Study Evaluating AMD3100 for Transplantation of Sibling Donor Stem Cells in Patients With Hematological Malignancies

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
Washington University School of Medicine
ClinicalTrials.gov Identifier:
NCT00241358
First received: October 17, 2005
Last updated: August 9, 2013
Last verified: August 2013
  Purpose

The purpose of this study is to determine if peripheral blood cells collected following AMD3100 mobilization can be used safely for hematopoietic cell transplantation into HLA-matched recipients.


Condition Intervention Phase
Leukemia, Myeloid, Acute
Leukemia, Myelogenous, Chronic
Leukemia, Lymphoblastic, Acute
Lymphocytic Leukemia, Chronic
Myelodysplastic Syndromes
Multiple Myeloma
Lymphoma, Non-Hodgkin
Hodgkin Disease
Drug: AMD3100
Procedure: Stem Cell Transplant
Phase 1
Phase 2

Study Type: Interventional
Study Design: Allocation: Non-Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: A Phase I/II Study Evaluating the Safety and Efficacy of AMD3100 for the Mobilization and Transplantation of HLA-Matched Sibling Donor Hematopoietic Stem Cells in Patients With Advanced Hematological Malignancies

Resource links provided by NLM:


Further study details as provided by Washington University School of Medicine:

Primary Outcome Measures:
  • Proportion of donors from whom a sufficient number of cells for transplantation are collected in no more than 2 LP procedures following mobilization with AMD3100 [ Time Frame: 30 days ] [ Designated as safety issue: No ]
  • Proportion of recipients who experience grade 2-4 acute GVHD [ Time Frame: Day 100 ] [ Designated as safety issue: Yes ]
  • Proportion of recipients who successfully engraft by day +21 after transplant [ Time Frame: Day 21 after transplant ] [ Designated as safety issue: No ]

Secondary Outcome Measures:
  • Proportion of recipients who experience chronic GVHD (any grade) [ Time Frame: 12 months after transplant ] [ Designated as safety issue: Yes ]
  • Percent recipient mortality at day 100 after transplant [ Time Frame: 100 days after transplant ] [ Designated as safety issue: No ]
  • Quality of life during stem cell mobilization [ Time Frame: 48-72 hours after last dose of AMD3100 ] [ Designated as safety issue: No ]
  • Toxicities during mobilization [ Time Frame: 30 days after completion of AMD3100 ] [ Designated as safety issue: Yes ]
  • Pharmacokinetics of SC and IV AMD3100 [ Time Frame: Day 1 ] [ Designated as safety issue: No ]

Enrollment: 92
Study Start Date: May 2004
Study Completion Date: February 2010
Primary Completion Date: December 2009 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Active Comparator: Subcutaneous (SC) Treatment Plan - Donor
AMD3100 SC 240 ug/kg/actual donor weight on Day 1 and possibly Day 3
Drug: AMD3100
Other Name: Plerixafor
Experimental: Intravenous Treatment Plan - Donor
Patients 21-39 will receive IV AMD3100 according to cohorts on day -3 (80 ug/kg cohort 1, 160 ug/kg cohort 2, 240 ug/kg cohort 3, 320 ug/kg cohort 4, 400 ug/kg cohort 5, 480 ug/kg cohort 6) and SC AMD3100 240 ug/kg/actual donor weight on day 1
Drug: AMD3100
Other Name: Plerixafor
Recepients
Stem Cell Transplantation Day 0
Procedure: Stem Cell Transplant

Detailed Description:

This study will determine if peripheral blood cells collected following AMD3100 mobilization can be used safely for hematopoietic cell transplantation into HLA-matched recipients.

  Eligibility

Ages Eligible for Study:   18 Years to 65 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

Donor criteria:

  • Donor is 18 to 70 years of age inclusive
  • If female and of child-bearing age, must be:

    • non-pregnant,
    • not breast feeding and
    • using adequate contraception
  • Donor is a 6/6 HLA-matched sibling willing to donate peripheral blood stem cell for transplant
  • Donor must be willing to provide written informed consent.
  • Adequate cardiac function with no history of congestive heart failure and no history of atrial fibrillation or ventricular tachyarrhythmia.
  • Adequate renal function as defined by a serum creatinine clearance of ≥75% of normal (Cockcroft-Gault equation)
  • Adequate hepatic function as defined by a total bilirubin <2x normal or absence of hepatic fibrosis/cirrhosis
  • Adequate neurologic function as defined by:

