Hematologic Disorders
Conditions
Keywords
Stem Cell Transplant, Umbilical Cord Blood Transplant, Haploidentical CD34+ Selected Cells
Brief summary
This is a study to evaluate the safety and efficacy of Miltenyi CliniMACS® CD34 Reagent System to promote engraftment of haploidentical CD34+ selected cells combined with single unit umbilical cord blood transplant for treatment of high-risk hematologic disorders.
Detailed description
In this clinical protocol, the CliniMACS® CD34 Reagent System will be used for processing hematopoietic progenitor cells collected by apheresis (HPC, Apheresis) from an allogeneic, HLA-haploidentical, related donor to obtain a CD34+ cell-enriched population for hematopoietic reconstitution. The haploidentical donor will be mobilized by G-CSF and undergo one apheresis to collect CD34+ stem cells. The products will be cryopreserved until the time of transplantation. Recipients with hematologic disorders who require transplant will receive a standard conditioning regimen and will receive an allograft on day 0 containing donor CD34+ cells that have been positively selected and T-cell depleted following G-CSF mobilization combined with a single UCB unit.
Interventions
DEVICEMiltenyi CliniMACS® CD34 Reagent System
Miltenyi CliniMACS® CD34 Reagent System will be used to prepare CD34+ enriched/T-cell depleted cells from haploidentical mobilized peripheral blood.
Sponsors
University of Colorado, Denver
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Intervention model description
This is a phase 1/phase 2 study.
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
Inclusion criteria
- Recipient * Ages 18-80 years inclusive * Diagnosed with high risk hematologic disorders warranting stem cell transplant per institutional standard of care * Lack HLA-identical related donor * Availability of at least one HLA- haploidentical (i.e. =\> 5/10 and \<= 8/10 HLA match) related donor (HLA-A, B, C, DR, and DQ loci) who is available to donate CD34+ cells. * Availability of at least one 4/6 HLA-matched (HLA-A, B, and DR loci) cord blood unit from the National Marrow Donor Program (NMDP). The cord blood unit must contain a minimum TNC (prior to thawing) of at least 2x107 cells per kilogram of recipient body weight * Ability to comprehend the nature of the treatment 6.2.
Exclusion criteria
- Recipient (any of the following) * HLA identical (6/6) related donor available and readily accessible at time of transplantation evaluation * Any patient not meeting institutional standard guidelines for transplant eligibility
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| The Number of Patients With Successful Engraftment | 42 days | To determine the rate of successful engraftment of patients who received haploidentical CD34+ selected cells combined with single unit umbilical cord blood transplant. Failure defined as failure of Miltenyi CliniMACS® CD34 Reagent System to select CD34+ cells, and/or failure to engraft by day 42. |
Countries
United States
Participant flow
Participants by arm
| Arm | Count |
|---|---|
| Miltenyi CliniMACS® CD34 Reagent System The haploidentical donor will be mobilized by G-CSF and undergo one apheresis to collect CD34+ selected stem cell product after Miltenyi CliniMACS® CD34+ selection. The products will be cryopreserved until the time of transplantation.
Recipients will receive a standard conditioning regimen. After the conditioning regimen, the subjects will receive an allograft on day 0 containing donor CD34+ cells that have been positively selected and T-cell depleted following G-CSF mobilization combined with a single UCB unit. UCB unit will not be manipulated, and will be prepared and infused separately following standard of care procedure.
Miltenyi CliniMACS® CD34 Reagent System: Miltenyi CliniMACS® CD34 Reagent System will be used to prepare CD34+ enriched/T-cell depleted cells from haploidentical mobilized peripheral blood. | 150 |
| Total | 150 |
Baseline characteristics
| Characteristic | Miltenyi CliniMACS® CD34 Reagent System |
|---|---|
| Age, Categorical <=18 years | 0 Participants |
| Age, Categorical >=65 years | 25 Participants |
| Age, Categorical Between 18 and 65 years | 125 Participants |
| Ethnicity (NIH/OMB) Hispanic or Latino | 20 Participants |
| Ethnicity (NIH/OMB) Not Hispanic or Latino | 126 Participants |
| Ethnicity (NIH/OMB) Unknown or Not Reported | 4 Participants |
| Sex: Female, Male Female | 57 Participants |
| Sex: Female, Male Male | 93 Participants |
Adverse events
| Event type | EG000 affected / at risk |
|---|---|
| deaths Total, all-cause mortality | 4 / 150 |
| other Total, other adverse events | 0 / 150 |
| serious Total, serious adverse events | 2 / 150 |
Outcome results
Primary
The Number of Patients With Successful Engraftment
To determine the rate of successful engraftment of patients who received haploidentical CD34+ selected cells combined with single unit umbilical cord blood transplant. Failure defined as failure of Miltenyi CliniMACS® CD34 Reagent System to select CD34+ cells, and/or failure to engraft by day 42.
Time frame: 42 days
Population: Participants who were eligible, received cell product, and were alive at D42.
| Arm | Measure | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|
| Miltenyi CliniMACS® CD34 Reagent System | The Number of Patients With Successful Engraftment | 144 Participants |