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Adjunctive GNX Treatment Compared With Placebo in Children and Adults With TSC-related Epilepsy

Study Purpose

This is a Phase 3, global, double-blind, randomized, placebo-controlled study of adjunctive GNX treatment in children and adults with TSC-related epilepsy. The study consists of a 4-week prospective baseline phase, defined as the first 28 days following screening, followed by a double-blind phase consisting of a 4-week titration period (with 2 additional weeks allowed, if necessary, for tolerance) and a 12-week maintenance period.

Recruitment Criteria

Accepts Healthy Volunteers

Healthy volunteers are participants who do not have a disease or condition, or related conditions or symptoms

No
Study Type

An interventional clinical study is where participants are assigned to receive one or more interventions (or no intervention) so that researchers can evaluate the effects of the interventions on biomedical or health-related outcomes.


An observational clinical study is where participants identified as belonging to study groups are assessed for biomedical or health outcomes.


Searching Both is inclusive of interventional and observational studies.

Interventional
Eligible Ages 1 Year - 65 Years
Gender All
More Inclusion & Exclusion Criteria

Inclusion Criteria:

1. Clinical or mutational diagnosis of TSC consistent with (Northrup and Krueger, 2013): 1. Molecular confirmation of a pathogenic mutation in TSC1 or TSC2. A pathogenic mutation is defined as a mutation that clearly prevents protein synthesis and/or inactivates the function of the TSC1 or TSC2 proteins (eg, nonsense mutation or frameshift mutations, large genomic deletions) or is a missense mutation whose effect on protein function has been established by functional assessment. The PI or designee must review the results of the genetic analysis and confirm that the causal relationship to the epilepsy syndrome is likely. OR. 2. Clinical diagnosis of definite TSC which includes 2 major features or 1 major feature with ≥ 2 minor features. 2. Male or female participants aged 1 through 65 years, inclusive. 3. Participant/parent or LAR willing to give written informed consent/assent, after being properly informed of the nature and risks of the study and prior to engaging in any study related procedures. 4. Assent for participants over 7 years of age should be obtained if appropriate. 5. Failure to control seizures despite appropriate trial of 2 or more AEDs at therapeutic doses and for adequate duration of treatment per PI judgment. 6. Participants should be on a stable regimen of AEDs (including moderate or strong inducer or inhibitor anti-seizure medications eg, carbamazepine, phenytoin, etc.) at therapeutic doses for ≥ 28 days prior to the screening visit, and without a foreseeable change in dosing for the duration of the study. (Note: Minor dose adjustment to address tolerability and safety events may be allowed on case-by-case basis and it should be discussed with the study medical monitor.) 7. A history of at least 8 countable seizures per month in the 2 months prior to screening with no more than 1 seizure free week in each month. 8. Have an average of at least 2 primary endpoint seizures per week in the 28 days following the screening visit. The primary endpoint seizure types are defined as the following: 1. focal motor seizures without impairment of consciousness or awareness. 2. focal seizures with impairment of consciousness or awareness with motor features. 3. focal seizures evolving to bilateral, tonic-clonic seizures. 4. generalized motor seizures including tonic-clonic, bilateral tonic, bilateral clonic, or atonic/drop seizures. Seizures that do not count towards the primary endpoint include: 1. Focal aware seizures without motor features. 2. Focal and generalized nonmotor seizures (eg, absence or focal nonmotor seizures with or without impairment of awareness) 3. Infantile or epileptic spasms. 4. Myoclonic seizures. 9. Participants with surgically implanted VNS will be allowed to enter the study provided that all of the following conditions are met: 1. The VNS has been in place for ≥ 1 year prior to the screening visit. 2. The settings must have remained constant for 3 months prior to the screening visit and are expected to remain constant throughout the study. 3. The battery is expected to last for the duration of the study. 10. Parent/caregiver or the participant, as appropriate, is able and willing to maintain an accurate and complete daily seizure eDiary for the duration of the study. 11. Able and willing to take IP (suspension) as directed with food TID. 12. Sexually active WOCBP must be using a medically acceptable method of birth control and have a negative quantitative serum β-HCG test collected at the initial screening visit. Childbearing potential is defined as a female who is biologically capable of becoming pregnant. A medically acceptable method of birth control includes intrauterine devices in place for 1 month prior to the screening visit, surgical sterilization, or adequate double barrier methods (eg, diaphragm or condom and foam). An oral contraceptive alone is not considered adequate for the purpose of this study. Use of oral contraceptives in combination with another method (eg, a spermicidal cream) is acceptable. In participants who are not sexually active, abstinence is an acceptable method of birth control. 13. Male participants must agree to take all necessary measures to avoid causing pregnancy in their sexual partners during the study and for 30 days after the last dose of IP. Medically acceptable contraceptives include surgical sterilization (such as a vasectomy) and a condom used with a spermicidal gel or foam.

