Octapharma发布儿童急性发作神经精神综合征3期优效性研究结果-动脉网

Octapharma Releases Results for Phase 3 Superiority Study of Pediatric Acute-onset Neuropsychiatric Syndrome

CISION 等信源发布 2025-05-28 21:06

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Clinical Global Impression Data Showed Statistically Significant & Clinically Relevant Improvement in Pediatric PANS Patients

临床全局印象数据显示，儿科PANS患者在统计学上显著且临床上有相关改善

PARAMUS, N.J.

新泽西州帕拉姆斯

，

May 28, 2025

2025年5月28日

/PRNewswire/ -- Octapharma

has released the results

已经发布了结果

from its prospective, randomized, double-blind, parallel group, crossover, placebo-controlled phase 3 study comparing the effectiveness of PANZYGA

从其前瞻性、随机、双盲、平行组、交叉、安慰剂对照的第3期研究，比较PANZYGA的有效性

(immune globulin intravenous, human - ifas) 10% Liquid Preparation versus placebo in patients with pediatric acute-onset neuropsychiatric syndrome (PANS).

静脉注射人免疫球蛋白（IFAS）10%液体剂型与安慰剂在儿童急性发作神经精神综合征（PANS）患者中的对比。

继续阅读

The CY-BOCS score (Children's Yale-Brown Obsessive-Compulsive Scale) measured at 9 weeks was utilized as the primary endpoint for assessing

第9周测量的CY-BOCS得分（儿童耶鲁-布朗强迫症量表）被用作评估的主要终点

PANZYGA

潘齐伽

effectiveness in reducing the severity of PANS symptoms. In addition, other secondary endpoints such as the Clinical Global Impression (CGI-I) were also measured. The endpoints were measured at week 9 and week 18.

在减轻PANS症状严重程度方面的有效性。此外，还测量了其他次要终点，如临床全局印象（CGI-I）。这些终点在第9周和第18周进行了测量。

Study results suggest that PANZYGA® may address the broader disease burden in pediatric neuropsychiatric syndromes

研究结果表明，PANZYGA® 可能会减轻儿童神经精神综合征的更广泛疾病负担。

Post this

发布这个

From baseline (week 0) to week 9, there was a mean improvement of 31.1% (SD 40.68) in the PANZYGA

从基线（第0周）到第9周，PANZYGA的平均改善率为31.1%（标准差40.68）。

group CY-BOCS score as compared to 12.1% (SD 68.39) in the placebo group. Researchers compared the CY-BOCS score of the participants in the two groups as a ratio at week 9 and week 0 (CY-BOCS score at week 9/CY-BOCS score at week 0). While the mean ratio of the total CY-BOCS score at week 9 improved in the PANZYGA.

CY-BOCS评分组与安慰剂组的12.1%（标准差68.39）相比。研究人员比较了两周参与者在第9周和第0周的CY-BOCS评分比值（第9周CY-BOCS评分/第0周CY-BOCS评分）。在PANZYGA组中，第9周的总CY-BOCS评分平均比值有所改善。

group as compared to the placebo group (primary endpoint), the ratio difference between the two groups was not statistically significant with a probability-value (p-value) of 0.072. The research team also observed that the difference in the Improvement of the Clinical Global Impression (CGI-I) score at week 9 (secondary endpoint) was statistically significant between both groups with a higher improvement in the IVIG group (p-value = 0.017).

与安慰剂组相比（主要终点），两组之间的比率差异无统计学意义，概率值（p值）为0.072。研究团队还观察到，在第9周临床全局印象改善（CGI-I）评分的差异（次要终点）在两组之间具有统计学意义，且IVIG组的改善更为显著（p值=0.017）。

Please visit .

请访问。

euclinicaltrials.eu

欧盟临床试验网站

for the study data.

用于研究数据。

The study utilized a crossover analysis from week 9 to week 18 where the PANZYGA

该研究在第9周到第18周进行了交叉分析，其中PANZYGA

group participants received the placebo and the participants who had received the placebo until week 9 were given PANZYGA

小组参与者接受了安慰剂，而那些在第9周之前接受安慰剂的参与者被给予PANZYGA。

through week 18. Researchers observed the mean total CY-BOCS score in both treatment arms continued to improve. The CY-BOCS score improvement was more prominent in the participant group that initially received PANZYGA

在第18周时，研究人员观察到两组治疗组的CY-BOCS总分均持续改善。最初接受PANZYGA的参与者组中，CY-BOCS评分的改善更为显著。

until week 9, which researchers attributed to a continued response to the PANZYGA

直到第9周，研究人员将其归因于对PANZYGA的持续反应。

treatment in the first half of the study.

