What study drug will be used?
We will be using megestrol, an appetite stimulant. Megestrol is used to treat loss of appetite and weight loss. Your child will either receive this drug or placebo. Both the drug and placebo will come in liquid form and can be given through your child's feeding tube. Through this study, we hope to find out if giving children megestrol helps them transition from being tube fed to eating by mouth.
Will I know if my child is receiving the study drug?
No. You and the study team will not know which drug your child is receiving until the end of the study. The reason you and the study team will not know is to make sure all participants in the study are treated the same. This will help us find out if the study drug helps children transition to oral feedings or if it does not.
If your child has significant changes to his or her medical care or health changes that require hospitalization, we will tell you and your child's medical team which drug your child is taking . If this happens, you will no longer be included in the study.
How many study visits will my child attend?
You and your child will attend 4 in-person visits at a clinic near you. These will happen at Weeks 1, 10, 14, and 24 of the study. These study visits will last about 1 hour each.
You and your child will also have remote sessions, which will happen over a computer or tablet on a video-conferencing app called Zoom or over the phone. These visits occur every 2 weeks and last about 30 minutes each. Over the study, you and your child will have 12 of these sessions.
What happens during the clinic visits?
The study physician will do a physical exam and ask about any symptoms your child may be having. Your child's weight and diet will be monitored closely at each visit. We will also ask you to collect a stool sample from your child at home prior to each visit.
At the Week 10 visit, you will be given either the megestrol or placebo and be asked to give the medication to your child for 4 weeks. You and the physician will come up with a plan to taper your child's tube feedings. This plan will reduce your child's tube feeding by 10% each day over 10 days, until your child is completely off the tube-feeding. Your child will have a blood draw this week and at Week 14 to make sure that your child's body is handling the study medication well. The study physician will continue to monitor your child's weight and diet to make sure they are getting enough calories by mouth.
What happens during the remote feeding sessions?
A feeding specialist from our team will review your child's feeding behaviors and provide coaching to improve your child's eating and mealtime skills. These calls will be scheduled around your child's typical mealtime so the feeding specialist can see your child eating. During the first 10 weeks of the study, you will work on improving your child's feeding skills and weight gain to get your child ready to transition off the tube feedings. The feeding specialist will also coach you through the transition process, help you problem-solve, and give you ideas for high-calorie foods and tips for successful mealtimes that will make the transition to eating by mouth easier. After your child transitions to eating by mouth, your feeding coach will help you plan high-calorie meals/snacks to maintain and increase your child's weight.
How will my child transition off the tube feedings?
During weeks 1-10, you will be working with your feeding specialist over the remote feeding calls to improve your child's feeding skills. This will help your child get ready to transition to oral eating. You and your child's study physician will discuss a plan to transition off tube feedings at the Week 10 visit. Your child will also be given either the megestrol appetite stimulant or placebo to take during this transition period.
You will be asked to reduce your child's tube feeding by 10% each day over 10 days, until your child is completely off the tube feeding. The study team and your feeding specialist will help you through the transition process and will be available to answer any questions or give feeding advice. Your child will be monitored closely after stopping the tube feedings to make sure they are maintaining their weight and getting enough food by mouth.
Does transitioning off tube feedings work?
Our study team did a similar study that showed children successfully transitioned off their tube feeds when asked to follow a similar protocol. This study was testing a different study drug called amitriptyline. All children received megestrol as well as amitriptyline or placebo. We found that children were able to transition to 100% eating by mouth by the end of the study regardless if they received the study drug or placebo. Because of this, we are doing the current study to test if the megestrol plays a role in helping children transition to oral feeding.
A copy of the study publication can be found here.
Is the study safe?
About 5% of children and adults have side effects while taking megestrol. We will be closely monitoring your child while on the medication and while transitioning off the tube feedings. If your child experiences side effects or is not tolerating the tube feeding transition well, they will be removed from the study.
Will I get paid to be part of this research study?
Yes. Participants will be paid $100 for completing each clinic visit. You may be paid up to $400 if you complete all four clinic visits.