Oxytocin Trial in Prader-Willi Syndrome

NCT ID: NCT02013258

Last Updated: 2018-04-05

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE1
• Total Enrollment: 24 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2015-03-31
• Study Completion Date: 2015-08-31

Brief Summary

Individuals with Prader-Willi syndrome (PWS) have been found to have a deficit of oxytocin-producing neurons and decreased oxytocin receptor gene function, so the purpose of this study is to determine if oxytocin (OT) administration will improve some of the aspects of Prader-Willi syndrome that are particularly troublesome for children and their families (the insatiable appetite and social behaviors).

The research questions are:

1. Does intranasal oxytocin cause any side effects in children with PWS?

2. Does intranasal oxytocin administration alter appetite or behaviors in PWS?

Detailed Description

This study is to investigate if intranasal oxytocin will improve hyperphagia, social skills, and behaviors in subjects with Prader-Willi syndrome. This will be a randomized placebo controlled pilot study. The primary outcome measure is to determine if intranasal administration of oxytocin will cause any adverse events in subjects with Prader-Willi syndrome. Secondarily, the investigators will also perform evaluations to determine if intranasal oxytocin has any effect on social skills, behaviors, or appetite in children with Prader-Willi syndrome.

Conditions

Prader Willi Syndrome

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: CROSSOVER
• Primary Study Purpose: TREATMENT
• Blinding Strategy: QUADRUPLE

Study Groups

Intranasal oxytocin

Intranasal oxytocin. 16 IU intranasal oxytocin x 5 days. One month interval between arms of treatment.

Group Type: ACTIVE_COMPARATOR

Intranasal oxytocin

Intervention Type: DRUG

This is a double-blind placebo controlled 2x2 study. Subjects will receive OT for 5 consecutive days during their 7 day stay. This will be followed by a wash out period of 4-6 weeks.

Placebo

Placebo will be administered via nasal spray - 1 spray in each nostril x5 days.

Group Type: PLACEBO_COMPARATOR

Placebo

Intervention Type: OTHER

This is a double-blind placebo controlled 2x2 study. Placebo will be given via intranasal spray, one spray in each nostril daily x 5 days. One month interval between arms of treatment.

Interventions

Intranasal oxytocin

This is a double-blind placebo controlled 2x2 study. Subjects will receive OT for 5 consecutive days during their 7 day stay. This will be followed by a wash out period of 4-6 weeks.

Intervention Type: DRUG

Placebo

This is a double-blind placebo controlled 2x2 study. Placebo will be given via intranasal spray, one spray in each nostril daily x 5 days. One month interval between arms of treatment.

Intervention Type: OTHER

Other Intervention Names

Pitocin; Syntocinon

Eligibility Criteria

Inclusion Criteria

• Children with genetically confirmed PWS
• Ages at ≥ 5 years and ≤ 11 years (must start treatment prior to 11th birthday)
• Child must be in nutritional phase 2b or 3, as determined by the PI at each site.
• Must currently be on growth hormone treatment, and have been receiving growth hormone treatment for at least one year prior to screening date.
• Treatment cannot have been interrupted for more than 1 week within 3 months prior to screening date.
• Priority will be given to children currently enrolled in the RDCRN Natural History study

Exclusion Criteria

• Inability to tolerate intranasal administration of medication
• Hepatic insufficiency (AST/ALT greater than 3 times the normal levels for age)
• Renal insufficiency (BUN/Creatinine greater than 3 times the normal levels for age)
• History of an abnormal ECG (as determined by a cardiologist). If there is any question about cardiac function, ECG reports will be reviewed with a cardiologist prior to enrollment in the study.
• Child not receiving growth hormone treatment
• Child with hypertension or hypotension for age and sex (blood pressure >97% for age and sex or blood pressure <3% for age and sex)
• Diabetes mellitus
• Pregnant or lactating.
• Schizophrenia or psychosis
• Taking any psychotropic medications

• Minimum Eligible Age: 5 Years
• Maximum Eligible Age: 11 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

National Institutes of Health (NIH)

NIH

Sponsor Role: collaborator

University of Florida

OTHER

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Jennifer L Miller, MD

Role: PRINCIPAL_INVESTIGATOR

University of Florida

Locations

University of California, Irvine

Orange, California, United States

University of Florida

Gainesville, Florida, United States

University of Kansas Medical Center

Kansas City, Kansas, United States

Countries

United States

References

Tauber M, Mantoulan C, Copet P, Jauregui J, Demeer G, Diene G, Roge B, Laurier V, Ehlinger V, Arnaud C, Molinas C, Thuilleaux D. Oxytocin may be useful to increase trust in others and decrease disruptive behaviours in patients with Prader-Willi syndrome: a randomised placebo-controlled trial in 24 patients. Orphanet J Rare Dis. 2011 Jun 24;6:47. doi: 10.1186/1750-1172-6-47.

Reference Type: BACKGROUND

PMID: 21702900 (View on PubMed)

Related Links

https://www.ncbi.nlm.nih.gov/pubmed/28371242

Oxytocin treatment in children with Prader-Willi syndrome: A double-blind, placebo-controlled, crossover study. Miller JL, Tamura R, Butler MG, Kimonis V, Sulsona C, Gold JA, Driscoll DJ. Am J Med Genet A. 2017 May;173(5):1243-1250.

Other Identifiers

5208-N

Identifier Type: -

Identifier Source: org_study_id