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Graft Versus Host DiseasePhase 2NCT00081055

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

A Phase 2 study testing autologous expanded mesenchymal stem cells OTI-010, busulfan and cyclophosphamide in people with Graft Versus Host Disease and Leukemia, run by Mesoblast International Sàrl at 1 site in United States.

Withdrawn

Withdrawn. The study stopped before anyone enrolled.

Official record
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Trial 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). Read more about this study

Who is it for?
Ages 18 to 55 years
What is being tested?
autologous expanded mesenchymal stem cells OTI-010, busulfan and cyclophosphamide
Type of treatment
Surgery or procedure
How many people
0 participants
Where
1 siteUnited States
Study dates
Start not givento not given
Phase
Phase 2
Are there results?
None posted in the registry

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 18 to 55 years
  • All sexes
  • No healthy volunteers
  • Adult

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)

Trial locations and contacts

1 site in 1 country.

United States1 site

  • Jonsson Comprehensive Cancer Center, UCLA

    Los Angeles, California, 90095-1678

Other trials in United States

About this study

Official title: 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

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.

Read the 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.

Being tested
autologous expanded mesenchymal stem cells OTI-010, busulfan, cyclophosphamide, cyclosporine, methotrexate
Keywords
graft versus host disease, adult acute lymphoblastic leukemia in remission, recurrent adult acute lymphoblastic leukemia, adult acute myeloid leukemia in remission, recurrent adult acute myeloid leukemia, accelerated phase chronic myelogenous leukemia, chronic phase chronic myelogenous leukemia, refractory anemia, refractory anemia with excess blasts, refractory anemia with ringed sideroblasts, de novo myelodysplastic syndromes, previously treated myelodysplastic syndromes, secondary myelodysplastic syndromes, adult acute myeloid leukemia with t(8;21)(q22;q22), adult acute myeloid leukemia with t(16;16)(p13;q22), adult acute myeloid leukemia with inv(16)(p13;q22), adult acute myeloid leukemia with 11q23 (MLL) abnormalities, adult acute myeloid leukemia with t(15;17)(q22;q12)
Medical subject headings
Graft vs Host Disease, Leukemia, Myelodysplastic Syndromes, Precursor Cell Lymphoblastic Leukemia-Lymphoma, Leukemia, Myeloid, Acute, Leukemia, Myeloid, Accelerated Phase, Leukemia, Myeloid, Chronic-Phase, Anemia, Refractory

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

What is being measured

The primary outcome is the main question the study is designed to answer.

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

    Measured: Day 84

Secondary outcomes (1)

  1. Safety as measured by infusional toxicity, relapse nd survival, formation of potential ectopic tissue foci

    Measured: 84 days

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted8 Apr 2004 (estimated)

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 2
Primary purpose
Supportive care
Allocation
Randomised
Masking
Double blind
Enrolment
0 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT00081055
Sponsor's protocol ID
Mesoblast
Other IDs
UCLA-0303036, CDR0000358809
Status last verified
Dec 2014
First submitted
7 Apr 2004
Last updated
4 Dec 2014
Results posted
No

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing autologous expanded mesenchymal stem cells OTI-010, busulfan and cyclophosphamide in people with Graft Versus Host Disease and Leukemia, run by Mesoblast International Sàrl at 1 site in United States.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: withdrawn, as of the sponsor's last update on 4 Dec 2014. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

The study was withdrawn before enrolling participants, so there are no results.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Incidence of acute GVHD grade II-IV of skin, liver and gut (stomach to rectum) through Day 84 post-PBSC transplantation (time frame: Day 84). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT00081055.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 4 Dec 2014.

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