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

Not Applicable

Recruiting

• Conditions: Graft Failure; Poor Graft Function
• Interventions: Device: CD34+ Selected Donor Cell Boost; Procedure: Blood Stem Cell Infusion

• Registration Number: NCT06569082
• Lead Sponsor: NYU Langone Health

Brief Summary

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.

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 Timeline

• Study First Submitted: 2024-08-22
• Study First Submitted QC: 2024-08-22
• Study First Posted: 2024-08-23
• Study Start: 2024-10-31
• Status Verified: 2024-11
• Last Update Submitted: 2024-11-14
• Last Update Posted: 2024-11-18
• Primary Completion: 2034-08
• Study Completion: 2035-08

Recruitment & Eligibility

• Status: RECRUITING
• Sex: All
• Target Recruitment: 21

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 & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Individuals with Poor Graft Function or Failure Following Allogeneic HSCT	CD34+ Selected Donor Cell Boost	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.
Individuals with Poor Graft Function or Failure Following Allogeneic HSCT	Blood Stem Cell Infusion	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.

Primary Outcome Measures

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

Secondary Outcome Measures

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

Trial Locations

Locations (1)

NYU Langone Health; 🇺🇸 New York, New York, United States