EUCTR2016-003611-35-FR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 have a 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
- 15
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). Provided
- •that the DMC has approved enrolling subjects younger than 5 years of
- •age, subjects younger than 5 years of age may be enrolled 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, 2014) with =8 transfusions of pRBCs per year in the 2 years
- •preceding enrollment (subjects =12 years).
- •3. Clinically stable.
- •4. Treated and followed for at least the past 2 years in a specialized
- •center that maintained detailed medical records on RBC transfusions, inpatient hospitalization, and iron chelation history.
- •Are the trial subjects under 18? yes
- •Number of subjects for this age range: 10
- •F.1.2 Adults (18-64 years) yes
- •F.1.2.1 Number of subjects for this age range 5
- •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 other than beta0 (e.g., beta+,
- •betaE, betaC) on at least one HBB allele.
- •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 as determined by the clinical investigator.
- •4. A white blood cell (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 (i.e. parent or siblings) 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
- •(corrected for Hb and/or alveolar volume, as clinically indicated).
- •13. A cardiac T2* <10 ms by MRI.
- •14. Any other evidence of severe iron overload that, in the investigator's
- •opinion, 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 human leukocyte antigen (HLA)-matched
- •family donor. Ifrequired by regional authority, patients with a known
- •and available matched unrelated donor will be excluded from the study.
- •22. Any contraindications to the use of G-CSF and plerixafor during the
- •mobilization of hematopoietic stem cells and any contraindications to the
- •use of busulfan and any other medicinal products used during the
- •myeloablative conditioning, including hypersensitivity to the active
- •substances or to any of the excipients.
研究者
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