Trial Outcomes & Findings for Haploidentical CD34+ Selected Cells Combined With Single Unit Umbilical Cord Blood Transplant for Treatment of High-risk Hematologic Disorders (NCT NCT03093844)

Last Updated: 2025-08-15

Results Overview

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.

Recruitment status

COMPLETED

Study phase

Target enrollment

150 participants

Primary outcome timeframe

42 days

Results posted on

2025-08-15

Participant Flow

Participant milestones

Participant milestones
Measure	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.
Overall Study STARTED	150
Overall Study COMPLETED	150
Overall Study NOT COMPLETED	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Haploidentical CD34+ Selected Cells Combined With Single Unit Umbilical Cord Blood Transplant for Treatment of High-risk Hematologic Disorders

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Miltenyi CliniMACS® CD34 Reagent System n=150 Participants 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.
Age, Categorical <=18 years	0 Participants n=99 Participants
Age, Categorical Between 18 and 65 years	125 Participants n=99 Participants
Age, Categorical >=65 years	25 Participants n=99 Participants
Sex: Female, Male Female	57 Participants n=99 Participants
Sex: Female, Male Male	93 Participants n=99 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	20 Participants n=99 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	126 Participants n=99 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	4 Participants n=99 Participants

PRIMARY outcome

Timeframe: 42 days

Population: Participants who were eligible, received cell product, and were alive at D42.

Outcome measures

Outcome measures
Measure	Miltenyi CliniMACS® CD34 Reagent System n=146 Participants 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.
The Number of Patients With Successful Engraftment	144 Participants

Adverse Events

Miltenyi CliniMACS® CD34 Reagent System

Serious events: 2 serious events

Other events: 0 other events

Deaths: 4 deaths

Serious adverse events

Serious adverse events
Measure	Miltenyi CliniMACS® CD34 Reagent System n=150 participants at risk 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.
General disorders General disorders and administration site conditions - Other, specify	1.3% 2/150 • Number of events 2 • Until engraftment (approximately 42 days).

Other adverse events

Adverse event data not reported

Additional Information

Dr. Jonathan Gutman

University of Colorado

Phone: 303-724-9562

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place