EUCTR2014-000584-41-GB进行中（未招募）1 期
Phase I/II study of CaspaCide T cells from an HLA-partially matched family donor after negative selection of TCR aß+ T cells in pediatric patients affected by hematological disorders - CaspaCide TCR aß haplo HSCT
适应症
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 入组人数
- 175
研究概览
简要总结
暂无简介。
研究设计
- 研究类型
- Interventional clinical trial of medicinal product
入排标准
- 性别
- All
入选标准
- •Patient Inclusion Criteria:
- •1. Males or females
- •2. Age = 18 years and = 1 month (<1 month upon approval by Sponsor)
- •3. Life expectancy > 10 weeks
- •4. Patients deemed clinically eligible for allogeneic stem cell transplantation.
- •5. Patients may have failed a prior allograft.
- •6. Patients with life-threatening acute leukemia (high-risk Acute Lymphoblastic leukemia (ALL) in 1st Complete remission (CR), ALL in 2nd CR, high-risk Acute Myeloid leukemia (AML) in 1st CR, AML in 2nd CR). Morphological CR must be documented and minimal residual disease measurement before transplantation is recommended.
- •7. Non-malignant disorders deemed curable by allogenic transplantation:
- •a. primary immune deficiencies,
- •b. severe aplastic anemia not responding to immune suppressive therapy,
- •c. osteopetrosis,
- •d. selected cases of erythoid disorders, such as ß0 ß0 thalassemia major, sickle cell disease, Diamond-Blackfan anemia,
- •e. congenital/hereditary cytopenia, including Fanconi Anemia before any clonal malignant evolution (Myelodysplastic syndrome (MDS), AML).
- •Note: subjects will be eligible if they meet either item 6 or 7.
- •8. Lack of suitable conventional donor (Human Leucocyte Antigene (HLA) identical sibling or HLA phenotypically identical relative or 9-10/10 unrelated donor evaluated using high resolution molecular typing) or presence of rapidly progressive disease not permitting time to identify an unrelated donor
- •9. A minimum genotypic identical match of 5/10 is required
- •10. The donor and recipient must be identical, as determined by high resolution typing, at least one allele of each of the following genetic loci: HLA-A, HLA-B, HLA-Cw, HLA- DRB1 and HLA-DQB1.
- •11. Lansky/Karnofsky score > 50
- •12. Signed written informed consent by the patient or the patient's parent or guardian for patients who are minors
- •1. Eligible donors include 5/10 HLA identical relative, including but not limited to biological parents, siblings, or half-siblings. Matching will be determined by class I and class II DNA typing. The donor of the BPX-501 T cells must be the HSCT donor.
- •2. Donor age must be > 18 and < 65 years.
- •3. The donor should be sufficiently healthy not to be at increased risk from the mobilization procedure.
- •4. Should more than one equally” MHC compatible donor be identified, other selection criteria may include natural killer cell (NK) alloreactivity, NK cell KIR-Haplotype, B-content for B-haplotype donors, size of the NK alloreactive subset, gender, age, CMV status, health status and body weight of donor. The physician treating the subject will make the final decision.
- •5. Donors must meet the selection criteria as defined by the European Directive 2006/17/CE and according to the FACT-JACIE International Standards and local regulations for donor selection.
- •6. The donor must have been informed of the investigational nature of the BPX-501 product and have signed an informed consent form that they will undergo a second pheresis procedure.
- •7. Donor must have adequate peripheral venous access for leukapheresis or must agree to placement of a central venous catheter.
- •8. The collection of donor T cells to be transduced with the suicide gene will be performed before the mobilization procedure with G-CSF in order to avoid any potential negative influence of this cytokine on function of genetically modified T cells.
- •9. Signed informed consent
- •Are the trial subjects under 18? yes
- •Number of subjects for this age range: 175
- •F.1.2 Adults (18-6
排除标准
- •1. Greater than grade II acute GVHD or chronic extensive GVHD due to a previous allograft at the time of inclusion
- •2. Patient receiving an immunosuppressive treatment for GVHD treatment due o a previous allograft at the time of inclusion
- •3. Dysfunction of liver (ALT/AST > 5 times normal value, or bilirubin > 3 times normal value), or of renal function (creatinine clearance < 30 ml / min)
- •4. Severe cardiovascular disease (arrhythmias requiring chronic treatment, congestive heart failure or left ventricular ejection fraction <40%)
- •5. Current uncontrolled clinically active infectious disease (including positive HIV serology or viral RNA
- •6. Serious concurrent uncontrolled medical disorder
- •7. Pregnant or breast feeding female patient
- •8. Lack of parents'/guardian's informed consent
- •Donor exclusion criteria
- •1. Evidence of active infection (including urinary tract infection, or upper respiratory tract infection) or viral hepatitis exposure (on screening), unless HBs Ab+ and HBV DNA negative.
- •2. Factors which place the donor at increased risk for complications from leukapheresis or G-CSF therapy (e.g., autoimmune disease, sickle cell trait, symptomatic coronary artery disease requiring therapy, previous thrombotic events).
- •3. Pregnancy at the time of normal leukapheresis for the T cells and at the time of the mobilization for the stem cell allograft collection.
- •4. Breastfeeding at the time of mobilization
- •Sponsor will be notified of any AEs (infections, etc.) occurring in the donor between the leukapheresis and stem cell apheresis.
研究者
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