Epilepsies, PartialPhase 2NCT00512317

Open-label Extension to Protocol 1042-0600

A Phase 2 study testing ganaxolone in people with Epilepsies, Partial, run by Marinus Pharmaceuticals at 25 sites in United States.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record

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Trial summary

To allow open-label extension to patients who have completed Protocol 1042-0600. Read more about this study

Who is it for?: Ages 18 to 69 years
Conditions the study is for: Epilepsies, Partial
What is being tested?: ganaxolone
Could I receive a placebo?: No, everyone receives an active treatment
How many people: 123 participants
Where: 25 sitesUnited States
Study dates: Jun 2007to Sep 2013
Phase: Phase 2
Are there results?: Yes, posted in the registrySummary in the doctors' view

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

Ages 18 to 69 years
All sexes
No healthy volunteers
Adult, Older adult

Who may be able to join

Participants who have completed all scheduled clinical study visits in the previous protocol 1042-0600 and have been deemed eligible (no major adverse events thought to be drug related) by the Investigator.
Diagnosis of epilepsy with CPS with or without secondarily generalized seizures according to the International League Against Epilepsy [ILAE] Classification of Epileptic Seizures (1981). Diagnosis should have been established by clinical history and computerized tomography (CT) or magnetic resonance imaging (MRI) of the brain to rule out progressive structural lesions and electroencephalogram (EEG) or video EEG with results consistent with partial-onset epilepsy.
Male or female, 18 to 69 years of age (inclusive). [Note: Participants who are > 69 years of age but are of good health condition may be allowed to enter the study after discussion with and approval by the Medical Monitor.]
A 12-lead electrocardiogram (ECG) without clinically significant abnormalities.
Be properly informed of the nature and risks of the study and give informed consent in writing, prior to entering the study.
Able to participate for the full term of study.

Show all 9Show fewer

Able to keep a seizure diary throughout the course of the study.
Sexually active women of childbearing potential must be using a medically acceptable method of birth control and have a negative qualitative serum beta-human chorionic growth hormone (beta HCG) pregnancy test result from a blood sample collected at the initial screening visit. A woman of 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 at least 3 months, surgical sterilization, or adequate barrier methods (e.g., diaphragm 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 (e.g., a spermicidal cream) is acceptable. In participants who are not sexually active, abstinence is an acceptable form of birth control and qualitative serum βHCG pregnancy tests must be tested per protocol.
Participants with a history of depression must be stable and may be taking one antidepressant medication

Who cannot join

Presence of non-motor simple partial seizures only.
History of pseudoseizures in the last 5 years.
History of a primary generalized seizure in the last 5 years.
Past use of vigabatrin without stable visual fields tested twice over the 12 months after the last dose of vigabatrin (Concomitant use of vigabatrin is not allowed).
Seizures secondary to illicit drug or alcohol use, infection, neoplasm, demyelinating disease, degenerative neurological disease, or CNS disease deemed progressive, metabolic illness, or progressive degenerative disease.
Status epilepticus within the last year prior to randomization in 1042-0600 study.

Show all 20Show fewer

Clinically unstable psychiatric disorder within the last 2 years.
Suicide attempt within the last 5 years or current significant suicidal ideation.
History of psychosis within the last 5 years.
Current use of neuroleptics for psychosis.
A significant medical or surgical condition at screening which might compromise the hematologic, cardiovascular, pulmonary, renal, gastrointestinal, or hepatic systems or other conditions that would place the participant at increased risk.
Known sensitivity or allergy to progesterone or related steroid compounds.
History of drug use or alcohol abuse within the past 5 years.
Sexually active women of childbearing potential (WCBP) who are unwilling to use a double-barrier method and establish that they are currently not pregnant by submitting to a serum pregnancy test.
A history of chronic noncompliance with drug regimens.
Females who are currently breastfeeding.
Exposure to any other investigational drug within 30 days prior to randomization in 1042-0600 study.
Aspartate transaminase (AST) or alanine transaminase (ALT) levels > 3 times the upper limit of normal (ULN) at screening.
Participant has history of repetitive seizures within the 12-month period preceding study entry where the individual seizures cannot be counted.
Inability to withhold grapefruit and grapefruit juice from diet during the entire clinical trial.

Trial locations and contacts

25 sites in 1 country.

