A Pilot Study of Alemtuzumab (Campath[R]) in Patients With Myelodysplastic Syndrome
Status:
Completed
Trial end date:
2017-02-06
Target enrollment:
Participant gender:
Summary
This study will evaluate the safety and effectiveness of a genetically engineered antibody,
alemtuzumab (Campath[R]) on patients with myelodysplastic syndrome. MDS is made up of
malignant stem cell disorders that can mean low levels of red blood cells-that is, anemia-and
low counts of white blood cells and platelets. Patients with MDS are at risk for infection,
spontaneous bleeding, and possible progression to leukemia, a cancer of bone marrow. Although
bone marrow can produce some blood cells, patients with MDS experience a decrease in
production of blood cells. Alemtuzumab recognizes specific types of white cells called
lymphocytes and destroys them. This study will examine not only the usefulness of the
medication but also the side effects in patients with MDS.
Patients ages 18 to 72 who have MDS that requires transfusions and who do not have HIV or a
life expectancy of less than 6 months may be eligible for this study. Screening tests include
a complete physical examination and medical history. There will be a collection of about 8
tablespoons of blood for analysis of blood counts as well as liver, kidney, and thyroid
function; a pregnancy test; an electrocardiogram (EKG) to measure electrical activity of the
heartbeat; an echocardiogram (ECHO), which uses sound waves to evaluate heart function;
wearing of a Holter monitor for 24 hours while the electrical activity of the heart is
recorded; and a bone marrow biopsy. Patients should not receive any vaccines when taking
alemtuzumab or for at least 12 months after the last dose. In addition, patients should not
take the herbal supplements Echinacea purpurea or Usnea 2 weeks before beginning the study
and during it.
For the study, all patients will receive a test dose of 1 mg of alemtuzumab infused into a
vein during the course of 1 hour. If the dose is tolerated, the medication will be given at
10 mg doses into the vein for 10 days, as an infusion of 2 hours. Blood samples of 2
tablespoons will be taken daily, and vital signs will be measured daily. The ECHO and 24-hour
Holter monitoring will be repeated after patients receive the last dose of the medication.
Because suppression of the immune system results from a decrease in white cells that fight
infections, patients will take medications to protect them against infections and to treat
them if infections occur. If needed, patients will receive blood transfusions for their MDS.
Side effects of alemtuzumab involve a temporarily significant lowering of the number of red
blood cells, white cells, and platelets. Side effects of the infusion can be rigidity, or
stiffness, and fever, as well as risks of infections resulting from the decrease of white
blood cells. Blood counts and reactions to all procedures will be carefully monitored
throughout the study. After patients receive the last dose of alemtuzumab, they will have
follow-up by their referring doctor or at NIH. They must be able to return to NIH after 1
month, 3 months, 6 months, and annually for 5 years after the study. At follow-up visits,
there will be blood tests to reevaluate blood counts and test for the presence of viruses.
Blood tests will be done weekly for the first 3 months after patients have completed taking
alemtuzumab, every other week until 6 months, and then annually for 5 years. There will also
be a repeat ECHO at the 3-month visit, and a repeat bone marrow biopsy at the 5-month and
12-month follow-up visits, and as needed after that.
This study may or may not have a direct benefit for participants. For some, the antibody may
improve blood counts and decrease the need for transfusions. Knowledge gained in the study
may help people in the future.