With this study we want to investigate the pharmacokinetic (PK) effect of a single injection of rhIGF-1 in patients with PAPP-A2 mutations compared to heterozygous carriers and healthy controls. This will be followed by treatment of PAPP-A2 deficient patients with IGF-1 for a period of one-year to assess growth velocity. Additionally, we want to further describe the phenotypic characteristics of patients with PAPP-A2 deficiency.

开始日期2015-12-21

申办/合作机构

Children's Hospital & Medical Center

NCT01777542

/ Completed临床2期IIT

Pharmacological Treatment of Rett Syndrome by Stimulation of Synaptic Maturation With Recombinant Human IGF-1(Mecasermin [rDNA] Injection)

Investigators are recruiting children for a clinical trial using the medication recombinant human IGF-1 (a.k.a. mecasermin or INCRELEX) to see if it improves the health of children with Rett syndrome (RTT). While IGF-1 is approved by the Food & Drug Administration (FDA) for certain use in children, it is considered an investigational drug in this trial because it has not previously been used to treat RTT. Information from this study will help determine if IGF-1 effectively treats RTT but will not necessarily lead to FDA approval of IGF-1 as a treatment for RTT.

开始日期2013-01-01

申办/合作机构

The Children's Hospital Corp.

[+1]

EUCTR2011-004104-38-ES

/ Recruiting临床3期IIT

ESTUDIO ALEATORIZADO, DOBLE CIEGO, CONTROLADO CON PLACEBO, DE EFICACIA, TOLERANCIA Y SEGURIDAD DEL TRATAMIENTO CON EL FACTOR TRÓFICO IGF-I EN LA ATAXIA DE FRIEDREICH

开始日期2012-02-17

申办/合作机构-

100 项与美卡舍明生（Ipsen SA）相关的临床结果

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100 项与美卡舍明生（Ipsen SA）相关的转化医学

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100 项与美卡舍明生（Ipsen SA）相关的专利（医药）

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项与美卡舍明生（Ipsen SA）相关的文献（医药）

2026-04-01·Ophthalmology science

A Deep Learning Framework for Predicting Teprotumumab Treatment Response in Thyroid Eye Disease

Article

作者: Langarica, Saul ; Lee, Nahyoung Grace ; Alkhadrawi, Adham M ; Kim, Young-Tak ; Lin, Lisa Y ; Ha, Sierra K ; Do, Synho

Purpose:

To develop and evaluate a deep learning-based framework for quantifying thyroid eye disease (TED) severity before and after teprotumumab treatment, an insulin-like growth factor-1 receptor inhibitor, and to create a predictive model for forecasting individual patient responses to therapy.

Design:

A retrospective cohort study was conducted at a single institution, utilizing an image-based deep learning TED severity quantification model and a regression-based prediction model for teprotumumab treatment response.

Subjects:

One hundred eighty-four patients with a clinical diagnosis of TED and 44 individuals with normal orbital anatomy (controls) used for model training and validation. Additionally, 19 patients with pretreatment and posttreatment imaging were analyzed for treatment response.

Methods:

A deep learning classification model integrating computed tomography-based orbital volumetric features and clinical data were developed to classify TED severity (normal, mild, and severe). A severity scoring model converted classification probabilities into a continuous severity metric. A regression-based model then predicted posttreatment severity using pretreatment clinical and imaging variables. Model performance was evaluated using accuracy, area under the receiver operating characteristic curve (AUC), root mean squared error, and coefficient of determination (R ² ).

Main Outcome Measures:

Thyroid eye disease severity scores and predicted treatment response.

Results:

The classification model achieved an accuracy of 0.81 and an AUC of 0.88 in the primary cohort and an accuracy of 0.81 with an AUC of 0.86 in the treatment response cohort. Within the treatment response group (n = 19), a mean improvement of 0.194 severity points (P < 0.001) was observed after teprotumumab. The regression-based prediction model achieved a root mean squared error of 0.117 and an R ² of 0.82 in leave-one-out cross-validation and showed 89% concordance with clinician-assessed improvement.

