Trial Outcomes & Findings for A Treatment Use Protocol for Subjects Continuing on From the Open-label Extension 0601 (NCT NCT01002820)
NCT ID: NCT01002820
Last Updated: 2022-09-07
Results Overview
Investigator's global assessment of the patient's response to ganaxolone treatment was collected during the study to support continued trial participation. The investigator's global assessment of the patient's response to ganaxolone treatment was collected during the study to support continued trial participation. Responses categories were Improved Markedly, Improved Moderately, Improved Slightly and No change from baseline.
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
11 participants
Primary outcome timeframe
Screening through 52 weeks
Results posted on
2022-09-07
Participant Flow
The study population was to have completed all scheduled clinical study visits in the previous protocol 1042-0601 and have been deemed eligible by the Investigator including no major adverse events \[AEs\] thought to be drug related and deriving benefit from ganaxolone treatment.
Participant milestones
| Measure |
Ganaxolone
liquid suspension, 50 mg/mL, up to 500 mg/day TID or 1500 mg/day, up to 1 year
|
|---|---|
|
Overall Study
STARTED
|
11
|
|
Overall Study
COMPLETED
|
7
|
|
Overall Study
NOT COMPLETED
|
4
|
Reasons for withdrawal
| Measure |
Ganaxolone
liquid suspension, 50 mg/mL, up to 500 mg/day TID or 1500 mg/day, up to 1 year
|
|---|---|
|
Overall Study
Adverse Event
|
1
|
|
Overall Study
Withdrawal by Subject
|
2
|
|
Overall Study
Lack of Efficacy
|
1
|
Baseline Characteristics
A Treatment Use Protocol for Subjects Continuing on From the Open-label Extension 0601
Baseline characteristics by cohort
| Measure |
Ganaxolone
n=11 Participants
liquid suspension, 50 mg/mL, up to 500 mg/day TID or 1500 mg/day, up to 1 year
|
|---|---|
|
Age, Categorical
<=18 years
|
0 Participants
n=5 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
11 Participants
n=5 Participants
|
|
Age, Categorical
>=65 years
|
0 Participants
n=5 Participants
|
|
Age, Continuous
|
44.5 years
STANDARD_DEVIATION 10.35 • n=5 Participants
|
|
Sex: Female, Male
Female
|
8 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
3 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
1 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
10 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Asian
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Black or African American
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
White
|
10 Participants
n=5 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
1 Participants
n=5 Participants
|
PRIMARY outcome
Timeframe: Screening through 52 weeksPopulation: ITT Population
Investigator's global assessment of the patient's response to ganaxolone treatment was collected during the study to support continued trial participation. The investigator's global assessment of the patient's response to ganaxolone treatment was collected during the study to support continued trial participation. Responses categories were Improved Markedly, Improved Moderately, Improved Slightly and No change from baseline.
Outcome measures
| Measure |
Ganaxolone
n=11 Participants
liquid suspension, 50 mg/mL, up to 500 mg/day TID or 1500 mg/day, up to 1 year
|
|---|---|
|
Investigators Global Assessment
No change from baseline - Visit 1 - Screening
|
1 Participants
|
|
Investigators Global Assessment
Improved Markedly - Visit 1 - Screening
|
6 Participants
|
|
Investigators Global Assessment
Improved Moderately - Visit 1 - Screening
|
4 Participants
|
|
Investigators Global Assessment
Improved Slightly - Visit 1 - Screening
|
0 Participants
|
|
Investigators Global Assessment
Improved Markedly - Visit 2 - Week 26
|
3 Participants
|
|
Investigators Global Assessment
Improved Moderately - Visit 2 - Week 26
|
4 Participants
|
|
Investigators Global Assessment
Improved Slightly - Visit 2 - Week 26
|
0 Participants
|
|
Investigators Global Assessment
No change from baseline - Visit 2 - Week 26
|
0 Participants
|
|
Investigators Global Assessment
Improved Markedly - Visit 3 - Week 52 or Taper Visit
|
2 Participants
|
|
Investigators Global Assessment
