MyelomaPhase 1NCT01526096
Stem Cell Transplantation for Patients With Multiple Myeloma
A Phase 1 study testing G-CSF, Plerixafor and Apheresis in people with Myeloma, run by University of Chicago at 1 site in United States.
Trial 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). Read more about this study
- Who is it for?
- Ages 21 to 70 years
- Conditions the study is for
- Myeloma
- What is being tested?
- G-CSF, Plerixafor and Apheresis
- Type of treatment
- Injection or infusion, Medical device, Surgery or procedure
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 30 participants
- Where
- 1 siteUnited States
- Study dates
- 12 Jul 2011to 28 Aug 2024
- Phase
- Phase 1
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 21 to 70 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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.
Show all 10Show fewer
- 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.
Who cannot join
- 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.
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Pilot Study T Cell Depletion in the Setting of Autologous Stem Cell Transplantation for Patients With Multiple Myeloma
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
- Medical subject headings
- Neoplasms, Plasma Cell
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
G-CSFDrug
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
PlerixaforDrug
Plerixafor (self-administered shot)prior to the beginning of the stem cell collection. Up to 4 doses of plerixafor may be given.
ApheresisProcedure or surgery
Stem cell collection begins on day 5 and can last up to 3 days depending on the number collected.
MelphalanDrug
Also called Alkeran
Melphalan chemotherapy 100mg/m2 for 2 days after your admission into the hospital for your ASCT procedure.
Stem cell re-infusionProcedure or surgery
Stem cells are thawed and reinfused back into the body via a catheter in the vein.
BasiliximabDrug
Also called Simulect
Basiliximab (20mg) given by IV infusion (through the vein) 20-30 minutes the day after ASCT.
CliniMACS CD25 microbeads and cell sorterDevice
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.
| Study group | Role | What they receive |
|---|---|---|
| Standard ASCT (Grp 1) | Active comparator | Standard autologous stem cell transplantation (ASCT) Receives: 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 Receives: 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. Receives: Drug: G-CSF; Drug: Plerixafor; Procedure: Apheresis; Drug: Melphalan; Procedure: Stem cell re-infusion; Device: CliniMACS CD25 microbeads and cell sorter |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: 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.
Measured: 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.
Measured: 180 days
Secondary outcomes (2)
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.
Measured: 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
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.
Measured: 100 days
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted3 Feb 2012 (estimated)
- Study start12 Jul 2011
- Primary completion28 Aug 2024
- Study completion28 Aug 2024
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for MyelomaStudies of the same condition, open and under way.
- More trials run by University of ChicagoThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 30 participants (actual)
Sponsor and investigators
- Lead sponsor
- University of Chicago
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Michael Bishop, MD, Principal investigator, University of Chicago
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01526096
- Sponsor's protocol ID
- 10-551-B
- Status last verified
- Nov 2025
- First submitted
- 10 Aug 2011
- Last updated
- 5 Nov 2025
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- Yes
Publications
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.
PubMed 31940591
Study at a glance
From the registry record, last updated by the sponsor on 5 Nov 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Pilot Study T Cell Depletion in the Setting of Autologous Stem Cell Transplantation for Patients With Multiple Myeloma
- ClinicalTrials.gov ID
- NCT01526096
- Acronym / sponsor ID
- 10-551-B
- Conditions
- Myeloma
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 30 participants (actual)
- Lead sponsor
- University of ChicagoUniversity, hospital or other
- Study start
- 12 Jul 2011 (actual)
- Primary completion
- 28 Aug 2024 (actual)
- Study completion
- 28 Aug 2024 (actual)
- First posted
- 3 Feb 2012 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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).
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| Standard ASCT (Grp 1) | Active comparator | Drug: G-CSF, Drug: Plerixafor, Procedure: Apheresis, Drug: Melphalan, Procedure: Stem cell re-infusionStandard autologous stem cell transplantation (ASCT) |
| Depletion of T-cells after ASCT (Grp 2) | Experimental | Drug: G-CSF, Drug: Plerixafor, Procedure: Apheresis, Drug: Melphalan, Procedure: Stem cell re-infusion, Drug: BasiliximabStandard 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) | Experimental | Drug: G-CSF, Drug: Plerixafor, Procedure: Apheresis, Drug: Melphalan, Procedure: Stem cell re-infusion, Device: CliniMACS CD25 microbeads and cell sorterBlood 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. |
Who may qualify
- Ages
- 21 to 70 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (10 listed)
- 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.
Key exclusion criteria (4 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| G-CSFG-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 | Drug | Depletion of T-cells after ASCT (Grp 2), Depletion of T-cells before ASCT(Grp 3), Standard ASCT (Grp 1) |
| PlerixaforPlerixafor (self-administered shot)prior to the beginning of the stem cell collection. Up to 4 doses of plerixafor may be given. | Drug | Depletion of T-cells after ASCT (Grp 2), Depletion of T-cells before ASCT(Grp 3), Standard ASCT (Grp 1) |
| ApheresisStem cell collection begins on day 5 and can last up to 3 days depending on the number collected. | Procedure or surgery | Depletion of T-cells after ASCT (Grp 2), Depletion of T-cells before ASCT(Grp 3), Standard ASCT (Grp 1) |
| MelphalanAlso: AlkeranMelphalan chemotherapy 100mg/m2 for 2 days after your admission into the hospital for your ASCT procedure. | Drug | Depletion of T-cells after ASCT (Grp 2), Depletion of T-cells before ASCT(Grp 3), Standard ASCT (Grp 1) |
| Stem cell re-infusionStem cells are thawed and reinfused back into the body via a catheter in the vein. | Procedure or surgery | Depletion of T-cells after ASCT (Grp 2), Depletion of T-cells before ASCT(Grp 3), Standard ASCT (Grp 1) |
| BasiliximabAlso: SimulectBasiliximab (20mg) given by IV infusion (through the vein) 20-30 minutes the day after ASCT. | Drug | Depletion of T-cells after ASCT (Grp 2) |
| CliniMACS CD25 microbeads and cell sorterThe 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. | Device | Depletion of T-cells before ASCT(Grp 3) |
Primary outcome
Purity of ex vivo depleted regulatory T cells prior to autologous stem cell transplant (arm 3 only)
Time frame: 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
Time frame: 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
Time frame: 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.
2 secondary outcomes
Kinetics of recovery of peripheral blood cellular elements
Time frame: 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
Time frame: 100 days
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 5 Nov 2025.
- Derived from this study: PubMed 31940591Derman 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.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Michael Bishop, MD principal investigator
University of Chicago
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing G-CSF, Plerixafor and Apheresis in people with Myeloma, run by University of Chicago at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 10 inclusion and 4 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 5 Nov 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Purity of ex vivo depleted regulatory T cells prior to autologous stem cell transplant (arm 3 only) (time frame: 1-3 days). The study lists 3 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT01526096, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 5 Nov 2025.