Diarrhea, Gastrointestinal Complications, Unspecified Childhood Solid Tumor, Protocol Specific
Conditions
Keywords
unspecified childhood solid tumor, protocol specific, gastrointestinal complications, diarrhea
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 autoimmune enteropathy.
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 effects of this drug in these patients. OUTLINE: Patients 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. After completion of study treatment, patients are followed periodically for up to 1½ years. PROJECTED ACCRUAL: A total of 7-11 patients will be accrued for this study.
Interventions
BIOLOGICALfilgrastim
Administered IV or subcutaneously once daily beginning on day 10 and continuing for 3 days or until blood counts recover
DRUGcyclophosphamide
Administered IV over 1 hour on days 1-4
Sponsors
National Cancer Institute (NCI)
Johns Hopkins University
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
SUPPORTIVE_CARE
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
1 Years to 21 Years
Healthy volunteers
No
Inclusion 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)
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Number of Participants Experiencing Intervention-related Adverse Events, as Defined by CTCAE at 1 Month | 1 month | — |
| Number of Participants With Treatment-free Remission at 1 Year After Study Completion | 1 year | Number of participants off therapy 1 year after study completion without relapse. |
Countries
United States
Participant flow
Pre-assignment details
1 subject was not assigned intervention due to PI decision.
Participants by arm
| Arm | Count |
|---|---|
| Severe Autoimmune Enteropathy 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. | 2 |
| Total | 2 |
Baseline characteristics
| Characteristic | Severe Autoimmune Enteropathy |
|---|---|
| Age, Categorical <=18 years | 2 Participants |
| Age, Categorical >=65 years | 0 Participants |
| Age, Categorical Between 18 and 65 years | 0 Participants |
| Race (NIH/OMB) American Indian or Alaska Native | 0 Participants |
| Race (NIH/OMB) Asian | 0 Participants |
| Race (NIH/OMB) Black or African American | 0 Participants |
| Race (NIH/OMB) More than one race | 0 Participants |
| Race (NIH/OMB) Native Hawaiian or Other Pacific Islander | 0 Participants |
| Race (NIH/OMB) Unknown or Not Reported | 0 Participants |
| Race (NIH/OMB) White | 2 Participants |
| Region of Enrollment United States | 2 Participants |
| Sex: Female, Male Female | 0 Participants |
| Sex: Female, Male Male | 2 Participants |
Adverse events
| Event type | EG000 affected / at risk |
|---|---|
| deaths Total, all-cause mortality | 0 / 2 |
| other Total, other adverse events | 0 / 2 |
| serious Total, serious adverse events | 0 / 2 |
Outcome results
Primary
Number of Participants Experiencing Intervention-related Adverse Events, as Defined by CTCAE at 1 Month
Time frame: 1 month
| Arm | Measure | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|
| Severe Autoimmune Enteropathy | Number of Participants Experiencing Intervention-related Adverse Events, as Defined by CTCAE at 1 Month | 0 Participants |
Primary
Number of Participants With Treatment-free Remission at 1 Year After Study Completion
Number of participants off therapy 1 year after study completion without relapse.
Time frame: 1 year
| Arm | Measure | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|
| Severe Autoimmune Enteropathy | Number of Participants With Treatment-free Remission at 1 Year After Study Completion | 2 Participants |