Exclusion Criteria:

1. Previous exposure to GNX. 2. Pregnant or breastfeeding. 3. Participants who have been taking felbamate for less than 1 year prior to screening. 4. Participants taking CBD preparations other than Epidiolex. 5. A positive result on plasma drug screen for CBD or THC at Visit 1 (screening), with the exception of results that are fully explained by Epidiolex, which is being prescribed and managed by the investigator. 6. Concurrent use of ACTH, prednisone or other glucocorticoid is not permitted, nor use of the strong inducers of CYP3A4, rifampin and St John's Wort. Participants on ACTH, prednisone, or other systemically (non-inhaled or topical) administered steroids should be off the product > 28 days prior to screening. Rifampin and St John's Wort must be discontinued at least 28 days before Visit 2, study drug initiation. Note: 1. Use of concomitant intranasal or PRN topical steroids for dermatologic reactions and allergic rhinitis are allowed during the study. 2. This exclusion criterion does not prohibit the use of approved AEDs. 7. Changes in any chronic medications within the 4 weeks prior to the screening visit. All chronic concomitant medications must be relatively stable in dose for at least 4 weeks prior to the screening visit unless otherwise noted. Small dose adjustment to manage tolerability and safety events is permitted and should be discussed with the study medical monitor. 8. Participants who have epilepsy surgery planned during the study or who have undergone surgery for epilepsy within the 6 months prior to screening. 9. An active CNS infection, demyelinating disease, degenerative neurological disease, or CNS disease deemed progressive as evaluated by brain imaging (MRI). This includes tumor growth which in the opinion of the investigator could affect primary endpoint seizure control. 10. Any disease or condition (medical or surgical; other than TSC) at the screening visit that might compromise the hematologic, cardiovascular (including any cardiac conduction defect), pulmonary, renal, gastrointestinal, or hepatic systems; or other conditions that might interfere with the absorption, distribution, metabolism, or excretion of the IP, or would place the participant at increased risk or interfere with the assessment of safety/efficacy. This may include any illness in the past 4 weeks which in the opinion of the investigator may affect seizure frequency. 11. An AST/SGOT or ALT/SGPT > 3 × the ULN at screening or baseline visits and confirmed by a repeat test. 12. Biliary impairment sufficient to affect patient safety, or total bilirubin levels > 1.5 × ULN at screening or baseline visit and confirmed by a repeat test. In cases of Gilbert's Syndrome, resulting in stable levels of total bilirubin greater than ULN, the medical monitor can determine if a protocol exception can be made. 13. Renal impairment sufficient to affect patient safety, or 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 (Levey et al, 2006). Cases of temporary renal insufficiency should be discussed with the medical monitor to determine the participant's study continuation. 14. Exposed to any other investigational drug or investigational device within 30 days or fewer than 5 half-lives prior to the screening visit. For therapies in which half-life cannot be readily established, the Sponsor's Medical Monitor should be consulted. 15. Unwillingness to avoid excessive alcohol use throughout the study. 16. Have active suicidal plan/intent, active suicidal thoughts or a suicide attempt in the past 6 months. 17. Known sensitivity or allergy to any component in the IP(s), progesterone, or other related steroid compounds.

