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

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ClinicalTrials.gov Identifier: NCT04285346
Recruitment Status : Recruiting
First Posted : February 26, 2020
Last Update Posted : October 19, 2020
Sponsor:
Information provided by (Responsible Party):
Marinus Pharmaceuticals

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.

Condition or disease Intervention/treatment Phase
Tuberous Sclerosis Drug: Ganaxolone Phase 2

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 parts: Part A consists of a 4-week baseline period followed by a 12-week treatment period (4-week titration and 8-week maintenance). For patients not continuing in the 24-week OLE period (Part B), a 2-week taper period followed by a 2-week safety period would follow. The main difference between Part A and Part B is the length of treatment, less frequent assessments, and the ability to alter drug doses (both GNX and other antiepileptic drug [AED] treatments which includes initiating and stopping other medications) based on investigator evaluation of the patient's clinical course during Part B. Patients with a seizure frequency reduction during the 12-week treatment period in Part A compared to baseline may continue into Part B ("OLE eligible"), to assess long-term safety, efficacy and tolerability in patients with TSC-related Epilepsy.

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 30 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A Phase 2 Open-label 12-Week Trial of Adjunctive Ganaxolone Treatment (Part A) in Tuberous Sclerosis Complex-related Epilepsy Followed by Long-term Treatment (Part B)
Actual Study Start Date : April 8, 2020
Estimated Primary Completion Date : April 30, 2021
Estimated Study Completion Date : December 31, 2021


Arm Intervention/treatment
Open-label
ganaxolone suspension (50 mg/ml) TID for 12 weeks with 24 week extension
Drug: Ganaxolone
titration followed by maintenance and extension period




Primary Outcome Measures :
  1. Percent Change in 28-day Seizure Frequency [ Time Frame: End of Open-label 12 weeks treatment period ]
    Percent change in 28-day seizure frequency during 12 weeks Open-label treatment period relative to the 4 week baseline period


Secondary Outcome Measures :
  1. Percentage of patients [ Time Frame: up to 24 weeks after end of Part A ]
    Percentage of patients experiencing a greater than or equal to 50% reduction in 28-day primary seizure frequency through the end of the 12-weeks treatment period compared to the 4-week Baseline Period.



Information from the National Library of Medicine

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Ages Eligible for Study:   2 Years to 65 Years   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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

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): NCT04285346


Contacts
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Contact: Mary Fragnito 484-801-5687 mfragnito@marinuspharma.com
Contact: Matt Hall 484-801-5633 mhall@marinuspharma.com

Locations
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United States, California
Marinus Research Site Not yet recruiting
Oakland, California, United States, 94609
Marinus Research Site Recruiting
Palo Alto, California, United States, 94304
Contact       marinus_ph2_tsc_calm_study@marinuspharma.com   
United States, Massachusetts
Marinus Research Site Recruiting
Boston, Massachusetts, United States, 02115
Contact       marinus_ph2_tsc_calm_study@marinuspharma.com   
United States, New Jersey
Marinus Research Site Not yet recruiting
Livingston, New Jersey, United States, 07039
United States, North Carolina
Marinus Research Site Recruiting
Durham, North Carolina, United States, 27710
Contact       marinus_ph2_tsc_calm_study@marinuspharma.com   
United States, Ohio
Marinus Research Site Recruiting
Cincinnati, Ohio, United States, 45229
Contact       marinus_ph2_tsc_calm_study@marinuspharma.com   
United States, Texas
Marinus Research Site Recruiting
Houston, Texas, United States, 77030
Contact       marinus_ph2_tsc_calm_study@marinuspharma.com   
Sponsors and Collaborators
Marinus Pharmaceuticals
Investigators
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Study Director: Maciej Gasior, MD Marinus Pharmaceuticals, Inc.
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Responsible Party: Marinus Pharmaceuticals
ClinicalTrials.gov Identifier: NCT04285346    
Other Study ID Numbers: 1042-TSC-2001
First Posted: February 26, 2020    Key Record Dates
Last Update Posted: October 19, 2020
Last Verified: October 2020
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
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Tuberous Sclerosis
Sclerosis
Pathologic Processes
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
Genetic Diseases, Inborn