Colitis Clinical Trial
Official title:
An Observational Study of the Immunopathogenesis of and Response to Step-Up Inflammatory Bowel Disease Therapy for Hermansky-Pudlak Syndrome-Associated Colitis
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.
The primary purpose of this study is to detect patterns of immune abnormalities in the
colitis associated with Hermansky-Pudlak Syndrome (HPS). Additionally we aim to document the
clinical response to and tolerance of conventional inflammatory bowel disease (IBD) therapy
for HPS patients with active colitis associated with Hermansky-Pudlak Syndrome (HPS). HPS is
a rare autosomal recessive disorder causing dysfunctional lysosome-related organelle
formation and trafficking resulting in oculocutaneous albinism and a bleeding diathesis
secondary to platelet dysfunction. Associated conditions include pulmonary fibrosis, IBD, and
systemic ceroid deposition. The associated IBD has been reported to occur at a higher
frequency in the HPS-1 and HPS-4 subtypes compared to the prevalence of IBD in non-HPS
populations. HPS IBD has clinical features of both ulcerative colitis (UC) and Crohn's
disease, but histologically resembles Crohn's disease in that granulomas are commonly seen in
the mucosa of the intestine. The pathogenesis of HPS IBD is not fully understood and little
data beyond descriptions of the clinical and histologic manifestations have been published.
Furthermore reports on treatment of the colitis in HPS patients are largely anecdotal, and
our own experience suggests that many patients may be under-treated.
HPS patients with active colitis will be enrolled into this prospective treatment study.
Endpoints for the immunopathogenesis studies will include baseline measurements of and
changes in immune cell populations, cytokine, and chemokine expression. In the blood and gut
mucosa. Endpoints for the study of response to treatment will include changes in clinical,
endoscopic, and histologic scores as well as the rate and severity of adverse events.
Descriptive summary statistical analysis (n, mean, median, standard deviation, minimum and
maximum range) and simple correlations of clinical response variables with immune parameters
will be done.
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