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Stem Cell Transplantation for Patients With Multiple Myeloma

Primary Purpose

Myeloma

Status
Active
Phase
Phase 1
Locations
United States
Study Type
Interventional
Intervention
G-CSF
Plerixafor
Apheresis
Melphalan
Stem cell re-infusion
Basiliximab
CliniMACS CD25 microbeads and cell sorter
Sponsored by
University of Chicago
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Myeloma

Eligibility Criteria

21 Years - 70 Years (Adult, Older Adult)All SexesDoes not accept healthy volunteers

Inclusion Criteria:

  • Symptomatic multiple myeloma of any subtype in any disease stage, providing that patient does not have smoldering myeloma.
  • Patient must otherwise be a candidate for ASCT as determined by treating physician.
  • No current CNS Myeloma at time of enrollment.
  • Life expectancy greater than 12 weeks.
  • Age greater than or equal to 21 and less than or equal to 70 years old.
  • EGOG performance status less than or equal to 2.
  • No cardiac, pulmonary, hepatic, or renal contraindications for high dose chemotherapy.
  • HIV Negative.
  • No active Hepatitis B or C.
  • Patients must be able to provide written informed, consent.

Exclusion Criteria:

  • Pregnant or nursing women. Women of child-bearing age must be tested for pregnancy.
  • Use of systemic immunosuppressive medications, including corticosteroids, tacrolimus, mycophenolate mofetil, sirolimus or cyclosporine A.
  • Psychiatric illness which may make compliance to the clinical protocol unmanageable or which may compromise the ability of the patient to give informed consent.
  • Active autoimmune disease including but not limited to: rheumatoid arthritis inflammatory bowel disease, celiac disease, systemic lupus erythematosis, scleroderma or multiple sclerosis.

Sites / Locations

  • University of Chicago

Arms of the Study

Arm 1

Arm 2

Arm 3

Arm Type

Active Comparator

Experimental

Experimental

Arm Label

Standard ASCT (Grp 1)

Depletion of T-cells after ASCT (Grp 2)

Depletion of T-cells before ASCT(Grp 3)

Arm Description

Standard autologous stem cell transplantation (ASCT)

Standard ASCT followed by treatment with basiliximab to remove certain immune cells (called regulatory T-cells or Tregs) from the blood

Blood collected for an ASCT will be processed using a special cell sorting machine (CliniMACS device) to remove Treg cells before the stem cells are infused back into the body during stem cell transplant.

Outcomes

Primary Outcome Measures

Purity of ex vivo depleted regulatory T cells prior to autologous stem cell transplant (arm 3 only)
Percentage of CD4+CD25+ regulatory T cells following ex vivo depletion in arm 3 will be analyzed by flow cytometry and compared to a pre-CD25-depletion sample. The depletion of CD25+ cells among the entire CD4+ population is expected to reach 80% efficiency.
Timing and duration of regulatory T cell depletion and recovery following autologous stem cell transplant
Timing and duration of regulatory T cell depletion and recovery following in vivo or ex vivo (arms 2 and 3) CD25+ T cell depletion will be performed at pre-defined timepoints prior to and following autologous stem cell transplant by flow cytometry on peripheral blood samples and directly compared to the percentages of regulatory T cells (CD4+CD25+FoxP3+ or CD4+CD25+CD127-) present at the same timepoints in patients enrolled onto arm 1 in which no regulatory T cell depletion is performed.
Incidence of autologous graft-versus-host disease following in vivo or ex vivo regulatory T cell depletion
The indicence of autologous graft-versus-host disease, as assessed by the development of skin rash, diarrhea and/or liver function test abnormalities consistent with autologous graft-versus-host disease following CD25+ T cell depletion and autologous stem cell transplant compared with the incidence of autologous graft-versus-host disease in patients enrolled onto arm 1 in which no regulatory T cell depletion is performed.

