Phase 2
N=2
Cyclophosphamide in Treating Young Patients With Severe Autoimmune Enteropathy
Diarrhea · Gastrointestinal Complications · Unspecified Childhood Solid Tumor, Protocol Specific
Bottom Line
View on ClinicalTrials.gov: NCT00258180 ↗Enrolled (actual)
2
Serious AEs
0.0%
Results posted
Apr 2019
Primary outcome: Primary: Number of Participants With Treatment-free Remission at 1 Year After Study Completion — 2 Participants
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 2
- Interventions
- filgrastim (Biological); cyclophosphamide (Drug)
- Age
- Pediatric, Adult · 1+ yrs
- Sex
- All
- Sponsor
- Johns Hopkins University
- Primary completion
- Feb 2009
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Number of Participants With Treatment-free Remission at 1 Year After Study Completion |
2 | — |
| PRIMARY Number of Participants Experiencing Intervention-related Adverse Events, as Defined by CTCAE at 1 Month |
— | — |
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.
Eligibility Criteria
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)
Data sourced from ClinicalTrials.gov (NCT00258180). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.