Trial Outcomes & Findings for Proof-Of-Concept Investigation With a Neurosteroid Analog (Ganaxolone) as a Smoking Cessation Candidate (NCT NCT01857531)
NCT ID: NCT01857531
Last Updated: 2014-09-08
Results Overview
To evaluate the effects of ganaxolone on ad lib smoking by looking at the percent change in expired air CO at the end of week two (relative to baseline).
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
36 participants
Primary outcome timeframe
Baseline and 2 Weeks
Results posted on
2014-09-08
Participant Flow
Participant milestones
| Measure |
Ganaxolone -- Nicotine Patch
Pre-Quit Period:
Ganaxolone -- 400mg daily (200mg bid) for the first 3 days, 800mg daily (400mg bid) for the next 3 days and 1200mg daily (600mg bid) for the remainder of the first 2 wks.
Nicotine Patches -- 21mg/24h nicotine patches applied daily during wks. 3 and 4.
Post-Quit Period:
Ganaxolone -- 1200mg daily for wk. 5, 800mg daily for 3 days, and 400mg daily for 3 days.
Nicotine Patches -- 21mg/24h for 4 wks., 14mg/24h for 1 wk., and 7mg/24h for 1 wk.
|
|---|---|
|
Overall Study
STARTED
|
16
|
|
Overall Study
COMPLETED
|
12
|
|
Overall Study
NOT COMPLETED
|
4
|
Reasons for withdrawal
| Measure |
Ganaxolone -- Nicotine Patch
Pre-Quit Period:
Ganaxolone -- 400mg daily (200mg bid) for the first 3 days, 800mg daily (400mg bid) for the next 3 days and 1200mg daily (600mg bid) for the remainder of the first 2 wks.
Nicotine Patches -- 21mg/24h nicotine patches applied daily during wks. 3 and 4.
Post-Quit Period:
Ganaxolone -- 1200mg daily for wk. 5, 800mg daily for 3 days, and 400mg daily for 3 days.
Nicotine Patches -- 21mg/24h for 4 wks., 14mg/24h for 1 wk., and 7mg/24h for 1 wk.
|
|---|---|
|
Overall Study
Withdrawal by Subject
|
4
|
Baseline Characteristics
Proof-Of-Concept Investigation With a Neurosteroid Analog (Ganaxolone) as a Smoking Cessation Candidate
Baseline characteristics by cohort
| Measure |
Ganaxolone -- Nicotine Patch
n=16 Participants
Pre-Quit Period:
Ganaxolone -- 400mg daily for the first 3 days, 800mg daily for the next 3 days and 1200mg daily for the remainder of the first 2 wks.
Nicotine Patches -- 21mg/24h nicotine patches applied daily during wks. 3 and 4.
Post-Quit Period:
Ganaxolone -- 1200mg daily for wk. 5, 800mg daily for 3 days, and 400mg daily for 3 days.
Nicotine Patches -- 21mg/24h for 4 wks., 14mg/24h for 1 wk., and 7mg/24h for 1 wk.
|
|---|---|
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Age, Continuous
|
47.44 years
STANDARD_DEVIATION 9.94 • n=99 Participants
|
|
Age, Categorical
<=18 years
|
0 Participants
n=99 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
16 Participants
n=99 Participants
|
|
Age, Categorical
>=65 years
|
0 Participants
n=99 Participants
|
|
Sex: Female, Male
Female
|
14 Participants
n=99 Participants
|
|
Sex: Female, Male
Male
|
2 Participants
n=99 Participants
|
|
Region of Enrollment
United States
|
16 participants
n=99 Participants
|
PRIMARY outcome
Timeframe: Baseline and 2 WeeksTo evaluate the effects of ganaxolone on ad lib smoking by looking at the percent change in expired air CO at the end of week two (relative to baseline).
Outcome measures
| Measure |
Ganaxolone -- Nicotine Patch
n=16 Participants
Pre-Quit Period:
Ganaxolone -- 400mg daily (200mg bid) for the first 3 days, 800mg daily (400mg bid) for the next 3 days and 1200mg daily (600mg bid) for the remainder of the first 2 wks.
