Related Donors Donating Peripheral Blood Stem Cells (PBSC) to a Family MemberPhase 2NCT01696461

A Phase II Study Evaluating the Safety and Efficacy of Subcutaneous Plerixafor

A Phase 2 study testing Plerixafor in people with Related Donors Donating Peripheral Blood Stem Cells (PBSC) to a Family Member and Acute Myelogenous Leukemia, run by Center for International Blood and Marrow Transplant Research at 12 sites in United States.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record

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Trial summary

This is a Phase II, open-label, two strata, multicenter, prospective study of plerixafor-mobilized HLA-identical sibling allografts in recipients with hematological malignancies. This study will establish the safety and efficacy of subcutaneous plerixafor for this purpose. Read more about this study

Who is it for?: Ages 18 to 65 years
Conditions the study is for: Related Donors Donating Peripheral Blood Stem Cells (PBSC) to a Family Member, Acute Myelogenous Leukemia, Acute Lymphoblastic Leukemia
What is being tested?: Plerixafor
Type of treatment: Injection or infusion
Could I receive a placebo?: No, everyone receives an active treatment
How many people: 127 participants
Where: 12 sitesUnited States
Study dates: May 2013to Aug 2016
Phase: Phase 2
Are there results?: Yes, posted in the registrySummary in the doctors' view

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 18 to 65 years
All sexes
No healthy volunteers
Adult, Older adult

Who may be able to join

Donor:
Donor eligibility will be determined according to applicable federal, state and local regulations and institutional standards
18-65 years of age
6/6 HLA-matched sibling
Fulfill individual Transplant Center criteria to serve as a mobilized blood cell donor
Serum creatinine <2.0mg/dl

Show all 22Show fewer

Recipient:
18 to 65 years of age
6/6 HLA antigen matched sibling willing to donate PBSC for transplant
Fulfill individual Transplant Center Criteria for transplant
One of the following diagnoses:
Acute myelogenous leukemia (AML) in 1st remission or beyond with <5% marrow blasts and no circulating blasts. Marrow must be done within 30 days of the start of transplant conditioning regimen in alignment with other pre-transplant assessments.
Acute lymphoblastic leukemia (ALL) in 1st remission or beyond with <5% marrow blasts and no circulating blasts
Myelodysplastic syndrome, either intermediate-1,2, or high risk by International Prognostic Scoring System or transfusion dependent
Chronic myelogenous leukemia (CML) failing or intolerant to tyrosine kinase inhibitor based therapy
Non-Hodgkin's lymphoma (NHL) or Hodgkin's disease (HD) in 2nd or greater complete remission, partial remission, or in relapse (but with at least stable disease after most recent therapy)
Chronic lymphocytic leukemia (CLL), relapsing after at least one prior regimen, or in remission with 17p deletion
Serum creatinine must be <2.0mg/dl
Total bilirubin and aspartate aminotransferase (AST) <3x normal
Infectious disease marker (IDM) monitoring will be performed per institutional standards
Karnofsky performance status of 70% or greater.
Patients who have undergone a prior autologous transplantation are eligible for a reduced intensity transplant only

Who cannot join

Donor:
Donor unwilling or unable to give informed consent, or unable to comply with the protocol including required follow-up and testing
Donor already enrolled on another investigational agent study
Pregnant or breast feeding females, or females not willing or able to use adequate contraception if sexually active
Recipient:
Patient unwilling or unable to give informed consent, or unable to comply with the protocol including required follow-up and testing

Show all 10Show fewer

Patients with active, uncontrolled infection at the time of the transplant preparative regimen
Pregnant or breast feeding females, or females not willing or able to use adequate contraception if sexually active
Patients with a history of previous central nervous system (CNS) tumor involvement showing active symptoms or signs along with documented disease on lumbar puncture and MRI of the brain within 30 days of start of conditioning
A condition, which, in the opinion of the clinical investigator, would interfere with the evaluation of primary and secondary endpoints.

Trial locations and contacts

12 sites in 1 country.