Secondary Outcome Measures

Kinetics of recovery of peripheral blood cellular elements
Time to recovery of neutrophils and platelets will be analyzed by daily complete blood counts following autologous stem cell transplant. Patients enrolled onto arms 2 and 3 (in vivo and ex vivo regulatory T cell depletion, respectively) will be directly compared to patients enrolled onto arm 1 in which no regulatory T cell depletion is performed.
Number of patients that experience a complete response following autologous stem cell transplant based upon the assigned study arm using International Myeloma Working Group definitions
The complete response rate following autologous stem cell transplant with or without regulatory T cell depletion will be analyzed and compared directly between study arms.

Full Information

First Posted
August 10, 2011
Last Updated
April 26, 2023
Sponsor
University of Chicago
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1. Study Identification

Unique Protocol Identification Number
NCT01526096
Brief Title
Stem Cell Transplantation for Patients With Multiple Myeloma
Official Title
Pilot Study T Cell Depletion in the Setting of Autologous Stem Cell Transplantation for Patients With Multiple Myeloma
Study Type
Interventional

2. Study Status

Record Verification Date
April 2023
Overall Recruitment Status
Active, not recruiting
Study Start Date
July 12, 2011 (Actual)
Primary Completion Date
September 12, 2023 (Anticipated)
Study Completion Date
September 12, 2023 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
University of Chicago

4. Oversight

Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
Yes
Device Product Not Approved or Cleared by U.S. FDA
Yes
Data Monitoring Committee
No

5. Study Description

Brief Summary
The purpose of this study is to test whether regulatory T-cell reduction is possible and safe in myeloma subjects undergoing autologous stem cell transplantation (ASCT).

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Myeloma

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 1
Interventional Study Model
Parallel Assignment
Masking
None (Open Label)
Allocation
Non-Randomized
Enrollment
15 (Actual)

8. Arms, Groups, and Interventions

Arm Title
Standard ASCT (Grp 1)
Arm Type
Active Comparator
Arm Description
Standard autologous stem cell transplantation (ASCT)
Arm Title
Depletion of T-cells after ASCT (Grp 2)
Arm Type
Experimental
Arm Description
Standard ASCT followed by treatment with basiliximab to remove certain immune cells (called regulatory T-cells or Tregs) from the blood
Arm Title
Depletion of T-cells before ASCT(Grp 3)
Arm Type
Experimental
Arm Description
Blood collected for an ASCT will be processed using a special cell sorting machine (CliniMACS device) to remove Treg cells before the stem cells are infused back into the body during stem cell transplant.
Intervention Type
Drug
Intervention Name(s)
G-CSF
Intervention Description
G-CSF will be self-administered shot daily for 4 days pre-transplant. Up to 8 doses of G-CSF may be given. G-CSF will also be administered once daily under the skin beginning 5 days after your stem cell infusion until your white blood cell count is high enough
Intervention Type
Drug
Intervention Name(s)
Plerixafor
Intervention Description
Plerixafor (self-administered shot)prior to the beginning of the stem cell collection. Up to 4 doses of plerixafor may be given.
Intervention Type
Procedure
Intervention Name(s)
Apheresis
Intervention Description
Stem cell collection begins on day 5 and can last up to 3 days depending on the number collected.
Intervention Type
Drug
Intervention Name(s)
Melphalan
Other Intervention Name(s)
Alkeran
Intervention Description
Melphalan chemotherapy 100mg/m2 for 2 days after your admission into the hospital for your ASCT procedure.
Intervention Type
Procedure
Intervention Name(s)
Stem cell re-infusion
Intervention Description
Stem cells are thawed and reinfused back into the body via a catheter in the vein.
Intervention Type
Drug
Intervention Name(s)
Basiliximab
Other Intervention Name(s)
Simulect
Intervention Description
Basiliximab (20mg) given by IV infusion (through the vein) 20-30 minutes the day after ASCT.
Intervention Type
Device
Intervention Name(s)
CliniMACS CD25 microbeads and cell sorter
Intervention Description
The stem cells collected during apheresis will be counted and treated with CD25 microbeads and processed by a special device called a CliniMACs machine which removes the regulatory T cells from you stem cell product.
Primary Outcome Measure Information:
Title
Purity of ex vivo depleted regulatory T cells prior to autologous stem cell transplant (arm 3 only)
Description
Percentage of CD4+CD25+ regulatory T cells following ex vivo depletion in arm 3 will be analyzed by flow cytometry and compared to a pre-CD25-depletion sample. The depletion of CD25+ cells among the entire CD4+ population is expected to reach 80% efficiency.
Time Frame
1-3 days
Title
Timing and duration of regulatory T cell depletion and recovery following autologous stem cell transplant
Description
Timing and duration of regulatory T cell depletion and recovery following in vivo or ex vivo (arms 2 and 3) CD25+ T cell depletion will be performed at pre-defined timepoints prior to and following autologous stem cell transplant by flow cytometry on peripheral blood samples and directly compared to the percentages of regulatory T cells (CD4+CD25+FoxP3+ or CD4+CD25+CD127-) present at the same timepoints in patients enrolled onto arm 1 in which no regulatory T cell depletion is performed.
Time Frame
180 days
Title
Incidence of autologous graft-versus-host disease following in vivo or ex vivo regulatory T cell depletion
Description
The indicence of autologous graft-versus-host disease, as assessed by the development of skin rash, diarrhea and/or liver function test abnormalities consistent with autologous graft-versus-host disease following CD25+ T cell depletion and autologous stem cell transplant compared with the incidence of autologous graft-versus-host disease in patients enrolled onto arm 1 in which no regulatory T cell depletion is performed.
Time Frame
180 days
Secondary Outcome Measure Information:
Title
Kinetics of recovery of peripheral blood cellular elements
Description
Time to recovery of neutrophils and platelets will be analyzed by daily complete blood counts following autologous stem cell transplant. Patients enrolled onto arms 2 and 3 (in vivo and ex vivo regulatory T cell depletion, respectively) will be directly compared to patients enrolled onto arm 1 in which no regulatory T cell depletion is performed.
Time Frame
180 days
Title
Number of patients that experience a complete response following autologous stem cell transplant based upon the assigned study arm using International Myeloma Working Group definitions
Description
The complete response rate following autologous stem cell transplant with or without regulatory T cell depletion will be analyzed and compared directly between study arms.
Time Frame
100 days

