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

Stanford University

Status and phase: Completed; Phase 2

Conditions: Fragile X Syndrome

Treatments: Drug: placebo; Drug: oxytocin 24IU; Drug: oxytocin 48IU

Study type: Interventional

Funder types: Other

Identifiers

NCT01254045

8618 (Other Identifier)

SU-11182010-7215

Details and patient eligibility

About

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.

Full description

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.

Enrollment: 10 patients

Sex: Male

Ages: 13 to 29 years old

Volunteers: No Healthy Volunteers

Inclusion criteria

1. Confirmed genetic diagnosis of Fragile X (FraX) (full mutation).
2. Male (who have more serious effects due to the X chromosome nature of the disorder)
3. Age 13-29 years.
4. Parent of adolescent must be willing to sign informed consent.
5. Intelligence Quotient (IQ) > 42.

Exclusion criteria

1. Cardiac risk factors.
2. Medication exclusions: opiates or opiate antagonists, corticosteroids, typical or atypical antipsychotics.

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Crossover Assignment

Masking: Quadruple Blind

---

10 participants in 6 patient groups

placebo, oxytocin 24IU, oxytocin 48IU

Experimental group

Description:

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

Treatment:

Drug: oxytocin 48IU

Drug: placebo

Drug: oxytocin 24IU

oxytocin 24IU, placebo, oxytocin 48IU

Experimental group

Description:

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

Treatment:

Drug: oxytocin 48IU

Drug: placebo

Drug: oxytocin 24IU

oxytocin 48IU, oxytocin 24IU, placebo

Experimental group

Description:

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

Treatment:

Drug: oxytocin 48IU

Drug: placebo

Drug: oxytocin 24IU

oxytocin 24IU, oxytocin 48IU, placebo

Experimental group

Description:

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

Treatment:

Drug: oxytocin 48IU

Drug: placebo

Drug: oxytocin 24IU

oxytocin 48IU, placebo, oxytocin 24IU

Experimental group

Description:

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

Treatment:

Drug: oxytocin 48IU

Drug: placebo

Drug: oxytocin 24IU

placebo, oxytocin 48IU, oxytocin 24IU

Experimental group

Description:

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

Treatment:

Drug: oxytocin 48IU

Drug: placebo

Drug: oxytocin 24IU