Oxytocin Trial in Prader-Willi Syndrome

NCT02013258 | Completed Phase 1 DMC

• 1 other identifier: 5208-N
• Study Type: interventional
• Target: 24
• Locations: 1 country
• Sites: 3

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. 1.Does intranasal oxytocin cause any side effects in children with PWS?
2. 2.Does intranasal oxytocin administration alter appetite or behaviors in PWS?

Conditions

Prader Willi Syndrome

Keywords

Prader Willi syndrome; Oxytocin

Outcome Measures

Primary Outcomes (1)

• Safety of intranasal oxytocin in children with Prader-Willi syndrome

  Occurrence of adverse event, description and quantification of clinical and behavior severity, pre- and post- intranasal oxytocin and placebo administration.

  3 months

Secondary Outcomes (1)

• Evaluation of food intake in Prader-Willi syndrome

  3 months

Other Outcomes (2)

• Evaluation of social effects of intranasal oxytocin in children with Prader-Willi syndrome

  3 months

• Effects of intranasal oxytocin on appetite-regulating hormones

  3 months

Study Arms (2)

Intranasal oxytocin

ACTIVE COMPARATOR

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

Drug: Intranasal oxytocin

Placebo

PLACEBO COMPARATOR

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

Other: Placebo

Interventions

Intranasal oxytocin 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.

Also known as: Pitocin, Syntocinon

Intranasal oxytocin

Placebo 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.

Placebo

Eligibility Criteria

• Age: 5 Years - 11 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• University of Florida: lead
• National Institutes of Health (NIH): collaborator

Study Sites (3)

University of California, Irvine

Orange, California, 92686, United States

Location

University of Florida

Gainesville, Florida, 32610, United States

Location

University of Kansas Medical Center

Kansas City, Kansas, 66160, United States

Location

Related Publications (1)

• 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.

  PMID: 21702900 BACKGROUND

Related Links

• 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.

MeSH Terms

Conditions

Prader-Willi Syndrome

Interventions

Oxytocin

Condition Hierarchy (Ancestors)

Intellectual Disability; Neurobehavioral Manifestations; Neurologic Manifestations; Nervous System Diseases; Abnormalities, Multiple; Congenital Abnormalities; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Chromosome Disorders; Genetic Diseases, Inborn; Imprinting Disorders; Obesity; Overweight; Overnutrition; Nutrition Disorders; Nutritional and Metabolic Diseases

Intervention Hierarchy (Ancestors)

Pituitary Hormones, Posterior; Pituitary Hormones; Peptide Hormones; Hormones; Hormones, Hormone Substitutes, and Hormone Antagonists; Peptides; Amino Acids, Peptides, and Proteins

Study Officials

• Jennifer L Miller, MD

  University of Florida

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: CROSSOVER
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: December 11, 2013
• First Posted: December 17, 2013
• Study Start: March 1, 2015
• Primary Completion: August 1, 2015
• Study Completion: August 1, 2015
• Last Updated: April 5, 2018

Record last verified: 2018-04

Locations

US (3)