研究前半部分的治疗。

'While this Phase III trial did not meet its primary efficacy outcome of OCD symptom improvement measured by CY-BOCS, it revealed promising insights: the key secondary endpoint of Clinical Global Impression (CGI) showed statistically significant and clinically relevant improvement,' said Principal Investigator .

“虽然这项 III 期试验未达到其主要疗效终点，即通过 CY-BOCS 测量的强迫症症状改善，但它揭示了令人鼓舞的见解：关键次要终点临床全局印象（CGI）显示出具有统计学意义且临床相关的改善，”首席研究员表示。

Michael Daines

迈克尔·戴恩斯

, M.D.

，医学博士

, Division Chief of Pediatric Allergy and Immunology at the

，儿科过敏与免疫科主任

University of Arizona

亚利桑那大学

College of Medicine. 'CGI reflects the holistic impact of PANS/PANDAS on a patient's life, encompassing behavioral, cognitive, and physical symptoms. These results suggest that PANZYGA

医学院。'CGI反映了PANS/PANDAS对患者生活的整体影响，包括行为、认知和身体症状。这些结果表明PANZYGA

may address the broader disease burden in pediatric acute-onset neuropsychiatric syndromes, offering a potential therapeutic pathway for families navigating this complex condition. Further research into this investigational use of PANZYGA

可能针对小儿急性发作神经精神综合征的更广泛疾病负担，为应对这一复杂状况的家庭提供潜在的治疗途径。进一步研究PANZYGA的这一试验性用途

is needed to confirm this suggestion.'

需要确认这个建议。

The

PACE Foundation

PACE基金会

, along with fellow advocacy groups, is proud to have supported recruitment for this important trial. 'We're grateful to Octapharma for their compassion and ongoing commitment to families affected by this challenging disease,' said

，与其他倡导团体一道，为这一重要试验的招募工作提供支持感到自豪。 “我们感谢Octapharma对受到这一棘手疾病影响的家庭所表现出的同情心和持续的承诺，”该人士表示。

Paul Ryan

保罗·瑞安

, PACE Foundation Co-Founder and President.

，PACE基金会联合创始人兼主席。

A Challenging Disease for Families & Providers

对家庭和医疗提供者而言都是一种具有挑战性的疾病

PANS is diagnosed in children who experience sudden dramatic, often overnight, onset of obsessive-compulsive symptoms and/or severe eating restrictions, along with at least two other cognitive, behavioral, or neurological symptoms. Brain inflammation can occur when the body's immune system mistakenly attacks healthy brain cells..

PANS 用于诊断那些突然出现戏剧性的、常常是隔夜之间就发作的强迫症状和/或严重饮食限制的儿童，同时还会伴随至少两种其他认知、行为或神经症状。当身体的免疫系统错误攻击健康的脑细胞时，可能会导致脑部炎症。

'PANS is a very challenging disease for the medical community and families,' said

‘PANS对于医学界和家庭来说都是一种非常具有挑战性的疾病，’

Octapharma

八因子制药

USA

美国

President

总统

Flemming Nielsen

弗莱明·尼尔森

. 'Parents are confronted with the challenge of seeing their children afflicted with sudden-onset, debilitating, and difficult to treat OCD as well as other behavioral and cognitive issues. Octapharma looks forward to bringing relief to children and their families.'

“父母面临着看到自己的孩子突然患上衰弱且难以治疗的强迫症（OCD）以及其他行为和认知问题的挑战。Octapharma期待为儿童及其家庭带来缓解。”

Octapharma enrolled 71 patients from age 6 to 17 with a confirmed diagnosis of moderate to severe PANS in the prospective, randomized, double-blind, parallel group, placebo-controlled superiority

Octapharma 在一项前瞻性、随机、双盲、平行组、安慰剂对照的优效性研究中，招募了71名年龄在6至17岁之间、确诊为中度至重度PANS的患者。

study

学习

. (ClinicalTrials.gov Identifier: NCT04508530). Participants received three infusions of PANZYGA

. (ClinicalTrials.gov 标识符：NCT04508530)。参与者接受了三次 PANZYGA 输注。

or placebo over two days every three weeks for a total of nine weeks, with an additional double-blind, crossover safety and efficacy follow-up phase of three infusions of PANZYGA

或在每三周的两天内使用安慰剂，总共九周，并额外进行一个双盲、交叉的安全性和有效性随访阶段，包括三次 PANZYGA 输注。

or placebo administered over two days every three weeks for a total of nine weeks.

