A Phase 3 interventional study of ganaxolone in Drug Resistant Partial Onset Seizure, sponsored by Marinus Pharmaceuticals. Terminated at 5 sites in United States. Open to participants aged 18 Years and older. Per ClinicalTrials.gov, last updated 2023-06-28.
Sponsored by Marinus Pharmaceuticals · Phase 3, Interventional, and Treatment
A follow-on, two-year open-label extension study of ganaxolone as add-on therapy in adult patients with drug-resistant partial-onset seizures
This study is a 2-year, open-label continuation for those patients benefiting from ganaxolone treatment after completing Protocol 1042-0603.
881 studies on the registry are indexed under Seizures; 143 are open to participants now.
This study's enrollment of 26 is below the median of 64 across 610 interventional studies indexed under Seizures.
Browse Seizures studies →Marinus Pharmaceuticals is the lead sponsor of 22 studies on the registry; none are open to participants now.
Of its 9 completed or terminated interventional studies of FDA-regulated products, 9 (100%) have results posted.
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Exclusion Criteria:
Up to a maximum of 1800 mg/day
Drug: ganaxolone
225 mg capsules 450 mg to 900 mg 2x/day
Also known as: CCD 1042
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.
Time frame: Baseline and at Day 28
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%.
Time frame: 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.
Time frame: 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.
Time frame: At Week 104
This was an open-label extension of Study 1042-0603, providing a two-year adjunctive ganaxolone treatment to adult participants with epilepsy consisting of partial-onset seizures (POS).
| Milestone | Ganaxolone |
|---|---|
| Started | 26 |
| Completed | 5 |
| Not completed | 21 |
| Withdrew: Study termination | 19 |
| Withdrew: Withdrawal by subject | 1 |
| Withdrew: Adverse event | 1 |
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.
| Percent change | Ganaxolone |
|---|---|
| Percent Change From Baseline in 28-day Seizure Frequency | -41.86 ± 44.913 |
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%.
| Participants | Ganaxolone |
|---|---|
| Number of Participants Who Showed Greater Than or Equal to 50% Reduction in 28-day Seizure Frequent From Baseline | 14 |
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.
| Participants | Ganaxolone |
|---|---|
| Very much improved | 1 |
| Much improved | 9 |
| Minimally improved | 11 |
| No change | 2 |
| Minimally worse | 0 |
| Much worse | 0 |
| Very much worse | 0 |
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.
| Participants | Ganaxolone |
|---|---|
| Very much improved | 4 |
| Much improved | 8 |
| Minimally improved | 10 |
| No change | 1 |
| Minimally worse | 0 |
| Much worse | 0 |
| Very much worse | 0 |
Collected over Up to Week 104. Non-serious events are listed at a 0% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| Ganaxolone | 0/26 (0%) | 1/26 (3.8%) | 10/26 (38.5%) |
| Event | Ganaxolone |
|---|---|
| Pulmonary embolism/Respiratory, thoracic and mediastinal disorders | 1/26 |
| Event | Ganaxolone |
|---|---|
| HeadacheNervous system disorders | 3/26 |
| DizzinessNervous system disorders | 1/26 |
| SyncopeNervous system disorders | 1/26 |
| CellulitisInfections and infestations | 1/26 |
| NasopharyngitisInfections and infestations | 1/26 |
| Tooth infectionInfections and infestations | 1/26 |
| Upper respiratory tract infectionInfections and infestations | 1/26 |
| FallInjury, poisoning and procedural complications | 1/26 |
| Ligament sprainInjury, poisoning and procedural complications | 1/26 |
| Lip injuryInjury, poisoning and procedural complications | 1/26 |
Safety Population: All participants who signed informed consent and took one dose of study medication in this study.
| Age, Continuous(Years) | Ganaxolone |
|---|---|
| Mean | 44.3 ± 15.03 |
| Sex: Female, Male(Participants) | Ganaxolone |
|---|---|
| Female | 11 |
| Male | 15 |
| Ethnicity (NIH/OMB)(Participants) | Ganaxolone |
|---|---|
| Hispanic or Latino | 3 |
| Not Hispanic or Latino | 23 |
| Unknown or Not Reported | 0 |
| Race (NIH/OMB)(Participants) | Ganaxolone |
|---|---|
| American Indian or Alaska Native | 0 |
| Asian | 0 |
| Native Hawaiian or Other Pacific Islander | 0 |
| Black or African American | 0 |
| White | 24 |
| More than one race | 0 |
| Unknown or Not Reported | 2 |
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Marinus Pharmaceuticals