Cenegermin for the Treatment of Moderate or Severe Neurotrophic Keratopathy: Results from a Prospective, Phase IV, Open-Label Study in China.

IF 3.2 3区医学 Q2 OPHTHALMOLOGY

Ophthalmology and Therapy Pub Date : 2025-10-05 DOI:10.1007/s40123-025-01253-5

Suxia Li, Jianjiang Xu, Jin Yuan, Zhiqiang Pan, Giacomo Siri, Scott G Hauswirth, Flavio Mantelli, Weiyun Shi

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引用次数: 0

Abstract

Introduction: Neurotrophic keratopathy (NK) is a rare degenerative disease that can lead to epithelial breakdown, corneal ulceration, and potentially perforation and vision loss. Cenegermin is a recombinant human nerve growth factor approved for stage 2/3 NK, though data on its efficacy outside of United States/European populations are limited.

Methods: This prospective, phase IV, open-label, multicenter study investigated the efficacy, safety, and pharmacokinetics (PK) of cenegermin eye drops in Chinese patients with stage 2/3 NK in a routine clinical setting (CTR20220066). Patients received cenegermin eye drops (20 mcg/mL; 6 times/day at 2-hour intervals) for 8 weeks with follow-up to week 56. The primary endpoint was corneal healing at week 8 per investigator assessment. Additional endpoints included lesion worsening, corneal healing through week 56, safety, and PK.

Results: Of the 28 patients receiving cenegermin, 22/26 (84.6%, 95% confidence interval 65.1-95.6%; 2 missing) achieved corneal healing at week 8; 3/22 (13.6%) reported recurrence during 48-week follow-up but 20/22 (90.9%) were healed at week 56; and 2/28 (7.1%) experienced lesion worsening during 8-week treatment. In addition, 25/28 (89.3%) experienced ≥ 1 treatment-emergent adverse event (TEAE); for the majority (23/28, 82.2%) of these patients, TEAEs were mild/moderate. None of the serious TEAEs reported by 10 (35.7%) patients were assessed as related to cenegermin or led to treatment discontinuation. Eye pain was the most common cenegermin-related TEAE (5/28, 17.9%), primarily limited to the treatment period.

Conclusions: These findings demonstrate that cenegermin eye drops are an effective, generally well-tolerated treatment in Chinese patients with stage 2/3 NK, supporting their use in this patient population.

Trial registration: CTR20220066.

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Cenegermin用于治疗中度或重度神经营养性角膜病变：来自中国一项前瞻性、IV期、开放标签研究的结果

神经营养性角膜病变（NK）是一种罕见的退行性疾病，可导致上皮破坏，角膜溃疡，潜在穿孔和视力丧失。Cenegermin是一种重组人神经生长因子，被批准用于2/3期NK，但其在美国/欧洲以外人群的疗效数据有限。方法：这项前瞻性、开放标签、多中心的IV期研究在常规临床环境（CTR20220066）中调查了genegermin滴眼液在中国2/3期NK患者中的疗效、安全性和药代动力学（PK）。患者接受genegermin滴眼液（20 mcg/mL， 6次/天，间隔2小时），持续8周，随访至第56周。主要终点是第8周的角膜愈合。其他终点包括病变恶化、第56周的角膜愈合、安全性和pkk。结果：在接受genegermin治疗的28例患者中，22/26（84.6%，95%置信区间65.1-95.6%，2例缺失）在第8周实现角膜愈合；3/22（13.6%）患者随访48周复发，20/22（90.9%）患者随访56周痊愈；2/28（7.1%）患者在8周治疗期间出现病变恶化。此外，25/28（89.3%）患者出现≥1次治疗不良事件（TEAE）；大多数患者（23/28,82.2%）teae为轻/中度。10例（35.7%）患者报告的严重teae均未被评估为与肾上腺素相关或导致停止治疗。眼部疼痛是最常见的与萌芽相关的TEAE(5/ 28,17.9%)，主要局限于治疗期间。结论：这些研究结果表明，绿芽明滴眼液在中国2/3期NK患者中是一种有效且耐受性良好的治疗方法，支持在这一患者群体中使用。试验注册号：CTR20220066。

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来源期刊

Ophthalmology and Therapy OPHTHALMOLOGY-

CiteScore

4.20

自引率

3.00%

发文量

157

审稿时长

6 weeks

期刊介绍： Aims and Scope Ophthalmology and Therapy is an international, open access, peer-reviewed (single-blind), and rapid publication journal. The scope of the journal is broad and will consider all scientifically sound research from preclinical, clinical (all phases), observational, real-world, and health outcomes research around the use of ophthalmological therapies, devices, and surgical techniques. The journal is of interest to a broad audience of pharmaceutical and healthcare professionals and publishes original research, reviews, case reports/series, trial protocols and short communications such as commentaries and editorials. Ophthalmology and Therapy will consider all scientifically sound research be it positive, confirmatory or negative data. Submissions are welcomed whether they relate to an international and/or a country-specific audience, something that is crucially important when researchers are trying to target more specific patient populations. 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