Cyclophosphamide in Treating Young Patients With Severe Autoimmune Enteropathy (NCT00258180) | Clinical Trial Compass
CompletedPhase 2
Cyclophosphamide in Treating Young Patients With Severe Autoimmune Enteropathy
United States3 participantsStarted 2005-08-15
Plain-language 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 autoimmune enteropathy.
Who can participate
Age range1 Year – 21 Years
SexALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
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 con…
What they're measuring
1
Number of Participants With Treatment-free Remission at 1 Year After Study Completion
Timeframe: 1 year
2
Number of Participants Experiencing Intervention-related Adverse Events, as Defined by CTCAE at 1 Month