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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
Results First Posted : April 16, 2019
Last Update Posted : April 16, 2019
Sponsor:
Collaborator:
National Cancer Institute (NCI)
Information provided by (Responsible Party):
Johns Hopkins University

Study Description
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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:

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.

Study Design
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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 3 participants
Allocation: N/A
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
Actual Study Start Date : August 15, 2005
Actual Primary Completion Date : February 24, 2009
Actual Study Completion Date : February 24, 2009

Resource links provided by the National Library of Medicine


Arms and Interventions
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Arm Intervention/treatment
Experimental: 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
 Biological: filgrastim
Administered IV or subcutaneously once daily beginning on day 10 and continuing for 3 days or until blood counts recover
Other Name: G-CSF

Drug: cyclophosphamide
Administered IV over 1 hour on days 1-4


Outcome Measures
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Primary Outcome Measures :
  1. Number of Participants With Treatment-free Remission at 1 Year After Study Completion [ Time Frame: 1 year ]
    Number of participants off therapy 1 year after study completion without relapse.

  2. Number of Participants Experiencing Intervention-related Adverse Events, as Defined by CTCAE at 1 Month [ Time Frame: 1 month ]

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


Locations
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United States, Maryland
Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins
Baltimore, Maryland, United States, 21231-2410
Sponsors and Collaborators
Johns Hopkins University
National Cancer Institute (NCI)
Investigators
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Principal Investigator: David M. Loeb, MD, PhD Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins
Principal Investigator: Maria Oliva-Hemker, MD Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins
More Information
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Responsible Party: Johns Hopkins University
ClinicalTrials.gov Identifier: NCT00258180    
Other Study ID Numbers: 03-07-08-04
P30CA006973 ( U.S. NIH Grant/Contract )
JHOC-J0326 ( Other Identifier: SKCCC )
J0326 ( Other Identifier: SKCCC )
CDR0000441133 ( Registry Identifier: NCI PDQ )
First Posted: November 24, 2005    Key Record Dates
Results First Posted: April 16, 2019
Last Update Posted: April 16, 2019
Last Verified: March 2019
Keywords provided by Johns Hopkins University:
unspecified childhood solid tumor, protocol specific
gastrointestinal complications
diarrhea
Additional relevant MeSH terms:
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Diarrhea
Signs and Symptoms, Digestive
Cyclophosphamide
Immunosuppressive Agents
Immunologic Factors
Physiological Effects of Drugs
 Antirheumatic Agents
Antineoplastic Agents, Alkylating
Alkylating Agents
Molecular Mechanisms of Pharmacological Action
Antineoplastic Agents
Myeloablative Agonists