Conclusions:

This deep learning framework demonstrates promising performance for objective TED severity quantification and exploratory prediction of teprotumumab treatment response. By integrating clinical and imaging data, this approach provides a proof-of-concept for data-driven tools that may support individualized treatment planning in TED.

Financial Disclosures:

Proprietary or commercial disclosure may be found in the Footnotes and Disclosures at the end of this article.

2026-01-21·JOURNAL OF CLINICAL ENDOCRINOLOGY & METABOLISM

Near-Adult Height Outcomes in Patients Treated With rhIGF-1 for Severe Growth Failure: Real-World IGFD Registry Data

Article

作者: Perrot, Valérie ; Maghnie, Mohamad ; Bang, Peter ; Sert, Caroline ; Ramon-Krauel, Marta ; Polak, Michel ; Woelfle, Joachim

Abstract:

Context:

The Global Increlex® Growth Forum Database (IGFD) Registry monitors real-world effectiveness and safety of recombinant human IGF-1 (rhIGF-1; Increlex® [mecasermin]) treatment in children and adolescents with severe growth failure due to severe primary IGF-I deficiency (SPIGFD).

Objective:

To report characteristics, effectiveness, and safety data from patients receiving rhIGF-1 treatment who achieved near-adult height (NAH), and determine factors that predict height gain to NAH.

Methods:

Descriptive analyses of patients included in the Global IGFD Registry (NCT00903110) who achieved NAH are reported for the overall population, treatment-naïve prepubertal (NPP) patients, and patients with Laron syndrome. Linear regression analyses of height gain to NAH are also reported.

Results:

One hundred and two patients enrolled in the Global IGFD Registry achieved NAH at data cut-off (April 20, 2023). Mean age at rhIGF-1 treatment initiation was 11.8 years; median treatment duration was 3.9 years. Mean (SD) height SD score (HtSDS) gain from rhIGF-1 initiation to NAH was 0.9 (1.1). In NPP patients, mean (SD) HtSDS gain was 1.4 (1.0). Almost half of NPP patients reached NAH within the normal range. Despite improved height in patients with Laron syndrome, only 10.5% reached NAH within the normal range; 3 patients with Laron syndrome were NPP. Treatment naivety was predictive of height gain in the overall NAH population. Safety data aligned with previous reports.

Conclusion:

In a real-world setting, despite patients with SPIGFD initiating rhIGF-1 treatment at a relatively advanced age, rhIGF-1 treatment resulted in improved NAH. The greatest improvements in height outcomes were observed in NPP patients.

Trial registration:

NCT00903110.

2025-11-01·Veterinary World

Optimization of insulin-like growth factor-1 supplementation enhances oocyte quality by modulating oxidative stress and apoptotic pathways during in vitro maturation of Kacang goat oocytes

Article

作者: Jaafar, Wan Nor Fitri Bin Wan ; Widjiati, Widjiati ; Kurniawati, Devia Yoanita ; Shabira, Zahra ; Luqman, Epy Muhammad ; Utama, Suzanita ; Aulanni’am, Aulanni’am ; Darsini, Ninik ; Hendrawan, Viski Fitri

