Medical Treatment of Colitis in Patients With Hermansky-Pudlak Syndrome
Status:
Withdrawn
Trial end date:
2011-03-08
Target enrollment:
Participant gender:
Summary
This study will determine if medical treatment of colitis (inflammation of the colon
resulting in loose bowel movements, rectal bleeding, and belly pain) that is used for other
colitis conditions, such as Crohn's disease and ulcerative colitis, is safe and effective for
treating colitis in patients with Hermansky-Pudlak syndrome (HPS). HPS is a hereditary
disorder that causes albinism, visual impairment, and abnormal bleeding. Some patients also
develop colitis, pulmonary fibrosis, and kidney disease.
Patients with HPS and colitis who are 18 years of age or older may be eligible for this
study. Participants receive treatment for their colitis symptoms with one or more of several
study drugs, which include mesalamine (5-ASA), corticosteroids, infliximab and
6-mercaptopurine, adalimumab and tacrolimus. The drugs are added to the treatment plan one at
a time to find the combination that works best for the individual patient. Patients who
respond to one or more of the medications may continue treatment with that same combination
for up to 6 months.
Regular clinic visits are scheduled for blood tests, symptoms ratings questionnaires and
periodic physical examinations and colonoscopies to measure the response to treatment and
evaluate any side effects.
Phase:
Phase 2
Details
Lead Sponsor:
National Institute of Allergy and Infectious Diseases (NIAID)