Intravenous Ganaxolone as Adjunctive Therapy to Treat Subjects With Status Epilepticus

NCT03350035 | Completed Phase 2 Results DMC FDA Drug

• 1 other identifier: 1042-SE-2001
• Study Type: interventional
• Target: 17
• Locations: 1 country
• Sites: 8

Brief Summary

This study will evaluate the effectiveness and safety of an investigational drug, IV ganaxolone, as adjunctive therapy to standard of care to treat subjects with status epilepticus.

Conditions

Status Epilepticus; Convulsive Status EPILEPTICUS; Non Convulsive Status Epilepticus; Epilepsy

Outcome Measures

Primary Outcomes (1)

• Number of Participants Who Did Not Require an IV Anesthetic Drug for SE Treatment

  Number of participants who did not require an intravenous (IV) Anesthetic Drug (a third-line Treatment) for Status Epilepticus (SE) within the first 24 hours after Study Drug Initiation.

  24 hours post study drug initiation

Secondary Outcomes (4)

• Time to Cessation of SE

  Time to SE Cessation, assessed up to 24 hours

• Number of Participants Who Required No Escalation of Treatment for Ongoing or Recurrent SE

  Drug initiation through follow-up period, up to approximately 4 weeks

• Number of Participants With No SE Recurrence Per Principal Investigator

  Baseline (within 24hrs of treatment) through follow up period, up to approximately 4 weeks.

• Seizure Burden

  Baseline (Pre-dose) to <-24hrs (Post Dose)

Study Arms (2)

IV Ganaxolone active

EXPERIMENTAL

Ganaxolone IV loading dose with continuous infusion (maintenance dose) for 2-4 days followed by an 18-hour taper.

Drug: IV Ganaxolone active

IV Placebo, non-active

PLACEBO COMPARATOR

Placebo IV loading dose with continuous infusion (maintenance dose) for 2-4 days followed by an 18-hour taper.

Drug: IV Placebo, non-active

Interventions

IV Ganaxolone active DRUG

IV

IV Ganaxolone active

IV Placebo, non-active DRUG

IV

IV Placebo, non-active

Eligibility Criteria

• Age: 12 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• Subjects 12 years of age and older
• Clinical and/or electrographic seizures

You may not qualify if:

• Life expectancy of less than 24 hours
• Anoxic brain injury as primary cause of SE
• Recent (\<24 hour) traumatic brain injury as the primary cause of SE
• Administered anesthesia for the treatment of SE

Sponsors & Collaborators

• Marinus Pharmaceuticals: lead

Study Sites (8)

Nemours/AI duPont Hospital for Children

Wilmington, Delaware, 19803, United States

Location

Nicklaus Children's Hospital

Miami, Florida, 33155, United States

Location

Grady Hospital

Atlanta, Georgia, 30303, United States

Location

Oschner Clinic Foundation

New Orleans, Louisiana, 70121, United States

Location

Brigham and Women's Hospital

Boston, Massachusetts, 02115, United States

Location

Henry Ford Hospital

Detroit, Michigan, 48202, United States

Location

Duke Medical Center

Durham, North Carolina, 27710, United States

Location

Allegheny General Hospital

Pittsburgh, Pennsylvania, 15212, United States

Location

Related Publications (1)

• Vaitkevicius H, Ramsay RE, Swisher CB, Husain AM, Aimetti A, Gasior M. Intravenous ganaxolone for the treatment of refractory status epilepticus: Results from an open-label, dose-finding, phase 2 trial. Epilepsia. 2022 Sep;63(9):2381-2391. doi: 10.1111/epi.17343. Epub 2022 Jul 10.

  PMID: 35748707 DERIVED

MeSH Terms

Conditions

Status Epilepticus; Epilepsy

Condition Hierarchy (Ancestors)

Seizures; Neurologic Manifestations; Nervous System Diseases; Signs and Symptoms; Pathological Conditions, Signs and Symptoms; Brain Diseases; Central Nervous System Diseases

Limitations and Caveats

2 deaths during the follow-up were extracted from the SAE reconciliation database.

Results Point of Contact

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

clinicaltrials@marinuspharma.com

484-801-4670

Study Officials

• Maciej Gasior, MD, PhD

  Marinus Pharmaceuticals

  STUDY DIRECTOR

Publication Agreements

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

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Model Details: Double-blind study that will randomize subjects to ganaxolone or placebo in a 1:1 ratio as adjunctive therapy to their standard of care.

Study Record Dates

• First Submitted: November 17, 2017
• First Posted: November 22, 2017
• Study Start: February 19, 2018
• Primary Completion: September 18, 2019
• Study Completion: September 18, 2019
• Last Updated: March 9, 2023
• Results First Posted: December 15, 2022

Record last verified: 2023-03

Locations

US (8)