A Phase 2 interventional study of Pregnenolone in Autistic Disorder, sponsored by Stanford University. Completed at 1 site in United States. Open to participants aged 18 Years to 45 Years. Per ClinicalTrials.gov, last updated 2017-03-29.
Sponsored by Stanford University · Phase 2, Interventional, and Treatment
This study will assess the tolerability and effectiveness of pregnenolone in the treatment of behavioral deficits in adults with autism. Pregnenolone is a naturally occurring hormone found in the body which has been shown to help with the function of nerve cells. It is also shown to modulate the activity of certain brain receptors implicated in autism. We hope to examine the tolerability of pregnenolone in adults with autism.
This study will assess the tolerability and effectiveness of pregnenolone in the treatment of behavioral deficits in adults with autism. Pregnenolone is a naturally occurring hormone found in the body which has been shown to help with the function of nerve cells. It is also shown to modulate the activity of certain brain receptors implicated in autism.
Pregnenolone has been used safely in research studies involving individuals with schizophrenia. In the proposed trial, we hope to examine the tolerability of pregnenolone in adults with autism. We hope to see improvement in behavioral outcomes as measured by standardized behavioral measures. Further, we will measure concentrations of pregnenolone and related neuroactive compounds in the blood. The use of pregnenolone has been studied in a number of mental disorders but not autism. Thus, we hope the study will identify new avenues of research for the treatment of autism.
1,344 studies on the registry are indexed under Autistic Disorder; 334 are open to participants now.
This study's enrollment of 15 is below the median of 45 across 1,044 interventional studies indexed under Autistic Disorder.
Browse Autistic Disorder studies →Stanford University is the lead sponsor of 2,117 studies on the registry; 425 are open to participants now.
Of its 259 completed or terminated interventional studies of FDA-regulated products, 197 (76%) have results posted.
Counted across the registry records on this site, refreshed daily.
Exclusion Criteria:
Pregnenolone up to 500 mg per day
Drug: Pregnenolone
With Baseline serving as approximately day 1, 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.
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)
Time frame: 2, 4, 6, 8, 10, 12, and 16 weeks
Social Responsiveness Scale (SRS) Total Score
SRS total score (total range 0-195); higher scores mean more abnormal social behaviors.
Time frame: 12 weeks
Sensory Profile Questionnaire Total Score
scores on a scale (range: 38-190); lower scores mean more abnormal sensory problems.
Time frame: 12
Vineland Adaptive Behavior Scale
Adaptive Behavior Composite Score (score range 20-160); higher scores mean more typical adaptive behaviors.
Time frame: 12 weeks
Repetitive Behavior Scale
Time frame: 12 weeks
Pregnenolone Level in Peripheral Blood as Measured at Baseline and After 12 Weeks
Time frame: 12 weeks
Participants recruited between November 2011 and September 2013 at Stanford University.
| Milestone | Pregnenolone |
|---|---|
| Started | 15 |
| Number screened | 15 |
| Completed | 10 |
| Not completed | 5 |
| Withdrew: Inclusion/exclusion criteria not met | 3 |
| Withdrew: Withdrawal by subject | 1 |
| Withdrew: Worsening of baseline behavior | 1 |
| participants | Pregnenolone |
|---|---|
| Tiredness | 1 |
| Diarrhea | 2 |
| Depressive Affect | 2 |
| Increased Excitement/Agitation | 3 |
| Sleep Problems | 1 |
| Drowsiness | 1 |
| Anorexia/Decreased Appetite | 2 |
| Increased Motor Activity | 1 |
| Sweating | 1 |
| Constipation | 1 |
| Tremor | 1 |
SRS total score (total range 0-195); higher scores mean more abnormal social behaviors.
| SRS total score (total range 0-195) | Pregnenolone |
|---|---|
| Baseline SRS Total Score | 84.9 ± 8.1 |
| Week 12 SRS Total Score | 84.5 ± 9.2 |
scores on a scale (range: 38-190); lower scores mean more abnormal sensory problems.
| scores on a scale (range: 38-190) | Pregnenolone |
|---|---|
| Baseline Score on the Sensory Profile | 137.7 ± 21.5 |
| Week 12 Score on the Sensory Profile | 147.6 ± 15.3 |
Adaptive Behavior Composite Score (score range 20-160); higher scores mean more typical adaptive behaviors.
| score (range 20-160) | Pregnenolone |
|---|---|
| Baseline Vineland Adaptive Behavior Score | 37.3 ± 13.1 |
| Week 12 Vineland Adaptive Behavior Score | 42.9 ± 16.5 |
No measurements were reported for this outcome.
| ng/ml | Pregnenolone |
|---|---|
| Baseline level | 1.9 ± 0.7 |
| Week 12 | 7.0 ± 4.1 |
Collected over Time Frame: Baseline, 2, 4, 6, 8, 10, 12, and 16 weeks. Non-serious events are listed at a 0% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| Pregnenolone | — | 0/12 (0%) | 6/12 (50%) |
| Event | Pregnenolone |
|---|---|
| Increased Excitement/AgitationPsychiatric disorders | 3/12 |
| DiarrheaGastrointestinal disorders | 2/12 |
| Depressive AffectPsychiatric disorders | 2/12 |
| Anorexia/Decreased AppetiteGastrointestinal disorders | 2/12 |
| TirednessGeneral disorders | 1/12 |
| Sleep ProblemsGeneral disorders | 1/12 |
| DrowsinessGeneral disorders | 1/12 |
| Increased Motor ActivityGeneral disorders | 1/12 |
| SweatingGeneral disorders | 1/12 |
| ConstipationGastrointestinal disorders | 1/12 |
15 subjects were consented; however, 3 of those subjects did not meet the inclusion/exclusion requirements so they could not participate in the trial and were not included in the baseline analysis population.
| Age, Categorical(Participants) | Pregnenolone |
|---|---|
| <=18 years | 0 |
| Between 18 and 65 years | 12 |
| >=65 years | 0 |
| Age, Continuous(years) | Pregnenolone |
|---|---|
| Mean | 22.5 ± 5.8 |
| Sex: Female, Male(Participants) | Pregnenolone |
|---|---|
| Female | 2 |
| Male | 10 |
| Region of Enrollment(participants) | Pregnenolone |
|---|---|
| United States | 12 |
Plan to share: Yes
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