Phase 2 N=12 Supportive Care

High-Dose Cyclophosphamide for Steroid Refractory GVHD

Graft Versus Host Disease

Bottom Line

View on ClinicalTrials.gov: NCT00492921 ↗

Enrolled (actual)

Serious AEs

33.3%

Results posted

Sep 2018

Primary outcome: Primary: Maximum Tolerated Dose of High-dose Cyclophosphamide as Determined by Number of Participants Who Tolerated Each Dose of Cyclophosphamide — 4; 4; 2 Participants

Study Design & Population

Study type: Interventional
Phase: Phase 2
Interventions: Cyclophosphamide (Drug)
Age: Pediatric, Adult, Older Adult
Sex: All
Sponsor: Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins
Primary completion: Jan 2013

Outcome Measures

Outcome	Result	p-value
PRIMARY Maximum Tolerated Dose of High-dose Cyclophosphamide as Determined by Number of Participants Who Tolerated Each Dose of Cyclophosphamide	4; 4; 2	—
PRIMARY GVHD Response Rate	1; 0; 1	—

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.

Eligibility Criteria

DISEASE CHARACTERISTICS:

Histologically confirmed acute graft-versus-host disease (GVHD) ≥ clinical grade II, that is steroid refractory
Steroid refractory GVHD is defined as GVHD that has progressed (increasing in grading) despite 49 hours of treatment with methylprednisolone of ≥ 2.0 mg/kg OR GVHD that has failed to improve (no change in grading stage) despite 4 days of treatment with methylprednisolone of ≥ 2.0 mg/kg
Prior allogeneic hematopoietic stem cell transplantation using either bone marrow, peripheral blood stem cells, or cord blood OR prior donor lymphocyte infusion required
Evidence of myeloid engraftment
No chronic GVHD

PATIENT CHARACTERISTICS:

ECOG (Eastern Cooperative Oncology Group) performance status (PS) 0-2 OR Karnofsky PS 60-100%
ANC (absolute neutrophil count) > 500/mm³
Not pregnant or nursing
Fertile patients must use effective contraception
Must be geographically accessible
No allergy or intolerance to cyclophosphamide or mesna
No HIV positivity
No mechanical ventilation
No active bleeding (excluding gastrointestinal bleeding) or history of hemorrhagic cystitis
No other uncontrolled illness including, but not limited to, the following:
Ongoing or active infection
Medical condition precluding patient from stopping azoles (e.g., fluconazole, itraconazole, or voriconazole) or other adequate antifungal therapy during cyclophosphamide administration
Symptomatic congestive heart failure
Unstable angina pectoris
Psychiatric illness/social situations that would preclude compliance

PRIOR CONCURRENT THERAPY:

See Disease Characteristics

View full record on ClinicalTrials.gov →

Data sourced from ClinicalTrials.gov (NCT00492921). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.