Drug Resistant Partial Onset SeizurePhase 3NCT02519439
A Two-year Open-label Extension Study of Ganaxolone in Patients With Drug-resistant Partial-onset Seizures
A Phase 3 study testing ganaxolone in people with Drug Resistant Partial Onset Seizure, run by Marinus Pharmaceuticals at 5 sites in United States.
Trial summary
A follow-on, two-year open-label extension study of ganaxolone as add-on therapy in adult patients with drug-resistant partial-onset seizures Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Drug Resistant Partial Onset Seizure
- What is being tested?
- ganaxolone
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 26 participants
- Where
- 5 sitesUnited States
- Study dates
- Feb 2015to Dec 2016
- Phase
- Phase 3
- 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 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Subjects who have completed all scheduled clinical study visits in the previous protocol 1042-0603 and have shown a minimum 35% improvement in mean 28-day seizure frequency over the last three 28-day periods in study 1042-603 as compared to the baseline of study 1042-603.
- Subjects whose daily study drug compliance in Study 1042-0603 was 90% or greater, and for whom the investigator feels that the subject was compliant with the full dose as prescribed.
- Able to give informed consent in writing, or have a legally authorized representative able to do so, after being properly informed of the nature and risks of the study and prior to engaging in any study-related procedures.
- Currently being treated and maintained with a stable regimen of 1, 2, or 3 anti-epileptic drugs (AED) at a consistent dose for one month prior to study entry.
- Implanted Vagus Nerve Stimulator (VNS) is permitted and will not count towards the number of concomitant AEDs.
- Able and willing to maintain an accurate and complete daily written seizure calendar or has a caregiver who is able and willing to maintain an accurate and complete daily written seizure calendar.
- Able and willing to take drug with food twice daily. Ganaxolone must be administered with food.
- Sexually active women of childbearing potential (WCBP) must be using a medically acceptable method of birth control and have a negative pregnancy test at Visit 1 and at subsequent visits.
Who cannot join
- Have any medical condition that, in the investigator's judgment, is considered to be clinically significant and could potentially affect subject safety or study outcome
- Experienced a Serious Adverse Event or a moderate or severe medically important adverse event judged probably or definitely related to open-label ganaxolone in the previous study, 1042-0603
- Have Alanine transferase (ALT; SGPT) or Aspartate transferase (AST; SGOT) levels > 3 times upper limits of normal (ULN), or total bilirubin >1.5 time ULN during Study 1042-0603.
- Have a history of malignancy within the past 2 years, with the exception of basal cell carcinoma.
- Seizures secondary to illicit drug or alcohol use, infection, neoplasm, demyelinating disease, degenerative neurological disease, or central nervous system (CNS) disease deemed progressive, metabolic illness, or progressive degenerative disease.
- Have active suicidal plan/intent, or have had active suicidal thoughts in the past 6 months. Have a history of an actual suicide attempt in the last 5 years or more than 1 lifetime actual suicide attempt as classified by the Columbia-Suicide Severity Rating Scale (C-SSRS).
Show all 11Show fewer
- Have a history of drug or alcohol abuse within the past 5 years. As with other AEDs, the use of alcohol is not advised.
- Are currently following or planning to follow a ketogenic diet.
- Current use of vigabatrin or ezogabine (retigabine; Potiga; Trobalt) is not permitted.
- Females who are pregnant, currently breastfeeding or planning to become pregnant during the study.
- Inability/unwillingness to withhold grapefruit and grapefruit juice from diet during the entire clinical trial.
Trial locations and contacts
5 sites in 1 country.
5 sites in 1 country
United States5 sites
Texas Epilepsy Group
Dallas, Texas, 75251
Minneapolis Clinic of Neurology
Golden Valley, Minnesota, 55422
Northeast Regional Epilepsy Group
Hackensack, New Jersey, 07601
Bluegrass Epilepsy Research, LLC
Lexington, Kentucky, 40504
Neurological Research Institute
Santa Monica, California, 90404
About this study
Official title: A follow-on, Two-year Open-label Extension Study of Ganaxolone as add-on Therapy in Adult Patients With Drug-resistant Partial-onset Seizures
A follow-on, two-year open-label extension study of ganaxolone as add-on therapy in adult patients with drug-resistant partial-onset seizures
Read the detailed description
This study is a 2-year, open-label continuation for those patients benefiting from ganaxolone treatment after completing Protocol 1042-0603.
