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

Transplant-Related Disorder Clinical Trial

Official title:

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

Clinical Trial Details — Status: Available

Administrative data

• NCT number: NCT06393660
• Other study ID #: STU-2024-0185
• Status: Available

Study information

• Verified date: June 2024
• Source: University of Texas Southwestern Medical Center
• Contact: Victor Aquino, MD
• Phone: 214-648-3896
• Email: Victor.Aquino[@]utsouthwestern.edu
• Is FDA regulated: No
• Study type: Expanded Access

Clinical Trial Summary

The purpose of this IDE protocol is to provide access to TCR Alpha/Beta+/CD19+ depleted allogeneic hematopoietic stem cell (HSC) in patients who require hematopoietic stem cell transplantation.

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

Recruitment information / eligibility

• Status: Available
• Enrollment: 0
• Accepts healthy volunteers: No
• Gender: All
• Age group: N/A to 17 Years

Eligibility

Recipient Inclusion Criteria:

• Age less than 18 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

Related Conditions & MeSH terms

• GVH - Graft Versus Host Reaction
• Transplant-Related Disorder

Intervention

Device:
• CliniMACS® CD34 TCR Alpha/Beta
CliniMACS System uses an automated antibody/magnetic beads column system that allows efficient depletion of TCRaß and CD19+ cells without relevant loss of cell viability. Hematopoietic stem cells are retained in sufficient numbers to permit engraftment and hematopoietic reconstitution. Humanitarian Exemption IDE approval has been obtained for CD34+ cell selection as a method of T-cell depletion.

Locations

Country	Name	City	State
United States	Children's Medical Center - Dallas	Dallas	Texas

Sponsors (1)

Lead Sponsor	Collaborator
Victor Aquino

Country where clinical trial is conducted

United States,