Trial Outcomes & Findings for A Study of Pregnenolone in the Treatment of Individuals With Autism (NCT NCT01881737)
NCT ID: NCT01881737
Last Updated: 2017-03-29
Results Overview
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
15 participants
Primary outcome timeframe
2, 4, 6, 8, 10, 12, and 16 weeks
Results posted on
2017-03-29
Participant Flow
Participants recruited between November 2011 and September 2013 at Stanford University.
Participant milestones
| Measure |
Pregnenolone
Open-Label Trial
|
|---|---|
|
Overall Study
STARTED
|
15
|
|
Overall Study
Number Screened
|
15
|
|
Overall Study
COMPLETED
|
10
|
|
Overall Study
NOT COMPLETED
|
5
|
Reasons for withdrawal
| Measure |
Pregnenolone
Open-Label Trial
|
|---|---|
|
Overall Study
Inclusion/Exclusion Criteria Not Met
|
3
|
|
Overall Study
Withdrawal by Subject
|
1
|
|
Overall Study
Worsening of baseline behavior
|
1
|
Baseline Characteristics
A Study of Pregnenolone in the Treatment of Individuals With Autism
Baseline characteristics by cohort
| Measure |
Pregnenolone
n=12 Participants
Twice daily intake of orally administered pregnenolone will occur on a schedule consisting of an up-titration followed by a down-titration as described below.
Week 1 and 2: 100 mg Week 3 and 4: 200 mg Week 5 and 6: 300 mg Week 7 and 8: 400 mg Week 9 -12: 500 mg At the end of Week 12, pregnenolone was decreased by 50 mg twice a day every 3 days until it was discontinued.
If the participant is unable to tolerate a specific dose then he/she will be maintained at the highest tolerated dose until down titration occurs.
|
|---|---|
|
Age, Categorical
<=18 years
|
0 Participants
n=5 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
12 Participants
n=5 Participants
|
|
Age, Categorical
>=65 years
|
0 Participants
n=5 Participants
|
|
Age, Continuous
|
22.5 years
STANDARD_DEVIATION 5.8 • n=5 Participants
|
|
Sex: Female, Male
Female
|
2 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
10 Participants
n=5 Participants
|
|
Region of Enrollment
United States
|
12 participants
n=5 Participants
|
PRIMARY outcome
Timeframe: 2, 4, 6, 8, 10, 12, and 16 weeksPopulation: During the 12-week treatment period, two participants dropped out of the study. The follow-up observations for the two participants who dropped out were included in the analyses.
Outcome measures
| Measure |
Pregnenolone
n=12 Participants
Open-Label study
|
|---|---|
|
Number of Participants With Adverse Events According to Dosage Record and Treatment Emergent Symptom (DOTES) as Assessed at All Follow-up Visits (2, 4, 6, 8, 10, 12, and 16 Weeks)
Tiredness
|
1 participants
|
|
Number of Participants With Adverse Events According to Dosage Record and Treatment Emergent Symptom (DOTES) as Assessed at All Follow-up Visits (2, 4, 6, 8, 10, 12, and 16 Weeks)
Diarrhea
|
2 participants
|
|
Number of Participants With Adverse Events According to Dosage Record and Treatment Emergent Symptom (DOTES) as Assessed at All Follow-up Visits (2, 4, 6, 8, 10, 12, and 16 Weeks)
Depressive Affect
|
2 participants
|
|
Number of Participants With Adverse Events According to Dosage Record and Treatment Emergent Symptom (DOTES) as Assessed at All Follow-up Visits (2, 4, 6, 8, 10, 12, and 16 Weeks)
Increased Excitement/Agitation
|
3 participants
|
|
Number of Participants With Adverse Events According to Dosage Record and Treatment Emergent Symptom (DOTES) as Assessed at All Follow-up Visits (2, 4, 6, 8, 10, 12, and 16 Weeks)
Sleep Problems
|
1 participants
|
|
Number of Participants With Adverse Events According to Dosage Record and Treatment Emergent Symptom (DOTES) as Assessed at All Follow-up Visits (2, 4, 6, 8, 10, 12, and 16 Weeks)
Drowsiness
|
1 participants
|
|
Number of Participants With Adverse Events According to Dosage Record and Treatment Emergent Symptom (DOTES) as Assessed at All Follow-up Visits (2, 4, 6, 8, 10, 12, and 16 Weeks)
Anorexia/Decreased Appetite
|
2 participants
|
|
Number of Participants With Adverse Events According to Dosage Record and Treatment Emergent Symptom (DOTES) as Assessed at All Follow-up Visits (2, 4, 6, 8, 10, 12, and 16 Weeks)
Increased Motor Activity
|
1 participants
|
|
Number of Participants With Adverse Events According to Dosage Record and Treatment Emergent Symptom (DOTES) as Assessed at All Follow-up Visits (2, 4, 6, 8, 10, 12, and 16 Weeks)
Sweating
|
1 participants
|
|
Number of Participants With Adverse Events According to Dosage Record and Treatment Emergent Symptom (DOTES) as Assessed at All Follow-up Visits (2, 4, 6, 8, 10, 12, and 16 Weeks)
Constipation
|
1 participants
|
|
Number of Participants With Adverse Events According to Dosage Record and Treatment Emergent Symptom (DOTES) as Assessed at All Follow-up Visits (2, 4, 6, 8, 10, 12, and 16 Weeks)
Tremor
|
1 participants
|
SECONDARY outcome
Timeframe: 12 weeksSRS total score (total range 0-195); higher scores mean more abnormal social behaviors.