- Conditions
- Drug Resistant Partial Onset Seizure
- Being tested
- ganaxolone
- Keywords
- Partial Onset Seizures, complex partial seizures, simple partial seizures, anticonvulsant, ganaxolone, neurosteroid, Marinus, epilepsy
- Medical subject headings
- Seizures, Epilepsies, Partial, Epilepsy
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
ganaxoloneDrug
Also called CCD 1042
225 mg capsules 450 mg to 900 mg 2x/day
| Study group | Role | What they receive |
|---|---|---|
| ganaxolone | Experimental | Up to a maximum of 1800 mg/day 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 28-day Seizure Frequency
Baseline 28-day seizure frequency was calculated as the number of seizures in the Baseline period of Study 1042-0603 (less than or equal to 56 days) divided by the number of days with available seizure data in the Baseline period and multiplied by 28. Post-baseline 28-day seizure frequency was calculated as the number of seizures in the entire treatment period divided by the number of days with available seizure data in the treatment period and multiplied by 28. Baseline was defined as the last non-missing value obtained before the first treatment in the preceding Study 1042-0603. The calculation for percent change from Baseline in 28-day seizure frequency was done as follows for each participant: post-Baseline 28-day seizure frequency minus Baseline 28-day seizure frequency whole divided by Baseline 28-day seizure frequency multiplied by 100 percent.
Measured: Baseline and at Day 28
Secondary outcomes (3)
Number of Participants Who Showed Greater Than or Equal to 50% Reduction in 28-day Seizure Frequent From Baseline
A 50% responder is an individual whose reduction of percent change from Baseline to the end of the open label extension period in 28-day partial-onset seizure (POS) seizure frequency is greater than or equal to 50%.
Measured: Baseline and at Day 28
Number of Participants With Clinical Global Impression of Improvement (CGI-I) Scores
The CGI-I scale is a clinician-rated 7-point scale used to assess how much the participant's illness had improved or worsened relative to a Baseline state at the beginning of the intervention. It was rated as: 1. "very much improved" 2. "much improved" 3. "minimally improved" 4. "no change" 5. "minimally worse" 6. "much worse" 7. "very much worse". Higher scores indicated worse condition. Participants who showed CGI improvement at Week 104 (End of treatment) has been presented.
Measured: At Week 104
Number of Participants With Patient/Caregiver Global Impression of Improvement (PGI-I) Scores
The participant is asked to rate the total improvement of their partial-onset seizures whether or not in the participant's judgment it is due entirely to drug treatment based on a 7-point scale using the markers "very much improved, much improved, slightly improved, no change, slightly worse, much worse, or very much worse" (1 = very much improved; 7 = very much worse). Higher scores indicated worse condition. Participants who showed PGI improvement at Week 104 (End of treatment) has been presented.
Measured: At Week 104
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted11 Aug 2015 (estimated)
- Study startFeb 2015
- Primary completionDec 2016
- Study completionDec 2016
Why it stopped: Ganaxolone missed its primary endpoint in the double-blind portion of the 1042-0603 study. Due to this outcome Marinus discontinued this extension study.
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 Drug Resistant Partial Onset SeizureStudies 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 3
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 26 participants (actual)
Sponsor and investigators
- Lead sponsor
- Marinus Pharmaceuticals
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02519439
- Sponsor's protocol ID
- 1042-0604
- Status last verified
- Jun 2023
- First submitted
- 4 Jun 2015
- Last updated
- 28 Jun 2023
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 28 Jun 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A follow-on, Two-year Open-label Extension Study of Ganaxolone as add-on Therapy in Adult Patients With Drug-resistant Partial-onset Seizures
- ClinicalTrials.gov ID
- NCT02519439
- Acronym / sponsor ID
- 1042-0604
- Conditions
- Drug Resistant Partial Onset Seizure
- Keywords
- Partial Onset Seizures, complex partial seizures, simple partial seizures, anticonvulsant, ganaxolone, neurosteroid, Marinus, epilepsy
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Ganaxolone missed its primary endpoint in the double-blind portion of the 1042-0603 study. Due to this outcome Marinus discontinued this extension study.
