Trial Outcomes & Findings for Unrelated Umbilical Cord Blood (UBC)Transplantation (NCT NCT01768845)

NCT ID: NCT01768845

Last Updated: 2021-06-14

Results Overview

Defined as neutrophil recovery associated with donor engraftment within the first 60 days of transplant

Recruitment status

COMPLETED

Study phase

Target enrollment

34 participants

Primary outcome timeframe

60 days

Results posted on

2021-06-14

Participant Flow

Participant milestones

Participant milestones
Measure	Transplant After a preparative regimen the patient will receive an infusion of one or two umbilical cord blood unit(s) (UBC). The UBC unit(s) will be thawed according to methods of Rubinstein et al. If two products are used, they will be administered sequentially on the same day 1-6 hours apart. Tacrolimus and mycophenolate mofetil (MMF) will be used for graft versus host disease (GVHD) prophylaxis. On day +30, +60, +100, +180, and +365 the chimeric status of patients will be interpreted by VNTR analysis. Immune reconstitution (Digeorge Panel) will also be checked at these time points. umbilical cord blood (UCB): Infusion will occur after preparative regimen in one or two UCB unit(s). If two products are used, they will be administered sequentially on the same day 1-6 hours apart. Tacrolimus and mycophenolate mofetil (MMF) will be used for GVHD prophylaxis.
Overall Study STARTED	34
Overall Study COMPLETED	11
Overall Study NOT COMPLETED	23

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Unrelated Umbilical Cord Blood (UBC)Transplantation

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Transplant n=34 Participants After a preparative regimen the patient will receive an infusion of one or two umbilical cord blood unit(s) (UBC). The UBC unit(s) will be thawed according to methods of Rubinstein et al. If two products are used, they will be administered sequentially on the same day 1-6 hours apart. Tacrolimus and mycophenolate mofetil (MMF) will be used for GVHD prophylaxis. On day +30, +60, +100, +180, and +365 the chimeric status of patients will be interpreted by variable number tandem repeat (VNTR) analysis. Immune reconstitution (Digeorge Panel) will also be checked at these time points. umbilical cord blood (UCB): Infusion will occur after preparative regimen in one or two UCB unit(s). If two products are used, they will be administered sequentially on the same day 1-6 hours apart. Tacrolimus and mycophenolate mofetil (MMF) will be used for GVHD prophylaxis.
Age, Categorical <=18 years	0 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	33 Participants n=5 Participants
Age, Categorical >=65 years	1 Participants n=5 Participants
Sex: Female, Male Female	16 Participants n=5 Participants
Sex: Female, Male Male	18 Participants n=5 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	0 Participants n=5 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	34 Participants n=5 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=5 Participants
Race (NIH/OMB) Asian	0 Participants n=5 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=5 Participants
Race (NIH/OMB) Black or African American	2 Participants n=5 Participants
Race (NIH/OMB) White	32 Participants n=5 Participants
Race (NIH/OMB) More than one race	0 Participants n=5 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants

PRIMARY outcome

Timeframe: 60 days

Defined as neutrophil recovery associated with donor engraftment within the first 60 days of transplant

Outcome measures

Outcome measures
Measure	Transplant n=34 Participants After a preparative regimen the patient will receive an infusion of one or two umbilical cord blood unit(s) (UBC). The UBC unit(s) will be thawed according to methods of Rubinstein et al. If two products are used, they will be administered sequentially on the same day 1-6 hours apart. Tacrolimus and mycophenolate mofetil (MMF) will be used for GVHD prophylaxis. On day +30, +60, +100, +180, and +365 the chimeric status of patients will be interpreted by VNTR analysis. Immune reconstitution (Digeorge Panel) will also be checked at these time points. umbilical cord blood (UCB): Infusion will occur after preparative regimen in one or two UCB unit(s). If two products are used, they will be administered sequentially on the same day 1-6 hours apart. Tacrolimus and mycophenolate mofetil (MMF) will be used for GVHD prophylaxis.
Number of Participants With Engraftment	29 Participants

SECONDARY outcome

Timeframe: 180 days

Number of Participants with Overall survival at day 180 transplant.

Outcome measures

Outcome measures
Measure	Transplant n=34 Participants After a preparative regimen the patient will receive an infusion of one or two umbilical cord blood unit(s) (UBC). The UBC unit(s) will be thawed according to methods of Rubinstein et al. If two products are used, they will be administered sequentially on the same day 1-6 hours apart. Tacrolimus and mycophenolate mofetil (MMF) will be used for GVHD prophylaxis. On day +30, +60, +100, +180, and +365 the chimeric status of patients will be interpreted by VNTR analysis. Immune reconstitution (Digeorge Panel) will also be checked at these time points. umbilical cord blood (UCB): Infusion will occur after preparative regimen in one or two UCB unit(s). If two products are used, they will be administered sequentially on the same day 1-6 hours apart. Tacrolimus and mycophenolate mofetil (MMF) will be used for GVHD prophylaxis.
Overall Survival at Day 180 Post-transplant	18 Participants

Adverse Events

Transplant

Serious events: 1 serious events

Other events: 0 other events

Deaths: 21 deaths

Serious adverse events

Serious adverse events
Measure	Transplant n=34 participants at risk After a preparative regimen the patient will receive an infusion of one or two umbilical cord blood unit(s) (UBC). The UBC unit(s) will be thawed according to methods of Rubinstein et al. If two products are used, they will be administered sequentially on the same day 1-6 hours apart. Tacrolimus and mycophenolate mofetil (MMF) will be used for GVHD prophylaxis. On day +30, +60, +100, +180, and +365 the chimeric status of patients will be interpreted by VNTR analysis. Immune reconstitution (Digeorge Panel) will also be checked at these time points. umbilical cord blood (UCB): Infusion will occur after preparative regimen in one or two UCB unit(s). If two products are used, they will be administered sequentially on the same day 1-6 hours apart. Tacrolimus and mycophenolate mofetil (MMF) will be used for GVHD prophylaxis.
Blood and lymphatic system disorders Infection	2.9% 1/34 • Number of events 1 • 6 months Systematic assessment of adverse events was completed through that participants all being followed per routine schedule for transplant patients with regular examinations by their care provider with adverse event discovery per standard assessment.

Other adverse events

Adverse event data not reported

Additional Information

Michael Craig, MD

West Virginia Universtiy

Phone: 304-293-4980

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place