Phase 2
Completed N=127
A Phase II Study Evaluating the Safety and Efficacy of Subcutaneous Plerixafor
Related Donors Donating Peripheral Blood Stem Cells (PBSC) to a Family Member · leukemia · Acute Lymphoblastic Leukemia · Myelodysplastic Syndromes
Source: ClinicalTrials.gov NCT01696461 ↗
Enrolled (actual)
127
Serious AEs
1.6%
Results posted
Sep 2023
Primary outcomePrimary: 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. — 63 Participants
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.
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY 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. |
63 | — |
| SECONDARY Incidence and Severity of Acute Toxicities |
19; 34; 10; 1 | — |
| SECONDARY Adverse Effects |
23; 28; 27; 24; 22; 7 | — |
| SECONDARY Incidence of and Kinetics of Neutrophil and Platelet Recovery After Transplantation of Hematopoietic Cells Mobilized With Plerixafor |
100; 100; 100; 97 | — |
| SECONDARY T-cell (CD3+) and Myeloid (CD33+) Chimerism After Transplantation of Hematopoietic Cells Mobilized With Plerixafor |
100; 100; 92; 80; 99; 100 | — |
| SECONDARY Primary Graft Failure After Transplantation of Hematopoietic Cells Mobilized With Plerixafor |
0; 0 | — |
| SECONDARY Incidence of Acute Graft-versus-host Disease (GVHD) |
53; 18; 17; 3 | — |
| SECONDARY Immune Reconstitution |
589; 478; 363; 214; 197; 127 | — |
| SECONDARY Incidence of Cytomegalovirus (CMV) Reactivation After Transplantation With Cells Mobilized With Plerixafor. |
3; 6 | — |
| SECONDARY Treatment-related Mortality and Disease Relapse/Progression |
7; 3; 17; 6; 30; 28 | — |
| SECONDARY Progression-free and Overall Survival |
63; 69; 53; 64; 87; 79 | — |
| SECONDARY Cellular Composition of Allografts |
5.96; 3.57; 2.18; 1.47; 0.38 | — |
| SECONDARY Incidence of Chronic Graft-versus-host Disease (GVHD) |
52; 39 | — |
| SECONDARY Secondary Graft Failure After Transplantation of Hematopoietic Cells Mobilized With Plerixafor |
0; 0 | — |
| SECONDARY CD34+ Cell Count of Allografts |
4.68 | — |
Eligibility Criteria
Inclusion Criteria
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
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
Exclusion Criteria
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
- 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.
Data sourced from ClinicalTrials.gov (NCT01696461). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication. Informational only — not medical advice.