Stem Cell Transplantation for Patients With Multiple Myeloma

NCT01526096 | Completed Phase 1 FDA Drug FDA Device

• 1 other identifier: 10-551-B
• Study Type: interventional
• Target: 30
• Locations: 1 country
• Sites: 1

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).

Conditions

Myeloma

Outcome Measures

Primary Outcomes (3)

• 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.

  1-3 days

• 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.

  180 days

• 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.

  180 days

Secondary Outcomes (2)

• Kinetics of recovery of peripheral blood cellular elements

  180 days

• 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

Study Arms (3)

Standard ASCT (Grp 1)

ACTIVE COMPARATOR

Standard autologous stem cell transplantation (ASCT)

Drug: G-CSF; Drug: Plerixafor; Procedure: Apheresis; Drug: Melphalan; Procedure: Stem cell re-infusion

Depletion of T-cells after ASCT (Grp 2)

EXPERIMENTAL

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

Drug: G-CSF; Drug: Plerixafor; Procedure: Apheresis; Drug: Melphalan; Procedure: Stem cell re-infusion; Drug: Basiliximab

Depletion of T-cells before ASCT(Grp 3)

EXPERIMENTAL

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.

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

Interventions

G-CSF DRUG

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

Depletion of T-cells after ASCT (Grp 2); Depletion of T-cells before ASCT(Grp 3); Standard ASCT (Grp 1)

Plerixafor DRUG

Plerixafor (self-administered shot)prior to the beginning of the stem cell collection. Up to 4 doses of plerixafor may be given.

Depletion of T-cells after ASCT (Grp 2); Depletion of T-cells before ASCT(Grp 3); Standard ASCT (Grp 1)

Apheresis PROCEDURE

Stem cell collection begins on day 5 and can last up to 3 days depending on the number collected.

Depletion of T-cells after ASCT (Grp 2); Depletion of T-cells before ASCT(Grp 3); Standard ASCT (Grp 1)

Melphalan DRUG

Melphalan chemotherapy 100mg/m2 for 2 days after your admission into the hospital for your ASCT procedure.

Also known as: Alkeran

Depletion of T-cells after ASCT (Grp 2); Depletion of T-cells before ASCT(Grp 3); Standard ASCT (Grp 1)

Stem cell re-infusion PROCEDURE

Stem cells are thawed and reinfused back into the body via a catheter in the vein.

Depletion of T-cells after ASCT (Grp 2); Depletion of T-cells before ASCT(Grp 3); Standard ASCT (Grp 1)

Basiliximab DRUG

Basiliximab (20mg) given by IV infusion (through the vein) 20-30 minutes the day after ASCT.

Also known as: Simulect

Depletion of T-cells after ASCT (Grp 2)

CliniMACS CD25 microbeads and cell sorter DEVICE

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.

Depletion of T-cells before ASCT(Grp 3)

Eligibility Criteria

• Age: 21 Years - 70 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• University of Chicago: lead

Study Sites (1)

University of Chicago

Chicago, Illinois, 60637, United States

Location

Related Publications (1)

• 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.

  PMID: 31940591 DERIVED

MeSH Terms

Conditions

Neoplasms, Plasma Cell

Interventions

Granulocyte Colony-Stimulating Factor; plerixafor; Blood Component Removal; Melphalan; Basiliximab

Condition Hierarchy (Ancestors)

Neoplasms by Histologic Type; Neoplasms

Intervention Hierarchy (Ancestors)

Colony-Stimulating Factors; Glycoproteins; Glycoconjugates; Carbohydrates; Hematopoietic Cell Growth Factors; Cytokines; Intercellular Signaling Peptides and Proteins; Peptides; Amino Acids, Peptides, and Proteins; Proteins; Biological Factors; Therapeutics; Nitrogen Mustard Compounds; Mustard Compounds; Hydrocarbons, Halogenated; Hydrocarbons; Organic Chemicals; Phenylalanine; Amino Acids, Aromatic; Amino Acids, Cyclic; Amino Acids; Antibodies, Monoclonal, Humanized; Antibodies, Monoclonal; Antibodies; Immunoglobulins; Immunoproteins; Blood Proteins; Serum Globulins; Globulins

Study Officials

• Michael Bishop, MD

  University of Chicago

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: August 10, 2011
• First Posted: February 3, 2012
• Study Start: July 12, 2011
• Primary Completion: August 28, 2024
• Study Completion: August 28, 2024
• Last Updated: November 5, 2025

Record last verified: 2025-11

Locations

US (1)