Deforolimus trial

I finally got to take a look at the informed consent documents for Ariad’s phase III trial of oral deforolimus. The trial is intended to see if the drug improves progression free survival for patients who have had successful chemotherapy for advanced sarcoma. 

It is a double-blind, placebo-controlled study: Participants are assigned randomly into a placebo group or a deforolimus group, and neither they nor their doctors know which group they are part of. The odds of getting the placebo are 50/50, a coin-flip -- a huge negative for electing to take part in this particular study, obviously. Ariad’s thinking is that the standard of care after completing sarcoma chemotherapy successfully is watchful waiting; therefore, study participants randomized into the placebo arm of the study are receiving standard treatment.

Here are some notes about how the study works and the side effects of the drug that I gleaned from the documents:

Patients must have completed at least four cycles of chemotherapy without any worsening of the cancer; 1, 2 or 3 previous treatments (chemo completed 3-8 weeks before being randomized into the study)

Randomized in either placebo arm or treatment arm; double-blind

14 days of pre-study screening; includes review of scans (process may take up to 3 weeks, I think; little confused by this part)

Study involves bloods every month; scans every 8 weeks

Kicked out of study if disease progresses or toxicity becomes unacceptable

No crossover from placebo arm to treatment arm

Ongoing monitoring

Greater than 20 percent side effects:

weakness and fatigue
appetite loss
mouth sores or sore throat
rash
low red blood cell counts
diarrhea
low white blood cell counts
nausea
increased blood fat levels
vomiting
low platelet counts
taste changes

Less frequent side effects:

headache
fever
weight loss
tingling sensation in the hands or feet
hair loss
high blood sugar levels
constipation
increased liver enzyme levels
low blood levels of sodium, potassium and phosphate
nail discoloration and brittleness

There are also some dire less than 2 percent and less than 1 percent side effects.