Nicotine Patches -- 21mg/24h nicotine patches applied daily during wks. 3 and 4.
Post-Quit Period:
Ganaxolone -- 1200mg daily for wk. 5, 800mg daily for 3 days, and 400mg daily for 3 days.
Nicotine Patches -- 21mg/24h for 4 wks., 14mg/24h for 1 wk., and 7mg/24h for 1 wk.
|
|---|---|
|
Percentage of Change in Expired Air Carbon Monoxide (CO) at End of Week 2
|
-0.52 percentage change
Standard Error 7.38
|
SECONDARY outcome
Timeframe: Baseline and 4 WeeksPopulation: Three of the original 16 subjects dropped out prior to week 4, so only 13 subjects had a week 4 CO reading and could be included in this analysis.
To evaluate the effects of ganaxolone as an augmentation treatment in conjunction with nicotine patch by looking at the percent change in expired air CO at the end of week four (relative to baseline).
Outcome measures
| Measure |
Ganaxolone -- Nicotine Patch
n=13 Participants
Pre-Quit Period:
Ganaxolone -- 400mg daily (200mg bid) for the first 3 days, 800mg daily (400mg bid) for the next 3 days and 1200mg daily (600mg bid) for the remainder of the first 2 wks.
Nicotine Patches -- 21mg/24h nicotine patches applied daily during wks. 3 and 4.
Post-Quit Period:
Ganaxolone -- 1200mg daily for wk. 5, 800mg daily for 3 days, and 400mg daily for 3 days.
Nicotine Patches -- 21mg/24h for 4 wks., 14mg/24h for 1 wk., and 7mg/24h for 1 wk.
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|---|---|
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Percentage of Change in Expired Air Carbon Monoxide (CO) at End of Week 4
|
-39.40 percentage change
Standard Error 8.96
|
OTHER_PRE_SPECIFIED outcome
Timeframe: 2 Weeks post quitContinuous two week abstinence from smoking at the first post-quit visit (approximately 2 weeks post quit date), based on self-reported abstinence confirmed by expired air CO.
Outcome measures
| Measure |
Ganaxolone -- Nicotine Patch
n=16 Participants
Pre-Quit Period:
Ganaxolone -- 400mg daily (200mg bid) for the first 3 days, 800mg daily (400mg bid) for the next 3 days and 1200mg daily (600mg bid) for the remainder of the first 2 wks.
Nicotine Patches -- 21mg/24h nicotine patches applied daily during wks. 3 and 4.
Post-Quit Period:
Ganaxolone -- 1200mg daily for wk. 5, 800mg daily for 3 days, and 400mg daily for 3 days.
Nicotine Patches -- 21mg/24h for 4 wks., 14mg/24h for 1 wk., and 7mg/24h for 1 wk.
|
|---|---|
|
Number of Participants Completing Continuous 2-week Abstinence From Smoking
|
3 participants
Interval 0.65 to 7.3
|
OTHER_PRE_SPECIFIED outcome
Timeframe: 7 day point abstinence from smoking at 2 weeks post quitPoint abstinence from smoking two weeks post quit, based on self-reported abstinence during last seven days confirmed by expired air CO at first post-quit visit.
Outcome measures
| Measure |
Ganaxolone -- Nicotine Patch
n=16 Participants
Pre-Quit Period:
Ganaxolone -- 400mg daily (200mg bid) for the first 3 days, 800mg daily (400mg bid) for the next 3 days and 1200mg daily (600mg bid) for the remainder of the first 2 wks.
Nicotine Patches -- 21mg/24h nicotine patches applied daily during wks. 3 and 4.
Post-Quit Period:
Ganaxolone -- 1200mg daily for wk. 5, 800mg daily for 3 days, and 400mg daily for 3 days.
Nicotine Patches -- 21mg/24h for 4 wks., 14mg/24h for 1 wk., and 7mg/24h for 1 wk.