Background and Aim: Insulin-like growth factor-1 (IGF-1) plays a crucial role in folliculogenesis and oocyte maturation by regulating oxidative stress and apoptotic pathways. However, the optimal IGF-1 concentration for small ruminant oocytes, particularly the native Kacang goat, remains undefined. This study aimed to determine the optimal IGF-1 dose for improving oocyte quality during in vitro oocyte maturation (IVM) by evaluating oxidative stress and apoptosis markers. Materials and Methods: Ovaries (n = 120) were collected from local slaughterhouses, and cumulus–oocyte complexes were aspirated, selected, and randomly divided into four groups: Control (0 ng/mL IGF-1) and treatments with 50, 100, and 150 ng/mL IGF-1 supplementation. Mature oocytes were analyzed for oxidative stress biomarkers, including superoxide dismutase (SOD-1), glutathione (GSH), and malondialdehyde (MDA), using an enzyme-linked immunosorbent assay, and for apoptosis regulators, B-cell lymphoma 2-associated X protein (BAX), B-cell lymphoma-2 (BCL-2), and cytochrome c, using immunocytochemistry. Data were analyzed using one-way analysis of variance and Duncan’s post hoc test (p < 0.05). Results: IGF-1 supplementation produced concentration-dependent effects. The 100 ng/mL group (T2) exhibited the highest SOD-1 (2.07 ± 0.60) and GSH (8.07 ± 1.79) levels (p < 0.05), while MDA increased with higher IGF-1 doses, indicating a threshold beyond which oxidative stress is induced. Anti-apoptotic BCL-2 and cytochrome c expressions peaked at 50 ng/mL IGF-1 (10.73 ± 1.56 and 11.73 ± 0.99, respectively), whereas the pro-apoptotic marker BAX was lowest in the same group and increased at higher doses. The findings suggest that 50 ng/mL IGF-1 effectively maintains redox balance and mitochondrial stability through phosphatidylinositol 3-kinase/protein kinase B-mediated regulation. Conclusion: This study demonstrates, for the 1st time, a dose-dependent, biphasic effect of IGF-1 on oxidative and apoptotic pathways in Kacang goat oocytes. An optimal concentration of 50 ng/mL IGF-1 enhances oocyte maturation by balancing antioxidant defense and anti-apoptotic mechanisms, whereas higher concentrations induce oxidative stress. These findings establish a breed-specific IVM optimization protocol that supports genetic preservation and sustainable reproductive biotechnology for indigenous goats. Keywords: apoptosis, in vitro oocyte maturation, Insulin-like growth factor-1, Kacang goat, oocyte quality, oxidative stress, phosphatidylinositol 3-kinase/protein kinase B.

项与美卡舍明生（Ipsen SA）相关的新闻（医药）

2026-09-28

·BioSpace

Eton Pharmaceuticals Announces U.S. Commercial Availability of IMPAVIDO® (miltefosine) Capsules

