Double-blind Placebo Controlled Study of Oxytocin in Fragile X Syndrome

Study Overview
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Brief Summary
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The purpose of this study is to determine whether the medication oxytocin is an effective and tolerable treatment in adolescent males with fragile X syndrome (FraX) for improving socially appropriate behaviors and reducing social anxiety.

Detailed Description
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Twelve male adolescent (13-24 years) subjects with confirmed genetic diagnosis of FraX (full mutation) will participate in this randomized double-blind placebo-controlled study. They will receive a dose of either 24 IU oxytocin, 48 IU oxytocin or placebo at each of three visits to the lab, with each visit spaced one week apart. The efficacy of each dose will be evaluated using behavioral, cognitive and physiological metrics. If individual subject results suggest that either of the oxytocin dosage levels (24 IU or 48 IU) is superior to placebo in the double-blind phase, a single-blind trial using the optimal dosage of oxytocin will then be administered daily for 14 days by parents at home. Subjects will then come into the lab for a final assessment on Day 30. Determination of beneficial response to oxytocin will be based on a 20% change (improvement) in behavior or test performance (see below). If both oxytocin dosage levels provide similar benefits compared to placebo, the lower dose will be chosen for the 14 day single-blind trial.

Official Title
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Double-blind Placebo Controlled Study of Oxytocin in Fragile X Syndrome

Conditions
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Fragile X Syndrome

Intervention / Treatment
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* Drug: placebo
* Drug: oxytocin 24IU
* Drug: oxytocin 48IU


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Confirmed genetic diagnosis of Fragile X (FraX) (full mutation). Male (who have more serious effects due to the X chromosome nature of the disorder) Age 13-29 years. Parent of adolescent must be willing to sign informed consent. Intelligence Quotient (IQ) > 42. Exclusion Criteria: Cardiac risk factors. Medication exclusions: opiates or opiate antagonists, corticosteroids, typical or atypical antipsychotics.

Ages Eligible for Study
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Minimum Age: 13 Years
Maximum Age: 29 Years

Sexes Eligible for Study
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Male

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Allocation: Randomized
Intervention Model: Crossover Assignment
Masking: Quadruple


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: placebo, oxytocin 24IU, oxytocin 48IU<br>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 intranasal 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 | Drug: placebo<br>* intranasal placebo (48 international units)<br>Drug: oxytocin 24IU<br>* intranasal oxytocin (24 international units) and intranasal placebo (24 international units)<br>* Other names: Syntocinon;Drug: oxytocin 48IU<br>* intranasal oxytocin (48 international units)<br>* Other names: Syntocinon;|
| Experimental: oxytocin 24IU, placebo, oxytocin 48IU<br>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 | Drug: placebo<br>* intranasal placebo (48 international units)<br>Drug: oxytocin 24IU<br>* intranasal oxytocin (24 international units) and intranasal placebo (24 international units)<br>* Other names: Syntocinon;Drug: oxytocin 48IU<br>* intranasal oxytocin (48 international units)<br>* Other names: Syntocinon;|
| Experimental: oxytocin 48IU, oxytocin 24IU, placebo<br>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 | Drug: placebo<br>* intranasal placebo (48 international units)<br>Drug: oxytocin 24IU<br>* intranasal oxytocin (24 international units) and intranasal placebo (24 international units)<br>* Other names: Syntocinon;Drug: oxytocin 48IU<br>* intranasal oxytocin (48 international units)<br>* Other names: Syntocinon;|
| Experimental: oxytocin 24IU, oxytocin 48IU, placebo<br>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 | Drug: placebo<br>* intranasal placebo (48 international units)<br>Drug: oxytocin 24IU<br>* intranasal oxytocin (24 international units) and intranasal placebo (24 international units)<br>* Other names: Syntocinon;Drug: oxytocin 48IU<br>* intranasal oxytocin (48 international units)<br>* Other names: Syntocinon;|
| Experimental: oxytocin 48IU, placebo, oxytocin 24IU<br>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) and intranasal placebo (24 international units; 4IU per puff) once at time 3, 6 puffs (3 puffs per nostril) from each of 2 bottles | Drug: placebo<br>* intranasal placebo (48 international units)<br>Drug: oxytocin 24IU<br>* intranasal oxytocin (24 international units) and intranasal placebo (24 international units)<br>* Other names: Syntocinon;Drug: oxytocin 48IU<br>* intranasal oxytocin (48 international units)<br>* Other names: Syntocinon;|
| Experimental: placebo, oxytocin 48IU, oxytocin 24IU<br>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 | Drug: placebo<br>* intranasal placebo (48 international units)<br>Drug: oxytocin 24IU<br>* intranasal oxytocin (24 international units) and intranasal placebo (24 international units)<br>* Other names: Syntocinon;Drug: oxytocin 48IU<br>* intranasal oxytocin (48 international units)<br>* Other names: Syntocinon;|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Eye Contact/Gaze During 10 Minute Social Challenge Task | 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. | baseline, week 2, and week 3 | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Salivary Cortisol | salivary 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) | baseline, week 2, and week 3 |