OTI-010 for Graft-Versus-Host Disease Prophylaxis in Treating Patients Who Are Undergoing Donor Peripheral Stem Cell Transplantation for Hematologic Malignancies

Phase 2

Withdrawn

• Conditions: Myelodysplastic Syndromes; Graft Versus Host Disease; Leukemia

• Registration Number: NCT00081055
• Lead Sponsor: Mesoblast International Sàrl

Brief Summary

RATIONALE: OTI-010 may be effective for graft-versus-host disease prophylaxis (prevention) in patients who are undergoing donor peripheral stem cell transplantation for hematologic malignancies (cancer of the blood or bone marrow).

PURPOSE: This randomized phase II trial is studying how well OTI-010 works in preventing graft-versus-host disease in patients who are undergoing donor peripheral stem cell transplantation for hematologic cancer.

Detailed Description

OBJECTIVES:

* Compare the safety and efficacy of OTI-010 vs placebo as graft-versus-host disease prophylaxis in patients with hematologic malignancies undergoing HLA-identical sibling matched peripheral blood stem cell transplantation.

OUTLINE: This is a randomized, double-blind, placebo-controlled, multicenter study. Patients are stratified according to age (18 to 34 vs 35 to 55) and donor/recipient gender (female donor/male recipient vs female donor/female recipient vs male donor/female recipient vs male donor/male recipient).

* Conditioning regimen: Patients receive cyclophosphamide IV once daily on days -5 and -4 and undergo total body irradiation twice daily on days -3 to -1 OR busulfan IV over 2 hours every 6 hours on days -7 to -4 and cyclophosphamide IV once daily on days -3 and -2.

* Graft-versus-host disease prophylaxis: Patients receive methotrexate IV on days 1, 3, 6, and 11. Patients also receive cyclosporine orally or IV (over 1-4 hours) twice daily beginning on day -1 and continuing for at least 6 months followed by a taper until 1 year after transplantation.

* OTI-010 therapy: Patients are randomized to 1 of 3 treatment arms.

* Arm I: Patients receive placebo IV 4 hours before peripheral blood stem cell transplantation (PBSCT) on day 0.

* Arm II: Patients receive OTI-010 IV 4 hours before PBSCT on day 0.

* Arm III: Patients receive a higher dose of OTI-010 IV 4 hours before PBSCT on day 0.

* Allogeneic stem cell transplantation: Patients undergo allogeneic PBSCT on day 0.

Patients are followed at 18 weeks, at 6, 9, and 12 months, every 6 months for 1 year, and then annually for 3 years.

PROJECTED ACCRUAL: A total of 99 patients (33 per treatment arm) will be accrued for this study within 5 months.

Study Timeline

• Study First Submitted: 2004-04-07
• Study First Submitted QC: 2004-04-07
• Study First Posted: 2004-04-08
• Status Verified: 2014-12
• Last Update Submitted: 2014-12-03
• Last Update Posted: 2014-12-04

Recruitment & Eligibility

• Status: WITHDRAWN
• Sex: All
• Target Recruitment: Not specified

Inclusion Criteria

Not provided

Exclusion Criteria

Not provided

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Incidence of acute GVHD grade II-IV of skin, liver and gut (stomach to rectum) through Day 84 post-PBSC transplantation	Day 84

Secondary Outcome Measures

Name	Time	Method
Safety as measured by infusional toxicity, relapse nd survival, formation of potential ectopic tissue foci	84 days

Trial Locations

Locations (1)

Jonsson Comprehensive Cancer Center, UCLA; 🇺🇸 Los Angeles, California, United States