Study Purpose
Carbetocin (LV-101) is an investigational drug that was created to have effects in the body like oxytocin. Carbetocin is not approved in the United States, but is approved in some other countries for treatment of women with excessive bleeding after giving birth via caesarean section. This Phase 3 study of intranasal carbetocin (LV-101) is a randomized, double-blind study with an 8-week, placebo controlled period, followed by a long-term follow-up period of 56 weeks during which all participants will receive active treatment with LV-101. At Week 8, participants who were randomized to placebo in the placebo-controlled period will be randomized to one of the two LV-101 doses, administered three times per day before meals.
Recruitment Criteria
Study Type: Interventional
Eligible Ages: 7-18 years (estimated)
Other Criteria: PWS Nutritional Phase 3
Trial Details
Trial Id: NCT03649477
Phase: 3
Duration: 8 weeks of double blind, placebo controlled study, followed by 56 weeks of open label treatment
Status: recruiting
Lead Sponsor: Levo
Countries: United States
Additional Details: Outcome measures include changes in hyperphagia behavior, obsessive and compulsive behaviors, anxiety and global impression
Eligibility Criteria
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Inclusion Criteria:
- Genetically-confirmed Prader-Willi syndrome
- Provide voluntary, written informed consent (parent(s) / legal guardian(s) of participant); provide voluntary, written assent (participants, as appropriate)
- PWS Nutritional Phase 3 (hyperphagic, rarely feels full)
Exclusion Criteria:
- Living in a group home
- Genetically diagnosed Schaaf-Yang syndrome or other genetic, hormonal, or chromosomal cognitive impairment
- New food-related interventions, including environment or dietary restrictions, within 1 month of screening
- Dose of any allowed chronic concomitant medications or supplements that have not been stable for ≥3 months prior to the study or is not expected to remain stable while participating in the study; adjustments in growth hormone dose ≤10% are not exclusionary
- Presence of cardiovascular disorders, epilepsy, frequent migraines, or severe asthma
- More than 3 episodes of sinusitis in the 12 months prior to Screening Visit or presence of nasal diseases that may affect deposition of intranasal medication
- Unwilling to abstain from nasal saline, other nasal irrigation, or other intranasal medications for 2 weeks prior to the Baseline visit and during the 8-week, placebo-controlled period of the study
- Use of weight loss medication, oxytocin, carbetocin, or vasopressin in the 6 months prior to screening
- Participation in an interventional research study involving another investigational medication or device in the 6 months prior to screening or during the study
- Based on the judgment of the Investigator, is unsuitable for the study for any reason, including but not limited to unstable medical condition, inability to comply with the protocol, or other risk to subject or to the integrity of the study
This Study is enrolling at the following sites:
University of Florida
Gainesville, Florida
Principal Investigator: Jennifer Miller, MD
Contact: Beverly Giordano 352-294-5280 bgiordano@peds.ufl.ed
Vanderbilt University School of Medicine
Nashville, Tennessee
Principal Investigator: Ronald Cowan, MD, Sub-Investigator: Elizabeth Roof
Contact: Hailee Hunt 615-343-0915 hailee.m.hunt@vanderbilt.edu
Additional sites coming soon.