City, postcode or hospital

12 sites in 1 country

United States12 sites

Emory University
Atlanta, Georgia
Massachusetts General Hospital
Boston, Massachusetts
University of Chicago
Chicago, Illinois
Cleveland Clinic
Cleveland, Ohio
Ohio State University
Columbus, Ohio
Duke University
Durham, North Carolina
Medical College of Wisconsin
Milwaukee, Wisconsin
University of Minnesota
Minneapolis, Minnesota
West Virginia University
Morgantown, West Virginia
Mayo Clinic
Rochester, Minnesota
Washington University
St Louis, Missouri
H. Lee Moffitt Cancer Center
Tampa, Florida

Other trials in United States

About this study

Official title: A Phase II Study Evaluating the Safety and Efficacy of Subcutaneous Plerixafor for the Mobilization and Transplantation of HLA-Matched Sibling Donor Hematopoietic Stem Cells in Recipients With Hematological Malignancies

Read the detailed description

The primary objective is to determine the proportion of donors whose cells can be successfully mobilized and collected with a sufficient CD34+ cell dose using plerixafor as the mobilizing agent, using an intention-to-treat analysis. Donor mobilization following plerixafor will be considered successful if ≥ 2.0x10e6 CD34+ cells/kg recipient weight are collected in no more than two leukapheresis collections.

All donors receiving plerixafor will be included in the analysis of the primary objective based on the intention-to-treat principle.Recipients will be classified into one of the two strata, myeloablative or reduced intensity, according to his/her conditioning regimen. The target enrollment is 64 donor/recipient pairs, 32 pairs per stratum.

Conditions: Related Donors Donating Peripheral Blood Stem Cells (PBSC) to a Family Member, Acute Myelogenous Leukemia, Acute Lymphoblastic Leukemia, Myelodysplastic Syndrome, Chronic Myelogenous Leukemia, Non-Hodgkin's Lymphoma
Being tested: Plerixafor
Medical subject headings: Leukemia, Myeloid, Acute, Precursor Cell Lymphoblastic Leukemia-Lymphoma, Myelodysplastic Syndromes, Leukemia, Myelogenous, Chronic, BCR-ABL Positive, Lymphoma, Non-Hodgkin, Hodgkin Disease, Leukemia, Lymphocytic, Chronic, B-Cell

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

PlerixaforDrug
Also called Mozobil, AMD3000

Study groups in NCT01696461
Study group	Role	What they receive
Related donors receiving plerixafor	Experimental	Collection of sufficient CD34+ cells using plerixafor as the mobilizing agent. Eligible donors determined according to institutional standards * 18-65 years of age * 6/6 HLA-matched sibling * Fulfill individual Transplant Center criteria to serve as a mobilized blood cell donor * Serum creatinine <1.5 x institution upper limit of normal (ULN) or estimated creatinine clearance (CLCR) >50 mL/min Treatment Description: * Receive subcutaneous plerixafor at 240 μg/kg and commence leukapheresis approximately 4 hours later. * Leukapheresis will be performed up to two consecutive days. The target CD34+ cell dose is > 4.0 x 106/kg with a minimum of > 2.0 x 106/kg. Receives: Drug: Plerixafor
Recipients, myeloablative regimen	No intervention	Patients undergoing conditioning under a myeloablative regimen Myeloablative (one of four general regimens): Busulfan (> 9 mg/kg po or iv total) with fludarabine Busulfan (> 9 mg/kg po or iv total) with cyclophosphamide Total body irradiation (> 1000 cGy) plus etoposide Total body irradiation (> 500 cGy) plus cyclophosphamide
Recipients, reduced intensity conditioning regimen.	No intervention	Patients undergoing conditioning using a reduced intensity conditioning regimen. Reduced Intensity (one of three general regimens): Busulfan (< 9 mg/kg po or iv total) plus fludarabine Melphalan (100-140 mg/m2 iv total) plus fludarabine Fludarabine plus cyclophosphamide (> 2000 mg/m2 total)

What is being measured

The primary outcome is the main question the study is designed to answer.

Percentage of Donors Whose Cells Were Successfully Mobilized and Collected With a Sufficient CD34+ Cell Dose Using Plerixafor as the Mobilizing Agent, Using an Intention-to-treat Analysis.
Donor mobilization following plerixafor was considered successful if ≥ 2.0x106 CD34+ cells/kg recipient weight was collected in no more than two leukapheresis collections.
Measured: donation

Secondary outcomes (14)

