Trial Outcomes & Findings for Double-blind Placebo Controlled Study of Oxytocin in Fragile X Syndrome (NCT NCT01254045)
NCT ID: NCT01254045
Last Updated: 2020-03-18
Results Overview
Number of times that eye gaze occurred (i.e., participant looked at female experimenter in the eyes) during 10 minute social challenge task (first 5 minutes social proximity, second 5 minutes social interaction). The social challenge task occurred 50 minutes after internasal dose (of placebo, placebo + oxytocin, or oxytocin) at baseline, week 2, and week 3 visits.
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
10 participants
Primary outcome timeframe
baseline, week 2, and week 3
Results posted on
2020-03-18
Participant Flow
Ten enrolled. One on antipsychotic mediation at baseline excluded, and one lost to follow-up after baseline. Eight participants included in data analysis. Each participant received a dose of either oxytocin(OT) 24 international units (IU)/intranasal placebo 24IU, OT 48IU, or placebo 48IU at each of 3 weekly visits.
Participant milestones
| Measure |
Placebo 48IU, Oxytocin 24IU/Placebo 24IU, Oxytocin 48IU
intranasal placebo (48 international units; 4IU per puff) once at baseline, 6 puffs (3 puffs per nostril) from each of 2 bottles; intranasal oxytocin (24 international units; 4IU per puff) and placebo (24 international units; 4IU per puff) once at time 2, 6 puffs (3 puffs per nostril) from each of 2 bottles; intranasal oxytocin (48 international units; 4IU per puff) once at time 3, 6 puffs (3 puffs per nostril) from each of 2 bottles
|
Oxytocin 24IU/Placebo 24IU, Placebo 48IU, Oxytocin 48IU
intranasal oxytocin (24 international units; 4IU per puff) and intranasal placebo (24 international units; 4IU per puff) once at baseline, 6 puffs (3 puffs per nostril) from each of 2 bottles; intranasal placebo (48 international units; 4IU per puff) once at time 2, 6 puffs (3 puffs per nostril) from each of 2 bottles; intranasal oxytocin (48 international units; 4IU per puff) once at time 3, 6 puffs (3 puffs per nostril) from each of 2 bottles
|
Oxytocin 48IU, Oxytocin 24IU/Placebo 24IU, Placebo 48IU
intranasal oxytocin (48 international units; 4IU per puff) once at baseline, 6 puffs (3 puffs per nostril) from each of 2 bottles; intranasal oxytocin (24 international units; 4IU per puff) and intranasal placebo (24 international units; 4IU per puff) once at time 2, 6 puffs (3 puffs per nostril) from each of 2 bottles; intranasal placebo (48 international units; 4IU per puff) once at time 3, 6 puffs (3 puffs per nostril) from each of 2 bottles
|
Oxytocin 24IU/Placebo 24IU, Oxytocin 48IU, Placebo 48IU
intranasal oxytocin (24 international units; 4IU per puff) and intranasal placebo (24 international units; 4IU per puff) once at baseline, 6 puffs (3 puffs per nostril) from each of 2 bottles; intranasal oxytocin (48 international units; 4IU per puff) once at time 2, 6 puffs (3 puffs per nostril) from each of 2 bottles; intranasal placebo (48 international units; 4IU per puff) once at time 3, 6 puffs (3 puffs per nostril) from each of 2 bottles
|
Oxytocin 48IU, Placebo 48IU, Oxytocin 24IU/Placebo 24IU
intranasal oxytocin (48 international units; 4IU per puff) once at baseline, 6 puffs (3 puffs per nostril) from each of 2 bottles; intranasal placebo (48 international units; 4IU per puff) once at time 2, 6 puffs (3 puffs per nostril) from each of 2 bottles ; intranasal oxytocin (24 international units; 4IU per puff) and intranasal placebo (24 international units; 4IU per puff) once at time 3, 6 puffs (3 puffs per nostril) from each of 2 bottles
|
Placebo 48IU, Oxytocin 48IU, Oxytocin 24IU/Placebo 24IU
intranasal placebo (48 international units; 4IU per puff) once at baseline, 6 puffs (3 puffs per nostril) from each of 2 bottles; intranasal oxytocin (48 international units; 4IU per puff) once at time 2, 6 puffs (3 puffs per nostril) from each of 2 bottles; intranasal oxytocin (24 international units; 4IU per puff) and intranasal placebo (24 international units; 4IU per puff) once at time 3, 6 puffs (3 puffs per nostril) from each of 2 bottles
|
|---|---|---|---|---|---|---|
