Cyclophosphamide in Treating Young Patients With Severe Autoimmune Enteropathy
This study has been completed.
Sponsor:
Sidney Kimmel Comprehensive Cancer Center
Collaborator:
Information provided by (Responsible Party):
Sidney Kimmel Comprehensive Cancer Center
ClinicalTrials.gov Identifier:
NCT00258180
First received: November 22, 2005
Last updated: June 13, 2012
Last verified: June 2012
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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 |
|---|---|---|
|
Diarrhea 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:
Genetics Home Reference related topics:
autoimmune polyglandular syndrome, type 1
Drug Information available for:
Cyclophosphamide
Filgrastim
Lenograstim
Granulocyte colony-stimulating factor
U.S. FDA Resources
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) |
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 | |
| Baltimore, Maryland, United States, 21231-2410 | |
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
Additional Information:
No publications provided
| Responsible Party: | Sidney Kimmel Comprehensive Cancer Center |
| ClinicalTrials.gov 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 diarrhea |
Additional relevant MeSH terms:
|
Diarrhea Intestinal Diseases Polyendocrinopathies, Autoimmune Signs and Symptoms, Digestive Signs and Symptoms Gastrointestinal Diseases Digestive System Diseases Endocrine System Diseases Autoimmune Diseases Immune System Diseases Cyclophosphamide Lenograstim |
Immunosuppressive Agents Immunologic Factors Physiological Effects of Drugs Pharmacologic Actions Antirheumatic Agents Therapeutic Uses Antineoplastic Agents, Alkylating Alkylating Agents Molecular Mechanisms of Pharmacological Action Antineoplastic Agents Myeloablative Agonists Adjuvants, Immunologic |
ClinicalTrials.gov processed this record on May 19, 2013