Incidence and Severity of Acute Toxicities
Incidence and severity of acute toxicities before and during apheresis experienced by donors receiving plerixafor. Acute toxicities are graded according to the NCI Common Terminology Criteria for Adverse Events, version 4.0. This outcome measure is descriptive. Grade of maximum toxicity across all time points is reported. Higher grades denote worse outcomes. 0 = None, 1 = Mild, 2 = Moderate, 3 = severe.
Measured: baseline, Day 1, Day 2, Day 3
Adverse Effects
Adverse effects experienced by donors receiving plerixafor up to one year post donation. Number of participants with a maximum MTC >0 reported at each individual time point. Adverse effects are graded according to the NCI Common Terminology Criteria for Adverse Events, version 4.0. This outcome measure is descriptive. Participants can experience adverse effects at more than one time point evaluated. Higher grades denote worse outcomes. 0 = None, 1 = Mild, 2 = Moderate, 3 = severe.
Measured: 30 minutes, 60 minutes, 120 minutes, 240 minutes, 1 month, 6 months, 12 months post donation for each subject
Incidence of and Kinetics of Neutrophil and Platelet Recovery After Transplantation of Hematopoietic Cells Mobilized With Plerixafor
Incidence of and kinetics of neutrophil and platelet recovery by day 100 in recipients after transplantation of hematopoietic cells mobilized with plerixafor.
Measured: Day +1 through neutrophil recovery or Day 21 (whichever is first)
T-cell (CD3+) and Myeloid (CD33+) Chimerism After Transplantation of Hematopoietic Cells Mobilized With Plerixafor
T-cell (CD3+) and myeloid (CD33+) chimerism in recipients after transplantation of hematopoietic cells mobilized with plerixafor. This outcome measure is descriptive.
Measured: Chimerism was evaluated at serial timepoints post HCT in patients in both RIC and MAC strata. Chimerism was assessed at Day +28, +100, +180, and +365
Primary Graft Failure After Transplantation of Hematopoietic Cells Mobilized With Plerixafor
Incidence of primary graft failure in recipients after transplantation of hematopoietic cells mobilized with plerixafor. This outcome measure is descriptive.
Measured: Day 28
Incidence of Acute Graft-versus-host Disease (GVHD)
Incidence of acute graft-versus-host disease (GVHD) in recipients after transplantation of hematopoietic cells mobilized with plerixafor
Measured: Day 100
Immune Reconstitution
Rate and quality of immune reconstitution as evidenced by peripheral blood immunophenotype after transplantation of hematopoietic cells mobilized with plerixafor.
Measured: Day 28, 100, 180, 365
Incidence of Cytomegalovirus (CMV) Reactivation After Transplantation With Cells Mobilized With Plerixafor.
Percentage of recipients with prior CMV infection whose CMV was reactivated after transplantation with cell mobilized with plerixafor
Measured: day 365
Treatment-related Mortality and Disease Relapse/Progression
Incidence of treatment-related mortality and disease relapse/progression in recipients after transplantation of hematopoietic cells mobilized with plerixafor
Measured: Day 180, 365
Progression-free and Overall Survival
Probability of progression-free and overall survival after transplantation of hematopoietic cells mobilized with plerixafor
Measured: Day 180, 365
Cellular Composition of Allografts
Cellular composition of allografts mobilized with plerixafor (stem/progenitor cells, T/B/ (Natural killer) NK-cells)
Measured: donation
Incidence of Chronic Graft-versus-host Disease (GVHD)
Incidence of chronic graft-versus-host disease (GVHD) in recipients after transplantation of hematopoietic cells mobilized with plerixafor
Measured: Day 365
Secondary Graft Failure After Transplantation of Hematopoietic Cells Mobilized With Plerixafor
Incidence of secondary graft failure in recipients after transplantation of hematopoietic cells mobilized with plerixafor. This outcome measure is descriptive.
Measured: Day 365
CD34+ Cell Count of Allografts
CD34+ cell count of allografts mobilized with plerixafor
Measured: donation

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

May 2013Aug 2016

First posted1 Oct 2012 (estimated)
Study startMay 2013
Primary completionAug 2016
Study completionAug 2016

Resources

Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
Publications (1)Papers the sponsor has linked to this study.
Other trials for Related Donors Donating Peripheral Blood Stem Cells (PBSC) to a Family MemberStudies of the same condition, open and under way.
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 2
Primary purpose: Treatment
Allocation: Non-randomised
Intervention model: Single group
Masking: None (open label)
Enrolment: 127 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT01696461
Sponsor's protocol ID: 09-PLEX
Status last verified: May 2023
First submitted: 24 Sep 2012
Last updated: 14 Sep 2023
Results posted: Yes, on ClinicalTrials.gov
Data monitoring committee: Yes

Publications

Chen YB, Le-Rademacher J, Brazauskas R, Kiefer DM, Hamadani M, DiPersio JF, Litzow MR, Craig M, Horwitz ME, Artz AS, McClune BL, Fernandez HF, Duong HK, Kobusingye H, Proue M, Drexler RJ, Horowitz MM, Shaw BE, Miller JP, Hosoba S, Waller EK, Devine SM. Plerixafor alone for the mobilization and transplantation of HLA-matched sibling donor hematopoietic stem cells. Blood Adv. 2019 Mar 26;3(6):875-883. doi: 10.1182/bloodadvances.2018027599.
PubMed 30890544

