EUCTR2015-004122-33-GB进行中（未招募）1 期
A Phase 3 Single Arm Study Evaluating the Efficacy and Safety of Gene Therapy in Subjects with Transfusion-dependent ß-Thalassemia, who do not have ß0/ß0 Genotype, by Transplantation of Autologous CD34+ Stem Cells Transduced Ex Vivo with a Lentiviral ßA-T87Q-Globin Vector in Subjects =50 Years of Age
适应症
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 入组人数
- 23
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
- •1. Subjects =50 years of age at the time of consent or assent (as applicable), and able to provide written consent (adults, or legal guardians, as applicable) or assent (adolescents or children). Pediatric subjects (<12 years of age) may only be enrolled at a given site if approved by the relevant regulatory authority. Provided that the DMC has approved enrolling subjects younger than 5 years of age, subjects younger than 5 years of age may be enrolled at sites with regulatory approval for the specified age range if they weigh a minimum of 6 kg and are reasonably anticipated to be able to provide at least the minimum number of cells required to initiate the manufacturing process.
- •2. Diagnosis of TDT with a history of at least 100 mL/kg/year of pRBCs in the 2 years preceding enrollment (all subjects), or be managed under standard thalassemia guidelines (e.g.,Thalassemia International Federation) with =8 transfusions of pRBCs per year in the 2 years preceding enrollment.
- •3. Clinically stable and eligible to undergo HSCT.
- •4. Treated and followed for at least the past 2 years in a specialized center that maintained detailed medical records on RBC transfusions, in-patient hospitalization, and iron chelation history.
- •Are the trial subjects under 18? yes
- •Number of subjects for this age range: 13
- •F.1.2 Adults (18-64 years) yes
- •F.1.2.1 Number of subjects for this age range 10
- •F.1.3 Elderly (>=65 years) no
- •F.1.3.1 Number of subjects for this age range
排除标准
- •1. Presence of a mutation characterized as beta0 on both HBB alleles. For the purpose of this study, the HBB mutation IVS I-110 (G -> A) will be considered equivalent to a beta0 mutation
- •2. Positive for presence of HIV-1 or HIV-2, HBV, or HCV
- •3. Clinically significant and active bacterial, viral, fungal, or parasitic infection
- •4. A WBC count < 3 × 10^9/L, and/or platelet count < 100 × 10^9/L not related to hypersplenism
- •5. Uncorrected bleeding disorder
- •6. Any prior or current malignancy or myeloproliferative or significant immunodeficiency disorder
- •7. Immediate family member with a known Familial Cancer Syndrome
- •8. Prior HSCT
- •9. Advanced liver disease
- •10. Baseline estimated glomerular filtration rate < 70 mL/min/1.73 m^2
- •11. Uncontrolled seizure disorder
- •12. Diffusion capacity of carbon monoxide (DLco) < 50% of predicted. If DLco cannot be assessed due to age or cognition-related restrictions, there must be a normal respiratory exam, chest radiograph without pulmonary infiltrates, and oxygen saturation by pulse oximetry >92% on room air. In the presence of clinically significant abnormal findings on chest radiograph, clinically significant abnormal findings on the respiratory exam, or oxygen saturation by pulse oximetry =92% on room air, the subject will be excluded.
- •13. A cardiac T2* < 10 ms by MRI
- •14. Any other evidence of severe iron overload that warrants exclusion
- •15. Participation in another clinical study with an investigational drug within 30 days of Screening
- •16. Any other condition that would render the subject ineligible for HSCT, as determined by the attending transplant physician or investigator
- •17. Prior receipt of gene therapy
- •18. Diagnosis of significant psychiatric disorder of the subject that could seriously impede the ability to participate in the study
- •19. Pregnancy or breastfeeding in a postpartum female or absence of adequate contraception for fertile subjects
- •20. An assessment by the investigator that the subject would not comply with the study procedures outlined in the protocol
- •21. A known and available HLA-matched family donor
- •22. Any contraindications to use of G-CSF and plerifaxor during the mobilization of hematopoietic stem cells and any contraindications to use of busulfan and any other medicinal products required during the myeloablative conditioning.
研究者
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