CD34+ Selected Stem Cell for Poor Graft Function or Graft Failure

CD34+ Selected Donor Cell Boost for Management of Poor Graft Function or Primary or Secondary Graft Failure Following Allogeneic Hematopoietic Stem Cell Transplantation

The proposed trial is a single arm, non-randomized, single center pilot study utilizing CliniMACS CD34 Reagent System for patients following allogeneic hematopoietic stem cell transplant (HSCT) requiring treatment of graft dysfunction or failure.

Study Overview

• Status: Recruiting
• Conditions: Graft Failure; Poor Graft Function
• Intervention / Treatment: Device: CD34+ Selected Donor Cell Boost; Procedure: Blood Stem Cell Infusion

Detailed Description

The primary objective of the study is to estimate the incidence of engraftment with sustained recovery of blood counts, including, absolute neutrophil cell (ANC) and platelet engraftment.

• ANC engraftment is defined as ANC of ≥ 0.5 × 10^9/L for three consecutive laboratory values obtained on different days. Date of ANC recovery is the date of the first of three consecutive laboratory values where the ANC is ≥ 0.5 × 10^9/L.
• Platelet engraftment should reflect no platelet transfusions administered for seven consecutive days. Report the date of the first of three consecutive laboratory values ≥ 20 × 10^9/L obtained on different days.

The secondary objective of the study is to estimate the incidence of Graft-versus-host disease (GvHD) and side effects that can be attributed to the CliniMACS CD34 Selected Cellular Product.

• Study Type: Interventional
• Enrollment (Estimated): 21
• Phase: Not Applicable

Contacts and Locations

• Study Contact: Name: Kiselle Mangalindan; Phone Number: 646-501-2973; Email: KiselleAnne.Mangalindan@nyulangone.org
• Study Contact Backup: Name: Kelsey Stocker; Phone Number: 646-501-4848; Email: Kelsey.Stocker@nyulangone.org

Study Locations

• United States
  • New York
    • New York, New York, United States, 10016
      • Recruiting
      • NYU Langone Health

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Adult; Older Adult
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

• Recipient of allogeneic transplantation, adult ≥18 years, from any type of donor including matched related, matched unrelated, mismatched related or mismatched unrelated or haploidentical donor transplant.

• Documented evidence of graft dysfunction or failure (a-c):

  1. Primary graft Failure: Graft failure is defined as failure to achieve neutrophil engraftment by day +28 or lack of donor chimerism > 50% by day 45 not due to the underlying malignancy;
  2. Poor graft function is defined by at least 2 of the following 3 criteria: Hemoglobin < 8 g/dL, ANC < 0.5x109/L, and platelets < 20x109/L. The cytopenia must be unexplained (such as by disease relapse) and unresponsive to hematopoietic growth factors and must last at least 4 weeks;
  3. Secondary graft failure is defined as poor graft function associated with donor chimerism < 5% after initial engraftment
• Transplanted donor availability
• Negative pregnancy test within seven (7) days of product infusion for women of childbearing potential.

Exclusion Criteria:

• Graft failure due to disease relapse or evidence of disease relapse or progression
• Donor unavailable or unable to collect peripheral HPC by apheresis
• Responsive to conventional measures (such as, hematopoietic growth factor)
• Allergic reaction to murine proteins or iron dextran
• Women of childbearing potential with positive serum HCG

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm

Intervention / Treatment

Experimental: Individuals with Poor Graft Function or Failure Following Allogeneic HSCT; The recipient will undergo evaluations, then undergo a single infusion of CD34 selected HSCT from the original donor. No additional GvHD prophylaxis will be administered. Patients who develop GvHD will receive the standard of care based on the discretion of the treating physician . Patients will receive supportive care per institutional standards and at the discretion of the treating physician.

Device: CD34+ Selected Donor Cell Boost; The CliniMACS CD34 Reagent System is used to separate CD34 cells from the remaining stem cell product, using a peripheral blood stem cell sample provided by the original donor. These CD34 cells are then infused into the recipient following selection with the intent to restore function of the blood forming cells.; Other Names: CliniMACS CD34 Reagent System; Procedure: Blood Stem Cell Infusion; The selected CD34 cells separated by the CliniMACS CD34 Reagent System are infused into the recipient.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Number of Participants with Absolute Neutrophil Cell (ANC) Engraftment	ANC engraftment is defined as ANC of ≥ 0.5 × 10^9/L for three consecutive laboratory values obtained on different days.	Day 100 Post-Procedure
Number of Participants with Platelet Engraftment	Platelet engraftment is defined as no platelet transfusions administered for seven consecutive days.	Day 100 Post-Procedure

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Number of Participants with Grade II-IV Acute GVHD	The diagnosis of acute GvHD is based on clinical and pathological evaluation by the principal investigator in collaboration with the treating physician.	Day 100 Post-Procedure
Number of Participants with Moderate to Severe Chronic GVHD	The diagnosis of chronic GvHD is based on clinical and pathological evaluation by the principal investigator in collaboration with the treating physician.	Day 365 Post-Procedure

Collaborators and Investigators

• Sponsor: NYU Langone Health
• Investigators: Principal Investigator: Jingmei Hsu, NYU Langone Health

Study record dates

Study Major Dates

• Study Start (Actual): October 31, 2024
• Primary Completion (Estimated): August 1, 2034
• Study Completion (Estimated): August 1, 2035

Study Registration Dates

• First Submitted: August 22, 2024
• First Submitted That Met QC Criteria: August 22, 2024
• First Posted (Actual): August 23, 2024

Study Record Updates

• Last Update Posted (Estimated): September 29, 2025
• Last Update Submitted That Met QC Criteria: September 23, 2025
• Last Verified: September 1, 2025

More Information

Terms related to this study

• Other Study ID Numbers: 23-00885

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: YES
• IPD Plan Description: All of the individual participant data collected during the trial, after deidentification, will be shared upon reasonable request provided the investigator whose proposed use of the data has been approved by an independent review committee ("learned intermediary") identified for this purpose executes a data use agreement with NYU Langone Health. Requests may be directed to: Jingmei.hsu@nyulangone.org. The protocol and statistical analysis plan will be made available on Clinicaltrials.gov only as required by federal regulation or as a condition of awards and agreements supporting the research.
• IPD Sharing Time Frame: No time limit.
• IPD Sharing Access Criteria: Requests should be directed to Jingmei.hsu@nyulangone.org. To gain access, data requestors will need to sign a data access agreement.
• IPD Sharing Supporting Information Type: STUDY_PROTOCOL; SAP; ICF; CSR

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: Yes
• product manufactured in and exported from the U.S.: No