Study at a glance

From the registry record, last updated by the sponsor on 14 Sep 2023. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title: A Phase II Study Evaluating the Safety and Efficacy of Subcutaneous Plerixafor for the Mobilization and Transplantation of HLA-Matched Sibling Donor Hematopoietic Stem Cells in Recipients With Hematological Malignancies
ClinicalTrials.gov ID: NCT01696461
Acronym / sponsor ID: 09-PLEX
Conditions: Related Donors Donating Peripheral Blood Stem Cells (PBSC) to a Family Member, Acute Myelogenous Leukemia, Acute Lymphoblastic Leukemia, Myelodysplastic Syndrome, Chronic Myelogenous Leukemia, Non-Hodgkin's Lymphoma
Phase: Phase 2
Study type: Interventional
Recruitment status: Completed
Enrolment: 127 participants (actual)
Lead sponsor: Center for International Blood and Marrow Transplant ResearchResearch network
Collaborators: Genzyme, a Sanofi Company, Sanofi
Study start: May 2013
Primary completion: Aug 2016 (actual)
Study completion: Aug 2016 (actual)
First posted: 1 Oct 2012 (estimated)
Results: Posted 14 Sep 2023

Clinical summary

The research question as the sponsor registered it. This describes what the study plans to test, not what it found.

Detailed description

Study design

Primary purpose: Treatment
Allocation: Non-randomised
Intervention model: Single group
Masking: None (open label)
Number of arms: 3

Arm	Type	Receives
Related donors receiving plerixafor	Experimental	Drug: PlerixaforCollection of sufficient CD34+ cells using plerixafor as the mobilizing agent. Eligible donors determined according to institutional standards * 18-65 years of age * 6/6 HLA-matched sibling * Fulfill individual Transplant Center criteria to serve as a mobilized blood cell donor * Serum creatinine <1.5 x institution upper limit of normal (ULN) or estimated creatinine clearance (CLCR) >50 mL/min Treatment Description: * Receive subcutaneous plerixafor at 240 μg/kg and commence leukapheresis approximately 4 hours later. * Leukapheresis will be performed up to two consecutive days. The target CD34+ cell dose is > 4.0 x 106/kg with a minimum of > 2.0 x 106/kg.
Recipients, myeloablative regimen	No intervention	–Patients undergoing conditioning under a myeloablative regimen Myeloablative (one of four general regimens): Busulfan (> 9 mg/kg po or iv total) with fludarabine Busulfan (> 9 mg/kg po or iv total) with cyclophosphamide Total body irradiation (> 1000 cGy) plus etoposide Total body irradiation (> 500 cGy) plus cyclophosphamide
Recipients, reduced intensity conditioning regimen.	No intervention	–Patients undergoing conditioning using a reduced intensity conditioning regimen. Reduced Intensity (one of three general regimens): Busulfan (< 9 mg/kg po or iv total) plus fludarabine Melphalan (100-140 mg/m2 iv total) plus fludarabine Fludarabine plus cyclophosphamide (> 2000 mg/m2 total)

Who may qualify

Ages: 18 to 65 years
Sex: All
Healthy volunteers: Not accepted
Age groups: Adult, Older adult

Key inclusion criteria (22 listed)

Donor eligibility will be determined according to applicable federal, state and local regulations and institutional standards
18-65 years of age
6/6 HLA-matched sibling
Fulfill individual Transplant Center criteria to serve as a mobilized blood cell donor
Serum creatinine <2.0mg/dl

Key exclusion criteria (10 listed)

Donor unwilling or unable to give informed consent, or unable to comply with the protocol including required follow-up and testing
Donor already enrolled on another investigational agent study
Pregnant or breast feeding females, or females not willing or able to use adequate contraception if sexually active
Patient unwilling or unable to give informed consent, or unable to comply with the protocol including required follow-up and testing
Patients with active, uncontrolled infection at the time of the transplant preparative regimen

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
PlerixaforAlso: Mozobil, AMD3000	Drug	Related donors receiving plerixafor

Primary outcome

Percentage of Donors Whose Cells Were Successfully Mobilized and Collected With a Sufficient CD34+ Cell Dose Using Plerixafor as the Mobilizing Agent, Using an Intention-to-treat Analysis.
Time frame: donation
Donor mobilization following plerixafor was considered successful if ≥ 2.0x106 CD34+ cells/kg recipient weight was collected in no more than two leukapheresis collections.