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|---|---|
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Number of Participants Completing Point Abstinence From Smoking Two Weeks After Quitting
|
4 participants
Interval 1.16 to 8.38
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OTHER_PRE_SPECIFIED outcome
Timeframe: 6 Weeks post quitPopulation: Because 6-week abstinence is counted backward from the final study visit, one additional subject (n=4) qualified as abstinent than at 2-weeks post-quit (n=3) due to the fact that the day(s) that one of the subjects smoked did not fall into the range evaluated at 6 weeks.
Continuous six week abstinence from smoking at final study visit (approximately 6 weeks post quit date), based on self-reported abstinence confirmed by expired air CO
Outcome measures
| Measure |
Ganaxolone -- Nicotine Patch
n=16 Participants
Pre-Quit Period:
Ganaxolone -- 400mg daily (200mg bid) for the first 3 days, 800mg daily (400mg bid) for the next 3 days and 1200mg daily (600mg bid) for the remainder of the first 2 wks.
Nicotine Patches -- 21mg/24h nicotine patches applied daily during wks. 3 and 4.
Post-Quit Period:
Ganaxolone -- 1200mg daily for wk. 5, 800mg daily for 3 days, and 400mg daily for 3 days.
Nicotine Patches -- 21mg/24h for 4 wks., 14mg/24h for 1 wk., and 7mg/24h for 1 wk.
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|---|---|
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Number of Participants Completing Continuous 6-week Abstinence From Smoking
|
4 participants
Interval 1.16 to 8.38
|
OTHER_PRE_SPECIFIED outcome
Timeframe: 4 Week abstinence from smoking at 6 weeks post quitEnd of treatment abstinence from smoking during the last four weeks of treatment, based on self-reported abstinence during last four weeks confirmed by expired air CO
Outcome measures
| Measure |
Ganaxolone -- Nicotine Patch
n=16 Participants
Pre-Quit Period:
Ganaxolone -- 400mg daily (200mg bid) for the first 3 days, 800mg daily (400mg bid) for the next 3 days and 1200mg daily (600mg bid) for the remainder of the first 2 wks.
Nicotine Patches -- 21mg/24h nicotine patches applied daily during wks. 3 and 4.
Post-Quit Period:
Ganaxolone -- 1200mg daily for wk. 5, 800mg daily for 3 days, and 400mg daily for 3 days.
Nicotine Patches -- 21mg/24h for 4 wks., 14mg/24h for 1 wk., and 7mg/24h for 1 wk.
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|---|---|
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Number of Participants Completing Abstinence From Smoking During the Last Four Weeks of Treatment
|
5 participants
Interval 1.76 to 9.39
|
Adverse Events
Ganaxolone -- Nicotine Patch
Serious events: 0 serious events
Other events: 16 other events
Deaths: 0 deaths
Serious adverse events
Adverse event data not reported
Other adverse events
| Measure |
Ganaxolone -- Nicotine Patch
n=16 participants at risk
Pre-Quit Period:
Ganaxolone -- 400mg daily (200mg bid) for the first 3 days, 800mg daily (400mg bid) for the next 3 days and 1200mg daily (600mg bid) for the remainder of the first 2 wks.
Nicotine Patches -- 21mg/24h nicotine patches applied daily during wks. 3 and 4.
Post-Quit Period:
Ganaxolone -- 1200mg daily for wk. 5, 800mg daily for 3 days, and 400mg daily for 3 days.
Nicotine Patches -- 21mg/24h for 4 wks., 14mg/24h for 1 wk., and 7mg/24h for 1 wk.
