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Allogeneic Transplantation for Pediatric Leukemias With Unrelated Donors

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ClinicalTrials.gov Identifier: NCT00679536
Recruitment Status : Unknown
Verified January 2014 by Sonali Chaudhury, MD, Ann & Robert H Lurie Children's Hospital of Chicago.
Recruitment status was:  Recruiting
First Posted : May 19, 2008
Last Update Posted : January 23, 2014
Sponsor:
Information provided by (Responsible Party):

Study Description
Brief Summary:
The study proposes the use of Fludarabine, Busulfan, Anti Thymocyte Globulin Rabbit (ATG) and Total Body Irradiation as a preparative regimen before hematopoietic stem cell transplant from unrelated donor peripheral blood stem cells (PBSC). The hypothesis states that the 100 day mortality after this type of transplant will be significantly below the accepted standards, which is about 30% for unrelated donors.

Condition or disease Intervention/treatment Phase
Leukemia Drug: Busulfan Drug: Fludarabine Drug: Thymoglobulin Radiation: Total Body Irradiation Phase 1 Phase 2

Detailed Description:
The primary objective of this study is to evaluate the toxicity (as measured as 100 day survival) after hematopoietic stem cell transplant from an unrelated donor with a novel preparative regimen of Fludarabine, Busulfan, Anti-Thymocyte Globulin, and Total Body Irradiation for pediatric patients with leukemia. The secondary objectives are to evaluate the relapse-free and overall survival after hematopoietic stem cell transplant as well as to evaluate the incidence of acute and chronic graft-versus-host disease after this preparative regimen.

Study Design

Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 30 participants
Allocation: Non-Randomized
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Allogeneic Transplantation for Pediatric Leukemias With Unrelated Donors Using Fludarabine, Busulfan, 400 cGy Total Body Irradiation, and Thymoglobulin
Study Start Date : May 2008
Estimated Primary Completion Date : May 2015
Estimated Study Completion Date : May 2015

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Leukemia
U.S. FDA Resources

Arms and Interventions

Arm Intervention/treatment
Experimental: A
All patients on this trial will receive a conditioning regimen of Busulfan, Fludarabine, Anti-Thymocyte Globulin and Total Body Irradiation (400 cGy)
Drug: Busulfan
Patient will receive a Test Dose of Busulfan on either Day -10 or Day -9. Patient will receive their Regimen Dose of Busulfan on Day -5 to Day -2. The regimen dose of Busulfan will be based off of the findings from their Test Dose.
Other Name: IV Busulfex
Drug: Fludarabine
Patient will receive Fludarabine from Day -6 to Day -2. The dose of Fludarabine will be 30 mg/m^2/day.
Other Name: Fludara
Drug: Thymoglobulin
The patient will also receive Thymoglobulin (rabbitATG) on Day -4 to Day -2. Each dose of rabbitATG will be 1.5 mg/kg/day.
Radiation: Total Body Irradiation
On Day -1 the patient will receive a total of 400 cGy of Total Body Irradiation.


Outcome Measures

Primary Outcome Measures :
  1. To evaluate the toxicity (as measures by 100 day survival) after hematopoietic stem cell transplant from an unrelated donor with a novel preparative regimen. [ Time Frame: 100 day mortality ]

Secondary Outcome Measures :
  1. To evaluate the relapse-free and overall survival after hematopoietic stem cell transplant with Fludarabine/Busulfan/ATG/TBI preparative regimen for pediatric patients with leukemia. [ Time Frame: 5 years ]
  2. To evaluate the incidence of acute and chronic graft-versus-host disease after hematopoietic stem cell transplant [ Time Frame: 5 years ]

Eligibility Criteria

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Ages Eligible for Study:   up to 21 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Ages 0-21
  • AML in one of the following stages:

    • Having preceding myelodysplasia (MDS)
    • High Risk cytogenetics
    • Requiring > 2 cycles chemotherapy to obtain complete remission
    • High allelic ratio FLT3/ITD+,
    • Standard risk cytogenetics with positive MRD at end of Induction
    • Second or greater CR
    • First relapse with < 25% blasts in bone marrow
    • With therapy-related AML whose prior malignancy has been in remission for at least 12 months
  • ALL in one of the following stages:
  • High risk first remission, defined as:

