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Safety and Efficacy of POL6326 for Mobilization/Transplant of Sibling Donor in Patients With Hematologic Malignancies

Phase 1
Completed
Conditions
Acute Myeloid Leukemia in Remission
Adult Acute Lymphoblastic Leukemia in Remission
Chronic Myelogenous Leukemia (CML)
Non-Hodgkin's Lymphoma (NHL) or Hodgkin's Disease (HD) in 2nd or Greater Complete Remission, Partial Remission
Chronic Lymphocytic Leukemia (CLL)
Multiple Myeloma (MM)
Myelodysplastic Syndrome (MDS)
Myeloproliferative Disorders
Interventions
Procedure: PBSC Transplant
Procedure: Leukapheresis
Registration Number
NCT01413568
Lead Sponsor
Polyphor Ltd.
Brief Summary

Determine the safety and tolerability of POL6326 when used as a single mobilization agent.

Detailed Description

Current protocols use G-CSF to mobilize hematopoietic progenitor cells from matched sibling donors. This process requires from four to six days of G-CSF injection and is associated with significant morbidity, most notably bone pain. POL6326 is associated with few side effects and collection of cells occurs on the same day as POL6326 administration.

This study will evaluate the safety and efficacy of this novel agent for hematopoietic progenitor cell mobilization and allogeneic transplantation based on the following hypotheses:

1. Donors mobilized with intravenous POL6326 will require fewer collections than have previously been seen for donors mobilized with subcutaneous plerixafor.

2. Healthy HLA-matched donors receiving one or two infusions of POL6326 will mobilize sufficient CD34+ cells (at least 2.0 x 106 CD34+ cells/kg recipient weights) following leukapheresis to support a hematopoietic cell transplant.

3. IV POL6326 will result in more rapid kinetics and a higher maximum (peak) of human CD34+ stem cells mobilized from human normal allogeneic donors compared to previous donors who were mobilized with plerixafor.

4. The hematopoietic cells mobilized by IV POL6326 will be functional and will result in prompt and durable hematopoietic engraftment following transplantation into HLA-identical siblings with advanced hematological malignancies using various non-myeloablative and myeloablative conditioning regimens and regimens for routine GVHD prophylaxis.

Recruitment & Eligibility

Status
COMPLETED
Sex
All
Target Recruitment
38
Inclusion Criteria

Not provided

Exclusion Criteria

Not provided

Study & Design

Study Type
INTERVENTIONAL
Study Design
PARALLEL
Arm && Interventions
GroupInterventionDescription
Donor (Phase I and Phase II)POL6326On Day 1 (and possibly Day 2) POL6326 IV Infusion with increasing dose levels in Phase I or with random dose assignment (from the 2 selected from Phase I) in phase II Leukapheresis collection on Day 1 (and possibly Day 2)
Donor (Phase I and Phase II)LeukapheresisOn Day 1 (and possibly Day 2) POL6326 IV Infusion with increasing dose levels in Phase I or with random dose assignment (from the 2 selected from Phase I) in phase II Leukapheresis collection on Day 1 (and possibly Day 2)
RecipientPBSC TransplantDay 0 - PBSC transplant with stem cells mobilized with IV POL6326
Primary Outcome Measures
NameTimeMethod
Phase I Study - safety and tolerability of POL6326 as a mobilization agent.30 days
Phase II Study - determine the number of allogeneic donors who require a second leukapheresis2 days

Determine the number of allogeneic donors which collect \>= 2 mill CD34+ cells with one or two leukapheresis procedures treated with IV POL6326. Comparison with historic group of donors who were mobilized with 240 µg/kg SC plerixafor.

Secondary Outcome Measures
NameTimeMethod
Phase I Study - define maximum tolerated dose of POL632630 days
Phase II Study - pharmacokinetics and pharmacodynamics of IV POL6326Day 1-3

Stem cell and T-cell phenotyping

Phase II Study - the proportion of HLA-identical sibling donors who experience grade 3-4 infusional toxicity and the proportion who are safely mobilized30 days

To estimate the proportion of HLA-identical sibling donors who experience grade 3-4 infusional toxicity and the proportion from whom \> 2 mill CD34+ cells/kg recipient weight are safely mobilized following one or two leukapheresis procedures

Phase II Study - rate of acute GVHD and chronic GVHD in patients who receive IV POL6326 mobilized peripheral blood stem cells.Day 100 (+/- 7 days) or Day 365 (+/-14 days)

Acute GVHD - Day 100 (+/- 7 days) Chronic GVHD - Day 365 (+/- 14 days)

Phase II Study - kinetics of neutrophil and platelet engraftment in recipients of POL6326 mobilized peripheral blood stem cells.Day 365 (+/- 14 days)

Trial Locations

Locations (2)

Washington University School of Medicine

🇺🇸

St. Louis, Missouri, United States

University of Kansas Cancer Center

🇺🇸

Kansas City, Kansas, United States

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