Prader-Willi syndrome (PWS) is a neurodevelopmental disorder which causes intellectual disability, a deficiency of growth hormones, compulsive behavior, a high risk of obesity, and hyperphagia, a symptom which can be life-threatening.
A new Phase 3 trial is currently enrolling patients (ages 7 to 18) to investigate the effect of intranasal carbetocin (LV-101) on managing some of the condition’s most debilitating symptoms- obsessiveness, compulsive behavior, and anxiety.
A Phase 2 trial of the drug showed the potential of its efficacy as a treatment. The Phase 3 trial will further examine its efficacy as well as its tolerability and safety.
About the trial
The Phase 3 trial is being conducted by Levo Therapeutics. It will occur at various sites across the United States but enrollment is currently open at Vanderbilt University and the University of Florida. To keep updated on the addition of future sites, keep a close eye on clinicaltrials.gov. Updates on enrollment for the trial will also be published on levotx.com.
The trial will be placebo-controlled for 8 weeks. Following that period, a long-term study will continue for 56 weeks. During this time, patients who first received placebo will be given a dose of LV-101. The drug will be given three times every day, before the patient eats their meals.
The effects of this treatment will be measured by data collected by the clinician as well as the caregiver of the patient. This data will include measures of hyperphagia and obsessive and compulsive behaviors. Adverse events, physical exams, and laboratory tests will also determine the tolerability of the drug.
Carbetocin has already been approved in more than 90 countries as a way to prevent excessive bleeding and uterine atony during c-sections. Hopefully, this Phase 3 trial will confirm its efficacy for the treatment of some of the most debilitating symptoms of PWS. Ultimately, this therapy has the potential to improve the quality of life for PWS patients and their families.
Stay tuned for updates on this trial! In the meantime you can read more on its development and previous studies concerning the drug here.
