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Cyclophosphamide in Treating Young Patients With Severe Autoimmune Enteropathy

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
ClinicalTrials.gov Identifier: NCT00258180
Recruitment Status : Completed
First Posted : November 24, 2005
Last Update Posted : June 14, 2012
National Cancer Institute (NCI)
Information provided by (Responsible Party):
Sidney Kimmel Comprehensive Cancer Center

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.

Condition or disease Intervention/treatment Phase
Diarrhea Gastrointestinal Complications Unspecified Childhood Solid Tumor, Protocol Specific Biological: filgrastim Drug: cyclophosphamide Phase 2

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.

Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 3 participants
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Supportive Care
Official Title: High-Dose Cyclophosphamide for the Treatment of Severe Autoimmune Enteropathy
Study Start Date : June 2005
Primary Completion Date : January 2009
Study Completion Date : January 2009

Resource links provided by the National Library of Medicine

U.S. FDA Resources

Primary Outcome Measures :
  1. Treatment-free remission at 1 year after study completion
  2. Toxicities during study treatment

Secondary Outcome Measures :
  1. Effect of treatment on anti-enterocyte antibody titers at 1 year after study completion

Information from the National Library of Medicine

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Ages Eligible for Study:   1 Year to 21 Years   (Child, Adult)
Sexes Eligible for Study:   All
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)

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): 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

Responsible Party: Sidney Kimmel Comprehensive Cancer Center
ClinicalTrials.gov Identifier: NCT00258180     History of Changes
Other Study ID Numbers: J0326, CDR0000441133
P30CA006973 ( U.S. NIH Grant/Contract )
First Posted: November 24, 2005    Key Record Dates
Last Update Posted: June 14, 2012
Last Verified: June 2012

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

Additional relevant MeSH terms:
Intestinal Diseases
Signs and Symptoms, Digestive
Signs and Symptoms
Gastrointestinal Diseases
Digestive System Diseases
Immunosuppressive Agents
Immunologic Factors
Physiological Effects of Drugs
Antirheumatic Agents
Antineoplastic Agents, Alkylating
Alkylating Agents
Molecular Mechanisms of Pharmacological Action
Antineoplastic Agents
Myeloablative Agonists