City, postcode or hospital

25 sites in 1 country

United States25 sites

Neurosciences Institute at Albany Medical Center
Albany, New York, 12208
Emory HealthCare
Atlanta, Georgia, 30322
Anchutz Outpatient Pavillion Neurosciences Clinic/ University of Colorado Hospital
Aurora, Colorado, 80010-0045
Mid-Atlantic Epilepsy and Sleep Center
Bethesda, Maryland, 20817
University of Alabama
Birmingham, Alabama, 35294-0021
Comprehensive Epilepsy Care Center for Children and Adults
Chesterfield, Missouri, 63017
Ohio State University Medical Center
Columbus, Ohio, 43210
Neurological Clinic of Texas, P.A.
Dallas, Texas, 75230
2799 West Grand blvd. CFP 071
Detroit, Michigan, 48202
University of Florida McKnight Brain Institute
Gainesville, Florida, 32610-0236
University of Iowa Hospitals and Clinics
Iowa City, Iowa, 52242
University of Kentucky, Dept. of Neurology
Lexington, Kentucky, 40536
Arkansas Epilepsy Program
Little Rock, Arkansas, 72205
University of Southern California Adult Comprehensive Epilepsy Center
Los Angeles, California, 90033
Riddle Health Care Center for Neuroscience
Media, Pennsylvania, 19063
Vanderbilt University Medical Center
Nashville, Tennessee, 37232
Yale University School of Medicine
New Haven, Connecticut, 06520
Thomas Jefferson University
Philadelphia, Pennsylvania, 19107
Barrow Neurological Institute
Phoenix, Arizona, 85013
Virginia Commonwealth University
Richmond, Virginia, 23298
University of California-Davis
Sacramento, California, 95817
Minnesota Epilepsy Group, PA
Saint Paul, Minnesota, 55102-2383
Intercoastal Neurology
Sarasota, Florida, 34232
Southern Illinois University Medical Center
Springfield, Illinois, 62702
SUNY Upstate Medical University
Syracuse, New York, 13210

Other trials in United States

About this study

Official title: An Open-label Extension Study to Evaluate the Safety, Tolerability, and Efficacy of Ganaxolone as add-on Therapy in Adult Patients With Epilepsy Consisting of Uncontrolled Partial-onset Seizures.

To allow open-label extension to patients who have completed Protocol 1042-0600.

Read the detailed description

This is an open-label study evaluating efficacy and safety of ganaxolone treatment in adults with partial onset epilepsy with or without secondary generalizations.

Conditions: Epilepsies, Partial
Being tested: ganaxolone
Keywords: partial onset epilepsy, adult epilepsy, partial seizures, anticonvulsant, catamenial epilepsy, adult partial onset epilepsy
Medical subject headings: Epilepsies, Partial, Epilepsy, Seizures

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

ganaxoloneDrug
liquid suspension dosed tid

Study groups in NCT00512317
Study group	Role	What they receive
ganaxolone	Experimental	active experimental drug Receives: Drug: ganaxolone

What is being measured

The primary outcome is the main question the study is designed to answer.

Percent Change From Baseline in Weekly Seizure Frequency During Weeks 1 Through 117
Percent Change in weekly seizure frequency by treatment group compared to Baseline at the beginning of the double-blind study 1042-0600 is presented. Weekly seizure frequency included partial-onset seizures (POS) with or without secondary generalization, but not non-motor simple partial seizure (SPS) during Weeks 1 through Week 117. Baseline was defined as the Day 0 assessment before study drug infusion of the double-blind study 1042-0600.
Measured: Baseline (Day 0) and Week 1 through Week 117

Secondary outcomes (4)

Number of Responders During Weeks 1 Through 117
Responders were defined as participants experiencing ≥50% of reduction in mean weekly seizure frequency from the Baseline. Baseline was defined as the Day 0 assessment before study drug infusion of the double-blind study 1042-0600.
Measured: Baseline (Day 0) and Week 1 through Week 117
Number of Seizure-free Days During Weeks 1 Through 117
Average number of seizure-free days per week for a given period was calculated as follows: (Total number of days with no seizures of any type during that period / number of days with seizure diary in that period) multiplied by 7.
Measured: Week 1 through Week 117
Number of Seizure-free Participants
Number of Seizure-free participants is presented.
Measured: Day 1 through Day 224 (Week 32)
Change From Baseline in Quality of Life in Epilepsy Inventory-31 (QOLIE-31) Questionnaire
QOLIE-31 was a survey of health-related QOL for adults with epilepsy and evaluated how much distress the participant feels about problems and worries related to epilepsy. It included 38 items grouped into eight multi-item subscales - Energy/Fatigue, Emotional Well-Being, Daily Activities/Social Functioning, Cognitive Functioning, Medication Effect, Seizure Worry, Overall Quality of Life (QoL) and Distress. The subscale scores and the total score were calculated according to the scoring algorithm defined by the author with scores ranging from 0 to 100; higher scores indicated better function. Baseline was defined as the last non-missing observation prior to the first dose in double-blind study 1042-0600. Change from Baseline was calculated by subtracting Baseline value from the post-dose visit value.
Measured: Baseline (Day 0) and up to Week 104

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

Jun 2007Sep 2013

First posted7 Aug 2007 (estimated)
Study startJun 2007
Primary completionDec 2009
Study completionSep 2013

Resources

Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
Other trials for Epilepsies, PartialStudies of the same condition, open and under way.
What the trial phases meanHow a treatment moves from first tests in people to approval.

