Back To Results

Study of Carbetocin Nasal Spray for the treatment of Hyperphagia in Prader-Willi Syndrome

UCI Specialty Area: Pediatrics
Principal Investigator: Virginia Kimonis
Official Title: A Phase 3, Randomized, Double-blind, Placebo-controlled, Parallel-group Study of Carbetocin Nasal Spray for the Treatment of Hyperphagia in Prader-Willi Syndrome
A Study On:
Pediatrics

Study Description

This is a 12-week, multicenter, randomized, double-blind, placebo-controlled, parallel-group study comparing carbetocin nasal spray 3.2 mg TID with placebo (matched placebo nasal spray TID) in subjects with PWS.

Eligibility

  1. Male or female and 5 through 30 years of age
  2. Prader-Willi syndrome with a documented disease-causing mutation
  3. Increased appetite with decreased satiety accompanied by food seeking (consistent with PWS Nutritional Phase 3)
  4. HQ-CT total score of ≥13 at Screening and Baseline
  5. CGI-S score for hyperphagia in PWS of ≥4 at Screening and Baseline
  6. Lives with a caregiver who understands and is willing and able to adhere to study-related procedures and is willing to participate in all study visits
  1. Genetically diagnosed with Schaaf-Yang syndrome or another genetic, hormonal, or chromosomal cognitive impairment besides PWS
  2. An active upper respiratory infection at the Screening visit or the Baseline visit
  3. Any clinically significant cardiovascular disorder, renal, hepatic, gastrointestinal, or respiratory disease, including severe asthma
  4. History of, or current, cerebrovascular disease, brain trauma, epilepsy, or frequent migraines. A history of febrile seizures is not exclusionary
  5. Nasal surgery within 1 month of Screening visit or planning to have nasal surgery during the study.
  6. Unwilling to abstain from nasal saline, other nasal irrigation, and other intranasal medications during the Screening period and through the treatment period of the study
  7. Clinically significant irritability or agitation, requiring initiation of or increase in the dose of antipsychotic medication, within the 6 months prior to the Screening visit
  8. Used prostaglandins, prostaglandin analogues, or prostaglandin agonists in the 3 months prior to the 9. Baseline visit. Inhibitors of prostaglandin synthesis, such as nonsteroidal anti-inflammatory drugs, are not exclusionary.
  9. Started a glucagon-like peptide 1 (GLP-1) agonist within the 6 months prior to the Screening visit. 11. Treatment with GLP-1 agonist is allowed if the subject has been taking it for more than 6 months prior to Screening.
  10. Used oxytocin, desmopressin (DDAVP), or tesofensine within 6 months prior to the Baseline visit
  11. Active psychotic symptoms, a history of psychotic symptoms, or a psychotic disorder
  12. History of suicide attempt or inpatient psychiatric hospitalization
  13. New food-related interventions, including environment or dietary restrictions, within 1 month of the Screening visit

Additional inclusion/exclusion criteria apply. Subjects will be evaluated at screening to ensure that all criteria for study participation are met.

A female nurse attentively holds a tablet, ready to assist with patient care and medical information.

Interested in Participating in this Trial?

Thank you for your interest with our team.

One of our specialists will be in contact with you soon.

Privacy Policy

University of California, Irvine - Study Information Sheet

UCI Center for Clinical Research Potential Participant Database

Lead Researcher
Maheswari Senthil, MD
UCI Center for Clinical Research

In the instance of parental permission, “You” refers to “Your child.”

  • Please read the information below and ask questions about anything that you do not understand. A
    researcher listed above will be available to answer your questions.
  • You are being asked to participate in a clinical research potential participant database to identify
    potential clinical research studies you may be eligible for. Participation in this database is voluntary.
    You may choose to skip any question. You may refuse to participate or discontinue your involvement
    at any time without penalty or loss of benefits. You are free to withdraw from this database at any
    time. If you decide to withdraw from this database you should notify the research team
    immediately    .
  • You are eligible to participate in this study if you may be interested in participating in a research trial at UCI Health
  • We would like to interview you to learn more about your eligibility for current clinical research studies at UCI. The interview will last about 5-30 minutes, depending on the extent of your medical history.
  • Possible risks/discomforts associated with the database are hearing about potential clinical research studies that may not be of interest to you, invasion of your privacy, or a potential breach of confidentiality.
  • There are no direct benefits from participation in the database. However, this database may help inform you on clinical research studies that you may be eligible for. Participation in UCI clinical research studies help us gain important data and knowledge that may lead to improved treatment options for people with similar conditions to you in the future.
  • There are no alternative procedures available. The only alternative is not to participate in this study.
  • There is no compensation for your participation in this database.
  • There is no cost to you for participation in this database.
  • All information collected will be stored confidentially in a world-class securely-hosted application, “RealTime CTMS,” with robust data protections and encryption. This application has HIPAA compliant architecture and application rules to ensure data privacy and regulatory compliance.
  • Researchers will use your information to assess your eligibility for current and future clinical research studies at UCI. You may opt out at any time.
  • The research team, authorized UCI personnel, and regulatory entities, may have access to your study records to protect your safety and welfare.
  • While the research team will make every effort to keep your personal information confidential, it is possible that an unauthorized person might see it. We cannot guarantee total privacy.
  • Future Research Use
    Researchers will use your information to conduct this study. Once the study is done using your information, we may share them with other researchers so they can use them for other studies in the future. We will not share your name or any other private identifiable information that would let the researchers know who you are. We will not ask you for additional permission to share this de-identified information
  • Data Retention - In accordance with UC Office of the President policy, information will be retained for 10 years after the end of the calendar year in which the research is completed.
  • If, during the course of this study, significant new information becomes available that may relate to your willingness to continue to participate, this information will be provided to you by the research team listed at the top of the form.
  • If you have any comments, concerns, or questions regarding the conduct of this research please contact the researchers listed at the top of this form
  • Please contact the UCI Institutional Review Board by phone, (949) 824-8170, by e-mail at IRB@research.uci.edu or at 160 Aldrich Hall, Irvine, CA 92697-7600 if you are unable to reach the researchers listed at the top of the form and have general questions; have concerns or complaints about the research; have questions about your rights as a research subject; or have general comments or suggestions.

What is an IRB? An Institutional Review Board (IRB) is a committee made up of scientists and nonscientists. The IRB’s role is to protect the rights and welfare of human subjects involved in research. The IRB also assures that the research complies with applicable regulations, laws, and institutional policies.