EUCTR2012-001725-26-DE进行中（未招募）1 期
A Phase I/II Gene Therapy trial for X-CGD with a SIN gamma retroviral vector - gene therapy for X-CGD
Johann Wolfgang Goethe-University0 个研究点目标入组 5 人开始时间: 2012年12月18日最近更新:
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 发起方
- 入组人数
- 5
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.Verified diagnosis of the X-linked form of chronic granulomatous disease, with loss of gp91phox expression (Western Blot). Evidence of less than 5% of normal oxidase production in circulating neutrophil granulocytes as measured by DHR- and NBT-assay.
- •2.History of severe chronic infections with life-threatening course or severe steroid- sensitive or steroid insensitive granulomatous disease, with necessity of inpatient treatment, without sustained improvement even under maximum conservative treatment measures.
- •3.18 years old or older
- •4.No HLA identical (10/10 match) sibling- or unrelated donor, or contraindications for allogenic stem cell transplantation in presence of a suitable donor. The lack of an HLA-identical (10/10 match) sibling- or unrelated donor has to be confirmed by an unsuccessful search in national and international donor registers for at leat 3 months.
- •5.Normal organ-function: GFR = 60ml/min., Bilirubin = 1,5-fold upper reference-level, normal parameters for liver enzymes and clotting (TPZ 75-100%, PTT 30-38sec, Fibrinogen 200-400mg/dl), Leukocytes > 3 x 109/l, Granulocytes > 1.5 x 109/l, Thrombocytes >100 x 109/l
- •6.Contraception from start of G-CF application until 1 year after retransfusion of the gene-corrected cells.
- •Women with childbearing potential or partners with childebearing potential of male patients with have to apply safe contraceptive measures (Pearlindex <1%), as sexual abstinence, combination of an oral contraceptive, IUS, vaginal ring with drug delivery, contraceptive pad, implanted contraceptive, injected depot-contraceptive; in combination with a second contraceptive method, in terms os a barrier-method, such as condom, diaphragm /cervical cap with spermicide or sterilization in male patients or partners.
- •a.A woman of childbearing potential is defined, as sexually mature woman without hysterectomy or surgical sterilization, who has not been postmenopausal for at least 12 months (no menstruation within the last 12 months).
- •7.No interferon-gamma injection within two weeks prior to hematopoietic stem cell mobilization
- •8.Karnofsky-Index > 70%
- •9.Signed informed consent
- •Are the trial subjects under 18? no
- •Number of subjects for this age range:
- •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.Patients with non-controlled acute infections
- •2.Severe cardiac or pulmonary malfuctions: ejection fraction < 60%, valvular heart disease > II°, arrhythmia requiring therapy, FEV1/VC < 75% , DLCO <60%
- •3.Bilirubin > 1,5-fold upper reference-level
- •4.Creatinine-clearance <60ml/min
- •5.HIV-, Hepatitis B- or C - infection
- •6.Contraindications for G-CSF administration, as autoimmune vasculitis.
- •7.Contraindications for stem cell apheresis, as low hemoglobin <8g/dl, cardiovascular instability or severe coagulapathy
- •8.Pregnacy or breast-feeding
- •9.Drug- or alcohol-abuse
- •10. Lack of search for an unrelated donor
- •11. Patients with an available HLA 9/10 MMUD will be excluded from the study if SCT is considered to be beneficial according to thorough risk-benefit-assessment
研究者
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