    • No evidence of a severe central or peripheral neurologic abnormality.
    • No history of cerebrovascular accident or seizure disorder requiring anticonvulsant medication
  • Must be HIV-1 & 2 antibody, HIV-1 antigen, and HTLV-I & II antibody sero-negative, by FDA licensed test.
  • Must have an ECOG performance status of 0 or 1
  • Must demonstrate ability to be compliant with study regimen.
  • Must not have an active infection at the time of study entry
  • Not have active alcohol or substance abuse within 6 months of study entry
  • Not currently enrolled in another investigational agent study
  • Not have any medical condition, which, in the opinion of the clinical investigator, would interfere with his/her evaluation

Recipient criteria:

  • 18 to 65 years of age inclusive
  • Willing and has a 6/6 HLA-matched sibling willing to donate PBSC for transplant
  • Provide signed informed consent
  • If female and of child-bearing age, must be:

    • non-pregnant,
    • not breast feeding, and
    • using adequate contraception

Patient must have one of the following diagnoses:

  • AML in 1st or subsequent remission or in relapse
  • ALL in 1st or subsequent remission or in relapse
  • MDS and intermediate 1 or 2, or high risk by the International Prognostic Scoring System
  • CML in accelerated or second chronic phase
  • NHL or HD in 2nd or greater complete remission, partial remission,or refractory relapse
  • CLL Rai Stage 2-4, failing at least 2 prior regimens
  • MM Stage 2-3
  • Adequate cardiac function with a left ventricular ejection fraction ≥ 40%
  • Adequate pulmonary function defined as:

    • No severe or symptomatic restrictive or obstructive lung disease, and
    • formal pulmonary function testing showing an forced expiratory volume at 1 second (FEV1) ≥50% of predicted and a diffusion capacity of the lung for carbon monoxide (DLCO) ≥40% of predicted, corrected for hemoglobin
  • Adequate renal function as defined by a serum creatinine clearance of ≥75% of normal (Cockcroft-Gault equation)
  • Adequate hepatic function as defined by a total bilirubin <2x normal or absence of hepatic fibrosis/cirrhosis
  • Adequate neurologic function as defined by no evidence of a severe central or peripheral neurologic abnormality. Patients with a history of previous central nervous system tumor involvement are eligible provided they are without symptoms or signs and the CNS is now free of disease on lumbar puncture and CT scan of the brain
  • No evidence of active infection at the time of the transplant preparative regimen or at the time of transplantation
  • Patient must be HIV-1 & 2 antibody, HIV-1 antigen, and HTLV-I & II antibody sero-negative, by FDA licensed test
  • ECOG performance status of 0 or 1
  • Must demonstrate ability to be compliant with medical regimen
  • Not have active alcohol or substance abuse within 6 months of study entry
  • Not be concurrently enrolled on another study involving an investigational agent
  • Not have any medical condition, which, in the opinion of the clinical investigator, would interfere with the evaluation of the patient
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT00241358

Locations
United States, Missouri
Washington University School of Medicine
St. Louis, Missouri, United States, 63110
Sponsors and Collaborators
Washington University School of Medicine
Investigators
Principal Investigator: John F. DiPersio, M.D., Ph.D. Washington University School of Medicine
  More Information

Additional Information:
Publications:
Responsible Party: Washington University School of Medicine
ClinicalTrials.gov Identifier: NCT00241358     History of Changes
Other Study ID Numbers: 03-0349
Study First Received: October 17, 2005
Last Updated: August 9, 2013
Health Authority: United States: Food and Drug Administration

Keywords provided by Washington University School of Medicine:
Plerixafor
Stem cell transplantation
Stem cells
Mobilization

Additional relevant MeSH terms:
Multiple Myeloma
Neoplasms, Plasma Cell
Myelodysplastic Syndromes
Preleukemia
Leukemia, Lymphoid
Leukemia
Hodgkin Disease
Lymphoma, Non-Hodgkin
Leukemia, Myeloid
Leukemia, Myeloid, Acute
Precursor Cell Lymphoblastic Leukemia-Lymphoma
Leukemia, Lymphocytic, Chronic, B-Cell
Leukemia, Myelogenous, Chronic, BCR-ABL Positive
Neoplasms by Histologic Type
Neoplasms
Hemostatic Disorders
Vascular Diseases
Cardiovascular Diseases
Paraproteinemias
Blood Protein Disorders
Hematologic Diseases
Hemorrhagic Disorders
Lymphoproliferative Disorders
Immunoproliferative Disorders
Immune System Diseases
Bone Marrow Diseases
Precancerous Conditions
Lymphatic Diseases
Lymphoma
Leukemia, B-Cell

ClinicalTrials.gov processed this record on September 18, 2014