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|---|---|
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Nervous system disorders
Headache
|
12.5%
2/16 • Number of events 2 • Subjects were receiving study drugs for ten weeks. At each study session during this period subjects completed a questionnaire about side effects. Subjects were also told to contact us between study sessions if side effects were bothersome.
|
|
Nervous system disorders
Fatigue
|
68.8%
11/16 • Number of events 15 • Subjects were receiving study drugs for ten weeks. At each study session during this period subjects completed a questionnaire about side effects. Subjects were also told to contact us between study sessions if side effects were bothersome.
|
|
Nervous system disorders
Dizziness
|
25.0%
4/16 • Number of events 5 • Subjects were receiving study drugs for ten weeks. At each study session during this period subjects completed a questionnaire about side effects. Subjects were also told to contact us between study sessions if side effects were bothersome.
|
|
Nervous system disorders
Loss of balance
|
6.2%
1/16 • Number of events 1 • Subjects were receiving study drugs for ten weeks. At each study session during this period subjects completed a questionnaire about side effects. Subjects were also told to contact us between study sessions if side effects were bothersome.
|
|
Psychiatric disorders
Anxiety
|
12.5%
2/16 • Number of events 2 • Subjects were receiving study drugs for ten weeks. At each study session during this period subjects completed a questionnaire about side effects. Subjects were also told to contact us between study sessions if side effects were bothersome.
|
|
Psychiatric disorders
Depression
|
6.2%
1/16 • Number of events 1 • Subjects were receiving study drugs for ten weeks. At each study session during this period subjects completed a questionnaire about side effects. Subjects were also told to contact us between study sessions if side effects were bothersome.
|
|
Nervous system disorders
Irritability
|
6.2%
1/16 • Number of events 1 • Subjects were receiving study drugs for ten weeks. At each study session during this period subjects completed a questionnaire about side effects. Subjects were also told to contact us between study sessions if side effects were bothersome.
|
|
Nervous system disorders
Insomnia
|
6.2%
1/16 • Number of events 3 • Subjects were receiving study drugs for ten weeks. At each study session during this period subjects completed a questionnaire about side effects. Subjects were also told to contact us between study sessions if side effects were bothersome.
|
|
Nervous system disorders
Nightmares
|
6.2%
1/16 • Number of events 1 • Subjects were receiving study drugs for ten weeks. At each study session during this period subjects completed a questionnaire about side effects. Subjects were also told to contact us between study sessions if side effects were bothersome.
|
|
Nervous system disorders
Vivid dreams
|
18.8%
3/16 • Number of events 5 • Subjects were receiving study drugs for ten weeks. At each study session during this period subjects completed a questionnaire about side effects. Subjects were also told to contact us between study sessions if side effects were bothersome.
|
|
Musculoskeletal and connective tissue disorders
Muscle / joint pain
|
6.2%
1/16 • Number of events 1 • Subjects were receiving study drugs for ten weeks. At each study session during this period subjects completed a questionnaire about side effects. Subjects were also told to contact us between study sessions if side effects were bothersome.
|
|
General disorders
Thirst
|
18.8%
3/16 • Number of events 4 • Subjects were receiving study drugs for ten weeks. At each study session during this period subjects completed a questionnaire about side effects. Subjects were also told to contact us between study sessions if side effects were bothersome.
|
|
General disorders
Mouth irritation
|
6.2%
1/16 • Number of events 2 • Subjects were receiving study drugs for ten weeks. At each study session during this period subjects completed a questionnaire about side effects. Subjects were also told to contact us between study sessions if side effects were bothersome.
|
|
General disorders
Dry mouth
|
6.2%
1/16 • Number of events 1 • Subjects were receiving study drugs for ten weeks. At each study session during this period subjects completed a questionnaire about side effects. Subjects were also told to contact us between study sessions if side effects were bothersome.
|
|
Skin and subcutaneous tissue disorders
Itching at patch site
|
31.2%
5/16 • Number of events 7 • Subjects were receiving study drugs for ten weeks. At each study session during this period subjects completed a questionnaire about side effects. Subjects were also told to contact us between study sessions if side effects were bothersome.
|
|
Skin and subcutaneous tissue disorders
Rash at patch site
|
12.5%
2/16 • Number of events 4 • Subjects were receiving study drugs for ten weeks. At each study session during this period subjects completed a questionnaire about side effects. Subjects were also told to contact us between study sessions if side effects were bothersome.
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Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place