Status:
COMPLETED
Cyclophosphamide in Treating Young Patients With Severe Autoimmune Enteropathy
Lead Sponsor:
Johns Hopkins University
Collaborating Sponsors:
National Cancer Institute (NCI)
Conditions:
Diarrhea
Gastrointestinal Complications
Eligibility:
All Genders
1-21 years
Phase:
PHASE2
Brief Summary
RATIONALE: Cyclophosphamide may help control the symptoms of autoimmune enteropathy . PURPOSE: This phase II trial is studying how well cyclophosphamide works in treating young patients with severe a...
Detailed Description
OBJECTIVES: Primary * Determine the rate of treatment-free remission in young patients with severe autoimmune enteropathy treated with high-dose cyclophosphamide. Secondary * Determine the toxic e...
Eligibility Criteria
Inclusion
- DISEASE CHARACTERISTICS:
- Diagnosis of severe autoimmune enteropathy
- Condition is resistant to conventional therapy
- Histologic evidence of severe villous atrophy with intense lymphocytic infiltrate of the lamina propria by small intestinal biopsy within the past 3 months
- Disease failed to respond after ≥ 2 months of corticosteroid therapy at a dose of ≥ 0.5 mg/kg/day or ≥ 40 mg/day for patients \> 20 kg AND 1 of the following therapies:
- Cyclosporine resulting in ≥ 1 whole blood level of \> 200 ng/mL
- Tacrolimus resulting in ≥ 1 whole blood level of 5 ng/mL
- At least 50% estimated caloric needs provided by parenteral nutrition
- History of intractable diarrhea, defined as frequent watery stools for \> 3 months that does not respond to dietary restriction
- No celiac disease, defined by a history of positive antiendomysial antibody or tissue transglutaminase antibody
- No primary immunodeficiency or x-linked autoimmunity-allergy dysregulation
- PATIENT CHARACTERISTICS:
- Performance status
- Lansky 60-100%
- Life expectancy
- Not specified
- Hematopoietic
- Not specified
- Hepatic
- Not specified
- Renal
- Not specified
- Cardiovascular
- Ejection fraction ≥ 40% OR shortening fraction ≥ 20%
- Pulmonary
- FVC or FEV\_1 ≥ 50% of predicted (for patients \> 8 years of age)
- No clinically abnormal pulmonary function or abnormal pulse oximetry (for patients ≤ 8 years of age)
- Other
- Not pregnant
- Negative pregnancy test
- Fertile patients must use effective contraception during and for at least 9 months after completion of study treatment
- No known chromosomal abnormality
- PRIOR CONCURRENT THERAPY:
- Biologic therapy
- No immunizations for at least 6 months after completion of study treatment
- Endocrine therapy
- See Disease Characteristics
- At least 5 days since prior corticosteroids
- No concurrent dexamethasone as an anti-emetic
- Other
- At least 5 days since other prior immunosuppressive medications (e.g., tacrolimus or cyclosporine)
Exclusion
Key Trial Info
Start Date :
August 15 2005
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
February 24 2009
Estimated Enrollment :
3 Patients enrolled
Trial Details
Trial ID
NCT00258180
Start Date
August 15 2005
End Date
February 24 2009
Last Update
April 16 2019
Active Locations (1)
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1
Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins
Baltimore, Maryland, United States, 21231-2410