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
试验速览
- 阶段
- 不适用
- 状态
- Available
- 发起方
- 试验地点
- 1
研究概览
简要总结
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.
详细描述
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.
研究设计
- 研究类型
- Expanded Access
入排标准
- 年龄范围
- — 至 21 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •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
排除标准
- •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
研究者
Victor Aquino
Professor
University of Texas Southwestern Medical Center