|
First/Baseline (1 Day at Week 1 )
STARTED
|
2
|
2
|
2
|
1
|
1
|
1
|
|
First/Baseline (1 Day at Week 1 )
COMPLETED
|
1
|
2
|
2
|
1
|
1
|
1
|
|
First/Baseline (1 Day at Week 1 )
NOT COMPLETED
|
1
|
0
|
0
|
0
|
0
|
0
|
|
Second/Time 2 (1 Day at Week 2)
STARTED
|
1
|
2
|
2
|
1
|
1
|
1
|
|
Second/Time 2 (1 Day at Week 2)
COMPLETED
|
1
|
2
|
2
|
1
|
1
|
1
|
|
Second/Time 2 (1 Day at Week 2)
NOT COMPLETED
|
0
|
0
|
0
|
0
|
0
|
0
|
|
Third/Time 3 (1 Day at Week 3)
STARTED
|
1
|
2
|
2
|
1
|
1
|
1
|
|
Third/Time 3 (1 Day at Week 3)
COMPLETED
|
1
|
2
|
2
|
1
|
1
|
1
|
|
Third/Time 3 (1 Day at Week 3)
NOT COMPLETED
|
0
|
0
|
0
|
0
|
0
|
0
|
Reasons for withdrawal
| Measure |
Placebo 48IU, Oxytocin 24IU/Placebo 24IU, Oxytocin 48IU
intranasal placebo (48 international units; 4IU per puff) once at baseline, 6 puffs (3 puffs per nostril) from each of 2 bottles; intranasal oxytocin (24 international units; 4IU per puff) and placebo (24 international units; 4IU per puff) once at time 2, 6 puffs (3 puffs per nostril) from each of 2 bottles; intranasal oxytocin (48 international units; 4IU per puff) once at time 3, 6 puffs (3 puffs per nostril) from each of 2 bottles
|
Oxytocin 24IU/Placebo 24IU, Placebo 48IU, Oxytocin 48IU
intranasal oxytocin (24 international units; 4IU per puff) and intranasal placebo (24 international units; 4IU per puff) once at baseline, 6 puffs (3 puffs per nostril) from each of 2 bottles; intranasal placebo (48 international units; 4IU per puff) once at time 2, 6 puffs (3 puffs per nostril) from each of 2 bottles; intranasal oxytocin (48 international units; 4IU per puff) once at time 3, 6 puffs (3 puffs per nostril) from each of 2 bottles
|
Oxytocin 48IU, Oxytocin 24IU/Placebo 24IU, Placebo 48IU
intranasal oxytocin (48 international units; 4IU per puff) once at baseline, 6 puffs (3 puffs per nostril) from each of 2 bottles; intranasal oxytocin (24 international units; 4IU per puff) and intranasal placebo (24 international units; 4IU per puff) once at time 2, 6 puffs (3 puffs per nostril) from each of 2 bottles; intranasal placebo (48 international units; 4IU per puff) once at time 3, 6 puffs (3 puffs per nostril) from each of 2 bottles
|
Oxytocin 24IU/Placebo 24IU, Oxytocin 48IU, Placebo 48IU
intranasal oxytocin (24 international units; 4IU per puff) and intranasal placebo (24 international units; 4IU per puff) once at baseline, 6 puffs (3 puffs per nostril) from each of 2 bottles; intranasal oxytocin (48 international units; 4IU per puff) once at time 2, 6 puffs (3 puffs per nostril) from each of 2 bottles; intranasal placebo (48 international units; 4IU per puff) once at time 3, 6 puffs (3 puffs per nostril) from each of 2 bottles
|
Oxytocin 48IU, Placebo 48IU, Oxytocin 24IU/Placebo 24IU
intranasal oxytocin (48 international units; 4IU per puff) once at baseline, 6 puffs (3 puffs per nostril) from each of 2 bottles; intranasal placebo (48 international units; 4IU per puff) once at time 2, 6 puffs (3 puffs per nostril) from each of 2 bottles ; intranasal oxytocin (24 international units; 4IU per puff) and intranasal placebo (24 international units; 4IU per puff) once at time 3, 6 puffs (3 puffs per nostril) from each of 2 bottles
|
Placebo 48IU, Oxytocin 48IU, Oxytocin 24IU/Placebo 24IU
intranasal placebo (48 international units; 4IU per puff) once at baseline, 6 puffs (3 puffs per nostril) from each of 2 bottles; intranasal oxytocin (48 international units; 4IU per puff) once at time 2, 6 puffs (3 puffs per nostril) from each of 2 bottles; intranasal oxytocin (24 international units; 4IU per puff) and intranasal placebo (24 international units; 4IU per puff) once at time 3, 6 puffs (3 puffs per nostril) from each of 2 bottles
|
|---|---|---|---|---|---|---|
|
First/Baseline (1 Day at Week 1 )
Lost to Follow-up
|
1
|
0
|
0
|
0
|
0
|
0
|
Baseline Characteristics
Double-blind Placebo Controlled Study of Oxytocin in Fragile X Syndrome
Baseline characteristics by cohort
| Measure |
All Study Participants
n=9 Participants
Includes groups randomized to one of six different sequences of three interventions (placebo, oxytocin 24IU, oxytocin 48IU).
