This study will examine the effectiveness of G-CSF in treating patients with Crohn's
disease-a long-term recurring inflammation of the small and large intestine. Patients may
have swelling and bleeding of the intestinal lining, which can lead to infection and
abdominal pain, weight loss, fever, diarrhea, bloody stools, fistula (connections between the
skin and intestine), intestinal blockages, and abscesses. Although there are various
treatments for Crohn's disease, many patients continue to have inflammation that is difficult
to control or severe side effects from the medications. G-CSF is an approved drug that is
used to increase white blood cell counts. Other cells, immune cells, exposed to G-CSF can
develop a specific immune action-a Th-2 response-that decreases the inflammatory response in
Crohn's disease-a Th-1 response.
Patients 18 years of age or older who have had mild to moderately severe Crohn's disease for
at least 4 months may be eligible for this study. Candidates will be screened with a medical
history and possible review of medical records, physical examination, blood tests,
electrocardiogram (EKG), urine and stool analyses and, for women, a pregnancy test. They will
fill out a Crohn's Disease Activity Index questionnaire daily for 7 days and an Inflammatory
Bowel Disease questionnaire.
Participants will have G-CSF therapy. Before starting therapy, they will have a series of
pre-treatment tests, including a colonoscopy and leukapheresis. Colonoscopy is an examination
of the colon. For the procedure, patients are given a medication to lessen anxiety and any
discomfort. An endoscope-a lighted flexible tube-is inserted into the rectum, allowing
examination of the extent of inflammation. The endoscope can also be used to take pictures of
the colon and extract tissue samples for testing (biopsy). Leukapheresis is a procedure for
collecting quantities of white blood cells. Whole blood is collected through a needle placed
in an arm vein and circulated through a machine that separates it into its components. The
white cells are removed, and the rest of the blood is returned to the body, either through
the same needle used to draw the blood or through another needle placed in the other arm.
After the colonoscopy and leukapheresis, patients receive G-CSF injections every day for 29
days. The patient or a caregiver, such as a family member, will be taught to give the
injections. Blood samples will be collected on treatment days 4, 8, 11 and 15, and a physical
examination and interview, blood tests and a stool exam will be done once a week. Patients
will have a repeat colonoscopy and leukapheresis 24 hours after the last treatment dose (day
29).
After the 29-day treatment, patients will be followed in the clinic as follows:
- Week 4 after treatment - physical exam and interview, routine blood work and stool exam
- Week 8 - interview and blood work
- Week 16 - interview, blood work and stool exam
- Week 24 - physical exam and interview, blood work, stool exam and colonoscopy
Phase:
Phase 1
Details
Lead Sponsor:
National Institute of Allergy and Infectious Diseases (NIAID)