EUCTR2006-003833-33-GB进行中（未招募）1 期
Restoring Dystrophin Expression in Duchenne Muscular Dystrophy: A Phase I/II Clinical Trial Using AVI-4658
适应症
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 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 clinical trial of medicinal product
入排标准
- 性别
- Male
入选标准
- •1. Subject is male = 12 years and = 17 years of age at the time of study drug administration.
- •2. Subject has clinical diagnosis compatible with Duchenne’s Muscular Dystrophy (DMD) and evidence of mutational and dystrophin defects from muscle biopsy consistent with DMD (out-of frame deletions, absent dystrophin).
- •3. Subject has had a muscle biopsy analysed, showing <5% revertant fibres present. Biopsy may be collected at the time of DMD diagnosis or as part of protocol screening procedures.
- •4. Subject is unable to ambulate or stand independently.
- •5. Subject has Stage 1 to 3 EDB muscle preservation determined by MRI (Mercuri et al., 2002; Hawley et al., 1984).
- •6. Subject has a forced vital capacity = 25% confirmed within 3 months from Day One.
- •7. Subject has mean oxygen saturation monitoring > 94% in overnight domiciliary overnight sleep study within 3 months of Day One.
- •8. Subject has the ability to comply with all study evaluations and return for all study.
- •9. Subject and parent have psychiatric adjustments, adequately supportive psychosocial circumstances and a full understanding of study aims process and likely outcomes.
- •Are the trial subjects under 18? yes
- •Number of subjects for this age range:
- •F.1.2 Adults (18-64 years) no
- •F.1.2.1 Number of subjects for this age range
- •F.1.3 Elderly (>=65 years) no
- •F.1.3.1 Number of subjects for this age range
排除标准
- •1. Subject has had external digitorum brevis (EDB) muscle removed.
- •2. Subject has Stage 4 EDB muscle preservation determined by MRI.
- •3. Subject has a left ventricular shortening fraction of < 25% and/or an ejection fraction of < 35% by echocardiography at visit one or within three months of visit one.
- •4. Subject has evidence of nocturnal hypoventilation (mean oxygen saturation at night of = 94%) confirmed via overnight sleep study at Visit One (as screening procedure) or within 3 months of Visit One by overnight sleep study.
- •5. Subject has severe respiratory insufficiency defined by the need for invasive or non-invasive mechanical ventilation (does not include nocturnal ventilatory support).
- •6. Subject has severe cognitive dysfunction rendering them unable to understand and collaborate with study protocol.
- •7. Subject has immune deficiency or autoimmune disease.
- •8. Subject has a known bleeding disorder or has received chronic anticoagulant treatment within three months of study entry.
- •9. Subject has received pharmacologic treatment, apart from corticosteroids, that might affect muscle strength or function within 8 weeks of study entry (viz., anabolic steroids, creatine protein supplementation, albuterol or other beta agonists).
- •10. Subject has had surgery within 3 months of study entry or planned for anytime during study.
- •11. Subject has active significant illness at time of study entry.
- •12. Subject has is unable to undergo MRI testing (viz., has metal implants).
- •13. Subject or parent has active psychiatric disorder, has adverse psychosocial circumstances, recent significant emotional loss, history of depressive or anxiety disorders that might interfere with protocol completion or compliance.
- •14. Subject has any known allergies to products likely to be used in the study (viz., antiseptics, anaesthetics).
- •15. Subject has used any experimental treatments or has participated in any clinical trial within 4 weeks of study entry.
- •16. Subject has used intranasal, inhaled or topical steroids for a condition other than muscular dystrophy within 1 weeks of study entry.
研究者
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