EUCTR2018-003842-18-IT进行中（未招募）1 期
A Single Arm, Open Label Clinical Study of Haematopoietic Stem Cell Gene Therapy with Cryopreserved Autologous CD34+ Cells Transduced with Lentiviral Vector encoding WAS cDNA in Subjects with Wiskott-Aldrich Syndrome (WAS). - Clinical study using cryopreserved OTL-103 for treatment of WAS.
Orchard Therapeutics Ltd.0 个研究点目标入组 6 人开始时间: 2018年11月19日最近更新:
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试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 发起方
- 入组人数
- 6
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
- Male
入选标准
- •Age: up to 65 years.
- •Diagnosis of WAS defined by genetic mutation and at least one of the following criteria:
- •Severe WASP mutation;
- •Absent WASP expression;
- •Severe clinical score (Zhu clinical score = 3);
- •Family member affected by WAS with life-threatening or fatal clinical events.
- •No human leukocyte antigen (HLA)-identical related donor available.
- •Parental/guardian/subject-signed informed consent, and subject assent (if appropriate).
- •For all subjects in the reproductive age range, agreement to use highly
- •effective and adequate method of contraception (as detailed in Appendix 4: Contraceptive Guidance and Collection of Pregnancy Information) while receiving treatment and for at least 12 months following drug administration.
- •Are the trial subjects under 18? yes
- •Number of subjects for this age range: 6
- •F.1.2 Adults (18-64 years) yes
- •F.1.2.1 Number of subjects for this age range 1
- •F.1.3 Elderly (>=65 years) no
- •F.1.3.1 Number of subjects for this age range
排除标准
- •1. End-organ dysfunction, severe active infection not responsive to treatment or other severe disease or clinical condition which, in the judgment of the investigator, would make the patient inappropriate for entry into this study.
- •In addition to the potential infections tested per protocol, the PI should Tissue Directive as clinically appropriate and discuss the results with the medical monitor prior to cell harvest.
- •Patients with ALT >2x upper limit of normal (ULN) or total bilirubin >1.5 x ULN may be included only after discussed and agreed with the medical monitor and considered in the context of the criterion for excluding patients with other severe disease.
- •Isolated elevation of total bilirubin >1.5 x ULN is acceptable if bilirubin is fractionated and direct bilirubin <35% of total.
- •2. Malignant neoplasia (except local skin cancer) or a documented history of hereditary cancer syndrome . Patients with a prior successfully treated malignancy and a sufficient follow-up to exclude recurrence (based on oncologist opinion) can be included after discussion and approval by the medical monitor.
- •3. Myelodysplasia, cytogenetic alterations characteristic of myelodysplastic syndrome and acute myeloid leukaemia , or other serious haematological disorders.
- •4. Prior allogeneic hematopoietic stem cell transplantation, with evidence of residual cells of donor origin.
- •5. Documented HIV infection (positive HIV RNA and/or anti-p24 antibodies).
- •6. Current participation in other interventional clinical trials.
- •7. Previous gene therapy.
- •8. Symptomatic herpes zoster, not responsive to specific treatment.
- •Patients with a recent history of herpes zoster may be included in the study. In such cases, inclusion, additional monitoring and treatment of the condition must be discussed and approved by the medical monitor.
- •9. Evidence of active tuberculosis (TB) based upon medical examination, chest imaging and TB testing i.e. QuantiFERON®-TB Gold test and microbiological evidence.
- •Patients with latent TB, as documented by medical history and/or TB testing may be included in the study if receiving antibiotic prophylaxis (e.g. isoniazid). Inclusion, monitoring and treatment of TB in such patients must be discussed and approved by the medical monitor.
- •10. Acute or chronic stable Hepatitis B as evidenced by positive Hepatitis B surface antigen (HBsAg) test result at screening or within 3 months prior to start of conditioning and/or positive HBV DNA.
- •Patients with positive Hepatitis B core antibody due to prior resolved disease may be eligible to be included, only if a confirmatory negative HBsAg and negative Hepatitis B DNA test are obtained. Inclusion, monitoring and treatment of hepatitis in such patients must be discussed and approved by the medical monitor.
- •11. Presence of positive Hepatitis C RNA test result at screening. Patients who have previously tested positive for HCV can be treated, provided they demonstrate absence of ongoing infection using a nucleic acid test with a limit of quantification of =15 international units/mL.
- •Negative test results are required on at least 3 sequential occasions over a period of at least 4 weeks, after completion of treatment for hepatitis C, with the final test conducted no more than 3 days prior to cell harvest.
- •Inclusion, monitoring and treatment of hepatitis in such subjects must be discussed and approved by the medical monitor.
- •12. Patients that are not eligible for mobilisation protocols in order to obtain CD34+ cells f
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