Outcome measures
| Measure |
Pregnenolone
n=12 Participants
Open-Label study
|
|---|---|
|
Social Responsiveness Scale (SRS) Total Score
Baseline SRS Total Score
|
84.9 SRS total score (total range 0-195)
Standard Deviation 8.1
|
|
Social Responsiveness Scale (SRS) Total Score
Week 12 SRS Total Score
|
84.5 SRS total score (total range 0-195)
Standard Deviation 9.2
|
SECONDARY outcome
Timeframe: 12Population: During the 12-week treatment period, two participants dropped out of the study. The follow-up observations for one of the participants who dropped out were included in the analyses.
scores on a scale (range: 38-190); lower scores mean more abnormal sensory problems.
Outcome measures
| Measure |
Pregnenolone
n=11 Participants
Open-Label study
|
|---|---|
|
Sensory Profile Questionnaire Total Score
Baseline Score on the Sensory Profile
|
137.7 scores on a scale (range: 38-190)
Standard Deviation 21.5
|
|
Sensory Profile Questionnaire Total Score
Week 12 Score on the Sensory Profile
|
147.6 scores on a scale (range: 38-190)
Standard Deviation 15.3
|
SECONDARY outcome
Timeframe: 12 weeksAdaptive Behavior Composite Score (score range 20-160); higher scores mean more typical adaptive behaviors.
Outcome measures
| Measure |
Pregnenolone
n=12 Participants
Open-Label study
|
|---|---|
|
Vineland Adaptive Behavior Scale
Baseline Vineland Adaptive Behavior Score
|
37.3 score (range 20-160)
Standard Deviation 13.1
|
|
Vineland Adaptive Behavior Scale
Week 12 Vineland Adaptive Behavior Score
|
42.9 score (range 20-160)
Standard Deviation 16.5
|
SECONDARY outcome
Timeframe: 12 weeksPopulation: Data were not collected for this Outcome Measure because the total score is not a very valid measure of receptive behaviors.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: 12 weeksPopulation: During the 12-week treatment period, two participants dropped out of the study. The follow-up observations for one of the participants who dropped out were included in the analyses.
Outcome measures
| Measure |
Pregnenolone
n=11 Participants
Open-Label study
|
|---|---|
|
Pregnenolone Level in Peripheral Blood as Measured at Baseline and After 12 Weeks
Baseline level
|
1.9 ng/ml
Standard Deviation 0.7
|
|
Pregnenolone Level in Peripheral Blood as Measured at Baseline and After 12 Weeks
Week 12
|
7.0 ng/ml
Standard Deviation 4.1
|
Adverse Events
Pregnenolone
Serious events: 0 serious events
Other events: 6 other events
Deaths: 0 deaths
Serious adverse events
Adverse event data not reported
Other adverse events
| Measure |
Pregnenolone
n=12 participants at risk
Pregnenolone was not associated with any severe adverse effects. Single episodes of tiredness (n = 1), diarrhea (n = 1), and depressive affect (n = 1) that could possibly be related to pregnenolone were reported. A few other adverse events with remote chance to be related to the medication were reported: increased excitement/agitation (n = 3), sleep problems (n = 1), drowsiness (n = 1), anorexia/decreased appetite (n = 2), increased motor activity (n = 1), sweating (n = 1), constipation (n = 1), diarrhea (n = 1), tremor (n = 1), and depressive affect (n = 1). No significant vital sign or EKG changes occurred in any study participants. No abnormal laboratory tests were caused by pregnenolone.
|
|---|---|
|
General disorders
Tiredness
|
8.3%
1/12 • Number of events 1 • Time Frame: Baseline, 2, 4, 6, 8, 10, 12, and 16 weeks
|
|
Gastrointestinal disorders
Diarrhea
|
16.7%
2/12 • Number of events 2 • Time Frame: Baseline, 2, 4, 6, 8, 10, 12, and 16 weeks
|
|
Psychiatric disorders
Depressive Affect
|
16.7%
2/12 • Number of events 2 • Time Frame: Baseline, 2, 4, 6, 8, 10, 12, and 16 weeks
|
|
Psychiatric disorders
Increased Excitement/Agitation
|
25.0%
3/12 • Number of events 3 • Time Frame: Baseline, 2, 4, 6, 8, 10, 12, and 16 weeks
|
|
General disorders
Sleep Problems
|
8.3%
1/12 • Number of events 1 • Time Frame: Baseline, 2, 4, 6, 8, 10, 12, and 16 weeks
|
|
General disorders
Drowsiness
|
8.3%
1/12 • Number of events 1 • Time Frame: Baseline, 2, 4, 6, 8, 10, 12, and 16 weeks
|
|
Gastrointestinal disorders
Anorexia/Decreased Appetite
|
16.7%
2/12 • Number of events 2 • Time Frame: Baseline, 2, 4, 6, 8, 10, 12, and 16 weeks
|
|
General disorders
Increased Motor Activity
|
8.3%
1/12 • Number of events 1 • Time Frame: Baseline, 2, 4, 6, 8, 10, 12, and 16 weeks
|
|
General disorders
Sweating
|
8.3%
1/12 • Number of events 1 • Time Frame: Baseline, 2, 4, 6, 8, 10, 12, and 16 weeks
|
|
Gastrointestinal disorders
Constipation
|
8.3%
1/12 • Number of events 1 • Time Frame: Baseline, 2, 4, 6, 8, 10, 12, and 16 weeks
|
|
General disorders
Tremor
|
8.3%
1/12 • Number of events 1 • Time Frame: Baseline, 2, 4, 6, 8, 10, 12, and 16 weeks
|
Additional Information
Antonio Hardan, MD
Stanford University School of Medicine
Phone: (650) 736-1235
Email: [email protected]
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place