Improved Moderately - Visit 3 - Week 52 or Taper Visit
|
4 Participants
|
|
Investigators Global Assessment
Improved Slightly - Visit 3 - Week 52 or Taper Visit
|
1 Participants
|
|
Investigators Global Assessment
No change from baseline - Visit 3 - Week 52 or Taper Visit
|
1 Participants
|
Adverse Events
Ganaxolone
Serious events: 1 serious events
Other events: 8 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
Ganaxolone
n=11 participants at risk
liquid suspension, 50 mg/mL, up to 500 mg/day TID or 1500 mg/day, up to 1 year
|
|---|---|
|
Nervous system disorders
Tension headache
|
9.1%
1/11 • Screening (0601 visit 12/last visit) through Visit 4 (week 56 - last visit)
|
|
Nervous system disorders
Transient ischemic attack
|
9.1%
1/11 • Screening (0601 visit 12/last visit) through Visit 4 (week 56 - last visit)
|
|
Vascular disorders
Aortic aneurysm
|
9.1%
1/11 • Screening (0601 visit 12/last visit) through Visit 4 (week 56 - last visit)
|
Other adverse events
| Measure |
Ganaxolone
n=11 participants at risk
liquid suspension, 50 mg/mL, up to 500 mg/day TID or 1500 mg/day, up to 1 year
|
|---|---|
|
Gastrointestinal disorders
Diarrhea
|
9.1%
1/11 • Screening (0601 visit 12/last visit) through Visit 4 (week 56 - last visit)
|
|
Gastrointestinal disorders
Vomiting
|
9.1%
1/11 • Screening (0601 visit 12/last visit) through Visit 4 (week 56 - last visit)
|
|
General disorders
Irritability
|
18.2%
2/11 • Screening (0601 visit 12/last visit) through Visit 4 (week 56 - last visit)
|
|
General disorders
Fatigue
|
9.1%
1/11 • Screening (0601 visit 12/last visit) through Visit 4 (week 56 - last visit)
|
|
General disorders
Oedema Peripheral
|
9.1%
1/11 • Screening (0601 visit 12/last visit) through Visit 4 (week 56 - last visit)
|
|
Infections and infestations
Nail Tinea
|
9.1%
1/11 • Screening (0601 visit 12/last visit) through Visit 4 (week 56 - last visit)
|
|
Infections and infestations
Tinea Pedis
|
9.1%
1/11 • Screening (0601 visit 12/last visit) through Visit 4 (week 56 - last visit)
|
|
Infections and infestations
Urinary Tract Infection
|
9.1%
1/11 • Screening (0601 visit 12/last visit) through Visit 4 (week 56 - last visit)
|
|
Injury, poisoning and procedural complications
Contusion
|
9.1%
1/11 • Screening (0601 visit 12/last visit) through Visit 4 (week 56 - last visit)
|
|
Injury, poisoning and procedural complications
Skin Laceration
|
9.1%
1/11 • Screening (0601 visit 12/last visit) through Visit 4 (week 56 - last visit)
|
|
Nervous system disorders
Headache
|
18.2%
2/11 • Screening (0601 visit 12/last visit) through Visit 4 (week 56 - last visit)
|
|
Nervous system disorders
Complex Partial Seizures
|
9.1%
1/11 • Screening (0601 visit 12/last visit) through Visit 4 (week 56 - last visit)
|
|
Nervous system disorders
Convulsion
|
9.1%
1/11 • Screening (0601 visit 12/last visit) through Visit 4 (week 56 - last visit)
|
|
Nervous system disorders
Intracranial Aneurysm
|
9.1%
1/11 • Screening (0601 visit 12/last visit) through Visit 4 (week 56 - last visit)
|
|
Nervous system disorders
Somnolence
|
9.1%
1/11 • Screening (0601 visit 12/last visit) through Visit 4 (week 56 - last visit)
|
|
Nervous system disorders
Syncope
|
9.1%
1/11 • Screening (0601 visit 12/last visit) through Visit 4 (week 56 - last visit)
|
|
Nervous system disorders
Tension Headache
|
9.1%
1/11 • Screening (0601 visit 12/last visit) through Visit 4 (week 56 - last visit)
|
|
Nervous system disorders
Transient Ischaemic Attack
|
9.1%
1/11 • Screening (0601 visit 12/last visit) through Visit 4 (week 56 - last visit)
|
|
Reproductive system and breast disorders
Menometrorrhagia
|
9.1%
1/11 • Screening (0601 visit 12/last visit) through Visit 4 (week 56 - last visit)
|
|
Skin and subcutaneous tissue disorders
Rash Erythematous
|
9.1%
1/11 • Screening (0601 visit 12/last visit) through Visit 4 (week 56 - last visit)
|
|
Vascular disorders
Aortic Aneurysm
|
9.1%
1/11 • Screening (0601 visit 12/last visit) through Visit 4 (week 56 - last visit)
|
Additional Information
Marinus Clinical Trials Submission Manager
Marinus Pharmaceuticals, Inc.
Phone: 484-801-4670
Email: [email protected]
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place