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

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ClinicalTrials.gov Identifier: NCT03093844
Recruitment Status : Recruiting
First Posted : March 28, 2017
Last Update Posted : May 1, 2018
Sponsor:
Information provided by (Responsible Party):
University of Colorado, Denver

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.

Condition or disease Intervention/treatment Phase
Hematologic Disorders Device: Miltenyi CliniMACS® CD34 Reagent System Not Applicable

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.

Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 50 participants
Intervention Model: Single Group Assignment
Intervention Model Description: This is a phase 1/phase 2 study.
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Safety and Efficacy of Miltenyi CliniMACS® CD34 Reagent System for Transplant Protocol Utilizing Haploidentical CD34+ Selected Cells Combined With Single Unit Umbilical Cord Blood Transplant for Treatment of High-risk Hematologic Disorders
Actual Study Start Date : October 12, 2017
Estimated Primary Completion Date : February 10, 2022
Estimated Study Completion Date : February 10, 2022

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Blood Disorders

Arm Intervention/treatment
Experimental: 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.

Device: 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.




Primary Outcome Measures :
  1. The number of patients with treatment-related serious adverse event rate (TRSAE) [ Time Frame: 42 days ]
    Safety will be monitored continuously with a stopping rule for toxicity based on the treatment-related serious adverse event rate (TRSAE), defined by failure of Miltenyi CliniMACS® CD34 Reagent System to select CD34+ cells, and/or failure to engraft that is higher than historical control (<10% for hematologic malignancies and 34% for severe aplastic anemia).

  2. The number of patients with successful engraftment [ Time Frame: 42 days ]
    Failure defined as failure of Miltenyi CliniMACS® CD34 Reagent System to select CD34+ cells, and/or failure to engraft by day 22 for patients with hematologic malignancies, and by day 42 for patients with severe aplastic anemia. Assuming a sample size of 50, if there is an engraftment success rate of 75% (or a failure rate of 25%), there would be a resulting 95% confidence interval of 63% to 87% (that is a width of 12% on either side of 75% successful engraftments



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Ages Eligible for Study:   18 Years to 80 Years   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

Recipient Eligibility

  • Ages 18-80 years inclusive
  • Diagnosed with high risk hematologic disorders warranting stem cell transplant pert institutional standard of care
  • Lack HLA-identical related donor
  • Availability of at least one HLA- haploidentical (i.e. greater than or equal to 5/10 and less than or equal to 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

Exclusion Criteria:

  • 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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03093844


Contacts
Contact: Constance Gorden 303-724-8649 CONSTANCE.GORDEN@UCDENVER.EDU

Locations
United States, Colorado
University of Colorado Recruiting
Aurora, Colorado, United States, 80045
Contact: Constance Gorden    303-724-8649    CONSTANCE.GORDEN@UCDENVER.EDU   
Principal Investigator: Enkhtsetseg Purev, MD, PhD         
Sponsors and Collaborators
University of Colorado, Denver
Investigators
Principal Investigator: Enkhtsetseg Purev, MD, PhD University of Colorado, Denver

Publications:
Responsible Party: University of Colorado, Denver
ClinicalTrials.gov Identifier: NCT03093844     History of Changes
Other Study ID Numbers: 16-1672.cc
First Posted: March 28, 2017    Key Record Dates
Last Update Posted: May 1, 2018
Last Verified: April 2018
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: Yes
Device Product Not Approved or Cleared by U.S. FDA: No
Pediatric Postmarket Surveillance of a Device Product: No

Keywords provided by University of Colorado, Denver:
Stem Cell Transplant
Umbilical Cord Blood Transplant
Haploidentical CD34+ Selected Cells

Additional relevant MeSH terms:
Disease
Hematologic Diseases
Pathologic Processes