EUCTR2021-006390-37-DE进行中（未招募）1 期
A Phase 3b Study to Evaluate Efficacy and Safety of a Single Dose of Autologous CRISPR Cas9 Modified CD34+ Human Hematopoietic Stem and Progenitor Cells (CTX001) in Subjects with Transfusion-Dependent ß-Thalassemia or Severe Sickle Cell Disease
适应症
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 入组人数
- 26
研究概览
简要总结
暂无简介。
研究设计
- 研究类型
- Interventional
入排标准
- 性别
- All
入选标准
- •Subjects with TDT:
- •Subjects 12 to 35 years of age, inclusive, on the date of informed consent
- •Eligible for autologous stem cell transplant as per investigator’s judgement
- •Diagnosis of transfusion-dependent ß-thalassemia (TDT) as defined by:
- •Documented homozygous ß-thalassemia or compound heterozygous ß-thalassemia including ß-thalassemia/hemoglobin E (HbE). Subjects can be enrolled based on historical data, but a confirmation of the genotype using the study central laboratory will be required before busulfan conditioning.
- •A history of at least 100mL/kg/year or 10 units/year of packed RBC transfusions in the prior 2 years before signing the consent or the last rescreening for patients going through re-screening
- •Subjects with SCD:
- •Subjects 12 to 35 years of age, inclusive, on the date of informed consent
- •Documented ßS/ßS, ßS/ß0, or ßS/ß+, subjects can be enrolled based on historical genotype results, but confirmation of genotype is required before busulfan conditioning
- •Eligible for autologous stem cell transplant as per investigator’s judgment
- •Subjects with severe SCD. Severe SCD defined aby the occurrence of at least 2 of the following events per year during the 2-year period before screening, while receiving appropriate supportive care (e.g. pain management plan, HU):
- •Acute pain events that require a visit to a medical facility and administration of pain medications (opioids or intravenous [IV] non-steroidal anti-inflammatory drugs [NSAIDs]) or RBC transfusions
- •Acute chest syndrome, as indicated by the presence of a new pulmonary infiltrate associated with pneumonia-like symptoms, pain, or fever
- •Priapism lasting >2 hours and requiring a visit to a medical facility
- •Splenic sequestration, as defined by an enlarged spleen, left upper quadrant pain, and an acute decrease in hemoglobin concentration of =2 g/dL.
- •Other protocol defined inclusion criteria may apply
- •Are the trial subjects under 18? yes
- •Number of subjects for this age range: 11
- •F.1.2 Adults (18-64 years) yes
- •F.1.2.1 Number of subjects for this age range 7
- •F.1.3 Elderly (>=65 years) no
- •F.1.3.1 Number of subjects for this age range
排除标准
- •Subjects with TDT:
- •A willing and healthy 10/10 Human Leukocyte Antigen (HLA)-matched related donor is
- •available per investigator’s judgement.
- •Prior hematopoietic stem cell transplant (HSCT).
- •Subjects with associated a-thalassemia and >1 alpha deletion, or alpha multiplications.
- •Subjects with sickle cell ß-thalassemia variant.
- •Clinically significant and active bacterial, viral, fungal, or parasitic infection as determined by the investigator.
- •White blood cell (WBC) count <3 × 109/L or platelet count <50 × 109/L not related to hypersplenism per investigator judgment.
- •Subjects with SCD:
- •A willing and healthy 10/10 Human Leukocyte Antigen (HLA)-matched related donor is available per investigator’s judgement.
- •Prior hematopoietic stem cell transplant (HSCT).
- •Clinically significant and active bacterial, viral, fungal, or parasitic infection as determined by the investigator.
- •Other protocol defined exclusion criteria may apply
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