或在每三周的两天内给予安慰剂，总共持续九周。

The primary objective of the study was to assess whether PANZYGA

这项研究的主要目的是评估PANZYGA是否

is superior to placebo (0.9% w/v sodium chloride) for reducing the severity of symptoms associated with PANS in pediatric patients. The secondary objectives were to evaluate the sustainability of the reduction of the severity of symptoms in pediatric patients treated with PANZYGA

在减轻儿童PANS相关症状的严重程度方面，优于安慰剂（0.9% w/v氯化钠）。次要目标是评估接受PANZYGA治疗的儿童患者症状严重程度减轻的持续性。

; and assess the efficacy of PANZYGA

；并评估PANZYGA的疗效

treatment in reducing functional impairment associated with PANS.

治疗与PANS相关的功能障碍的减轻。

About PANZYGA

关于PANZYGA

PANZYGA

潘齐伽

is an immune globulin intravenous, (human) - ifas 10% liquid preparation indicated for:

是一种静脉注射用人免疫球蛋白，IFAS 10% 液体剂型，适用于：

- Primary humoral immunodeficiency (PI) in patients 2 years of age and older;

- 2岁及以上患者的原发性体液免疫缺陷（PI）；

- Chronic immune thrombocytopenia (ITP) in adults; and

- 成人慢性免疫性血小板减少症 (ITP)；以及

- Chronic inflammatory demyelinating polyneuropathy (CIDP) in adults.

成人慢性炎性脱髓鞘性多发性神经病（CIDP）。

WARNING: THROMBOSIS, RENAL DYSFUNCTION, and ACUTE RENAL FAILURE

警告：血栓形成、肾功能障碍和急性肾衰竭

– Thrombosis may occur with immune globulin intravenous (IGIV) products, including PANZYGA

- 静脉注射免疫球蛋白（IGIV）产品，包括PANZYGA，可能会发生血栓

. Risk factors may include: advanced age, prolonged immobilization, hypercoagulable conditions, history of venous or arterial thrombosis, use of estrogens, indwelling vascular catheters, hyperviscosity, and cardiovascular risk factors.

风险因素可能包括：高龄、长期制动、高凝状态、静脉或动脉血栓病史、使用雌激素、留置血管导管、高粘滞度以及心血管风险因素。

– Renal dysfunction, acute renal failure, osmotic nephropathy, and death may occur with the administration of IGIV products in predisposed patients. Renal dysfunction and acute renal failure occur more commonly in patients receiving IGIV products containing sucrose. PANZYGA

- 易感患者使用IGIV产品可能会导致肾功能障碍、急性肾衰竭、渗透性肾病甚至死亡。接受含蔗糖的IGIV产品的患者更常发生肾功能障碍和急性肾衰竭。PANZYGA

does not contain sucrose.

不含蔗糖。

– For patients at risk of thrombosis, renal dysfunction, or renal failure, administer PANZYGA

对于有血栓形成风险、肾功能不全或肾衰竭的患者，给予PANZYGA。

at the minimum infusion rate practicable. Ensure adequate hydration in patients before administration. Monitor for signs and symptoms of thrombosis and assess blood viscosity in patients at risk for hyperviscosity.

以最低的输注速率进行。在给药前确保患者充分补水。监测血栓形成的迹象和症状，并评估高粘滞度风险患者的血液粘稠度。

Please see full prescribing information at

请参阅完整的处方信息 tại

hcp.panzygausa.com/pi

。

About Octapharma

关于Octapharma

Headquartered in Lachen,

总部位于拉亨，

Switzerland

瑞士

, Octapharma is one of the largest human protein manufacturers in the world, developing and producing human proteins from human plasma and human cell lines.

，Octapharma是全球最大的人类蛋白质制造商之一，致力于从人类血浆和人类细胞系中开发和生产人类蛋白质。

Octapharma employs nearly 12,000 employees worldwide to support the treatment of patients in 118 countries with products across three therapeutic areas: Immunotherapy, Hematology and Critical Care.

Octapharma 在全球拥有近 12,000 名员工，通过免疫治疗、血液学和重症监护三大治疗领域的产品，为 118 个国家的患者提供治疗支持。

Octapharma has seven R&D sites and five state-of-the-art manufacturing facilities in

Octapharma在以下地区拥有七个研发中心和五个最先进的生产设施：

Austria

奥地利

，

France

法国

，

Germany

德国

and