HUMAN FGF 4 GENE TRANSFER IN PATIENTS W/ ANGINA

心绞痛患者中的人类 FGF 4 基因转移

• 批准号: 6264076
• 负责人: Jeffrey Alan Brinker
• 金额: $ 0.23万
• 依托单位: JOHNS HOPKINS UNIVERSITY
• 依托单位国家: 美国
• 财政年份: 1998
• 资助国家: 美国
• 起止时间: 1998-12-01 至 1999-11-30
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/6264076
• 关键词: Adenoviridae; angina pectoris; clinical research; clinical trial phase I; disease /disorder classification; dosage; gene therapy; human subject; human therapy evaluation; myocardial ischemia /hypoxia; transfection /expression vector

This is a Phase I/II clinical trial. The objective of this study is to evaluate the safety and anti-ischemic effects of adenovirus mediated hFGF-4 gene transfer in patients with stable exertional angina (Class II and Class III) so that a potentially safe and effective dose may be selected for a subsequent study. This is a double-blind, ascending dose, placebo controlled study. Each dose group consists of at least nine active and three placebo patients. The lowest dose (3.2 x 10^9 viral particles) will be studied first. For each dose group, safety observations during the first two weeks are reviewed and safety confirmed for the first patient randomized to active medication before dosing the second patient randomized to active medication. Safety observations for two weeks after the second patient are reviewed and safety confirmed before dosing the remaining patients in each dose group. Sponsor unblinding for all patients is allowed, but the patient, investigator, and his staff all remain blinded throughout the study. Core laboratories read, in a blinded fashion, exercise ECGs, stress echocardiograms, and coronary angiography. There have been no adverse events in any of the subjects enrolled at Johns Hopkins. Nationally, one patient had a transient rise in liver enzymes with no sequelae. As the study is still in progress, efficacy data is not yet available. Other research into the use of gene therapy for angiogenesis has shown remarkable results in the area of blood vessel growth. However, this area of study is still in the very early stages.

这是一项I/II期临床试验。 本研究的目的是评估腺病毒介导的hFGF-4基因转移在稳定性劳累性心绞痛（II级和III级）患者中的安全性和抗缺血作用，以便为后续研究选择潜在安全有效的剂量。这是一项双盲、剂量递增、安慰剂对照研究。 每个剂量组由至少9名活性药物和3名安慰剂患者组成。将首先研究最低剂量（3.2 x 10^9个病毒颗粒）。 对于每个剂量组，审查前两周期间的安全性观察结果，并在第二名随机分配至活性药物的患者给药前确认第一名随机分配至活性药物的患者的安全性。 审查第二例患者后两周的安全性观察结果，并在每个剂量组的其余患者给药前确认安全性。 允许申办者对所有患者揭盲，但患者、研究者及其工作人员在整个研究期间均保持盲态。核心实验室以盲法读取运动心电图、负荷超声心动图和冠状动脉造影。在约翰霍普金斯入组的任何受试者中均未发生不良事件。 在全国范围内，1例患者出现一过性肝酶升高，无后遗症。 由于研究仍在进行中，尚未获得疗效数据。 其他研究使用基因治疗血管生成显示出显着的成果，在该地区的血管生长。 然而，这一领域的研究仍处于非常早期的阶段。