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design

Study type: Interventional
Phase: Phase 2
Primary purpose: Treatment
Allocation: Not applicable
Intervention model: Single group
Masking: None (open label)
Enrolment: 123 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT00512317
Sponsor's protocol ID: 1042-0601
Other IDs: V1.6
Status last verified: Dec 2022
First submitted: 3 Aug 2007
Last updated: 17 Jan 2023
Results posted: Yes, on ClinicalTrials.gov
Data monitoring committee: No

Want to know more?

The sponsor has not listed a contact. The official record may have more details.

Talk to your doctor first and take this page with you. Only the study team can confirm whether you can join.

Questions to ask the study team

What is the study trying to find out?
What would I need to do, and how often would I visit?
Could I receive a placebo instead of the treatment?
What are the possible risks and benefits for me?
Will it cost me anything? Are travel or time paid for?
Can I keep seeing my own doctor during the study?
What happens when the study ends?

See locations

RecruitingObservational study
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Active, not recruitingPhase 4
Open-label Study of Cenobamate Monotherapy in Adult Subjects With Newly Diagnosed or Recurrent Partial-Onset Epilepsy
Focal Onset Seizure
Enrolling by invitationPhase 2
SPN-817 Open-Label Extension Study in Adults With Focal Onset Seizures
Focal Onset Seizures
RecruitingObservational study
Investigating Epilepsy: Screening and Evaluation
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All Epilepsies, Partial trials

Study at a glance

From the registry record, last updated by the sponsor on 17 Jan 2023. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title: An Open-label Extension Study to Evaluate the Safety, Tolerability, and Efficacy of Ganaxolone as add-on Therapy in Adult Patients With Epilepsy Consisting of Uncontrolled Partial-onset Seizures.
ClinicalTrials.gov ID: NCT00512317
Acronym / sponsor ID: 1042-0601
Conditions: Epilepsies, Partial
Keywords: partial onset epilepsy, adult epilepsy, partial seizures, anticonvulsant, catamenial epilepsy, adult partial onset epilepsy
Phase: Phase 2
Study type: Interventional
Recruitment status: Completed
Enrolment: 123 participants (actual)
Lead sponsor: Marinus PharmaceuticalsIndustry (pharma, biotech, devices)
Study start: Jun 2007
Primary completion: Dec 2009 (actual)
Study completion: Sep 2013 (actual)
First posted: 7 Aug 2007 (estimated)
Results: Posted 17 Jan 2023

Clinical summary

The research question as the sponsor registered it. This describes what the study plans to test, not what it found.

To allow open-label extension to patients who have completed Protocol 1042-0600.

Detailed description

This is an open-label study evaluating efficacy and safety of ganaxolone treatment in adults with partial onset epilepsy with or without secondary generalizations.

Study design

Primary purpose: Treatment
Allocation: Not applicable
Intervention model: Single group
Masking: None (open label)
Number of arms: 1

Arm	Type	Receives
ganaxolone	Experimental	Drug: ganaxoloneactive experimental drug

Who may qualify

Ages: 18 to 69 years
Sex: All
Healthy volunteers: Not accepted
Age groups: Adult, Older adult

Key inclusion criteria (9 listed)

Participants who have completed all scheduled clinical study visits in the previous protocol 1042-0600 and have been deemed eligible (no major adverse events thought to be drug related) by the Investigator.
Diagnosis of epilepsy with CPS with or without secondarily generalized seizures according to the International League Against Epilepsy [ILAE] Classification of Epileptic Seizures (1981). Diagnosis should have been established by clinical history and computerized tomography (CT) or magnetic resonance imaging (MRI) of the brain to rule out progressive structural lesions and electroencephalogram (EEG) or video EEG with results consistent with partial-onset epilepsy.
Male or female, 18 to 69 years of age (inclusive). [Note: Participants who are > 69 years of age but are of good health condition may be allowed to enter the study after discussion with and approval by the Medical Monitor.]
A 12-lead electrocardiogram (ECG) without clinically significant abnormalities.
Be properly informed of the nature and risks of the study and give informed consent in writing, prior to entering the study.

Key exclusion criteria (20 listed)

Presence of non-motor simple partial seizures only.
History of pseudoseizures in the last 5 years.
History of a primary generalized seizure in the last 5 years.
Past use of vigabatrin without stable visual fields tested twice over the 12 months after the last dose of vigabatrin (Concomitant use of vigabatrin is not allowed).
Seizures secondary to illicit drug or alcohol use, infection, neoplasm, demyelinating disease, degenerative neurological disease, or CNS disease deemed progressive, metabolic illness, or progressive degenerative disease.

Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.

Interventions

Intervention	Type	Arms
ganaxoloneliquid suspension dosed tid	Drug	ganaxolone

Primary outcome

Percent Change From Baseline in Weekly Seizure Frequency During Weeks 1 Through 117
Time frame: Baseline (Day 0) and Week 1 through Week 117
Percent Change in weekly seizure frequency by treatment group compared to Baseline at the beginning of the double-blind study 1042-0600 is presented. Weekly seizure frequency included partial-onset seizures (POS) with or without secondary generalization, but not non-motor simple partial seizure (SPS) during Weeks 1 through Week 117. Baseline was defined as the Day 0 assessment before study drug infusion of the double-blind study 1042-0600.

4 secondary outcomes

Number of Responders During Weeks 1 Through 117
Time frame: Baseline (Day 0) and Week 1 through Week 117
Number of Seizure-free Days During Weeks 1 Through 117
Time frame: Week 1 through Week 117
Number of Seizure-free Participants
Time frame: Day 1 through Day 224 (Week 32)
Change From Baseline in Quality of Life in Epilepsy Inventory-31 (QOLIE-31) Questionnaire
Time frame: Baseline (Day 0) and up to Week 104

Results and safety

Results posted to the registry on 17 Jan 2023, as reported by the sponsor. Figures are copied from the record, not re-analysed. Full results on ClinicalTrials.gov.

Participant flow

Group	Started	Completed
Ganaxolone	123	6

Primary outcome: Percent Change From Baseline in Weekly Seizure Frequency During Weeks 1 Through 117

Time frame: Baseline (Day 0) and Week 1 through Week 117. Mean (Standard Deviation). Unit: Percent Change

Measure	GNX/GNX n = 79	PBO/GNX n = 41
Weeks 1-10	-14.8 (54.86)	-12.3 (64.08)
Weeks 1-13	-14.0 (52.91)	-13.7 (65.60)
Weeks >13-26	-19.6 (42.38)	-19.8 (56.43)
Weeks >26-39	-37.7 (33.79)	-48.6 (37.54)
Weeks >39-52	-29.1 (51.26)	-42.9 (59.81)
Weeks >52-65	-29.9 (51.85)	-43.1 (59.45)
Weeks >65-78	-41.4 (38.21)	-67.7 (23.09)
Weeks >78-91	-38.8 (66.24)	-43.6 (37.60)

Population analysed: Intent-To-Treat (ITT) Population included all participants who received at least 1 dose of open-label study medication. Only participants who received at least 1 dose of ganaxolone in the open-label (OL) study (1042-0601), had seizure diary data for Baseline (from double-blind study 1042-0600), and who had data from at least 7 seizure diaries in the open-label study (1042-0601) were included. Only those participants with data available at the specified data points were analyzed.

4 secondary outcomes are reported in the full results.

Adverse events

Time frame: Up to Week 117. Other adverse events reported at 0% frequency or more

Group	Serious adverse events	Other adverse events	Deaths
Ganaxolone	15 of 123 (12.2%)	103 of 123 (83.7%)	0 of 123

Loading the posted results from ClinicalTrials.gov…

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 17 Jan 2023.
Posted resultsParticipant flow, baseline, all outcome measures and adverse events.

Related trials for Epilepsies, Partial

RecruitingObservational study
Rare Disease Patient Registry & Natural History Study - Coordination of Rare Diseases at Sanford
Rare Disorders, Undiagnosed Disorders
Not yet recruiting
Peri-procedural Weighted Blanket Use for ECT
Post-ect Agitation, Post-ictal Agitation
Active, not recruitingPhase 4
Open-label Study of Cenobamate Monotherapy in Adult Subjects With Newly Diagnosed or Recurrent Partial-Onset Epilepsy
Focal Onset Seizure
Enrolling by invitationPhase 2
SPN-817 Open-Label Extension Study in Adults With Focal Onset Seizures
Focal Onset Seizures
RecruitingObservational study
Investigating Epilepsy: Screening and Evaluation
Seizures, Epilepsy

Referral and contact

AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.

The record lists no contact. Use the official record to reach the sponsor.

25 sites in 1 country: see every site and its status.

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing ganaxolone in people with Epilepsies, Partial, run by Marinus Pharmaceuticals at 25 sites in United States.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 9 inclusion and 20 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 17 Jan 2023. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 25 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

Yes. Results are posted in the registry (first posted 17 Jan 2023). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Percent Change From Baseline in Weekly Seizure Frequency During Weeks 1 Through 117 (time frame: Baseline (Day 0) and Week 1 through Week 117). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT00512317.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 17 Jan 2023.