Will not share

Locations

US (7)