Medical Treatment of Colitis in Patients With Hermansky-Pudlak Syndrome

Withdrawn before enrolling · Phase 2

Conditions studied: Hermanski-Pudlak Syndrome, Colitis, Cytokines, Lymphocytes, Drug Evaluation

In brief

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.

Key facts

Study ID

NCT00514982

Run by

National Institute of Allergy and Infectious Diseases (NIAID)

People needed

0

Starts

2007-08-07

Expected to finish

2011-03-08

Last updated by the study team

2017-07-02

Who can join

Age: 18 and older. Sex: any. Healthy volunteers: not accepted.

You may not qualify if…

• A subject is excluded from the study if any of the following criteria are met:
• GENERAL CRITERIA:
• Has any clinically significant disease (e.g., renal, hepatic, neurological, cardiovascular, pulmonary, endocrinology, psychiatric, hematological, urologic, or other acute or chronic illness) that in the opinion of the investigator would make the subject an unsuitable candidate for this trial.
• Inability to meet any of the above inclusion criteria.
• Is a woman who has a positive serum pregnancy test or who is breast-feeding.
• Has any of the following clinical chemistry values:
• AST greater than 2.5 times the upper limit of normal (ULN).
• ALT greater than 2.5 times the ULN.
• Serum bilirubin greater than 1.5 times the ULN.
• Serum creatinine greater than 1.5 times the ULN.
• Alkaline phosphatase greater than 2.5 times the ULN.
• Has a hemoglobin level less than 8.0 g/dL or hematocrit less than 26 percent.
• Has a PT INR greater than 1.3 or a PTT greater than 3 seconds compared to control value.
• Has the following cell counts:
• Platelet count less than 80,000 or greater than 950,000.
• White blood cell count less than 1200.
• Neutrophil count less than 700.
• Has a current infection requiring intravenous antibiotics, a serious local infection (e.g., cellulitis, abscess) or systemic infection (e.g., pneumonia, septicemia).
• Has a history of cancer within the past 5 years, with the exception of excised basal cell carcinoma, squamous cell carcinoma of the skin, or cervical carcinoma in situ.
• Had a dependency for any illicit drug, chemical or alcohol within the past 5 years.
• Has a history of active tuberculosis (TB) (or a positive PPD skin test or chest x-ray with findings suggestive of old TB infection including calcified nodular lesions, apical fibrosis, or pleural scarring), acute or chronic hepatitis B, hepatitis C, human immunodeficiency virus (HIV).
• History of central nervous system demyelinating disease, or systemic lupus erythematosus.
• Unable to keep to the scheduled appointments and other test to watch for changes in symptoms and side effects of treatment.
• GASTROINTESTINAL CRITERIA:
• History of colectomy, partial colectomy, current ostomy, pouchitis, or small bowel resection within the past 6 months, or short gut syndrome.

Where it is running

• National Institutes of Health Clinical Center, 9000 Rockville Pike — Bethesda, Maryland, United States

Full record on ClinicalTrials.gov

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