Clinical trial · Interventional
Cyclophosphamide in Treating Young Patients With Severe Autoimmune Enteropathy
High-Dose Cyclophosphamide for the Treatment of Severe Autoimmune Enteropathy
NCT00258180CI-TRIAL-00038143completedPhase 2Results postedClinicalTrials.gov clinicaltrialsProvenance
- Source
- ClinicalTrials.gov
- Retrieved
- Sep 8, 2026
- Layer
- normalized (units and labels harmonized; values unchanged)
- Run
- ING-CLINICALTRIALS-20260908-000001
Summary
Brief summary (as posted)
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.
Conditions
Conditions (3)
Free-text conditions as registered, with the CancerIndex entity they were reconciled to and the match type.
| Condition (as posted) | Mapped entity | Match | Confidence |
|---|---|---|---|
| Diarrhea | — | UNRESOLVED | — |
| Gastrointestinal Complications | — | UNRESOLVED | — |
| Unspecified Childhood Solid Tumor, Protocol Specific | Childhood Solid Neoplasm | ALIAS | 0.85 |
Interventions
Interventions (2)
| Intervention | Type | Mapped drug | Match |
|---|---|---|---|
| cyclophosphamide | Drug | Cyclophosphamide | ALIAS |
| filgrastim | Biological | Filgrastim | ALIAS |
Design
Arms and outcomes
Arms (1)
- type
- EXPERIMENTAL
- label
- severe autoimmune enteropathy
- description
- Young patients with severe autoimmune enteropathy receive cyclophosphamide IV over 1 hour on days 1-4. Patients then receive filgrastim (G-CSF) IV or subcutaneously once daily beginning on day 10 and continuing for 3 days or until blood counts recover
- interventionNames
- Biological: filgrastim
- Drug: cyclophosphamide
Primary outcomes (2)
- measure
- Number of Participants With Treatment-free Remission at 1 Year After Study Completion
- timeFrame
- 1 year
- description
- Number of participants off therapy 1 year after study completion without relapse.
- measure
- Number of Participants Experiencing Intervention-related Adverse Events, as Defined by CTCAE at 1 Month
- timeFrame
Eligibility
Eligibility (as posted)
- Sex
- All
- Minimum age
- 1 Year
- Maximum age
- 21 Years
Show eligibility criteria text
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)
References
Publications (0)
Data not yet available
No reference posted for this study.