Trial Details

Trial ID:

This trial id was obtained from ClinicalTrials.gov, a service of the U.S. National Institutes of Health, providing information on publicly and privately supported clinical studies of human participants with locations in all 50 States and in 196 countries.

NCT05323734
Phase

Phase 1: Studies that emphasize safety and how the drug is metabolized and excreted in humans.

Phase 2: Studies that gather preliminary data on effectiveness (whether the drug works in people who have a certain disease or condition) and additional safety data.

Phase 3: Studies that gather more information about safety and effectiveness by studying different populations and different dosages and by using the drug in combination with other drugs.

Phase 4: Studies occurring after FDA has approved a drug for marketing, efficacy, or optimal use.

Phase 3
Lead Sponsor

The sponsor is the organization or person who oversees the clinical study and is responsible for analyzing the study data.

Marinus Pharmaceuticals
Principal Investigator

The person who is responsible for the scientific and technical direction of the entire clinical study.

N/A
Principal Investigator Affiliation N/A
Agency Class

Category of organization(s) involved as sponsor (and collaborator) supporting the trial.

Industry
Overall Status Recruiting
Countries United States
Conditions

The disease, disorder, syndrome, illness, or injury that is being studied.

Tuberous Sclerosis
Arms & Interventions

Arms

Experimental: GNX

GNX oral suspension, TID

Placebo Comparator: Placebo

Placebo oral suspension, TID

Interventions

Drug: - GNX oral suspension, TID

Ganaxalone

Drug: - Placebo

Placebo (for Ganaxolone)

Contact a Trial Team

If you are interested in learning more about this trial, find the trial site nearest to your location and contact the site coordinator via email or phone. We also strongly recommend that you consult with your healthcare provider about the trials that may interest you and refer to our terms of service below.

Phoenix Children's Hospital, Phoenix, Arizona

Status

Not yet recruiting

Address

Phoenix Children's Hospital

Phoenix, Arizona, 85106

Site Contact

Angus Wilfong

clinicaltrials@marinuspharma.com

484-801-4670

Arkansas Children's Hospital, Little Rock, Arkansas

Status

Not yet recruiting

Address

Arkansas Children's Hospital

Little Rock, Arkansas, 72202

Site Contact

Debopam Samanta

clinicaltrials@marinuspharma.com

484-801-4670

Los Angeles, California

Status

Not yet recruiting

Address

UCLA Mattel Children's Hospital Los Angeles Tuberous Sclerosis Clinic

Los Angeles, California, 90095

Site Contact

Rajeskar Rajaraman

clinicaltrials@marinuspharma.com

484-801-4670

University of Colorado Denver, Aurora, Colorado

Status

Not yet recruiting

Address

University of Colorado Denver

Aurora, Colorado, 80045

Yale University School of Medicine, New Haven, Connecticut

Status

Not yet recruiting

Address

Yale University School of Medicine

New Haven, Connecticut, 06510

Wilmington, Delaware

Status

Not yet recruiting

Address

Nemours Children's Hospital of Delaware (TSC Clinic at Nemours/duPont Hospital for Children)

