Cyclophosphamide in Treating Young Patients With Severe Autoimmune Enteropathy

This study has been completed.
National Cancer Institute (NCI)
Information provided by (Responsible Party):
Sidney Kimmel Comprehensive Cancer Center Identifier:
First received: November 22, 2005
Last updated: June 13, 2012
Last verified: June 2012

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.

Condition Intervention Phase
Gastrointestinal Complications
Unspecified Childhood Solid Tumor, Protocol Specific
Biological: filgrastim
Drug: cyclophosphamide
Phase 2

Study Type: Interventional
Study Design: Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Supportive Care
Official Title: High-Dose Cyclophosphamide for the Treatment of Severe Autoimmune Enteropathy

Resource links provided by NLM:

Further study details as provided by Sidney Kimmel Comprehensive Cancer Center:

Primary Outcome Measures:
  • Treatment-free remission at 1 year after study completion [ Designated as safety issue: No ]
  • Toxicities during study treatment [ Designated as safety issue: Yes ]

Secondary Outcome Measures:
  • Effect of treatment on anti-enterocyte antibody titers at 1 year after study completion [ Designated as safety issue: No ]

Enrollment: 3
Study Start Date: June 2005
Study Completion Date: January 2009
Primary Completion Date: January 2009 (Final data collection date for primary outcome measure)
Detailed Description:



  • Determine the rate of treatment-free remission in young patients with severe autoimmune enteropathy treated with high-dose cyclophosphamide.


  • 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.


Ages Eligible for Study:   1 Year to 21 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No


  • 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


Performance status

  • Lansky 60-100%

Life expectancy

  • Not specified


  • Not specified


  • Not specified


  • Not specified


  • Ejection fraction ≥ 40% OR shortening fraction ≥ 20%


  • 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)


  • 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


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


  • At least 5 days since other prior immunosuppressive medications (e.g., tacrolimus or cyclosporine)
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its identifier: NCT00258180

United States, Maryland
Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins
Baltimore, Maryland, United States, 21231-2410
Sponsors and Collaborators
Sidney Kimmel Comprehensive Cancer Center
National Cancer Institute (NCI)
Principal Investigator: David M. Loeb, MD, PhD Sidney Kimmel Comprehensive Cancer Center
Principal Investigator: Maria Oliva-Hemker, MD Sidney Kimmel Comprehensive Cancer Center
  More Information

Additional Information:
No publications provided

Responsible Party: Sidney Kimmel Comprehensive Cancer Center Identifier: NCT00258180     History of Changes
Other Study ID Numbers: J0326, CDR0000441133, P30CA006973, JHOC-J0326, JHOC-03070804
Study First Received: November 22, 2005
Last Updated: June 13, 2012
Health Authority: United States: Food and Drug Administration

Keywords provided by Sidney Kimmel Comprehensive Cancer Center:
unspecified childhood solid tumor, protocol specific
gastrointestinal complications

Additional relevant MeSH terms:
Polyendocrinopathies, Autoimmune
Autoimmune Diseases
Endocrine System Diseases
Immune System Diseases
Alkylating Agents
Antineoplastic Agents
Antineoplastic Agents, Alkylating
Antirheumatic Agents
Immunologic Factors
Immunosuppressive Agents
Molecular Mechanisms of Pharmacological Action
Myeloablative Agonists
Pharmacologic Actions
Physiological Effects of Drugs
Therapeutic Uses processed this record on November 30, 2015