14 secondary outcomes

Incidence and Severity of Acute Toxicities
Time frame: baseline, Day 1, Day 2, Day 3
Adverse Effects
Time frame: 30 minutes, 60 minutes, 120 minutes, 240 minutes, 1 month, 6 months, 12 months post donation for each subject
Incidence of and Kinetics of Neutrophil and Platelet Recovery After Transplantation of Hematopoietic Cells Mobilized With Plerixafor
Time frame: Day +1 through neutrophil recovery or Day 21 (whichever is first)
T-cell (CD3+) and Myeloid (CD33+) Chimerism After Transplantation of Hematopoietic Cells Mobilized With Plerixafor
Time frame: Chimerism was evaluated at serial timepoints post HCT in patients in both RIC and MAC strata. Chimerism was assessed at Day +28, +100, +180, and +365
Primary Graft Failure After Transplantation of Hematopoietic Cells Mobilized With Plerixafor
Time frame: Day 28
Incidence of Acute Graft-versus-host Disease (GVHD)
Time frame: Day 100
Immune Reconstitution
Time frame: Day 28, 100, 180, 365
Incidence of Cytomegalovirus (CMV) Reactivation After Transplantation With Cells Mobilized With Plerixafor.
Time frame: day 365
Treatment-related Mortality and Disease Relapse/Progression
Time frame: Day 180, 365
Progression-free and Overall Survival
Time frame: Day 180, 365
Cellular Composition of Allografts
Time frame: donation
Incidence of Chronic Graft-versus-host Disease (GVHD)
Time frame: Day 365
Secondary Graft Failure After Transplantation of Hematopoietic Cells Mobilized With Plerixafor
Time frame: Day 365
CD34+ Cell Count of Allografts
Time frame: donation

Results and safety

Results posted to the registry on 14 Sep 2023, as reported by the sponsor. Figures are copied from the record, not re-analysed. Full results on ClinicalTrials.gov.

Participant flow

Group	Started	Completed
Related Donors Receiving Plerixafor	64	64
Recipients, Reduced Intensity	33	33
Recipients Myeloablative Regimen	30	30

Primary outcome: Percentage of Donors Whose Cells Were Successfully Mobilized and Collected With a Sufficient CD34+ Cell Dose Using Plerixafor as the Mobilizing Agent, Using an Intention-to-treat Analysis.

Time frame: donation. Count of participants. Unit: Participants

Measure	Related Donors Receiving Plerixafor n = 64
Value	63

Population analysed: Recipients were not mobilized with plerixafor.

14 secondary outcomes are reported in the full results.

Adverse events

Other adverse events reported at 0% frequency or more

Group	Serious adverse events	Other adverse events	Deaths
Related Donors Receiving Plerixafor	1 of 64 (1.6%)	44 of 64 (68.8%)	Not reported

Limitations and caveats (from the sponsor): Although this was a prospective multicenter trial, conclusions are limited by the single arm design and small sample size.

Loading the posted results from ClinicalTrials.gov…

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 14 Sep 2023.
Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
Derived from this study: PubMed 30890544Chen YB, Le-Rademacher J, Brazauskas R, Kiefer DM, Hamadani M, DiPersio JF, Litzow MR, Craig M, Horwitz ME, Artz AS, McClune BL, Fernandez HF, Duong HK, Kobusingye H, Proue M, Drexler RJ, Horowitz MM, Shaw BE, Miller JP, Hosoba S, Waller EK, Devine SM. Plerixafor alone for the mobilization and transplantation of HLA-matched sibling donor hematopoietic stem cells. Blood Adv. 2019 Mar 26;3(6):875-883. doi: 10.1182/bloodadvances.2018027599.

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.

Steve Devine, MD principal investigator

NMDP/BeTheMatch

12 sites in 1 country: see every site and its status.

Questions about this trial

What is this clinical trial studying?

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 22 inclusion and 10 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 14 Sep 2023. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 12 sites 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 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

Yes. Results are posted in the registry (first posted 14 Sep 2023). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

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?

Percentage of Donors Whose Cells Were Successfully Mobilized and Collected With a Sufficient CD34+ Cell Dose Using Plerixafor as the Mobilizing Agent, Using an Intention-to-treat Analysis. (time frame: donation). 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 NCT01696461, 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 14 Sep 2023; AzTrials last refreshed its copy on 29 Oct 2026.