Eton Pharmaceuticals now holds exclusive commercialization rights for IMPAVIDO in the United States Eton has integrated full Eton Cares ® patient support, including $0 co-pay for eligible commercially insured patients and expanded patient assistance programs Centralized distribution through Anovo Specialty Pharmacy is designed to simplify access and therapy initiation for outpatient prescribing, inpatient consignment, and urgent access coordination DEER PARK, Ill., Sept. 28, 2026 (GLOBE NEWSWIRE) -- Eton Pharmaceuticals, Inc (“Eton” or “the Company”) (Nasdaq: ETON), an innovative pharmaceutical company focused on developing and commercializing treatments for rare diseases, today announced that the Company has launched IMPAVIDO ® (miltefosine) capsules. “We are excited to be relaunching IMPAVIDO and making Eton Cares available to all patients. By bringing IMPAVIDO into Eton's rare disease commercial infrastructure, we can pair this important therapy with high-touch services designed to help support healthcare providers and expand access for patients. We believe this comprehensive approach will help ensure that patients who need IMPAVIDO can access and initiate treatment without unnecessary delays. We are also working to expand hospital on-site availability across the United States through our consignment inventory stocking program,” said Sean Brynjelsen, CEO of Eton Pharmaceuticals. Clinicians seeking to prescribe IMPAVIDO can e-prescribe by selecting Anovo #5 (Memphis) or fax a patient referral form to 855-813-2039. Patients with questions regarding their prescription or healthcare providers can call Anovo at the dedicated IMPAVIDO line 877-469-4078. Hospital pharmacies seeking IMPAVIDO for inpatient use may contact Anovo Specialty Pharmacy to establish access through the hospital network. For urgent or time-sensitive patient needs, Anovo will coordinate an emergency access pathway with treating clinicians, CDC representatives, and hospital pharmacies. The pathway is designed to evaluate the fastest available source of product and delivery option based on the patient’s need-by time and the treating institution’s location. Additional product details can be found on the product website, . Important Safety Information for IMPAVIDO INDICATION IMPAVIDO® (miltefosine) is an antileishmanial drug indicated in adults and adolescents ≥12 years of age weighing ≥30 kg (66 lbs) for treatment of: • Visceral leishmaniasis due to Leishmania donovani. • Cutaneous leishmaniasis due to Leishmania braziliensis, Leishmania guyanensis, and Leishmania panamensis. • Mucosal leishmaniasis due to Leishmania braziliensis. Limitations of Use: Leishmania species evaluated in clinical trials were based on epidemiologic data. There may be geographic variation in the response of the same Leishmania species to IMPAVIDO. The efficacy of IMPAVIDO in the treatment of other Leishmania species has not been evaluated. IMPORTANT SAFETY INFORMATION Contraindications IMPAVIDO is contraindicated in pregnancy because it may cause fetal harm; in patients with Sjögren-Larsson syndrome; and in patients with hypersensitivity to miltefosine or any component of the formulation. Warnings and Precautions Embryo-Fetal Toxicity: IMPAVIDO may cause fetal harm when administered during pregnancy. Obtain a pregnancy test before initiating therapy in females of reproductive potential. Advise females of reproductive potential to use effective contraception during treatment and for 5 months after completion of therapy. If vomiting or diarrhea occurs during treatment, oral contraceptive effectiveness may be reduced; advise use of an additional non-hormonal contraceptive method. Reproductive Effects: Miltefosine caused impaired fertility in animal studies. The potential effects on human fertility have not been adequately evaluated. Renal Effects: Monitor serum creatinine during treatment and for 4 weeks after completion of therapy. Hepatic Effects: Monitor liver transaminases and bilirubin during treatment. Gastrointestinal Effects: Vomiting and diarrhea are common and may result in dehydration. Encourage adequate fluid intake during treatment . Thrombocytopenia: Monitor platelet counts during therapy in patients treated for visceral leishmaniasis. Stevens-Johnson Syndrome: Stevens-Johnson syndrome has been reported. Discontinue IMPAVIDO if an exfoliative or bullous rash develops. Adverse Reactions The most common adverse reactions (≥2%) are nausea, vomiting, diarrhea, headache, decreased appetite, dizziness, abdominal pain, pruritus, somnolence, elevated transaminases, and elevated serum creatinine. To report a suspected adverse event related to IMPAVIDO, contact Eton Pharmaceuticals, Inc. at 1-855-224-0233 or the U.S. Food and Drug Administration (FDA) at or call 1-800-FDA-1088. Please see Full Prescribing Information , including Boxed Warning regarding Embryo-Fetal Toxicity. About Eton Pharmaceuticals Eton is an innovative pharmaceutical company focused on developing and commercializing treatments for rare diseases. The Company currently has eleven commercial rare disease products: KHINDIVI ® (hydrocortisone), INCRELEX ® (mecasermin), ALKINDI SPRINKLE ® (hydrocortisone), DESMODA ™ (desmopressin acetate), GALZIN ® (zinc acetate), HEMANGEOL ® (propranolol hydrochloride), PKU GOLIKE ® , IMPAVIDO ® (miltefosine), Carglumic Acid, Betaine Anhydrous, and Nitisinone. The Company has five additional product candidates in late-stage development: AMGLIDIA ® (glyburide), ASN-001, ET-700, ET-800, and ZENEO ® hydrocortisone autoinjector. For more information, please visit our website at . Investor Relations: Lisa M. Wilson, In-Site Communications, Inc. T: 212-452-2793 E: lwilson@insitecony.com

