A Clinical Evaluation of Non-Invasive Vagus Nerve Stimulation for Temper Outbursts in People With PWS

NCT ID: NCT06144645

Last Updated: 2025-11-25

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

RECRUITING

Clinical Phase

PHASE3

Total Enrollment

102 participants

Study Classification

INTERVENTIONAL

Study Start Date

2024-01-08

Study Completion Date

2027-12-01

Brief Summary

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The goal of the VNS4PWS clinical study is to test the efficacy, safety, and acceptability of transcutaneous vagus nerve stimulation (tVNS) treatment in people with PWS.

Detailed Description

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The main questions the VNS4PWS study seeks to answer are: (1) is tVNS treatment safe in people with PWS, (2) is tVNS treatment acceptable to people with PWS, and (3) is tVNS an effective treatment to reduce temper outbursts in people with PWS. Participants will wear the tVNS device daily for 4 hours over a period of 9 months. Two different doses of tVNS will be compared. During the final three months of the trial, the effect of stopping treatment will be studied. After the first year of the study, participants will have the opportunity to continue on to a 1-year open label extension period during which active tVNS treatment will be resumed.

Conditions

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Prader-Willi Syndrome

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

DOUBLE

Participants Investigators

Study Groups

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tVNS, Intermittent Stimulation

28 seconds on, 32 seconds off

Group Type ACTIVE_COMPARATOR

tVNS, intermittent stimulation

Intervention Type DEVICE

transcutaneous vagus nerve stimulation, intermittent stimulation

tVNS, Continuous Stimulation

continuous stimulation

Group Type ACTIVE_COMPARATOR

tVNS, continuous stimulation

Intervention Type DEVICE

transcutaneous vagus nerve stimulation, continuous stimulation

Interventions

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tVNS, intermittent stimulation

transcutaneous vagus nerve stimulation, intermittent stimulation

Intervention Type DEVICE

tVNS, continuous stimulation

transcutaneous vagus nerve stimulation, continuous stimulation

Intervention Type DEVICE

Other Intervention Names

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tVNS R, manufactured by tVNS Technologies, intermittent stimulation tVNS R, manufactured by tVNS Technologies, continuous stimulation

Eligibility Criteria

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Inclusion Criteria

1. Genetically proven diagnosis of PWS.
2. Age 10-40 years.
3. History in the last six months of an average of at least two temper outbursts per week.
4. Able to comply with requirements of study and provide consent or assent; If consent is to be provided by the participant's guardian, same applies to the guardian.
5. Concomitant use of psychiatric medication is allowed; participant must be on a stable dose 90 days prior to screening with no plans for dose modification during the course of the study.
6. Concomitant use of psychotherapy/counseling is allowed. Therapies for mental health or behavior challenges, including applied behavior analysis (ABA) are also allowed; however, participant must be on a stable regimen 90 days prior to screening with no plans for modification during the course of the study.
7. Living with family or in another setting with family members or staff willing to support the participant and the required data collection.
8. Agree to share tVNS patient application compliance and daily temper outburst data with the sponsor.
9. Access to cellular data or Wi-Fi.
10. Participant and caregiver speak American English as first language or are fluent in American English.

Exclusion Criteria

1. Positive pregnancy test at screening, baseline, or at any point in the study.
2. Evidence of active or recent unstable serious mental illness, including, psychosis, mania, severe depression, or suicidality.
3. Moved to present residential placement in last three months or less.
4. Likely move in residential placement during the course of the study.
5. Cardiac abnormalities including medically documented history of cardiac disease or cardiac arrhythmia, documented resting heart rate ≤ 50 beats per minute (BPM); or history of 2° type 2 or 3° heart block on electrocardiogram (ECG).
6. Evidence of clinically significant abnormalities of blood, liver, or kidney function from clinical safety laboratory assessments as determined by the Site Investigator.
7. History of blood clot, pulmonary embolism, or deep vein thrombosis.
8. Prior diagnosis of epilepsy or currently active seizures.
9. Current enrollment in the active phase of different clinical trial or interventional study.
10. Current use of hearing aids or implantable medical devices including implanted vagus nerve stimulation (iVNS) device, implanted cardiac pacemaker, implanted cardiac defibrillator, cochlear implants, cerebral shunt or cardiac implantable electrical devices.
11. Presence of dermal abnormalities at the stimulation site that would interfere with the ability of the tVNS device to function properly.
12. Presence of an allergy to titanium, titanium-iridium, thermoplastic elastomers, perfluoroethylene propylene, or polyurethane elastomers, or components of / preservatives present in the device electrode cream (Ceteareth-20, Propylene Glycol, Bentonite, Polysorbate 20, Phenoxyethanol, Ethylhexylglycerin).
13. Severe, untreated sleep apnea, as self-reported by subject or caregiver or suspected by Site Investigator.
14. Subject is, in the opinion of the Investigator, not suitable to participate in the study.
Minimum Eligible Age

