High-Dose Cyclophosphamide for Steroid Refractory GVHD

Phase 2

Completed

• Conditions: Graft Versus Host Disease
• Interventions: Drug: Cyclophosphamide

• Registration Number: NCT00492921
• Lead Sponsor: Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins

Brief Summary

RATIONALE: High-dose cyclophosphamide may be an effective treatment for acute graft-versus-host disease that did not respond to steroid therapy.

PURPOSE: This phase II trial is studying the side effects, best dose, and how well high-dose cyclophosphamide works in treating patients with acute graft-versus-host disease that did not respond to steroid therapy.

Detailed Description

OBJECTIVES:

* Determine the maximum tolerated dose of high-dose cyclophosphamide in patients with steroid refractory acute graft-versus-host disease (GVHD).

* Determine the efficacy of this regimen at 28 days post-treatment in these patients.

OUTLINE: This is a dose-escalation study.

Patients receive high-dose cyclophosphamide once daily for 1-4 days beginning on day 1 and filgrastim (G-CSF) subcutaneously once daily beginning on day 10 and continuing until blood counts recover.

Cohorts of 3-6 patients receive escalating doses of high-dose cyclophosphamide until the maximum tolerated dose (MTD) is determined. The MTD is defined as the dose preceding that at which 2 of 3 or 2 of 6 patients experience dose-limiting toxicity.

After completion of study treatment, patients are followed weekly for 4 weeks.

Study Timeline

• Study Start: 2007-05
• Study First Submitted: 2007-06-25
• Study First Submitted QC: 2007-06-25
• Study First Posted: 2007-06-27
• Primary Completion: 2013-01
• Study Completion: 2013-01
• Results First Submitted: 2018-07-27
• Status Verified: 2018-09
• Results First Submitted QC: 2018-09-11
• Last Update Submitted: 2018-09-11
• Results First Posted: 2018-09-14
• Last Update Posted: 2018-09-14

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 12

Inclusion Criteria

Not provided

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Exclusion Criteria

Not provided

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Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SEQUENTIAL

Arm && Interventions

Group	Intervention	Description
Cyclophosphamide 50	Cyclophosphamide	Treatment with cyclophosphamide 50 mg/kg/d x 1 days.
Cyclophosphamide 100	Cyclophosphamide	Treatment with cyclophosphamide 50 mg/kg/d x 2 days.
Cyclophosphamide 150	Cyclophosphamide	Treatment with cyclophosphamide 50 mg/kg/d x 3 days.

Primary Outcome Measures

Name	Time	Method
Maximum Tolerated Dose of High-dose Cyclophosphamide as Determined by Number of Participants Who Tolerated Each Dose of Cyclophosphamide	Day 28
GVHD Response Rate	Day 28	Percentage of patients whose GVHD (as defined by Przepiorka criteria) responded to cyclophosphamide (complete response). Acute GVHD is defined by the Przepiorka criteria, which stages the degree of organ involvement in the skin, liver, and gastrointestinal (GI) tract, based on severity, with Stage 1+ being least severe and stage 4+ being the most severe. Grading of acute GVHD is as follows: Grade I (skin involvement stages 1+ to 2+, with no liver or GI involvement), Grade II (skin involvement stages 1+ to 3+, liver 1+, GI tract 1+), Grade III (skin involvement stages 2+ to 3+, liver 1+, GI tract 2+ to 4+), Grade IV (skin involvement stages 4+, Liver 4+).

Trial Locations

Locations (1)

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