Wilmington, Delaware, 19803

NW FL Clinical Research Group, LLC, Gulf Breeze, Florida

Status

Recruiting

Address

NW FL Clinical Research Group, LLC

Gulf Breeze, Florida, 32561

Nicklaus Children's Hospital, Miami, Florida

Status

Not yet recruiting

Address

Nicklaus Children's Hospital

Miami, Florida, 33155

Arnold Palmer Hospital, Orlando, Florida

Status

Not yet recruiting

Address

Arnold Palmer Hospital

Orlando, Florida, 32806

Site Contact

Vikram Prakash

clinicaltrials@marinuspharma.com

484-801-4670

Comprehensive Neurology Clinic, Orlando, Florida

Status

Not yet recruiting

Address

Comprehensive Neurology Clinic

Orlando, Florida, 32825

Site Contact

Refaat El-Said

clinicaltrials@marinuspharma.com

484-801-4670

Chicago, Illinois

Status

Not yet recruiting

Address

Ann & Robert H. Lurie Children's Hospital of Chicago

Chicago, Illinois, 60611

Site Contact

Priyamvada Tatachar

clinicaltrials@marinuspharma.com

484-801-4670

Mid Atlantic Epilepsy & Sleep Centre, Bethesda, Maryland

Status

Not yet recruiting

Address

Mid Atlantic Epilepsy & Sleep Centre

Bethesda, Maryland, 20817

Boston, Massachusetts

Status

Not yet recruiting

Address

Boston Children's Hospital, Harvard Medical School

Boston, Massachusetts, 02115

Site Contact

Jurriaan Peters

clinicaltrials@marinuspharma.com

484-801-4670

Detroit, Michigan

Status

Not yet recruiting

Address

TSC Clinic at Children's Hospital of Michigan

Detroit, Michigan, 48201

Mayo Clinic, Rochester, Minnesota

Status

Not yet recruiting

Address

Mayo Clinic

Rochester, Minnesota, 55905

Site Contact

Elaine Wirrell

clinicaltrials@marinuspharma.com

484-801-4670

University of Missouri Child Health, Columbia, Missouri

Status

Not yet recruiting

Address

University of Missouri Child Health

Columbia, Missouri, 65201

TSC Clinic at Children's Mercy Hosptial, Kansas City, Missouri

Status

Not yet recruiting

Address

TSC Clinic at Children's Mercy Hosptial

Kansas City, Missouri, 64108

Site Contact

Mohammed Ilyas

clinicaltrials@marinuspharma.com

484-801-4670

NYU Comprehensive Epilepsy Center, New York, New York

Status

Not yet recruiting

Address

NYU Comprehensive Epilepsy Center

New York, New York, 10016

Site Contact

Josiane Lajoie

clinicaltrials@marinuspharma.com

484-801-4670

Chapel Hill, North Carolina

Status

Not yet recruiting

Address

University of North Carolina at Chapel Hill

Chapel Hill, North Carolina, 27599

Charlotte, North Carolina

Status

Not yet recruiting

Address

Atrium Healthcare TSC Clinic at Levine Children's Hospital

Charlotte, North Carolina, 28025

Duke University Medical Center, Durham, North Carolina

Status

Not yet recruiting

Address

Duke University Medical Center

Durham, North Carolina, 27710

Site Contact

Muhammad Zafar

clinicaltrials@marinuspharma.com

484-801-4670

Cincinnati, Ohio

Status

Not yet recruiting

Address

Cincinnati Children's Hospital Medical Center

Cincinnati, Ohio, 45229

Medical Univ. of SC (MUSC), Charleston, South Carolina

Status

Not yet recruiting

Address

Medical Univ. of SC (MUSC)

Charleston, South Carolina, 29425

Le Bonheur Children's Hospital, Memphis, Tennessee

Status

Not yet recruiting

Address

Le Bonheur Children's Hospital

Memphis, Tennessee, 38103

Site Contact

James Wheless

clinicaltrials@marinuspharma.com

484-801-4670

Austin, Texas

Status

Recruiting

Address

Child Neurology Consultants of Austin (CNCA)

Austin, Texas, 78749

U of TX Health Sciences Center, Houston, Texas

Status

Recruiting

Address

U of TX Health Sciences Center

Houston, Texas, 77030

Site Contact

Mary Kay Koenig

clinicaltrials@marinuspharma.com

484-801-4670

Salt Lake City, Utah

Status

Not yet recruiting

Address

University of Utah Health Care-Pediatric Neurology

Salt Lake City, Utah, 84113

Site Contact

Matthew Sweeney

clinicaltrials@marinuspharma.com

484-801-4670

Seattle, Washington

Status

Not yet recruiting

Address

Seattle Children's Hospital, Research and Foundation

Seattle, Washington, 98105

Site Contact

Russell Saneto

clinicaltrials@marinuspharma.com

484-801-4670

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