Treatment Use of the CliniMACS® CD34 TCR Alpha/Beta for Allogenic Transplant (Expanded Access Use)

October 11, 2025 updated by: Victor Aquino

Treatment Use of the CliniMACS® CD34 TCR Alpha/Beta to Prepare Cells for an Unlabeled Indication Using an HLA-Compatible Related or Unrelated Donor for Allogenic Transplant

The protocol is designed to provide access to patients to the Clinimacs® CD34 TCRαβ system to prepare cells for an unlabeled indication using an HLA-compatible related or unrelated donor for allogenic transplant.

Study Overview

Status

Available

Conditions

Intervention / Treatment

Detailed Description

Allogeneic hematopoietic stem cell transplant (HSCT) is the only known curative therapy for a variety of non-malignant and malignant disorders1-4. Only about 25% of patients have an HLA-matched sibling and in about 16-75% (depending on ethnicity), a 10/10 HLA-matched unrelated donor can be identified. Engraftment failure is a dynamic complex process that may occur with this product due to unbalanced depletion of T-cells between recipient and donor, not only due to low number of CD34 cells. The use of mis-matched or haploidentical donors is associated with a higher risk of post-transplant mortality due to the development of severe graft versus host disease, graft failure and the development of infection due to the use of immunosuppression or T cell depletion. A total of 10 patients will be enrolled. The patients will be divided into 2 strata (haploidentical 7, unrelated donor 3).

Study Type

Expanded Access

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • United States
    • Texas
      • Dallas, Texas, United States, 75235
        • Available
        • Children's Medical Center - Dallas

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Child
  • Adult

Accepts Healthy Volunteers

No

Description

Recipient Inclusion Criteria:

  • Age less than 22 years of age
  • No available genotypically matched related donor (sibling)
  • Availability of a suitable donor and graft source
  • Haploidentical related mobilized peripheral blood cells
  • 9/10 or 10/10 allele matched (HLA-A, -B, -C, -DRB1, -DQB1) volunteer unrelated donor mobilized peripheral blood cells

Donor Inclusion Criteria:

  • Matching as described in the Recipient and Donor Suitability Criteria
  • Size and vascular access appropriate by center standard for peripheral blood stem cell (PBSC) collection. Donors with inadequate peripheral access will require placement of an apheresis catheter for collection.

  • Must meet appropriate screening/eligibility requirements:

    • Haploidentical matched family members: screened by center health screens and found to be eligible.
    • Unrelated donors: meet suitability criteria to donate PBSC
    • HIV negative
    • Not pregnant or lactating
    • Recipient must not have high-level donor specific anti-HLA antibodies according to institutional practices.
    • Must agree to donate PBSC
    • Must give informed consent

Recipient Suitability:

- Adequate organ functioning as demonstrated by:

  • MUGA ≥ 50% or ECHO >30%
  • 24-hour Creatinine Clearance or Glofil ≥ 50 ml/min or Serum Creatinine ≤2 times the upper limit of normal
  • DLCO > 65% of predicted value. FEV1 >65% of predicted
  • AST / ALT ≤ 2.5 and/or Bilirubin ≤ 1.5 times the upper limit of normal
  • Pregnancy test negative
  • HIV 1 / 2 (HIV screen) negative with a negative qualitative HIV PCR.
  • HTLV I negative
  • RPR or VDRL negative
  • RVP (Respiratory Viral PCR) with a negative result
  • Positive hepatitis testing does not make the patient ineligible. Hepatitis results will be evaluated on a case-by-case basis by the transplant physician to determine donor suitability/eligibility.

Donor Suitability:

- Allowed Donor Sources

  • Fully matched sibling donors are not allowed.
  • Unrelated donors. HLA typing of at least 10 alleles is required. Donor must be matched at 9/10 or 10/10 alleles (HLA-A, -B, -C, -DRB1, -DQB1). Haploidentical matched family members. Minimum match level full haploidentical (at least 5/10; HLA-A, -B, -C, -DRB1, -DQB1 alleles), but use of haploidentical donors with extra matches (e.g., 6, 7, or 8/10) encouraged.
  • Cord blood is not allowed as a stem cell source on this IDE.

Recipient and Donor Exclusion Criteria:

  • Pregnant or lactating females are ineligible as the risk to unborn children and infants is unknown
  • Presence of life-threatening, uncontrolled opportunistic infection (fungal, bacterial, or viral infections). Patients with history of fungal disease during induction therapy may proceed if they have a significant response to antifungal therapy with no or minimal evidence of disease remaining by CT evaluation.
  • HIV or HTLV I/II infection

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Victor Aquino

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Registration Dates

First Submitted

April 9, 2024

First Submitted That Met QC Criteria

April 26, 2024

First Posted (Actual)

May 1, 2024

Study Record Updates

Last Update Posted (Estimated)

October 14, 2025

Last Update Submitted That Met QC Criteria

October 11, 2025

Last Verified

October 1, 2025

More Information

Terms related to this study

Keywords

Other Study ID Numbers

  • STU-2024-0185

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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