Fragile X SyndromePhase 2NCT01254045
Double-blind Placebo Controlled Study of Oxytocin in Fragile X Syndrome
A Phase 2 study testing oxytocin 24IU and oxytocin 48IU in people with Fragile X Syndrome, run by Stanford University at 1 site in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages 13 to 29 years, men only
- Conditions the study is for
- Fragile X Syndrome
- What is being tested?
- oxytocin 24IU and oxytocin 48IU
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 10 participants
- Where
- 1 siteUnited States
- Study dates
- Feb 2007to Jan 2010
- Phase
- Phase 2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 13 to 29 years
- Men only
- No healthy volunteers
- Child, Adult
Who may be able to join
- 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.
Who cannot join
- Cardiac risk factors.
- Medication exclusions: opiates or opiate antagonists, corticosteroids, typical or atypical antipsychotics.
Trial locations and contacts
1 site in 1 country.
United States1 site
Stanford University School of Medicine
Stanford, California, 94305
About this study
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.
Read the 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.
- Conditions
- Fragile X Syndrome
- Being tested
- placebo, oxytocin 24IU, oxytocin 48IU
- Medical subject headings
- Fragile X Syndrome
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
placeboDrug
intranasal placebo (48 international units)
oxytocin 24IUDrug
Also called Syntocinon
intranasal oxytocin (24 international units) and intranasal placebo (24 international units)
oxytocin 48IUDrug
Also called Syntocinon
intranasal oxytocin (48 international units)
| Study group | Role | What they receive |
|---|---|---|
| placebo, oxytocin 24IU, oxytocin 48IU | Experimental | 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 Receives: Drug: placebo; Drug: oxytocin 24IU; Drug: oxytocin 48IU |
| oxytocin 24IU, placebo, oxytocin 48IU | Experimental | 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 Receives: Drug: placebo; Drug: oxytocin 24IU; Drug: oxytocin 48IU |
| oxytocin 48IU, oxytocin 24IU, placebo | Experimental | 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 Receives: Drug: placebo; Drug: oxytocin 24IU; Drug: oxytocin 48IU |
| oxytocin 24IU, oxytocin 48IU, placebo | Experimental | 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 Receives: Drug: placebo; Drug: oxytocin 24IU; Drug: oxytocin 48IU |
| oxytocin 48IU, placebo, oxytocin 24IU | Experimental | 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 Receives: Drug: placebo; Drug: oxytocin 24IU; Drug: oxytocin 48IU |
| placebo, oxytocin 48IU, oxytocin 24IU | Experimental | 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 Receives: Drug: placebo; Drug: oxytocin 24IU; Drug: oxytocin 48IU |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: baseline, week 2, and week 3
Secondary outcomes (1)
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)
Measured: baseline, week 2, and week 3
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted6 Dec 2010 (estimated)
- Study startFeb 2007
- Primary completionNov 2009
- Study completionJan 2010
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Fragile X SyndromeStudies of the same condition, open and under way.
- More trials run by Stanford UniversityThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 10 participants (actual)
Sponsor and investigators
- Lead sponsor
- Stanford University
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Allan Reiss, Stanford University
- Investigators
- Allan L Reiss, Principal investigator, Stanford University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01254045
- Sponsor's protocol ID
- SU-11182010-7215
- Other IDs
- 8618
- Status last verified
- Mar 2020
- First submitted
- 2 Dec 2010
- Last updated
- 18 Mar 2020
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
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.
PubMed 21862226
Study at a glance
From the registry record, last updated by the sponsor on 18 Mar 2020. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Double-blind Placebo Controlled Study of Oxytocin in Fragile X Syndrome
- ClinicalTrials.gov ID
- NCT01254045
- Acronym / sponsor ID
- SU-11182010-7215
- Conditions
- Fragile X Syndrome
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 10 participants (actual)
- Lead sponsor
- Stanford UniversityUniversity, hospital or other
- Study start
- Feb 2007
- Primary completion
- Nov 2009 (actual)
- Study completion
- Jan 2010 (actual)
- First posted
- 6 Dec 2010 (estimated)
- Results
- Posted 29 Aug 2014
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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
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
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 6
| Arm | Type | Receives |
|---|---|---|
| placebo, oxytocin 24IU, oxytocin 48IU | Experimental | Drug: placebo, Drug: oxytocin 24IU, Drug: oxytocin 48IUintranasal 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 |
| oxytocin 24IU, placebo, oxytocin 48IU | Experimental | Drug: placebo, Drug: oxytocin 24IU, Drug: oxytocin 48IUintranasal 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 | Experimental | Drug: placebo, Drug: oxytocin 24IU, Drug: oxytocin 48IUintranasal 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, oxytocin 48IU, placebo | Experimental | Drug: placebo, Drug: oxytocin 24IU, Drug: oxytocin 48IUintranasal 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, oxytocin 24IU | Experimental | Drug: placebo, Drug: oxytocin 24IU, Drug: oxytocin 48IUintranasal 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 |
| placebo, oxytocin 48IU, oxytocin 24IU | Experimental | Drug: placebo, Drug: oxytocin 24IU, Drug: oxytocin 48IUintranasal 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 |
Who may qualify
- Ages
- 13 to 29 years
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (5 listed)
- 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.
Key exclusion criteria (2 listed)
- Cardiac risk factors.
- Medication exclusions: opiates or opiate antagonists, corticosteroids, typical or atypical antipsychotics.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| placebointranasal placebo (48 international units) | Drug | oxytocin 24IU, oxytocin 48IU, placebo, oxytocin 24IU, placebo, oxytocin 48IU, oxytocin 48IU, oxytocin 24IU, placebo, oxytocin 48IU, placebo, oxytocin 24IU, placebo, oxytocin 24IU, oxytocin 48IU, placebo, oxytocin 48IU, oxytocin 24IU |
| oxytocin 24IUAlso: Syntocinonintranasal oxytocin (24 international units) and intranasal placebo (24 international units) | Drug | oxytocin 24IU, oxytocin 48IU, placebo, oxytocin 24IU, placebo, oxytocin 48IU, oxytocin 48IU, oxytocin 24IU, placebo, oxytocin 48IU, placebo, oxytocin 24IU, placebo, oxytocin 24IU, oxytocin 48IU, placebo, oxytocin 48IU, oxytocin 24IU |
| oxytocin 48IUAlso: Syntocinonintranasal oxytocin (48 international units) | Drug | oxytocin 24IU, oxytocin 48IU, placebo, oxytocin 24IU, placebo, oxytocin 48IU, oxytocin 48IU, oxytocin 24IU, placebo, oxytocin 48IU, placebo, oxytocin 24IU, placebo, oxytocin 24IU, oxytocin 48IU, placebo, oxytocin 48IU, oxytocin 24IU |
Primary outcome
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.
1 secondary outcome
Salivary Cortisol
Time frame: baseline, week 2, and week 3
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 18 Mar 2020.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Results publication: PubMed 21862226Hall 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.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Allan L Reiss principal investigator
Stanford University
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing oxytocin 24IU and oxytocin 48IU in people with Fragile X Syndrome, run by Stanford University at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 5 inclusion and 2 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 18 Mar 2020. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 29 Aug 2014). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Eye Contact/Gaze During 10 Minute Social Challenge Task (time frame: baseline, week 2, and week 3). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT01254045, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 18 Mar 2020.