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Note: This record shows only 22 elements of the WHO Trial Registration Data Set. To view changes that have been made to the source record, or for additional information about this trial, click on the URL below to go to the source record in the primary register.
Register: ClinicalTrials.gov
Last refreshed on: 16 December 2017
Main ID:  NCT00514982
Date of registration: 09/08/2007
Prospective Registration: Yes
Primary sponsor: National Institute of Allergy and Infectious Diseases (NIAID)
Public title: Medical Treatment of Colitis in Patients With Hermansky-Pudlak Syndrome
Scientific title: An Observational Study of the Immunopathogenesis of and Response to Step-Up Inflammatory Bowel Disease Therapy for Hermansky-Pudlak Syndrome-Associated Colitis
Date of first enrolment: August 7, 2007
Target sample size: 0
Recruitment status: Withdrawn
URL:  https://clinicaltrials.gov/show/NCT00514982
Study type:  Interventional
Study design:  Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).  
Phase:  Phase 2
Countries of recruitment
United States
Contacts
Key inclusion & exclusion criteria

- INCLUSION CRITERIA:

A subject is eligible for the study if all of the following criteria are met:

- Has given written informed consent prior to screening.

- Age 18 years old or greater.

- Has confirmed diagnosis of HPS prior to screening.

- Has confirmed diagnosis of IBD prior to screening.

- The presence of active disease as defined by a SCCAI score greater than or equal to 5.

- Negative results on stool examination for culture of enteric pathogens (Salmonella,
Shigella, Yersinia, Campylobacter, Vibrio, E. coli O157/H7), Clostridium difficile
toxin assay, enteric parasites and their ova (including Giardia and Cryptosporidia).

- If currently receiving medication for their IBD, patients may be on a stable regimen
of one or a combination of the following drug doses and durations:

Corticosteroids (less than or equal to 25 milligrams Prednisone or Prednisone equivalent
per day) - greater than or equal to 4 weeks.

5-ASA Sulfasalazine - greater than or equal to 4 weeks.

Azathioprine/6-MP/thioguanine with stable dose for eight weeks - greater than or equal to
12 weeks. (NOTE: Patients receiving azathioprine/6-MP/thioguanine must have been on a
stable dose of this medication for greater than or equal to 8 weeks)

Probiotics (live bacterial dietary supplements) - greater than or equal to 4 weeks.

Prebiotics (dietary supplements to produce biologically active substances) - greater than
or equal to 4 weeks.

Infliximab (5 to 10 mg/kg IV) - greater than 8 weeks or no response within 4 weeks of an
induction dose of 3 infusions.

Adalimumab (40 to 80 mg subq) - greater than or equal to 4 weeks or no response within 2
weeks after induction dose of 2 injections.

EXCLUSION CRITERIA:

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:

1. AST greater than 2.5 times the upper limit of normal (ULN).

2. ALT greater than 2.5 times the ULN.

3. Serum bilirubin greater than 1.5 times the ULN.

4. Serum creatinine greater than 1.5 times the ULN.

5. 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:

1. Platelet count less than 80,000 or greater than 950,000.

2. White blood cell count less than 1200.

3. 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.

- Presence of current active bowel obstruction, intestinal perforation, known presence
of high grade stricture, history of toxic megacolon, history of colonic epithelium
high-grade dysplasia or a dysplasia-associated lesion or mass that does not resemble
an adenoma (that is a mass lesion, stricture, or broad-based tumor with dysplasia).



Age minimum: 18 Years
Age maximum: N/A
Gender: All
Health Condition(s) or Problem(s) studied
Colitis
Cytokines
Drug Evaluation
Hermanski-Pudlak Syndrome
Lymphocytes
Intervention(s)
Drug: 6-Mercaptopurine
Drug: Adalimumab
Drug: Corticosteroids
Drug: Infliximab
Drug: Mesalamine
Drug: Tacrolimus
Primary Outcome(s)
To detect patterns of immune abnormalities in the colitis associated with Hermansky-Pudlak Syndrome (HPS). [Time Frame: 60 weeks]
Secondary Outcome(s)
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). [Time Frame: 60 weeks]
Secondary ID(s)
07-I-0205
070205
Source(s) of Monetary Support
Please refer to primary and secondary sponsors
Secondary Sponsor(s)
Ethics review
Results
Results available:
Date Posted:
Date Completed:
URL:
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