上市批准

2026-08-30

·AI医疗观察

速递｜牛津博士创办长寿抗衰公司，融资近亿元押注犬类抗衰，为人类抗衰寻找新线索

2026年7月17日，深圳AI生物科技公司无尽方舟宣布，在一个月内连续完成种子轮和种子加轮融资，累计金额接近1亿元。创始人赵思邈正在牛津大学攻读博士，研究方向是用跨物种人工智能寻找造血干细胞衰老机制。与多数直接面向人类疾病的医疗AI公司不同，无尽方舟先把目光投向犬猫：从伴侣动物的衰老差异中寻找可干预靶点，再判断这些发现能否跨越物种。公司公开管线中的第一个项目指向IGF-1，一条与生长、体型和衰老有关的信号通路。融资和“让狗活得更久”的目标足够醒目，只是距离一款真正的药物还很远：现有材料里可以看到抗体专利和研发管线，却还看不到犬只药效、安全性和寿命数据。拿到近亿元融资，为什么先从狗开始种子轮的参与方包括Monolith、博裕资本、九合创投和未来光锥；到了种子加轮，九合创投、Monolith和博裕资本继续投入，星连资本加入。两轮各自融了多少，公司没有披露，只给出了累计近亿元这一总数，投资机构也没有分别公布交易细节。这笔钱将主要用于研发和扩充团队。赵思邈目前仍在牛津大学攻读博士。牛津大学医学科学部介绍，他正在利用跨物种AI寻找造血干细胞衰老中可以干预的机制，并曾获得2024年罗氏与HDR UK组织的AI医学竞赛冠军及长寿生物科技奖学金。他在学校里研究的问题，后来也成了无尽方舟的技术起点。联合创始人曾京昆负责药物研发。北京大学生命科学学院资料显示，他博士毕业于弗朗西斯·克里克研究所，随后在加州大学伯克利分校和Gladstone研究所从事博士后研究。2026年，他作为第一作者之一在《Nature》发表论文，利用Cas12a2识别癌细胞特异性RNA并触发细胞清除。这段经历解释了团队为什么强调模型输出必须回到实验室。公司的H2P模型试图从不同物种、器官和单细胞组学数据中寻找共同的衰老特征，再定位可能被干预的基因网络节点。实验结果重新进入模型，形成“数据—模型—实验—再学习”的循环。公司还没有公布模型的数据规模、外部测试集、预测准确率或同行评审论文，这套方法究竟能不能提高药物研发效率，还要等外部测试和实验结果。无尽方舟还把甲基化检测列为较早可能产生收入的业务，用于观察伴侣动物的生物年龄、遗传风险和健康变化。持续复测有机会积累年龄、疾病与干预结果相对应的纵向数据。不过，官网没有公布产品价格、付费用户、合作医院或销售收入，这项业务目前做到了多大规模，外界还无从得知。这项专利离真正的延寿药还有很长距离。中国授权专利CN118791608B的发明人包括曾京昆、赵思邈等。实施例完成了犬IGF-1重组蛋白制备、抗体筛选、表达纯化和亲和力优化，部分抗体的亲和力提高2倍以上。团队由此得到了一批可以结合犬IGF-1的候选抗体，下一步才是犬只给药、剂量摸索和安全性研究。专利中没有这些数据，也没有疾病改善或生存曲线。还有一个问题是专利权属。登记的权利人为深圳仁盈生物科技有限公司和上海津伦克咨询管理有限公司，并非无尽方舟，目前也没有公开的转让或许可文件。无尽方舟把IGF-1列为ARK-001，并在管线图中写入未来获批和上市时间，但这些日期只是公司设定的研发目标。获得专利、形成候选药物和得到监管批准，仍是三个不同阶段。从一支抗体到一款药，中间还缺什么犬的品种差异提供了罕见的天然对照。覆盖超过1000家宠物医院、约1329万只犬临床记录的研究显示，犬的预期寿命总体随体型减小而上升。另一项覆盖118个品种、超过4万只犬的研究发现，大型犬的寿命劣势与更高的癌症死亡风险有关。犬又与人共同生活，比实验室小鼠更接近真实环境。 IGF-1因此成为一个有依据但没有定论的入口。犬类研究发现，血清IGF-1水平总体随体重增加而升高，相关遗传变异也是犬体型差异的重要因素。但关联不等于干预效果。IGF-1还参与生长、代谢和组织修复，给药时间、剂量、性别和副作用都可能改变结果。小鼠研究已经显示这种分歧。2003年的Igf1r杂合敲除实验报告雌鼠平均寿命增加33%，雄鼠增加16%但没有达到统计学显著；后来的复核没有观察到雄鼠获益，雌鼠增幅低于5%。2018年，针对IGF-1受体的单克隆抗体使老年雌鼠中位寿命增加9%。无尽方舟专利针对的是IGF-1本身，并非IGF-1受体，药物到了犬身上会产生什么结果，仍要通过犬只试验回答。 AI在这里更实际的作用是缩小搜索范围。某个靶点在小鼠中有效，进入犬或人后可能失效。跨物种模型如果能优先找出在犬、猫、小鼠和人类细胞中共同出现的变化，就可能减少一部分无效实验。这套模型有没有用，最终要看它能否提前找到经得住跨物种实验的靶点，而不是一次能列出多少候选基因。甲基化检测则承担另一项任务。药物可能多年不能产生收入，连续检测却能较早连接宠物主、医院和实验室，并为模型补充真实世界数据。如果策略奏效，公司出售的是生物年龄和健康风险评估，积累的则是同一只动物在不同年龄、疾病和干预阶段的连续记录。一次性体检和单点化验可能因此向长期跟踪转变。接下来12个月，公司需要跨过几道具体门槛：说明ARK-001的权属或授权关系，拿出犬只药代、剂量和安全性数据，让模型接受独立测试，并让甲基化检测进入可以核实的付费场景。在这些节点跨过去之前，管线图中的获批和上市日期仍只是企业设定的目标。未来1至3年，即使候选抗体显示效果，首个产品也未必以“延长寿命”获批。关节炎、慢性肾病、胰腺炎等疾病拥有更明确的临床终点，更容易判断治疗是否有效。无尽方舟已经把这些疾病列入管线。先改善具体疾病，再评估健康寿命，可能比直接证明犬能多活几年更现实。这条路径也会牵动国内的宠物医院、检测实验室、抗体生产企业和兽药公司。国内并不缺生物制造和医疗渠道，缺的是可以长期跟踪、标准一致的数据。家庭自费、兽药注册、数据合规、跨医院标准和复诊意愿都会决定产品能走多远。犬类结果即使成功，也不能直接推到人身上，只能帮助研究人员判断哪些机制更值得进入人体研究。近亿元融资买到的是时间；最终答案仍要写在动物生存曲线、安全性数据、独立模型验证和监管文件里。全展商分析医疗大模型医学影像药物研发 · 康复机器人与智能诊疗设备脑机接口与神经医疗中国公司 · AI医疗公司数字健康公司 · 减重降糖进展大会前瞻 · 大会Day1~2回顾大会Day 3回顾 · 大会Day 4回顾全展商分析 · 可穿戴设备综合健康管理 · 康复与功能改善慢病管理 · 精准诊疗英伟达AI医疗布局 · GTC中国公司盘点 AI医疗底座 · 手术机器人 · 蛋白质设计

