Cyclophosphamide in Treating Young Patients With Severe Autoimmune Enteropathy

Phase 2

Completed

• Conditions: Unspecified Childhood Solid Tumor, Protocol Specific; Diarrhea; Gastrointestinal Complications
• Interventions: Biological: filgrastim; Drug: cyclophosphamide

• Registration Number: NCT00258180
• Lead Sponsor: Johns Hopkins University

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.

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 Timeline

• Study Start: 2005-08-15
• Study First Submitted: 2005-11-22
• Study First Submitted QC: 2005-11-22
• Study First Posted: 2005-11-24
• Primary Completion: 2009-02-24
• Study Completion: 2009-02-24
• Results First Submitted: 2016-11-28
• Status Verified: 2019-03
• Results First Submitted QC: 2019-03-26
• Last Update Submitted: 2019-03-26
• Results First Posted: 2019-04-16
• Last Update Posted: 2019-04-16

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 3

Inclusion Criteria

Not provided

Exclusion Criteria

Not provided

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
severe autoimmune enteropathy	filgrastim	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
severe autoimmune enteropathy	cyclophosphamide	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

Primary Outcome Measures

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

Trial Locations

Locations (1)

Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins; 🇺🇸 Baltimore, Maryland, United States