Cyclophosphamide in Treating Young Patients With Severe Autoimmune Enteropathy

Diarrhea Clinical Trial

Official title:

High-Dose Cyclophosphamide for the Treatment of Severe Autoimmune Enteropathy

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT00258180
• Other study ID #: 03-07-08-04
• Secondary ID: P30CA006973JHOC-
• Status: Completed
• Phase: Phase 2
• Start date: August 15, 2005
• Est. completion date: February 24, 2009

Study information

• Verified date: March 2019
• Source: Johns Hopkins University
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 3
• Est. completion date: February 24, 2009
• Est. primary completion date: February 24, 2009
• Accepts healthy volunteers: No
• Gender: All
• Age group: 1 Year to 21 Years

Eligibility

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)

Related Conditions & MeSH terms

• Diarrhea
• Gastrointestinal Complications
• Intestinal Diseases
• Unspecified Childhood Solid Tumor, Protocol Specific

Intervention

Biological:
• filgrastim
Administered IV or subcutaneously once daily beginning on day 10 and continuing for 3 days or until blood counts recover

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

Locations

Country	Name	City	State
United States	Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins	Baltimore	Maryland

Sponsors (2)

Lead Sponsor	Collaborator
Johns Hopkins University	National Cancer Institute (NCI)

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Number of Participants With Treatment-free Remission at 1 Year After Study Completion	Number of participants off therapy 1 year after study completion without relapse.	1 year
Primary	Number of Participants Experiencing Intervention-related Adverse Events, as Defined by CTCAE at 1 Month		1 month