2010年将会率先获得上市批准的是免疫球蛋白（Gammagard，百特）。该药在一项为期6个月的Ⅱ期临床试验中已显示出良好的疗效和耐受性。静脉免疫球蛋白已于几年前被批准用于免疫排斥的预防和治疗。目前该药处于Ⅲ期临床阶段。同样进入Ⅲ期临床试验的还包括bapineuzumab（惠氏）、solanezumab（礼来）、dimebon（辉瑞）和LY450139（礼来）。

Indication & Usage
GAMMAGARD LIQUID is indicated for the treatment of primary immunodeficiency disorders associated with defects in humoral immunity. These include but are not limited to congenital X-linked agammaglobulinemia, common variable immunodeficiency, Wiskott-Aldrich syndrome, and severe combined immunodeficiencies.

Important Risk Information
GAMMAGARD LIQUID is contraindicated in patients with known anaphylactic or severe hypersensitivity responses to Immune Globulin (Human). Patients with severe selective IgA deficiency (IgA < 0.05 g/L) may develop anti-IgA antibodies that can result in a severe anaphylactic reaction.

Immune Globulin Intravenous (Human) products have been reported to be associated with renal dysfunction, acute renal failure, osmotic nephrosis, and death. Patients predisposed to acute renal failure include patients with any degree of pre-existing renal insufficiency, diabetes mellitus, age greater than 65, volume depletion, sepsis, paraproteinemia, or patients receiving known nephrotoxic drugs. Especially in such patients, IGIV products should be administered at the minimum concentration available and the minimum rate of infusion practicable. While these reports of renal dysfunction and acute renal failure have been associated with the use of many of the licensed IGIV products, those containing sucrose as a stabilizer accounted for a disproportionate share of the total number.

Glycine, an amino acid, is used as a stabilizer. GAMMAGARD LIQUID does not contain sucrose.

GAMMAGARD LIQUID is made from human plasma. It may carry a risk of transmitting infectious agents, e.g. viruses, and theoretically, the Creutzfeldt-Jakob disease (CJD) agent.

Components used in the packaging of this product are latex-free.

Thrombotic events have been reported in association with IGIV. Patients at risk may include those with a history of atheroscle¬rosis, multiple cardiovascular risk factors, advanced age, impaired cardiac output, and/or known or suspected hyperviscosity, hypercoagulable disorders, and prolonged periods of immobilization.

IGIV products can contain blood group antibodies that may cause a positive direct antiglobulin reaction and, rarely, hemolysis.

Aseptic meningitis syndrome (AMS) has been reported to occur infrequently in association with IGIV treatment. Discontinua¬tion of IGIV treatment has resulted in remission of AMS within several days without sequelae.

Various mild and moderate reactions, such as headache, fever, fatigue, chills, flushing, dizziness, urticaria, wheezing or chest tightness, nausea, vomiting, rigors, back pain, chest pain, muscle cramps, and changes in blood pressure may occur with infusions of Immune Globulin Intravenous (Human).

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百特启动阿尔茨海默氏症治疗三期临床试验

DEERFIELD, Ill., September 22, 2008 — Baxter Healthcare Corporation today announced initiation of a Phase III clinical trial following U.S. Food and Drug Administration (FDA) review of its investigational new drug application to evaluate GAMMAGARD LIQUID [Immune Globulin Intravenous (IGIV)], marketed as KIOVIG in European Union, for the treatment of mild-to-moderate Alzheimer’s disease. This trial is expected to be the first of two pivotal Phase III trials required to support filing for regulatory approval for GAMMAGARD LIQUID as a potential treatment for the disease.
美国伊利诺伊州迪尔菲尔德2008年9月22日讯 — 在GAMMAGARD LIQUID [（人体）免疫球蛋白静脉注射液（IGIV），在欧盟市场上称为KIOVIGTM，用于治疗轻中度阿尔茨海默氏症]通过美国食品与药品监管局（FDA）的新药应用调查性评估后，百特医疗公司今日宣布启动该药物三期临床试验。此次试验被认为是提供GAMMAGARD LIQUID申请批准成为阿尔茨海默氏症治疗药物需要的支持材料的三期临床试验中两个关键性试验之一。

The Phase III trial is a prospective, 18-month, randomized, double-blind, placebo-controlled, two dose-arm, parallel study in 360 subjects of both genders, ages 50 to 89 years old, with dementia of mild-to-moderate severity. The study will determine whether GAMMAGARD LIQUID treatment results in a significantly slower rate of decline of cognitive and other functions compared to placebo. Approximately 40 U.S. leading academic centers have been identified and will begin clinical trial enrollment within the next several weeks.
三期临床试验是一次前瞻性18个月随机双盲安慰剂控制组高剂量平行研究试验，将对不同性别、年龄在50-89岁之间的具有轻度到中度老年痴呆症状的360个病例进行临床试验性研究。该项研究旨在确定GAMMAGARD LIQUID疗法与安慰剂相比是否能明显减缓患者大脑认知和其他功能的衰退速度。目前已有大约40家美国主要学术中心已经确认将在未来数周内招募试验病人进行此项临床试验。