2026-08-27

·BioSpace

Eton Pharmaceuticals to Participate at 2026 Wells Fargo Healthcare Conference

DEER PARK, Ill., Aug. 27, 2026 (GLOBE NEWSWIRE) -- Eton Pharmaceuticals, Inc (“Eton” or the “Company”) (Nasdaq: ETON), an innovative pharmaceutical company focused on developing and commercializing treatments for rare diseases, today announced that members of the Company’s executive leadership team will host 1x1 meetings on September 9, 2026 at the 2026 Wells Fargo Healthcare Conference being held September 8-10, 2026 in Boston, MA. To schedule a 1x1 meeting with the Company, please contact your Wells Fargo institutional sales representative. About Eton Pharmaceuticals Eton is an innovative pharmaceutical company focused on developing and commercializing treatments for rare diseases. The Company currently has eleven commercial rare disease products: KHINDIVI ® , INCRELEX ® , ALKINDI SPRINKLE ® , DESMODA™, GALZIN ® , HEMANGEOL ® , PKU GOLIKE ® , IMPAVIDO ® (September 2026 launch), Carglumic Acid, Betaine Anhydrous, and Nitisinone. The Company has five additional product candidates in late-stage development: ASN-001, AMGLIDIA ® , ET-700, ET-800, and ZENEO ® hydrocortisone autoinjector. For more information, please visit our website at . Investor Relations: Lisa M. Wilson, In-Site Communications, Inc. T: 212-452-2793 E: lwilson@insitecony.com Source: Eton Pharmaceuticals.