    • Ph+ ALL; or,
    • MLL rearrangement with slow early response [defined as having M2 (5-25% blasts) or M3 (>25% blasts on bone marrow examination on Day 14 of induction therapy)]; or,
    • Hypodiploidy (< 44 chromosomes or DNA index < 0.81); or,
    • End of induction M3 bone marrow; or,
    • End of induction M2 marrow or MRD>1% with M2-3 marrow or MRD>1% at Day 42.
    • High-risk infant ALL defined as age <6 months at diagnosis with MLL (11q23) translocation.
  • High risk second remission, defined as:

    • Bone marrow relapse < 36 months from induction; or >36 mths if a matched sibling donor is available
    • T-lineage relapse at any time; or,
    • Very early isolated CNS relapse (<18 months from diagnosis); or,
    • Slow reinduction (M2-3 at Day 28) after relapse at any time. − Any third or subsequent CR.
  • Biphenotypic or undifferentiated leukemia in any CR or if in first relapse must have < 25% blasts in bone marrow
  • MDS at any stage; prior therapies allowed
  • CML in chronic or accelerated phase; prior therapies allowed
  • Patient also must have the following organ requirements:

    • Adequate renal function defined as serum creatinine <2x normal, or creatinine clearance > 40 ml/min/m^2 or 70 ml/min.
    • Adequate liver function as defined by total bilirubin less than or equal to 2 times normal and AST and ALT less than or equal to 4 times normal.
    • Adequate cardiac function as defined by: shortening fraction > 24% by echocardiogram, or ejection fraction > 30% by radionuclide angiogram.
    • Adequate pulmonary function as defined by DLCO, FEV1/FVC > 60% by pulmonary function tests. For children who are uncooperative for PFTs and have no evidence of dyspnea at rest or exercise intolerance, pulse oximetry > 94% on room air is considered acceptable, with a normal chest xray.
    • Adequate venous access; a double lumen central vascular access device or its equivalent and an additional PICC line will be required for all patients.
  • Women of childbearing potential and sexually active males should use effective contraception while on study.

Exclusion Criteria:

  • Inability to give informed consent or assent
  • Inability to obtain a suitable donor
  • Patient who is HIV-positive
  • Patient who has active Hepatitis B
  • Patient who is pregnant
  • Patient who is otherwise considered unsuitable for transplant at the discretion of the principal investigator.
Contacts and Locations

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT00679536


Contacts
Contact: Sonali Chaudhury, MD 312-227-4863 schaudhury@luriechildrens.org
Contact: Colleen E Rosen, BS, RN 312-227-4870 crosen@luriechildrens.org

Locations
United States, Illinois
Ann & Robert H. Lurie Children's Hospital of Chicago Recruiting
Chicago, Illinois, United States, 60611
Contact: Sonali Chaudhury, MD    312-227-4863    schaudhury@luriechildrens.org   
Contact: Colleen E Rosen, BS, RN    312-227-4870    crosen@luriechildrens.org   
Sponsors and Collaborators
Ann & Robert H Lurie Children's Hospital of Chicago
Investigators
Principal Investigator: Sonali Chaudhury, MD Ann & Robert H Lurie Children's Hospital of Chicago
More Information

Responsible Party: Sonali Chaudhury, MD, PI, Ann & Robert H Lurie Children's Hospital of Chicago
ClinicalTrials.gov Identifier: NCT00679536     History of Changes
Other Study ID Numbers: SCT 0208
First Posted: May 19, 2008    Key Record Dates
Last Update Posted: January 23, 2014
Last Verified: January 2014

Keywords provided by Sonali Chaudhury, MD, Ann & Robert H Lurie Children's Hospital of Chicago:
Reduced Toxicity
Toxicity
Event Free Survival
Graft vs Host Disease

Additional relevant MeSH terms:
Leukemia
Neoplasms by Histologic Type
Neoplasms
Fludarabine
Fludarabine phosphate
Busulfan
Thymoglobulin
Antilymphocyte Serum
Vidarabine
Antineoplastic Agents
Antimetabolites, Antineoplastic
Antimetabolites
Molecular Mechanisms of Pharmacological Action
Immunosuppressive Agents
Immunologic Factors
Physiological Effects of Drugs
Antiviral Agents
Anti-Infective Agents
Alkylating Agents
Antineoplastic Agents, Alkylating
Myeloablative Agonists