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标题: QLT公司公布维甲酸治疗初步结果 [打印本页]

作者: 雪山飞狐 时间: 2012-3-2 08:31 标题: QLT公司公布维甲酸治疗初步结果

QLT Announces Positive Preliminary Results From Phase 1b Trial of QLT091001 in Subjects With Retinitis Pigmentosa Due to RPE65 and LRAT Mutations

VANCOUVER, British Columbia, Mar 1, 2012 (GlobeNewswire via COMTEX) -- QLT Inc. /quotes/zigman/13615/quotes/nls/qlti QLTI +2.24% /quotes/zigman/13629 CA

LT +2.27% ("QLT" or the "Company") today announced positive preliminary results from its international multi-center Phase 1b proof-of-concept clinical trial of QLT091001 for the treatment of Retinitis Pigmentosa (RP) due to inherited genetic mutations in retinal pigment epithelium protein 65 (RPE65) or lecithin:retinol acyltransferase (LRAT) (also known as early-onset RP).

The Phase 1b study showed rapid, statistically significant and clinically meaningful changes in visual fields (VF) from baseline values, as well as improvements in visual acuity (VA), in the study of 17 RP subjects. In addition, small subsets of RP subjects were investigated for secondary effects on other key vision parameters impacted by RP, such as decreased retinal sensitivity, and the data available in these subsets showed notable and promising increases in average sensitivity levels. The single-course treatment data with QLT091001 represents the first stage of dose regimen testing as the basis for a longer term multiple course regimen in RP due to mutations in RPE65 and LRAT.

RP is a disabling group of genetic eye diseases associated with progressive loss of vision including night blindness, constricted peripheral vision resulting in difficulties with daily activities, and in later life, reduced central vision, inability to read, and in many cases progression to severe blindness. RP can be caused by many different gene defects and symptoms can start at varying ages; patients with mutations in the RPE65 and LRAT genes tend to show vision loss very early in life (this type of RP is also known as early-onset RP).

In the open-label, multi-center Phase 1b clinical study, 17 subjects (ranging in age from 6 to 55 years, mean 29 years) with either RPE65 (12 subjects) or LRAT (5 subjects) mutations received a 40 mg/m2/day dose of QLT091001 once daily for seven days with post-treatment follow-up at 7, 14, and 30 days. Visual fields and visual acuity are key measures of clinically relevant visual function. VF was assessed using Goldmann Visual Fields (GVF) and VA was assessed using best-corrected visual acuity (BCVA, ETDRS letters); GVF maps were converted to assess the remaining functional retinal area for analysis. After a single 7-day course of treatment with QLT091001, the average retinal areas from baseline showed statistically significant improvements of 34% at day 7 (p=0.005), 29% at day 14 (p=0.02) and trended towards a statistically significant improvement of 23% at day 30 (p=0.07) in the evaluable subjects meeting GVF test criteria (n=14 subset). In the intent-to-treat (ITT; all subjects enrolled) analysis (n=17), the average retinal area from baseline improved by 22% at day 7 (p=0.03, statistically significant), 16% at day 14 (p=0.13) and 18% at day 30 (p=0.096). The evaluable subset of 14 subjects excludes three patients in the VF analysis because they did not meet criteria as determined by a third-party reader. Nine of 17 subjects (53%) showed an improvement in VA over baseline in at least one eye by greater than or equal to five ETDRS letters.