10. Eligibility

Sex
All
Minimum Age & Unit of Time
21 Years
Maximum Age & Unit of Time
70 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria: Symptomatic multiple myeloma of any subtype in any disease stage, providing that patient does not have smoldering myeloma. Patient must otherwise be a candidate for ASCT as determined by treating physician. No current CNS Myeloma at time of enrollment. Life expectancy greater than 12 weeks. Age greater than or equal to 21 and less than or equal to 70 years old. EGOG performance status less than or equal to 2. No cardiac, pulmonary, hepatic, or renal contraindications for high dose chemotherapy. HIV Negative. No active Hepatitis B or C. Patients must be able to provide written informed, consent. Exclusion Criteria: Pregnant or nursing women. Women of child-bearing age must be tested for pregnancy. Use of systemic immunosuppressive medications, including corticosteroids, tacrolimus, mycophenolate mofetil, sirolimus or cyclosporine A. Psychiatric illness which may make compliance to the clinical protocol unmanageable or which may compromise the ability of the patient to give informed consent. Active autoimmune disease including but not limited to: rheumatoid arthritis inflammatory bowel disease, celiac disease, systemic lupus erythematosis, scleroderma or multiple sclerosis.
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Michael Bishop, MD
Organizational Affiliation
University of Chicago
Official's Role
Principal Investigator
Facility Information:
Facility Name
University of Chicago
City
Chicago
State/Province
Illinois
ZIP/Postal Code
60637
Country
United States

12. IPD Sharing Statement

Citations:
PubMed Identifier
31940591
Citation
Derman BA, Zha Y, Zimmerman TM, Malloy R, Jakubowiak A, Bishop MR, Kline J. Regulatory T-cell depletion in the setting of autologous stem cell transplantation for multiple myeloma: pilot study. J Immunother Cancer. 2020 Jan;8(1):e000286. doi: 10.1136/jitc-2019-000286.
Results Reference
derived

Learn more about this trial

Stem Cell Transplantation for Patients With Multiple Myeloma

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