- Enrolment
- 26 participants (actual)
- Lead sponsor
- Marinus PharmaceuticalsIndustry (pharma, biotech, devices)
- Study start
- Feb 2015
- Primary completion
- Dec 2016 (actual)
- Study completion
- Dec 2016 (actual)
- First posted
- 11 Aug 2015 (estimated)
- Results
- Posted 14 Feb 2023
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
A follow-on, two-year open-label extension study of ganaxolone as add-on therapy in adult patients with drug-resistant partial-onset seizures
Detailed description
This study is a 2-year, open-label continuation for those patients benefiting from ganaxolone treatment after completing Protocol 1042-0603.
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: ganaxoloneUp to a maximum of 1800 mg/day |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (8 listed)
- Subjects who have completed all scheduled clinical study visits in the previous protocol 1042-0603 and have shown a minimum 35% improvement in mean 28-day seizure frequency over the last three 28-day periods in study 1042-603 as compared to the baseline of study 1042-603.
- Subjects whose daily study drug compliance in Study 1042-0603 was 90% or greater, and for whom the investigator feels that the subject was compliant with the full dose as prescribed.
- Able to give informed consent in writing, or have a legally authorized representative able to do so, after being properly informed of the nature and risks of the study and prior to engaging in any study-related procedures.
- Currently being treated and maintained with a stable regimen of 1, 2, or 3 anti-epileptic drugs (AED) at a consistent dose for one month prior to study entry.
- Implanted Vagus Nerve Stimulator (VNS) is permitted and will not count towards the number of concomitant AEDs.
Key exclusion criteria (11 listed)
- Have any medical condition that, in the investigator's judgment, is considered to be clinically significant and could potentially affect subject safety or study outcome
- Experienced a Serious Adverse Event or a moderate or severe medically important adverse event judged probably or definitely related to open-label ganaxolone in the previous study, 1042-0603
- Have Alanine transferase (ALT; SGPT) or Aspartate transferase (AST; SGOT) levels > 3 times upper limits of normal (ULN), or total bilirubin >1.5 time ULN during Study 1042-0603.
- Have a history of malignancy within the past 2 years, with the exception of basal cell carcinoma.
- Seizures secondary to illicit drug or alcohol use, infection, neoplasm, demyelinating disease, degenerative neurological disease, or central nervous system (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 |
|---|---|---|
| ganaxoloneAlso: CCD 1042225 mg capsules 450 mg to 900 mg 2x/day | Drug | ganaxolone |
Primary outcome
Percent Change From Baseline in 28-day Seizure Frequency
Time frame: Baseline and at Day 28
Baseline 28-day seizure frequency was calculated as the number of seizures in the Baseline period of Study 1042-0603 (less than or equal to 56 days) divided by the number of days with available seizure data in the Baseline period and multiplied by 28. Post-baseline 28-day seizure frequency was calculated as the number of seizures in the entire treatment period divided by the number of days with available seizure data in the treatment period and multiplied by 28. Baseline was defined as the last non-missing value obtained before the first treatment in the preceding Study 1042-0603. The calculation for percent change from Baseline in 28-day seizure frequency was done as follows for each participant: post-Baseline 28-day seizure frequency minus Baseline 28-day seizure frequency whole divided by Baseline 28-day seizure frequency multiplied by 100 percent.
3 secondary outcomes
Number of Participants Who Showed Greater Than or Equal to 50% Reduction in 28-day Seizure Frequent From Baseline
Time frame: Baseline and at Day 28
Number of Participants With Clinical Global Impression of Improvement (CGI-I) Scores
Time frame: At Week 104
Number of Participants With Patient/Caregiver Global Impression of Improvement (PGI-I) Scores
Time frame: At Week 104
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 28 Jun 2023.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
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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.
5 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing ganaxolone in people with Drug Resistant Partial Onset Seizure, run by Marinus Pharmaceuticals at 5 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 8 inclusion and 11 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: stopped early, as of the sponsor's last update on 28 Jun 2023. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 5 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 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
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 14 Feb 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 28-day Seizure Frequency (time frame: Baseline and at Day 28). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 28 Jun 2023.