作者: chming2011 时间: 2012-3-2 08:40

谢谢飞狐兄，这个是对所有的RP都有点效果吗

作者: rwmlau2004 时间: 2012-3-2 08:45

本帖最后由 rwmlau2004 于 2012-3-2 09:04 编辑

1. Positive Preliminary Results
2. RP due to RPE65 and LRAT Mutations
3. Both VF & VA are improved

作者: muse 时间: 2012-3-2 08:46

谢谢飞狐大哥的分享

作者: newzix 时间: 2012-3-2 08:58

看不懂，请高手解答一下呀！

作者: a280055 时间: 2012-3-2 09:13

这个药物就是只针对RPE65异常引起的RP，什么时候所有的RP才能得到治疗呢？

作者: 南国乞儿 时间: 2012-3-2 09:26

谢谢飞狐大哥的分享.好像也只针对RPE65吧？

作者: rwmlau2004 时间: 2012-3-2 10:19

内文其实已有暗示说明
这药因是针对缺失某一个蛋白质的RP
即是现时常见的RPE65和LCA患者
这并非基因疗法, 只算是一种特效补充剂

作者: 余米亚 时间: 2012-3-2 11:01

嗯其实我是黄斑病变患者，也非医学专业，就我所能大致了解如下，如有错误或者不当请指正：
• QLT091001的多中心临床试验针仅仅对RPE65和LRAT引起的RP
• 试验阶段为Ib（根据之前读到的资料，FDA一般临床试验分为I / II / III期，分别为少数人参与评估安全性与耐受性，更多参与者持续评价并对不同剂量的反应加以评估，大量参与者测试广泛效果并允许调查机构参与跟进）
• Ib的试验效果统计显示，QLT在提高视野和视力方面临床效果十分显著，而其对于提升视网膜敏感性等视觉参数的作用尚在调查中
• 从6岁到55岁的17个病例参与了试验，包括对其OCT、ERG和生活质量的考量，对少部分病例，还检查了其夜视、瞳孔反射、视觉刺激的大脑皮层反应等情况
• QLT091001相关安全性的分析仍在继续，参与试验的病人将继续给药以便观察其持续的药效（耐受性）
• 目前项目在欧美加拿大的多个领先的RP治疗中心开展
• QLT的CEO表示，正式的试验结果有望于第二季度完成并与欧美的监管当局商议进一步项目进展
原文见：http://www.qltinc.com/documents/120301.pdf
说点其他的，由于这是公司网站的新闻，对于其试验效果的用词显得比较鼓舞人心，相对而言ACT发表在柳叶刀上文章措辞就比较谨慎。相信效果是必须有的，请坐等第二季度正式的报告，用数据和事实说话。
。

作者: 雪山飞狐 时间: 2012-3-2 11:07

9# 余米亚

谢谢你的翻译，辛苦了！

作者: 余米亚 时间: 2012-3-2 11:12

10# 雪山飞狐

rwmlau2004也能翻的呢，哈哈。我只是无耻的孔雀了一把。顺便谢谢您的乐观和鼓励。

作者: YESCHONG 时间: 2012-3-2 13:16

哎，对于另外的RP，没有用。

作者: rwmlau2004 时间: 2012-3-2 13:23

12# YESCHONG

对于另类的RP
干细胞理并会先成功
但维续性未知
而且预先估计的费用超出一般人负担能力
如社保包括?了
实在难行

作者: 凤凰涅盘 时间: 2012-3-16 23:28

Drug for RP and LCA Continues to Perform Well in Clinical Trial

March 15, 2011 – A drug designed to save and restore vision in people with retinitis pigmentosa (RP) and Leber congenital amaurosis (LCA) caused by defects in the RPE65 or LRAT genes continues to show positive preliminary results in a Phase Ib clinical trial taking place in several locations worldwide. The study, which began in 2010 and was made possible by earlier studies funded by the Foundation, is evaluating both safety and effectiveness of the treatment.

QLT, Inc., the maker of the drug, reported on March 1, 2012, that visual field improved on average by 22 percent for 17 people with RP after they took a seven-day course of the treatment. Nine of the 17 subjects also had improvement in visual acuity of at least one line on an eye chart.

In spring 2011, QLT announced positive preliminary trial results for 12 people with LCA. All patients responded well, reporting some gains in acuity and/or sizes of their visual fields. The investigators were encouraged that the effect of the week-long oral treatment persisted for several months.

Known as QLT091001, the drug replaces a retinoid, a form of vitamin A, which is necessary for vision, but is missing in people with RP or LCA caused by RPE65 or LRAT mutations.

“Vitamin A is an essential ingredient in the visual cycle, the biochemical process that makes vision possible. But for it to work, it needs to get processed and recycled into different forms called retinoids, including the retinoid 11-cis-retinal,” says Dr. Stephen Rose, the Foundation’s chief research officer. “QLT’s drug replaces the 11-cis-retinal that’s missing in certain forms of RP and LCA. And early study results show that it’s working.”

Scientists funded by the Foundation have conducted extensive research to understand the role of LRAT and RPE65 mutations in LCA and RP, giving targets for related treatments to companies such as QLT.

QLT is evaluating increased and longer term dosing of the drug for both safety and effectiveness. The company continues to recruit participants for the trial, which will conclude in 2015. Seven clinical research centers are conducting the trial for autosomal recessive forms of RP and LCA. The Montreal Children’s Hospital is also recruiting patients with the autosomal dominant form of RP caused by RPE65 mutations.

作者: 坐听松涛 时间: 2012-3-18 14:32

2012年3月15日，OLC报告17位RP患者在经过一周治疗后，视野平均提高了22%，17个人中9人视力至少提高了一行。

在2011年春天，OLC宣布，12位LCA患者临床治疗结果乐观。

OLC 公司正在评估更长期用药的安全性和有效性。公司继续征集参与者，实验将在2015年结束。

感谢凤凰涅槃。

作者: 坐听松涛 时间: 2012-3-18 14:33

2012年3月15日，OLC报告17位RP患者在经过一周治疗后，视野平均提高了22%，17个人中9人视力至少提高了一行。

在2011年春天，OLC宣布，12位LCA患者临床治疗结果乐观。

OLC 公司正在评估更长期用药的安全性和有效性。公司继续征集参与者，实验将在2015年结束。

感谢凤凰涅槃。

作者: 坐听松涛 时间: 2012-3-18 14:37

这是一种维生素A。对这种药我一直很期待。

作者: passway 时间: 2012-3-18 17:02

还要到2015年啊·~？

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