EUCTR2016-002959-17-GB进行中（未招募）1 期
An exploratory, open-label study to evaluate the safety and feasibility of ATIR201, a T-lymphocyte enriched leukocyte preparation depleted ex vivo of host alloreactive T-cells (using photodynamic treatment), as adjuvant treatment to a T-cell depleted haploidentical hematopoietic stem cell transplantation in patients with beta-thalassemia major
适应症
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 入组人数
- 10
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
入选标准
- •Patient Inclusion Criteria:
- •Confirmed diagnosis of beta-thalassemia major:
- •- Hypertransfusion program with eight or more red blood cell (RBC) transfusions a year and a confirmed annual transfusion of = 100 ml/kg/year.
- •- Genotype consistent with clinical diagnosis of beta-thalassemia major (including hemoglobin E-beta-thalassemia genotype)
- •- Iron chelation therapy initiated, or not yet initiated but at risk of transfusional iron overload
- •Lansky / Karnofsky performance score > 70%
- •Eligible for haploidentical stem cell transplantation according to the investigator taking into account the feasibility of transplanting patients with pre-existing antibodies (e.g. anti-A, anti-B, and/or other anti-donor red cell antibodies or known presence of HLA antibodies against the non-shared donor haplotype)
- •Male or female, age = 2 years and = 25 years
- •Donor Inclusion Criteria:
- •Haploidentical family donor with at least 4 out of 8 or 5 out of 10 matches at the HLA- A, -B , -C, -DR, and/or DQ loci as determined by high resolution typing
- •Male or female, age = 16 and = 75 years (If applicable, local legal requirements for
- •donors under the age of 18 will be followed)
- •Eligible for donations of human blood and blood components according to local requirements and regulations
- •Fit to receive granulocyte colony-stimulating factor (G-CSF) and to give peripheral blood stem cells (blood counts and blood pressure within standards)
- •Eligible for donation according to the transplantation center
- •Are the trial subjects under 18? yes
- •Number of subjects for this age range: 7
- •F.1.2 Adults (18-64 years) yes
- •F.1.2.1 Number of subjects for this age range 3
- •F.1.3 Elderly (>=65 years) no
- •F.1.3.1 Number of subjects for this age range
排除标准
- •Exclusion Criteria Patient:
- •Availability of a fully matched related or unrelated donor following a donor search, which might have been initiated before screening.
- •Patient assigned to Pesaro risk class 3
- •Symptomatic cardiac failure: left ventricular ejection fraction at rest < 50% and no improvement with exercise, or shortening fraction < 26% (evaluated by echocardiogram or MUGA)
- •Hepatic failure: > 5 times the upper limit of normal range (ULN) for age of AST or > 2.5 times the ULN for age of total serum bilirubin
- •Renal function (creatinine clearance or GFR) < 50% of the lower limit of normal (LLN) for age
- •Symptomatic pulmonary failure: DLCO, FEV1, or FEC (diffusion capacity) < 50% of predicted (corrected for hemoglobin); if unable to perform a pulmonary function test, oxygen saturation < 85% on room air
- •Positive pregnancy test (females of childbearing age only)
- •Positive test for HIV-1 or HIV-2
- •Active viral, bacterial, fungal, or parasitic infection, including hepatitis B, hepatitis C
- •Prior HSCT for beta-thalassemia major
- •Known allergy to any of the components of ATIR201 (e.g., dimethyl sulfoxide)
- •Any other condition which, in the opinion of the investigator, makes the patient ineligible for the study
- •Donor Exclusion Criteria:
- •Positive pregnancy test or nursing (females of childbearing age only)
- •Positive viral test for HIV-1, HIV-2, HBV, HCV, or Treponema pallidum
- •Hemoglobin S = 50%, or presence of beta-thalassemia intermedia
- •History of congestive heart failure, unstable angina, or history of stroke
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