A Randomized, Double Blind, Placebo-Controlled Trial of Megestrol Acetate as an Appetite Stimulant in Malnourished Children With Cancer
Overview of the study phases Prior to the randomization phase, we will draw some blood work
(1 teaspoon) with regular blood work to see if you/your child meet the final eligibility
requirements. If the results are within normal limits, we will proceed on to the
Randomization Phase. If the results are not within normal limits for your/your child's age,
then you/your child will not be able to proceed with the study and we will pass these test
results on to your regular oncologist.
Randomization Phase If you/your child agree to take part in this study you/your child will
have an equal chance of receiving one of two possible liquids: Megace (7.5 mg/kg/day taken
once in the morning) or the same amount of a placebo (a placebo is a substance that looks
and tastes exactly like the real medicine, however it is an inactive substance that contains
no drug). This assigning to the drug or the placebo is called randomization. Neither you
nor your physician can choose or know which treatment you are receiving. This "blinding" is
necessary to fairly test these drugs. In an emergency, your physician can immediately find
out which of the drugs/treatments you are receiving.
Once randomized, you/your child will be given a supply of the drug or the placebo. Both the
drug and the placebo are a lemon-lime flavored liquid. There are no pills or tablets to
take. You/your child are to take the liquid by mouth in the amount prescribed once per day.
You will take the liquid every day for 90 days (3 months). The liquid is to be taken half
an-hour before breakfast.
Pre-treatment Information Collection Phase Before you/your child start taking the liquid, we
will collect some initial information. This will include taking your/your child's weight
and height. A dietitian will perform an assessment of you/your child's body composition
using a tool called a caliper. This will briefly pinch the skin at the back of your/your
child's arm. It will not hurt. The dietitian will also measure your/your child's upper
arms using a tape measure.
A research nurse will ask a series of questions called a Quality of Life assessment. This
will tell us about how you/your child feels before starting the study.
Afterwards, you/your child will have a scan called a DEXA (stands for Dual Energy X-Ray
Absorptiometry) scan done to assess your/your child's body composition. A DEXA scan does
not hurt but requires that you/your child lie still on the table while the scanner moves
over you/your child. Like all scans, there is some radiation exposure. However, the total
amount of radiation is very small (about half the radiation exposure of a normal chest
xray). This should not cause any problem for you/your child.
We will also draw some additional blood (approximately 1 teaspoon) from your central line.
You/your child will not have extra pokes for the purposes of this study.
Follow-up Phase You/your child will be followed closely by our team of doctors and
dieticians during the 90 days you/your child are on the study. Follow-up will occur when
you are normally scheduled to see your doctor. This can occur at either the Pediatric
Oncology outpatient clinics or when you are admitted to the BC Children's Hospital
(Vancouver) or Stollery Children's Hospital (Edmonton). You do not have to make special
appointments for the purpose of this study.
We will watch your weight closely during the 3 months of the study. If you/your child lose
an additional 15% from your/your child's baseline (starting) weight we will stop you taking
the medication or placebo. You will then receive regular medical care. This may include
starting tube feeding or TPN.
We will also take additional blood work at least every 2 weeks during the 3 months. This
can be done through your/your child's central line at times when regular blood work is being
drawn. Once a month our research nurse will contact you by phone to see how things are
going and ask questions about possible side effects.
Study Conclusion Phase At the end of the study we will again collect all the information as
we did in the Pre-treatment Information Collection Phase. This will include your/your
child's weight, height, caliper and arm measurements as well as asking the same quality of
life questions. You will then have a second DEXA scan done.
After 90 days you will be told whether you received the drug (Megace) or the inactive
The time commitment for you/your child to take part in this research should be minimal.
Each DEXA scan (you/your child are asked to complete 2 DEXA scans during the study) will
take approximately 1 hour. Each study nurse interview (3) should take approximately 10-15
minutes each time. The quality of life questions will be asked three times and take
approximately 5-10 minutes each time to complete.
Allocation: Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Parallel Assignment, Masking: Double Blind (Subject, Investigator), Primary Purpose: Treatment
To determine if daily administration of megestrol acetate (MA) for 3 months compared to placebo prevents weight loss or improves weight gain in children with cancer-associated anorexia-cachexia.
David Dix, MD
University of British Columbia
Canada: Health Canada