Stem Cell Transplantation for Patients With Multiple Myeloma

Phase 1

Active, not recruiting

• Conditions: Myeloma
• Interventions: Drug: G-CSF; Drug: Plerixafor; Procedure: Apheresis; Drug: Melphalan; Procedure: Stem cell re-infusion; Device: CliniMACS CD25 microbeads and cell sorter; Drug: Basiliximab

• Registration Number: NCT01526096
• Lead Sponsor: University of Chicago

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).

Detailed Description

Not available

Study Timeline

• Study Start: 2011-07-12
• Study First Submitted: 2011-08-10
• Study First Submitted QC: 2012-01-31
• Study First Posted: 2012-02-03
• Status Verified: 2025-02
• Last Update Submitted: 2025-02-04
• Last Update Posted: 2025-02-05
• Primary Completion: 2025-09-12
• Study Completion: 2025-09-12

Recruitment & Eligibility

• Status: ACTIVE_NOT_RECRUITING
• Sex: All
• Target Recruitment: 15

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.

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
Depletion of T-cells before ASCT(Grp 3)	Apheresis	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.
Depletion of T-cells before ASCT(Grp 3)	Stem cell re-infusion	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.
Depletion of T-cells before ASCT(Grp 3)	CliniMACS CD25 microbeads and cell sorter	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.
Standard ASCT (Grp 1)	Apheresis	Standard autologous stem cell transplantation (ASCT)
Standard ASCT (Grp 1)	Stem cell re-infusion	Standard autologous stem cell transplantation (ASCT)
Depletion of T-cells after ASCT (Grp 2)	Apheresis	Standard ASCT followed by treatment with basiliximab to remove certain immune cells (called regulatory T-cells or Tregs) from the blood
Depletion of T-cells after ASCT (Grp 2)	Stem cell re-infusion	Standard ASCT followed by treatment with basiliximab to remove certain immune cells (called regulatory T-cells or Tregs) from the blood
Standard ASCT (Grp 1)	G-CSF	Standard autologous stem cell transplantation (ASCT)
Standard ASCT (Grp 1)	Plerixafor	Standard autologous stem cell transplantation (ASCT)
Standard ASCT (Grp 1)	Melphalan	Standard autologous stem cell transplantation (ASCT)
Depletion of T-cells after ASCT (Grp 2)	G-CSF	Standard ASCT followed by treatment with basiliximab to remove certain immune cells (called regulatory T-cells or Tregs) from the blood
Depletion of T-cells after ASCT (Grp 2)	Plerixafor	Standard ASCT followed by treatment with basiliximab to remove certain immune cells (called regulatory T-cells or Tregs) from the blood
Depletion of T-cells after ASCT (Grp 2)	Basiliximab	Standard ASCT followed by treatment with basiliximab to remove certain immune cells (called regulatory T-cells or Tregs) from the blood
Depletion of T-cells after ASCT (Grp 2)	Melphalan	Standard ASCT followed by treatment with basiliximab to remove certain immune cells (called regulatory T-cells or Tregs) from the blood
Depletion of T-cells before ASCT(Grp 3)	G-CSF	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.
Depletion of T-cells before ASCT(Grp 3)	Plerixafor	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.
Depletion of T-cells before ASCT(Grp 3)	Melphalan	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.

Primary Outcome Measures

Name	Time	Method
Purity of ex vivo depleted regulatory T cells prior to autologous stem cell transplant (arm 3 only)	1-3 days	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	180 days	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	180 days	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

Name	Time	Method
Kinetics of recovery of peripheral blood cellular elements	180 days	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	100 days	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.

Trial Locations

Locations (1)

University of Chicago; 🇺🇸 Chicago, Illinois, United States