100 项与美卡舍明生（Ipsen SA）相关的药物交易

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外链

KEGG	Wiki	ATC	Drug Bank
-	美卡舍明生（Ipsen SA）	H01AC03	DB01277

研发状态

批准上市

10 条最早获批的记录,

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适应症	国家/地区	公司	日期
成长不全	加拿大	Ipsen Biopharmaceuticals, Inc.	2020-12-17
原发性胰岛素样生长因子-1 缺乏症	加拿大	Ipsen Biopharmaceuticals, Inc.	2020-12-17
侏儒症	澳大利亚	Ipsen SA	2019-11-22
生长障碍	美国	Ipsen, Inc.	2005-08-30
生长激素缺乏症	美国	Ipsen, Inc.	2005-08-30
胰岛素样生长因子I缺乏症	美国	Ipsen, Inc.	2005-08-30

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适应症	最高研发状态	国家/地区	公司	日期
克罗恩病	临床3期	美国	Tercica, Inc.	2008-10-01
Laron综合征	临床3期	美国	Ipsen SA	1991-05-20
杜氏肌营养不良症	临床2期	美国	Ipsen SA	2010-11-01
糖尿病	临床2期	美国	Tercica, Inc.	-
多发性硬化症	药物发现	美国	Genentech, Inc.	-

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研究	分期	人群特征	评价人数	分组	结果	评价	发布日期