|
|---|---|
|
Age, Categorical
<=18 years
|
2 Participants
n=5 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
7 Participants
n=5 Participants
|
|
Age, Categorical
>=65 years
|
0 Participants
n=5 Participants
|
|
Age, Continuous
|
20.89 years
STANDARD_DEVIATION 4.73 • n=5 Participants
|
|
Sex: Female, Male
Female
|
0 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
9 Participants
n=5 Participants
|
|
Region of Enrollment
United States
|
9 participants
n=5 Participants
|
PRIMARY outcome
Timeframe: baseline, week 2, and week 3Number of times that eye gaze occurred (i.e., participant looked at female experimenter in the eyes) during 10 minute social challenge task (first 5 minutes social proximity, second 5 minutes social interaction). The social challenge task occurred 50 minutes after internasal dose (of placebo, placebo + oxytocin, or oxytocin) at baseline, week 2, and week 3 visits.
Outcome measures
| Measure |
Placebo
n=8 Participants
placebo (48IU)
|
Oxytocin 24IU/Placebo 24IU
n=8 Participants
24 international units of intranasal oxytocin and 24 international units of placebo
|
48IU OT
n=8 Participants
48 international units of intranasal oxytocin
|
|---|---|---|---|
|
Eye Contact/Gaze During 10 Minute Social Challenge Task
|
15.4 eye contact/gaze events per 10 minutes
Standard Error 3.5
|
20.9 eye contact/gaze events per 10 minutes
Standard Error 3.6
|
17.0 eye contact/gaze events per 10 minutes
Standard Error 4.5
|
SECONDARY outcome
Timeframe: baseline, week 2, and week 3salivary cortisol level measured immediately before social challenge task and 20 minutes following social challenge task at each time point (i.e., baseline, week 2, and week 3)
Outcome measures
| Measure |
Placebo
n=8 Participants
placebo (48IU)
|
Oxytocin 24IU/Placebo 24IU
n=8 Participants
24 international units of intranasal oxytocin and 24 international units of placebo
|
48IU OT
n=8 Participants
48 international units of intranasal oxytocin
|
|---|---|---|---|
|
Salivary Cortisol
|
6.33 nmol/L
Standard Error .86
|
4.67 nmol/L
Standard Error .56
|
4.37 nmol/L
Standard Error .92
|
Adverse Events
Intervention: Placebo 48IU
Serious events: 0 serious events
Other events: 0 other events
Deaths: 0 deaths
Intervention: Oxytocin 24IU/Placebo 24IU
Serious events: 0 serious events
Other events: 1 other events
Deaths: 0 deaths
Intervention: Oxytocin 48IU
Serious events: 0 serious events
Other events: 0 other events
Deaths: 0 deaths
Serious adverse events
Adverse event data not reported
Other adverse events
| Measure |
Intervention: Placebo 48IU
n=8 participants at risk
Two participants received placebo 48IU at baseline. Three participants received placebo 48IU at Time 2. Three participants received placebo 48IU at Time 3. This study is a cross-over design.
|
Intervention: Oxytocin 24IU/Placebo 24IU
n=8 participants at risk
Three participants received Oxytocin 24IU/Placebo 24IU at baseline. Three participants received Oxytocin 24IU/Placebo 24IU at Time 2. Two participants received Oxytocin 24IU/Placebo 24IU at Time 3. This study is a cross-over design.
|
Intervention: Oxytocin 48IU
n=8 participants at risk
Three participants received Oxytocin 48IU at baseline. Two participants received Oxytocin 48IU at Time 2. Three participants received Oxytocin 48IU at Time 3. This study is a cross-over design.
|
|---|---|---|---|
|
Nervous system disorders
Bell's palsy
|
0.00%
0/8
|
12.5%
1/8 • Number of events 1
|
0.00%
0/8
|
Additional Information
Scott S. Hall, PhD
Stanford University School of Medicine
Phone: 650-498-4799
Email: [email protected]
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place