ISRCTN22225367已完成不适用
Phase I clinical study of AAV1-gamma-sarcoglycan gene therapy for limb girdle muscular dystrophy type 2C
Genethon (France)0 个研究点目标入组 9 人开始时间: 2011年4月8日最近更新:
适应症
试验速览
- 阶段
- 不适用
- 状态
- 已完成
- 发起方
- 入组人数
- 9
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
- •1. Confirmed diagnosis of LGMD 2C including:
- •1.1. Molecular analysis proving del525T mutation on ?-sarcoglycan gene (chromosome 13) at homozygous state
- •1.2. Muscle biopsy with immunohistochemical and/or Western blot analysis showing marked decrease or absence of ?-sarcoglycan staining in muscle, as well as a fibrosis assessment should be available. If not, an initial muscular biopsy may be performed during the pre-enrolment period
- •2. Minimum age of 15 years
- •3. Males and females may be equally enrolled
- •4. Adequate carpi radialis muscle bulk for muscle biopsy as assessed by examination
- •5. Subjects should be able to communicate with the investigation staff
- •6. Subjects should be able to understand, to comply with and to perform all needed evaluations during the trial period including muscle strength tests
- •7. Forearm muscle strength should be of at least 3+ as assessed through the British Medical Research Council (MRC) Manual Muscle Testing (MMT) scale
- •8. Subjects should also have already lost ambulation
- •9. Subjects should be able and willing to return for follow up
- •10. Subjects should be able and willing to give signed informed consent
- •11. For minor subjects, a signed informed consent will be given by a legally authorised representative
- •12. Eligible subjects belonging to a multiplex family should not be enrolled in the same cohort
排除标准
- •1. Severity of disease and presence of ill-prognosis complications:
- •1.1. Severe respiratory dysfunction such as subjects with tracheostomy or forced vital capacity (FVC) < 1000ml and/or < 30%
- •1.2. Uncompensated heart failure
- •1.3. An ejection fraction (EF) < 30% as measured on either echocardiography or scintigraphy
- •1.4. Severe rhythm disturbances and/or high degree conduction defect in the absence of a pacemaker insertion
- •2. Underlying conditions, diseases or active viral infections likely to increase risk of complications or to interfere with the investigational treatment:
- •2.1. Contraindications for injections and muscle biopsies
- •2.2. Platelet count < 100,000/mm3
- •2.3. Total bilirubin > 10 mg/l (> 17 µmol/l)
- •2.4. Serum creatinine > 110 µmol/l
- •2.5. Lymphocytes CD4+ < 250/mm3 (< 15%)
- •2.6. History of diabetes mellitus
- •2.7. Current infectious diseases, including known positive human immunodeficiency virus (HIV) serology, hepatitis B and C
- •2.8. Abnormal profile on protein immunoelectrophoresis
- •2.9. Immunisations of any kind within the past month
- •2.10. Receipt of another investigational agent within 4 weeks of study enrolment
- •2.11. History of or current steroid medication for indications other than muscular dystrophy, chemotherapy, radiotherapy or other immunosuppressive therapy
- •2.12. Steroid medication, if any, should be discontinued at least 3 months before entering the protocol and not received during the study
- •2.13. Pregnant or lactating women
- •2.14. Females or males of childbearing age must be willing to employ adequate contraception, that is to use condoms during the 3 months following the administration of the product
- •2.15. Pre-injection neutralising anti-AAV1 antibodies titer (on pre-enrolment / D-30 visit) superior or equal to 1/800
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