Suspended

A 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

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What is being tested

Data Collection

Who is being recruted

Bone Marrow Diseases+12

+ Disease

+ Graft vs Host Disease

From 18 to 55 Years
See all eligibility criteria
How is the trial designed

Supportive Care Study

Phase 2
Interventional
See protocol details

Summary

Principal SponsorMesoblast International Sàrl
Last updated: January 14, 2026
Sourced from a government-validated database.Claim as a partner

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 
NCT00081055
Principal SponsorMesoblast International Sàrl
Last updated: January 14, 2026
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.

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.

Eligibility

Researchers look for people who fit a certain description, called eligibility criteria: person's general health condition or prior treatments.
Conditions
Criteria

Any sex

Biological sex of participants that are eligible to enroll.

From 18 to 55 Years

Range of ages for which participants are eligible to join.

Healthy volunteers not allowed

If individuals who are healthy and do not have the condition being studied can participate.

Conditions

Pathology

Bone Marrow DiseasesDiseaseGraft vs Host DiseaseHematologic DiseasesImmune System DiseasesLeukemiaMyelodysplastic SyndromesNeoplasmsNeoplasms by Histologic TypeNeoplasms by SitePathologic ProcessesPrecancerous ConditionsPreleukemiaSyndromeHematologic Neoplasms

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, UCLA

Los Angeles, United StatesSee the location
SuspendedOne Study Center