Intranasal Oxytocin vs. Placebo for the Treatment of Hyperphagia in Prader-Willi Syndrome

NCT03197662 | Completed Phase 2 Results DMC FDA Drug

• 2 other identifiers: 2017-8076FD-R-005106-01
• Study Type: interventional
• Target: 32
• Locations: 1 country
• Sites: 1

Brief Summary

This study is a phase 2 randomized double blind 8-week treatment trial of intranasal OXT vs. placebo in 50 subjects aged 5 to 17 years with PWS in order to assess IN-OXT's affect on measurements of (1) eating behaviors (2) repetitive behaviors (3) weight and body composition (4) quality of life (5) salivary OXT and hormone levels (including ghrelin, pancreatic polypeptide, peptide YY, Glucagon-Like Peptide-1 (GLP-1), insulin, glucagon, testosterone, and estrogen). If superior to placebo, this data will add to the current knowledge that OXT is an effective treatment for hyperphagia as well as other symptoms of PWS. Funding Source- FDA OOPD

Conditions

Prader-Willi Syndrome; Hyperphagia

Keywords

Prader-Willi Syndrome; Hyperphagia

Outcome Measures

Primary Outcomes (1)

• Change in Efficacy of Peptide Intranasal Oxytocin (IN-OXT)

  Change in efficacy of peptide IN-OXT as measured by changes in hyperphagia from baseline will be assessed using the Hyperphagia Questionnaire for Clinical Trials (HQ-CT). HQ-CT is a caregiver-rated assessment comprised of 9-items that measures the frequency and intensity of hyperphagic behaviors, over the preceding two weeks, in participants with Prader-Willi Syndrome (PWS). The HQ-CT total score is created by summing the 9 item-level responses (ranging from 0 to 4) for a possible range of 0-36 with higher scores indicate more extreme hyperphagia. For purposes of this outcome measure, negative scores represent a decrease in hyperphagia from baseline and positive scores represent an increase in hyperphagia from baseline.

  From Randomization at Baseline until Endpoint at Week 8 (Change over 8 weeks)

Secondary Outcomes (13)

• Change in Repetitive Behavior

  From Randomization at Baseline until Endpoint at Week 8 (Change over 8 weeks)

• Change in Rigid Behavior

  From Randomization at Baseline until Endpoint at Week 8 (Change over 8 weeks)

• Change in Body Weight

  From Randomization at Baseline until Endpoint at Week 8 (Change over 8 weeks)

• Body Composition

  8 weeks

• Change in Body Mass Index (BMI)

  From Randomization at Baseline until Endpoint at Week 8 (Change over 8 weeks)

Study Arms (2)

Experimental: Intranasal Oxytocin (IN-OXT)

EXPERIMENTAL

Syntocinon (synthetic oxytocin) will be used in this protocol. Each subject will receive a dose of 16 IU QD ("quaque die" or once a day) and will be instructed to inhale 2 puffs per nostril (4 IU each).

Drug: Intranasal Oxytocin (IN-OXT)

Placebo Comparator: Matched Placebo

PLACEBO COMPARATOR

Each subject will receive a dose of 16 IU QD, and will be instructed to inhale 2 puffs per nostril (4 IU each).

Drug: Matched Placebo

Interventions

Intranasal Oxytocin (IN-OXT) DRUG

Each subject will receive a dose of 16 IU QD, and will be instructed to inhale 2 puffs per nostril every day (4 IU each). If needed, treatment will be titrated due to side effects or non-response.

Also known as: Syntocinon

Experimental: Intranasal Oxytocin (IN-OXT)

Matched Placebo DRUG

Each subject will receive a dose of 16 IU QD, and will be instructed to inhale 2 puffs per nostril every day (4 IU each). If needed, treatment will be titrated due to side effects or non-response.

Placebo Comparator: Matched Placebo

Eligibility Criteria

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

You may qualify if:

• Male or female pediatric outpatients aged 5 to 17 years
• Must be in PWS nutritional phase 2b or 3 as determined by PI
• Must be on growth hormone treatment and have been receiving stable doses of growth hormone treatment for at least 3 months prior to screening date. Treatment cannot have been interrupted for more than one week within 3 months of screening.
• Diagnosis of PWS confirmed by patient medical records.
• A score of at least moderate severity on the Hyperphagia Questionnaire for Clinical Trials at both screening and baseline visits.
• Stable dosages of hormone treatments (including testosterone and estrogen supplements) for 4 weeks prior to randomization and for the duration of the study.
• Stable dosages of metabolic treatments that could affect appetite (including metformin) for 4 weeks prior to randomization and for the duration of the study.
• Physical exam and laboratory results that are within the normal range for individuals with PWS.
• Presence of a parent/caregiver/guardian that is able to consent for their participation and complete assessments regarding the child's development and behavior change throughout the study.

You may not qualify if:

• Exposure to any investigational agent in the 30 days prior to randomization.
• Child not receiving growth hormone treatment
• Children weighing less than 40 lbs
• Children with unstable Type 2 Diabetes confirmed by Hemoglobin A1C levels at screening
• Children with unstable medical co-morbidities at baseline.
• Children with active upper respiratory infections at screening.
• A primary psychiatric diagnosis other than Autism Spectrum Disorder (ASD), including bipolar disorder, psychosis, schizophrenia, Post-traumatic stress disorder (PTSD) or major depressive disorder (MDD). These patients will be excluded due to potential confounding results.
• Pregnant or lactating patients or patients who will not agree to use a double barrier method of contraception. IN-OXT has not been studied in pregnant or lactating women.
• Females using an estrogen-based contraceptive. As an alternative to an estrogen based contraceptive, subjects will be counseled to use progesterone-based contraceptives; cervical cap; cervical sponges; or spermicidal foam in combination with a condom. Subjects will need to use a double barrier method to be in the study.
• A medical condition that might interfere with the conduct of the study, confound interpretation of study results or endanger the subject's well-being.
• A known diagnosis of Rett's Syndrome of Childhood Disintegrative Disorder or marked sensory impairment such as deafness or blindness.
• Subjects who have changes in allied health therapies, behavioral or educational interventions within four weeks prior to randomization other than those associated with school holidays.
• Subjects who have had changes in medications or medication doses of risperidone, aripiprazole, other antipsychotic medications, clonidine, guanfacine, stimulants or anti-convulsants within four weeks of randomization.

Sponsors & Collaborators

• Eric Hollander: lead

Study Sites (1)

Montefiore Medical Center, Albert Einstein College of Medicine

The Bronx, New York, 10461, United States

Location

MeSH Terms

Conditions

Prader-Willi Syndrome; Hyperphagia

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; Signs and Symptoms, Digestive; Signs and Symptoms; Pathological Conditions, Signs and Symptoms

Intervention Hierarchy (Ancestors)

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

Results Point of Contact

• Title: Dr. Eric Hollander
• Organization: Montefiore Medical Center

eholland@montefiore.org

718-839-7516

Study Officials

• Eric Hollander, MD

  Albert Einstein College of Medicine

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: Yes

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR INVESTIGATOR
• PI Title: Director, Autism and Obsessive Compulsive Spectrum Program and Professor, Department of Psychiatry and Behavioral Sciences

Study Record Dates

• First Submitted: June 19, 2017
• First Posted: June 23, 2017
• Study Start: April 11, 2018
• Primary Completion: August 29, 2022
• Study Completion: August 29, 2022
• Last Updated: February 7, 2025
• Results First Posted: February 7, 2025

Record last verified: 2025-02

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)