Ferring Pharmaceuticals concluded a Phase II trial in 2014. The goal of the study was to evaluate the safety and effectiveness of an intranasal oxytocin analogue in treating hyperphagia in children and young adults with Prader-Willi Syndrome. Participants were between the ages of 10-18, in nutritional phase 3, and with a genetic confirmation of PWS.
This study is now complete, and we look forward to seeing the results soon. If the study shows positive effects and a good safety profile, we would expect Ferring to next develop a Phase III study to demonstrate efficacy in a larger number of patients.
Below is a description of the study From Elizabeth Roof at Vanderbilt University
We have been approved to do an investigational drug treatment study. We are working with Ferring Pharmaceuticals to try a new nasal spray with carbetocin, an selective oxytocin agonist. We are seeing 20 people here and 20 people with PWS between the ages of 10-18 in NY and about half will get the study drug and half will get a placebo that looks and smells like the study drug. We will not know, nor will the doctor or you or your child know.
We are scheduling people now for every week in the coming 6 months. The study involves 2 visits 15 days apart and the time line is very strict with new patients being enrolled M-W. You will be pre-screened on the phone to make sure your child fits all the criteria which includes HYPERPHAGIA which is one of the possible targets of the drug. We would ask about medication changes, recent surgeries and any chronic nose problem or severe asthma that requires daily treatment. We would make sure that you understand all of the parts of the study by sending you a consent form to read and ask us all questions before your child enrolled.
If your child qualified, you would come to Vanderbilt on a Monday morning with your child and we would ask a lot of questions about behavior and health, they would get their blood drawn, give a urine sample, have an EKG and get height weight and vitals taken. A doctor would do a physical and make sure they were healthy enough to participate. If so, you would leave that afternoon, go back to the hotel and early the next morning we would see you child again, give them the first dose of spray and teach you how to give it. We would monitor their vital signs and take 3 blood samples to see how much of the medicine was in their blood at certain times. Your child could bring their schoolwork, games and movies to watch and we would have some fun games for them to do during downtime. They would get a dose before each meal on Tuesday and Wednesday and we would continue to monitor all vital signs before we sent you home on Wednesday. You would sign for and get daily delivery of the drug and give it to your child before breakfast, lunch and dinner. You would write down what time you gave it every day in a diary and give it to the person who picks up your used medication bottles. On Tuesday, Day 8, I would call you and find out how your child is doing. On Day 15, Tuesday, you would come back in and let us do a physical and EKG and draw one last sample of blood. Then you would return home and at the end of the week, I would give one more call to see how your child is doing.
I know this study involves a lot of time and travel, but many of our families handle more than this every day with Drs. appointments and daily injections of GH. We would love to hear from you if you think your child qualifies and you are willing to commit to the this short, but pretty intense schedule.