Suspended

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

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What is being tested

autologous expanded mesenchymal stem cells OTI-010

+ busulfan
+ cyclophosphamide
Biological
Drug
Procedure
Radiation
Who is being recruted

Graft Versus Host Disease

+ Leukemia
+ Myelodysplastic Syndromes
From 18 to 55 Years
How is the trial designed

Supportive Care Study

Phase 2
Interventional

Summary

Principal SponsorMesoblast International Sàrl
Last updated: December 4, 2014
Sourced from a government-validated database.Claim as a partner

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. 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.

Official TitleA Phase II, Double-Blind, Randomized, Placebo-Controlled Study to Evaluate the Safety and Efficacy of OTI-010 in Subjects Who Receive HLA-Identical Sibling Peripheral Blood Stem Cell Transplantation for Hematologic Malignancies 
Principal SponsorMesoblast International Sàrl
Last updated: December 4, 2014
Sourced from a government-validated database.Claim as a partner

Protocol

This section provides details of the study plan, including how the study is designed and what the study is measuring.
Design Details
Supportive Care Study
These studies explore ways to improve comfort and daily life for people living with a condition. They may focus on easing symptoms, reducing treatment side effects, or supporting overall well-being.

How participants are assigned to different groups/arms
In this clinical study, participants are placed into groups randomly, like flipping a coin. This ensures that the study is fair and unbiased, making the results more reliable. By assigning participants by chance, researchers can better compare treatments without external influences.

Other Ways to Assign Participants
Non-randomized allocation
: Participants are assigned based on specific factors, such as their medical condition or a doctor's decision.

None (Single-arm trial)
: If the study has only one group, all participants receive the same treatment, and no allocation is needed.

How the interventions assigned to participants is kept confidential
Neither participants nor researchers know who is receiving which treatment. This is the most rigorous way to reduce bias, ensuring that expectations do not influence the results.

Other Ways to Mask Information
Open-label
: Everyone knows which treatment is being given.

Single-blind
: Participants do not know which treatment they are receiving, but researchers do.

Triple-blind
: Participants, researchers, and outcome assessors do not know which treatment is given.

Quadruple-blind
: Participants, researchers, outcome assessors, and care providers all do not know which treatment is given.

Eligibility

Researchers look for people who fit a certain description, called eligibility criteria: person's general health condition or prior treatments.
Conditions
Criteria
Any sexBiological sex of participants that are eligible to enroll.
From 18 to 55 YearsRange of ages for which participants are eligible to join.
Healthy volunteers not allowedIf individuals who are healthy and do not have the condition being studied can participate.
Conditions
Pathology
Graft Versus Host Disease
Leukemia
Myelodysplastic Syndromes
Criteria

DISEASE CHARACTERISTICS: * Histologically confirmed diagnosis of 1 of the following hematologic malignancies: * Acute lymphoblastic leukemia, meeting 1 of the following criteria: * In first or second remission * In early first or second relapse\* * Acute myeloid leukemia, meeting 1 of the following criteria: * In first or second remission * In early first or second relapse\* * Chronic myelogenous leukemia * Chronic or accelerated phase * Any of the following myelodysplastic syndromes: * Refractory anemia (RA) * RA with ringed sideroblasts * RA with excess blasts NOTE: \*\< 24% marrow blasts and \< 5% peripheral blood blasts (within 10 days of beginning conditioning regimen) * No secondary acute leukemia * Prior CNS tumor involvement allowed provided patient is asymptomatic and there is no evidence of CNS disease on lumbar puncture and CT scan of the brain * Must have a 6/6 HLA-identical sibling donor available PATIENT CHARACTERISTICS: Age * 18 to 55 Performance status * Karnofsky 70-100% Life expectancy * Not specified Hematopoietic * Not specified Hepatic * Bilirubin \< 2 times upper limit of normal (ULN) * SGOT \< 10 times ULN * Hepatitis B core antigen, surface antigen, and e-antigen negative * Hepatitis B DNA negative * Hepatitis C RNA negative Renal * Creatinine clearance ≥ 60 mL/min Cardiovascular * LVEF ≥ 50% by MUGA or echocardiogram * No right sided heart failure Pulmonary * FEV_1 \> 50% of predicted * DLCO ≥ 50% of predicted (corrected for anemia) * Oxygen saturation ≥ 97% on room air * No pulmonary hypertension Immunologic * HIV-1 and 2 antibody negative * HIV-1 antigen negative * HTLV-I and II antibody negative * No active infection Other * CNS function normal * No uncontrolled alcohol or substance abuse within the past 6 months * No other concurrent underlying medical condition that would preclude study participation * Not pregnant * Negative pregnancy test * Fertile patients must use 2 effective methods of contraception PRIOR CONCURRENT THERAPY: Biologic therapy * No prior allogeneic or autologous hematopoietic stem cell transplantation * No concurrent medication to accelerate neutrophil or platelet engraftment except filgrastim (G-CSF) Chemotherapy * Not specified Endocrine therapy * Not specified Radiotherapy * Not specified Surgery * No prior solid organ transplantation Other * More than 30 days since prior investigational agents or devices * No other concurrent investigational agents or devices * No concurrent anti-infective therapy except prophylactic therapy * No other concurrent conditioning regimen agents * No concurrent herbal remedies except multivitamins * No other concurrent graft-versus-host disease prophylaxis medications (e.g., ursodeoxycholic acid)


Study Plan

Find out more about all the medication administered in this study, their detailed description and what they involve.
Study Objectives
Study Objectives
Primary Objectives

Secondary Objectives


Study Centers

These are the hospitals, clinics, or research facilities where the trial is being conducted. You can find the location closest to you and its status.
This study has 1 location
Suspended
Jonsson Comprehensive Cancer Center, UCLALos Angeles, United StatesSee the location

SuspendedOne Study Center