10 Years

Maximum Eligible Age

40 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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RTI International

OTHER

Sponsor Role collaborator

Foundation for Prader-Willi Research

OTHER

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Theresa Strong, PhD

Role: PRINCIPAL_INVESTIGATOR

Foundation for Prader-Willi Research

Deepan Singh, MD

Role: PRINCIPAL_INVESTIGATOR

Maimonides Medical Center

Locations

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Stanford University

Palo Alto, California, United States

Site Status RECRUITING

Rady Children's Hospital San Diego

San Diego, California, United States

Site Status RECRUITING

Children's Hospital Colorado

Aurora, Colorado, United States

Site Status RECRUITING

Emory University

Atlanta, Georgia, United States

Site Status RECRUITING

Rare Disease Research

Atlanta, Georgia, United States

Site Status RECRUITING

University of Iowa

Iowa City, Iowa, United States

Site Status RECRUITING

Johns Hopkins Hospital

Baltimore, Maryland, United States

Site Status RECRUITING

University of Minnesota

Minneapolis, Minnesota, United States

Site Status RECRUITING

Children's Mercy Kansas City

Kansas City, Missouri, United States

Site Status RECRUITING

Maimonides Medical Center

Brooklyn, New York, United States

Site Status RECRUITING

NYU Langone Health

Garden City, New York, United States

Site Status RECRUITING

Montefiore Medical Center and Albert Einstein College of Medicine

The Bronx, New York, United States

Site Status RECRUITING

University Hospitals Cleveland Medical Center

Cleveland, Ohio, United States

Site Status RECRUITING

Nationwide Children's Hospital

Columbus, Ohio, United States

Site Status RECRUITING

Vanderbilt University Medical Center

Nashville, Tennessee, United States

Site Status RECRUITING

Christus Children'S

San Antonio, Texas, United States

Site Status RECRUITING

University of Utah

Salt Lake City, Utah, United States

Site Status RECRUITING

University of Virginia

Charlottesville, Virginia, United States

Site Status RECRUITING

Countries

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United States

Central Contacts

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Lisa Matesevac, AuD

Role: CONTACT

(760)420-5878

Caroline Vrana-Diaz, PhD

Role: CONTACT

Facility Contacts

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Diane Stafford, MD

Role: primary

650-721-1811

Lynne Bird, MD

Role: primary

858-966-5808

Lukas Jacyniuk Moskalis

Role: primary

720-777-5698

Jean L McColl

Role: primary

404-778-8619

Christian Hanson

Role: primary

(470) 666-1884

Emma Carlson

Role: primary

319-467-0634

Ann Scheimann, MD

Role: primary

410-955-8769

Kayla Arndt, MA

Role: primary

612-626-0240

Krystin Sanford, MAEd

Role: primary

816-601-4493

Deepan Singh, MD

Role: primary

(718) 283-7864

Marilyn Richardson

Role: primary

516-969-6120

Casara Ferretti, MD

Role: primary

718-839-7542

Mehjabeen Hossain, MS

Role: primary

216-844-5663

Colin Peachey, BSN, RN

Role: primary

(614) 722-2606

Elizabeth Roof, M.A., H.S.P.

Role: primary

(615)343-333

Elizabeth Roeder, MD

Role: primary

210-704-0407

Annabelle Wilcox, PA-C

Role: primary

801-587-3605

Gabrielle Laskey, MA

Role: primary

434-243-8236

Other Identifiers

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FPWR-VNS-R-3-01

Identifier Type: -

Identifier Source: org_study_id

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