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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


Phase 2
18 Years
55 Years
Open (Enrolling)
Both
Graft Versus Host Disease, Leukemia, Myelodysplastic Syndromes

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Trial Information

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


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.

Inclusion 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)

Type of Study:

Interventional

Study Design:

Allocation: Randomized, Masking: Double-Blind, Primary Purpose: Supportive Care

Principal Investigator

Mary C. Territo, MD

Investigator Role:

Principal Investigator

Investigator Affiliation:

Jonsson Comprehensive Cancer Center

Authority:

United States: Federal Government

Study ID:

CDR0000358809

NCT ID:

NCT00081055

Start Date:

December 2003

Completion Date:

Related Keywords:

  • Graft Versus Host Disease
  • Leukemia
  • Myelodysplastic Syndromes
  • 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)
  • Graft vs Host Disease
  • Leukemia
  • Myelodysplastic Syndromes
  • Preleukemia
  • Hematologic Neoplasms

Name

Location

Jonsson Comprehensive Cancer Center, UCLALos Angeles, California  90095-1781