Efficacy will be assessed by two primary endpoints:
•Cognitive outcomes using the Alzheimer’s Disease Assessment Scale-Cognitive Subscale score (ADAS-Cog), and
•Global clinical outcome as assessed by the Alzheimer’s Disease Cooperative Study-Clinical Global Impression of Change rating (ADCS-CGIC). Secondary endpoints to be assessed at 18 months include behavioral, functional and quality of life outcome measures. Other secondary endpoints will include several
试验药物的药效将通过以下两个一级指标进行评估：
•使用阿尔茨海默氏症评估量表 — 认知分量表得分（ADAS-Cog）测得的患者认知能力得分.
•使用阿尔茨海默氏症合作研究 – 临床总体印象改变评分（ADCS-CGIC）测得的临床总体得分。

plasma, cerebrospinal fluid, and imaging biomarkers to assess disease progression and response to therapy.
在18个月期间还将在对患者包括行为、功能和生活质量量表在内的二级指标例进行评估。其他二级指标有血浆、脑脊髓液、成像生物标志物，用于观察病情的进展和机体药物反应。

The trial is sponsored by Baxter and partially funded by the National Institutes of Health (NIH) through the Alzheimer’s Disease Cooperative Study (ADCS). The ADCS is a cooperative agreement between the National Institute of Aging and the University of California San Diego (UCSD). The ADCS was developed in response to a perceived need to advance research in the development of therapies that might be useful for treating patients with Alzheimer's disease. The trial will be managed by Paul Aisen, M.D., director of the Alzheimer’s Disease Cooperative Study and professor, department of Neurosciences, UCSD. The involvement of the ADCS and NIH in the conduct of the Phase III trial should help ensure the highest level of independent scientific evaluation of the potential role of GAMMAGARD LIQUID in the treatment of Alzheimer’s disease.
试验由百特企业赞助，一部分资金来自美国国立卫生研究院（NIH）的阿尔茨海默氏症合作研究（ADCS）合作协议。ADCS是美国国立老化研究院和美国加利福尼亚大学圣地亚哥分校（UCSD）签订的一项合作协议，旨在推动可能对阿尔茨海默氏症治疗有用的治疗方法的研究和开发。试验由阿尔茨海默氏症合作研究主任，加利福尼亚大学圣地亚哥分校（UCSD）神经系统科学系教授，医学博士Paul Aisen统一管理。ADCS和NIH的介入将有助于确保GAMMAGARD LIQUID在阿尔茨海默氏症治疗中潜在功效的独立科学评估的最高级别。

The project leader for the trial is Norman Relkin, M.D., PhD, director of the Memory Disorders Program and behavioral neurologist and neuroscientist at New York-Presbyterian/Weill Cornell Medical Center, and associate professor of clinical neurology at Weill Cornell Medical College in New York City. Dr. Relkin is also the lead investigator for the Phase I and II studies with GAMMAGARD LIQUID for the same indication.
项目由记忆障碍研究计划主任，纽约-长老会医院/威尔康乃尔医学中心行为神经学医师兼精神分析学家，纽约市威尔康乃尔医学院临床神经学副教授，医学博士Norman Relkin主持。Relkin博士曾担任用于相同治疗目的的GAMMAGARD LIQUID的第一、二阶段试验的主导调研员。

GAMMAGARD LIQUID contains a broad spectrum of immunoglobulins (antibodies). The therapy is approved for use as an immunoglobulin replacement therapy that boosts the immune system in patients with primary immunodeficiency disorders. The precise mechanisms of the therapy’s effects in Alzheimer’s disease are not yet known.
GAMMAGARD LIQUID含有多种形式的免疫球蛋白（抗体）。该药物已被批准用于免疫球蛋白替代治疗，增强原发性免疫缺陷病人的免疫系统功能。这种药物治疗阿尔茨海默氏症的准确药理尚且未知。

About GAMMAGARD LIQUID

关于GAMMAGARD LIQUID

GAMMAGARD LIQUID is indicated for the treatment of primary immunodeficiency disorders associated with defects in humoral immunity. These include, but are not limited to, congenital X-linked agammaglobulinemia, common variable immunodeficiency, Wiskott-Aldrich syndrome, and severe combined immunodeficiencies.
GAMMAGARD LIQUID主要用于治疗与体液免疫缺陷相关的原发性免疫缺陷，包括，但不限于，先天性X连锁无丙种球蛋白血症、常见变异型免疫缺陷病（CVID）、维-奥二氏综合征和严重复合型免疫不全症（SCID）。