NCT00903110 (Global IGFD Registry, ENDO2025)	N/A	胰岛素样生长因子I缺乏症	45	rhIGF-1 (Naive Prepubertal)	鹽糧構簾襯衊選繭鏇簾(範鏇繭獵壓襯艱醖觸襯) = 窪餘醖衊蓋衊網顧獵蓋網積網構膚艱鹹廠範獵 (選廠顧蓋艱願獵糧蓋膚 )	积极	2025-07-12
NCT00903110 (ESPE2024)	N/A	原发性胰岛素样生长因子-1 缺乏症	215	Increlex (mecasermin) (GH-naïve patients)	獵壓簾積鹽網選鏇鑰淵(製淵遞醖構鏇窪蓋構鹹) = 壓壓築獵壓積繭餘蓋齋構壓憲範壓鑰遞簾築醖 (襯壓鏇網壓網觸獵鏇衊 )	积极	2024-11-16
				Increlex (mecasermin) (with previous GH treatment)	獵壓簾積鹽網選鏇鑰淵(製淵遞醖構鏇窪蓋構鹹) = 艱餘鹹艱範鏇淵夢網構構壓憲範壓鑰遞簾築醖 (襯壓鏇網壓網觸獵鏇衊 )
NCT00684957 (CTgov)	临床1/2期	生长激素缺乏症	5	Recombinant Human Growth Hormone (Recombinant Human Growth Hormone First, Then Recombinant Human IGF-1)	窪醖窪願積憲鹹網膚壓(獵範蓋鹽膚夢夢願夢襯) = 壓膚糧繭夢醖築憲範壓壓壓選艱繭夢衊餘觸糧 (餘願顧鹽鹹憲齋鬱觸鑰, 網顧觸夢構餘範顧廠夢 ~ 夢鹽鬱壓鏇鹽窪廠艱壓) 更多	-	2024-07-17
				Recombinant Human Growth Hormone (Recombinant Human IGF-1 First, Then Recombinant Human Growth Hormone)	窪醖窪願積憲鹹網膚壓(獵範蓋鹽膚夢夢願夢襯) = 壓衊觸齋願築蓋齋淵壓壓壓選艱繭夢衊餘觸糧 (餘願顧鹽鹹憲齋鬱觸鑰, 窪築襯鹹糧觸鬱糧獵築 ~ 鑰簾鬱鏇簾簾願廠糧積) 更多
NCT00571727 (CTgov)	临床2/3期	Laron综合征	92	Mecasermin	壓製壓築構築築壓鹽觸(齋觸觸顧觸糧艱窪築選) = 壓築廠廠積製鑰觸窪積夢壓鑰築構觸淵醖夢壓 (積積糧衊網鏇範蓋願範, 1.7) 更多	-	2023-07-06
NCT00903110 (ESPE2022)	N/A	胰岛素样生长因子I缺乏症 GH levels \| IGF binding protein-3 (IGFBP-3) \| GH binding protein (GHBP) ... 更多	306	mecasermin (Increlex ®)	遞築鹹繭積鹹齋齋廠膚(衊醖鹽餘願窪遞積鏇範) = 顧製繭膚顧選齋壓糧艱壓襯憲積獵簾積鏇齋願 (網觸網簾憲糧窪築窪餘 )	-	2022-09-15
NCT01525901 (CTgov)	临床2期	Phelan-McDermid 综合征	19	Insulin-Like Growth Factor-1 (IGF-1) (Insulin-Like Growth Factor-1 (IGF-1))	衊簾夢衊網鏇鹽獵繭憲(繭築壓襯簾齋窪淵壓願) = 衊簾餘醖淵範襯醖蓋鹹窪鏇夢鏇夢鑰壓鏇餘範 (鏇餘範範糧顧憲鹽衊窪, 鑰餘艱觸願顧衊艱鏇膚 ~ 鹹顧積積衊艱鑰膚艱艱) 更多	-	2022-05-12
				Normal saline (Normal Saline)	衊簾夢衊網鏇鹽獵繭憲(繭築壓襯簾齋窪淵壓願) = 窪遞願網鏇願憲構積糧窪鏇夢鏇夢鑰壓鏇餘範 (鏇餘範範糧顧憲鹽衊窪, 廠醖獵觸鬱網醖夢獵願 ~ 鹽廠鏇鹹獵鹹齋淵壓簾) 更多
NCT00903110 (European Increlex® Growth Forum Database Registry, Pubmed \| Eur J Endocrinol)	N/A	Laron综合征	242	rhIGF1 therapy	鹹鹽選齋範蓋醖積鬱糧(襯廠廠襯窪鹽網憲衊遞) = experienced by 65.3% of patients; hypoglycaemia was most common 製淵廠簾廠鑰鏇鹽窪醖 (廠觸鑰艱醖廠觸願製醖 )	-	2021-02-01
NCT01207908 (CTgov)	临床1/2期	杜氏肌营养不良症	44	IGF-1 (IGF-1)	觸獵觸齋選簾顧醖遞鹹(憲鑰壓鬱鏇鹽鹽鹽製鏇) = 積蓋艱膚繭夢獵觸淵鹽鏇憲蓋構鏇簾範壓構鹽 (鏇獵壓糧鹹鹹糧簾簾醖, 32.4) 更多	-	2021-01-20
				steroid+Prednisone+Deflazacort (Standard Steroid Treatment Alone)	觸獵觸齋選簾顧醖遞鹹(憲鑰壓鬱鏇鹽鹽鹽製鏇) = 衊鬱夢觸窪鏇膚鬱築窪鏇憲蓋構鏇簾範壓構鹽 (鏇獵壓糧鹹鹹糧簾簾醖, 50.2) 更多
NCT00764699 (CTgov)	临床2/3期	克罗恩病	3	rhIGF (Increlex)	簾繭膚鑰鹹蓋簾製窪願(積鹹襯襯衊窪網選蓋夢) = 廠願鬱鹹獵蓋鑰製憲範鹹鬱糧鑰窪顧製艱製繭 (獵鬱積淵繭廠遞獵選襯, 襯壓積淵範蓋網廠遞獵 ~ 淵窪獵網構醖構鑰窪獵) 更多	-	2018-03-02
NCT00903110 (EU-IGFD, ESPE2016)	N/A	原发性胰岛素样生长因子-1 缺乏症	221	Increlex	構繭艱醖蓋鑰鹹觸蓋艱(選遞願淵製選淵遞壓醖) = 13% vs 11% 選廠餘窪製齋遞製醖窪 (製襯膚鏇選選夢襯襯選 ) 更多	-	2016-09-10
				Increlex