NCT03639844No Longer Available不适用
Expanded Access Protocol for CaspaCIDe T Cells From An HLA-Partially Matched Related Donor After Negative Selection of TCR αβ+T Cells In Pediatric Patients Affected by Hematological and Other Disorders
Bellicum Pharmaceuticals2 个研究点 分布在 1 个国家开始时间: 2018年8月21日最近更新:
适应症
相关药物
试验速览
- 阶段
- 不适用
- 状态
- No Longer Available
- 发起方
- 试验地点
- 2
研究概览
简要总结
Providing access of BPX-501 gene modified T cells and rimiducid to pediatric patients who do not meet the eligibility criteria of the BP-U-004 study.
详细描述
This is an expanded access protocol of BPX-501 T cells infused after T cell-depleted HSCT in pediatric patients with non-malignant hematologic disorders eligible for treatment on the BP-U-004 study.
The purpose of this protocol is to provide access to the CaspaCIDe system combination product (BPX-501 gene modified T cells and rimiducid) to patients on a case by case basis who do not meet the BP-U-004 protocol eligibility criteria. BPX-501 infusion can enhance immune reconstitution with the potential for reducing the severity and duration of severe acute GVHD.
研究设计
- 研究类型
- Expanded Access
入排标准
- 年龄范围
- 3 Months 至 21 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Males or females
- •Age < 21 years and > 3 months
- •Life expectancy > 10 weeks
- •Patients deemed eligible for allogeneic stem cell transplantation.
- •Non-malignant disorders including:
- •inherited metabolic disorders such as adrenal leukodystrophy;
- •lysosomal storage disorders such as Hurler syndrome or metachromatic leukodystrophy
- •other inborn errors of metabolism
- •Lack of suitable conventional donor (HLA identical sibling or HLA phenotypically identical relative evaluated using high resolution molecular typing).
- •A minimum genotypic identical match of 5/10 is required.
- •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, and HLA- DRB
- •Lansky/Karnofsky score > 50
- •Signed written informed consent
- •3.2 Subject exclusion criteria
- •Age < 3 months or >21 years
- •Patients with non-malignant disorders eligible for treatment on the BP-U-004 study:
- •primary immune deficiencies,
- •severe aplastic anemia not responding to immune suppressive therapy,
- •osteopetrosis,
- •selected cases of hemoglobinopathies and
- •congenital/hereditary cytopenia, including Fanconi Anemia before any clonal malignant evolution (MDS, AML)
- •Greater than Grade II acute GVHD or chronic extensive GVHD due to a previous allograft at the time of inclusion
- •Patient receiving an immunosuppressive treatment for GVHD treatment due to a previous allograft at the time of inclusion
- •Dysfunction of liver (ALT/AST > 5 times normal value, or bilirubin > 3 times normal value), or of renal function (creatinine clearance < 30 ml / min)
- •Severe cardiovascular disease (arrhythmias requiring chronic treatment, congestive heart failure or left ventricular ejection fraction < 40%)
- •Current active infectious disease (including positive HIV serology or viral RNA)
- •Serious concurrent uncontrolled medical disorder
- •Pregnant or breast feeding female patient
- •Lack of parents'/guardian's informed consent.
排除标准
- 未提供
研究者
研究点 (2)
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