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

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ClinicalTrials.gov Identifier: NCT01254045
Recruitment Status : Completed
First Posted : December 6, 2010
Results First Posted : August 29, 2014
Last Update Posted : August 29, 2014
Sponsor:
Information provided by (Responsible Party):
Stanford University

Study Description
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Study Description Study Design Arms and Interventions Outcome Measures Eligibility Criteria Contacts and Locations More Information
Brief Summary:
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.

Condition or disease Intervention/treatment Phase
Fragile X Syndrome Drug: placebo Drug: oxytocin 24IU Drug: oxytocin 48IU Phase 2

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 Design
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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 10 participants
Allocation: Randomized
Intervention Model: Crossover Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Official Title: Double-blind Placebo Controlled Study of Oxytocin in Fragile X Syndrome
Study Start Date : February 2007
Actual Primary Completion Date : November 2009
Actual Study Completion Date : January 2010

Resource links provided by the National Library of Medicine

Drug Information available for: Oxytocin
U.S. FDA Resources

Arms and Interventions
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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 Name: Syntocinon
Drug: oxytocin 48IU
intranasal oxytocin (48 international units)
Other Name: 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 Name: Syntocinon
Drug: oxytocin 48IU
intranasal oxytocin (48 international units)
Other Name: 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 Name: Syntocinon
Drug: oxytocin 48IU
intranasal oxytocin (48 international units)
Other Name: 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 Name: Syntocinon
Drug: oxytocin 48IU
intranasal oxytocin (48 international units)
Other Name: 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 Name: Syntocinon
Drug: oxytocin 48IU
intranasal oxytocin (48 international units)
Other Name: 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 Name: Syntocinon
Drug: oxytocin 48IU
intranasal oxytocin (48 international units)
Other Name: Syntocinon


Outcome Measures
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Study Description Study Design Arms and Interventions Outcome Measures Eligibility Criteria Contacts and Locations More Information

Primary Outcome Measures :
  1. Eye Contact/Gaze During 10 Minute Social Challenge Task [ Time Frame: baseline, week 2, and week 3 ]
    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.


Secondary Outcome Measures :
  1. Salivary Cortisol [ Time Frame: baseline, week 2, and week 3 ]
    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)


Eligibility Criteria
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Study Description Study Design Arms and Interventions Outcome Measures Eligibility Criteria Contacts and Locations More Information

Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


Ages Eligible for Study:   13 Years to 29 Years   (Child, Adult)
Sexes Eligible for Study:   Male
Accepts Healthy Volunteers:   No
Criteria

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.
Contacts and Locations
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Study Description Study Design Arms and Interventions Outcome Measures Eligibility Criteria Contacts and Locations More Information

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01254045


Locations
United States, California
Stanford University School of Medicine
Stanford, California, United States, 94305
Sponsors and Collaborators
Stanford University
Investigators
Principal Investigator: Allan L Reiss Stanford University
More Information
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Study Description Study Design Arms and Interventions Outcome Measures Eligibility Criteria Contacts and Locations More Information

Publications of Results:

Responsible Party: Stanford University
ClinicalTrials.gov Identifier: NCT01254045     History of Changes
Other Study ID Numbers: SU-11182010-7215
8618 ( Other Identifier: Stanford University IRB eProtocol )
First Posted: December 6, 2010    Key Record Dates
Results First Posted: August 29, 2014
Last Update Posted: August 29, 2014
Last Verified: August 2014

Additional relevant MeSH terms:
Fragile X Syndrome
Syndrome
Disease
Pathologic Processes
Mental Retardation, X-Linked
Intellectual Disability
Neurobehavioral Manifestations
Neurologic Manifestations
Nervous System Diseases
Sex Chromosome Disorders
 Chromosome Disorders
Congenital Abnormalities
Genetic Diseases, Inborn
Genetic Diseases, X-Linked
Heredodegenerative Disorders, Nervous System
Oxytocin
Oxytocics
Reproductive Control Agents
Physiological Effects of Drugs