NCT03245762

Brief Summary

The purpose of this study is to compare the change in suck and swallow competency from baseline to morning of day 6 with intranasal oxytocin spray vs placebo in infants/children with Prader-Willi Syndrome who are in nutritional phase 1a. Videofluoroscopic swallow studies will be performed on treatment day 1 and on the day following treatment morning of day 6.

Trial Health

87
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
15

participants targeted

Target at below P25 for phase_1

Timeline
Completed

Started Aug 2017

Shorter than P25 for phase_1

Geographic Reach
1 country

1 active site

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

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Study Timeline

Key milestones and dates

Study Start

First participant enrolled

August 1, 2017

Completed
6 days until next milestone

First Submitted

Initial submission to the registry

August 7, 2017

Completed
3 days until next milestone

First Posted

Study publicly available on registry

August 10, 2017

Completed
5 months until next milestone

Primary Completion

Last participant's last visit for primary outcome

January 4, 2018

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

January 4, 2018

Completed
1.5 years until next milestone

Results Posted

Study results publicly available

June 25, 2019

Completed
Last Updated

March 26, 2020

Status Verified

March 1, 2020

Enrollment Period

5 months

First QC Date

August 7, 2017

Results QC Date

January 2, 2019

Last Update Submit

March 24, 2020

Conditions

Outcome Measures

Primary Outcomes (1)

  • Suck and Swallow Competency in Infants/Children With PWS Who Are in Nutritional Phase 1a

    Swallow study Overall improvement

    baseline to day 5

Study Arms (2)

Intranasal oxytocin

ACTIVE COMPARATOR

Intervention: 4 IU/day of intranasal oxytocin via a nasal spray device each morning.

Drug: Oxytocin

IN-placebo

PLACEBO COMPARATOR

Intervention: 4 IU/day of placebo via nasal spray device each morning.

Drug: Placebo

Interventions

4 IU/day of oxytocin administered via nasal spray device each morning.

Also known as: Syntocinon
Intranasal oxytocin

4 IU/day of placebo administered via nasal spray device each morning

Also known as: Saline
IN-placebo

Eligibility Criteria

Age1 Week - 6 Months
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17)

You may qualify if:

  • Individuals with genetically confirmed PWS who are in nutritional phase 1a, as determined by PI
  • Physical exam and laboratory results that are within the normal range.
  • Presence of a parent/caregiver/guardian that is able to consent for their participation.

You may not qualify if:

  • Exposure to any investigational agent in the 30 days prior to randomization.
  • Prior chronic treatment with oxytocin.
  • A medical condition that might interfere with the conduct of the study, confound interpretation of study results or endanger the subject's well-being.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

University of Florida

Gainesville, Florida, 32610, United States

Location

MeSH Terms

Conditions

Prader-Willi Syndrome

Interventions

OxytocinSodium Chloride

Condition Hierarchy (Ancestors)

Intellectual DisabilityNeurobehavioral ManifestationsNeurologic ManifestationsNervous System DiseasesAbnormalities, MultipleCongenital AbnormalitiesCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesChromosome DisordersGenetic Diseases, InbornImprinting DisordersObesityOverweightOvernutritionNutrition DisordersNutritional and Metabolic Diseases

Intervention Hierarchy (Ancestors)

Pituitary Hormones, PosteriorPituitary HormonesPeptide HormonesHormonesHormones, Hormone Substitutes, and Hormone AntagonistsPeptidesAmino Acids, Peptides, and ProteinsChloridesHydrochloric AcidChlorine CompoundsInorganic ChemicalsSodium Compounds

Limitations and Caveats

Short study with small number of participants

Results Point of Contact

Title
Jennifer Miller, MD
Organization
University of Florida

Study Officials

  • Jennifer Miller, MD

    University of Florida

    PRINCIPAL INVESTIGATOR

Publication Agreements

PI is Sponsor Employee
No
Restrictive Agreement
No

Study Design

Study Type
interventional
Phase
phase 1
Allocation
RANDOMIZED
Masking
DOUBLE
Who Masked
CARE PROVIDER, OUTCOMES ASSESSOR
Masking Details
This will be a double-blinded study, as no studies to date have compared this treatment in infants with placebo. Board-certified speech and language pathologist who will be blinded to the treatment arm of each patient.
Purpose
BASIC SCIENCE
Intervention Model
PARALLEL
Sponsor Type
OTHER
Responsible Party
SPONSOR

Study Record Dates

First Submitted

August 7, 2017

First Posted

August 10, 2017

Study Start

August 1, 2017

Primary Completion

January 4, 2018

Study Completion

January 4, 2018

Last Updated

March 26, 2020

Results First Posted

June 25, 2019

Record last verified: 2020-03

Locations