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Cyclophosphamide in Treating Young Patients With Severe Autoimmune Enteropathy
This study is currently recruiting participants.
Verified by National Cancer Institute (NCI), February 2007
Sponsors and Collaborators: Sidney Kimmel Comprehensive Cancer Center
National Cancer Institute (NCI)
Information provided by: National Cancer Institute (NCI)
ClinicalTrials.gov Identifier: NCT00258180
  Purpose

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
Cancer-Related Problem/Condition
Unspecified Childhood Solid Tumor, Protocol Specific
Drug: cyclophosphamide
Drug: filgrastim
Phase II

MedlinePlus related topics: Cancer
Drug Information available for: Cyclophosphamide Filgrastim
U.S. FDA Resources
Study Type: Interventional
Study Design: Supportive Care, Open Label
Official Title: High-Dose Cyclophosphamide for the Treatment of Severe Autoimmune Enteropathy

Further study details as provided by National Cancer Institute (NCI):

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

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

Estimated Enrollment: 11
Study Start Date: June 2005
Estimated Primary Completion Date: June 2011 (Final data collection date for primary outcome measure)
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.

  Eligibility

Ages Eligible for Study:   1 Year to 21 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
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)
  Contacts and Locations
Please refer to this study by its ClinicalTrials.gov identifier: NCT00258180

Locations
United States, Maryland
Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins Recruiting
Baltimore, Maryland, United States, 21231-2410
Contact: Clinical Trials Office - Sidney Kimmel Comprehensive Cancer Ce     410-955-8804     jhcccro@jhmi.edu    
Sponsors and Collaborators
Sidney Kimmel Comprehensive Cancer Center
Investigators
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

Clinical trial summary from the National Cancer Institute's PDQ® database  This link exits the ClinicalTrials.gov site

Study ID Numbers: CDR0000441133, JHOC-J0326, JHOC-03070804
Study First Received: November 22, 2005
Last Updated: October 18, 2008
ClinicalTrials.gov Identifier: NCT00258180  
Health Authority: Unspecified

Keywords provided by National Cancer Institute (NCI):
unspecified childhood solid tumor, protocol specific
gastrointestinal complications
diarrhea

Study placed in the following topic categories:
Diarrhea
Autoimmune enteropathy
Cyclophosphamide
Intestinal Diseases

Additional relevant MeSH terms:
Molecular Mechanisms of Pharmacological Action
Immunologic Factors
Antineoplastic Agents
Therapeutic Uses
Physiological Effects of Drugs
Myeloablative Agonists
Antineoplastic Agents, Alkylating
Antirheumatic Agents
Alkylating Agents
Immunosuppressive Agents
Pharmacologic Actions

ClinicalTrials.gov processed this record on January 14, 2009