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High-Dose Cyclophosphamide for the Treatment of Severe Autoimmune Enteropathy


Phase 2
1 Year
21 Years
Not Enrolling
Both
Diarrhea, Gastrointestinal Complications, Unspecified Childhood Solid Tumor, Protocol Specific

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Trial Information

High-Dose Cyclophosphamide for the Treatment of Severe Autoimmune Enteropathy


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.

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)

Type of Study:

Interventional

Study Design:

Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Supportive Care

Outcome Measure:

Treatment-free remission at 1 year after study completion

Safety Issue:

No

Principal Investigator

David M. Loeb, MD, PhD

Investigator Role:

Principal Investigator

Investigator Affiliation:

Sidney Kimmel Comprehensive Cancer Center

Authority:

United States: Food and Drug Administration

Study ID:

J0326, CDR0000441133

NCT ID:

NCT00258180

Start Date:

June 2005

Completion Date:

January 2009

Related Keywords:

  • Diarrhea
  • Gastrointestinal Complications
  • Unspecified Childhood Solid Tumor, Protocol Specific
  • unspecified childhood solid tumor, protocol specific
  • gastrointestinal complications
  • diarrhea
  • Diarrhea
  • Intestinal Diseases
  • Polyendocrinopathies, Autoimmune

Name

Location

Sidney Kimmel Comprehensive Cancer Center at Johns HopkinsBaltimore, Maryland  21231-2410