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

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.

Study Overview

• Status: Completed
• Conditions: Fragile X Syndrome
• Intervention / Treatment: Drug: placebo; Drug: oxytocin 24IU; Drug: oxytocin 48IU

Detailed 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.

• Study Type: Interventional
• Enrollment (Actual): 10
• Phase: Phase 2

Contacts and Locations

Study Locations

• United States
  • California
    • Stanford, California, United States, 94305
      • Stanford University School of Medicine

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 13 years to 29 years (Child, Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: Male

Description

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.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Crossover Assignment
• Masking: Quadruple

Number of Arms

6

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: placebo, oxytocin 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 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; intranasal placebo (48 international units); Drug: oxytocin 24IU; intranasal oxytocin (24 international units) and intranasal placebo (24 international units); Other Names: Syntocinon; Drug: oxytocin 48IU; intranasal oxytocin (48 international units); Other Names: Syntocinon
Experimental: oxytocin 24IU, placebo, 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	Drug: placebo; intranasal placebo (48 international units); Drug: oxytocin 24IU; intranasal oxytocin (24 international units) and intranasal placebo (24 international units); Other Names: Syntocinon; Drug: oxytocin 48IU; intranasal oxytocin (48 international units); Other Names: Syntocinon
Experimental: oxytocin 48IU, oxytocin 24IU, placebo; 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; intranasal placebo (48 international units); Drug: oxytocin 24IU; intranasal oxytocin (24 international units) and intranasal placebo (24 international units); Other Names: Syntocinon; Drug: oxytocin 48IU; intranasal oxytocin (48 international units); Other Names: Syntocinon
Experimental: oxytocin 24IU, oxytocin 48IU, placebo; 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; intranasal placebo (48 international units); Drug: oxytocin 24IU; intranasal oxytocin (24 international units) and intranasal placebo (24 international units); Other Names: Syntocinon; Drug: oxytocin 48IU; intranasal oxytocin (48 international units); Other Names: Syntocinon
Experimental: oxytocin 48IU, placebo, oxytocin 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) 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; intranasal placebo (48 international units); Drug: oxytocin 24IU; intranasal oxytocin (24 international units) and intranasal placebo (24 international units); Other Names: Syntocinon; Drug: oxytocin 48IU; intranasal oxytocin (48 international units); Other Names: Syntocinon
Experimental: placebo, oxytocin 48IU, oxytocin 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	Drug: placebo; intranasal placebo (48 international units); Drug: oxytocin 24IU; intranasal oxytocin (24 international units) and intranasal placebo (24 international units); Other Names: Syntocinon; Drug: oxytocin 48IU; intranasal oxytocin (48 international units); Other Names: Syntocinon

What is the study measuring?

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

Collaborators and Investigators

• Sponsor: Stanford University
• Investigators: Principal Investigator: Allan L Reiss, Stanford University

Publications and helpful links

General Publications

• Hall SS, Lightbody AA, McCarthy BE, Parker KJ, Reiss AL. Effects of intranasal oxytocin on social anxiety in males with fragile X syndrome. Psychoneuroendocrinology. 2012 Apr;37(4):509-18. doi: 10.1016/j.psyneuen.2011.07.020. Epub 2011 Aug 20.

Study record dates

Study Major Dates

• Study Start: February 1, 2007
• Primary Completion (Actual): November 1, 2009
• Study Completion (Actual): January 1, 2010

Study Registration Dates

• First Submitted: December 2, 2010
• First Submitted That Met QC Criteria: December 3, 2010
• First Posted (Estimate): December 6, 2010

Study Record Updates

• Last Update Posted (Actual): March 18, 2020
• Last Update Submitted That Met QC Criteria: March 16, 2020
• Last Verified: March 1, 2020

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Pathologic Processes; Nervous System Diseases; Neurologic Manifestations; Neurobehavioral Manifestations; Disease; Congenital Abnormalities; Genetic Diseases, Inborn; Genetic Diseases, X-Linked; Mental Retardation, X-Linked; Intellectual Disability; Heredodegenerative Disorders, Nervous System; Chromosome Disorders; Sex Chromosome Disorders; Syndrome; Fragile X Syndrome; Physiological Effects of Drugs; Reproductive Control Agents; Oxytocics; Oxytocin
• Other Study ID Numbers: SU-11182010-7215; 8618 (Other Identifier: Stanford University IRB eProtocol)