The Effectiveness of NP001 on the Long-Term Survival of Patients with Amyotrophic Lateral Sclerosis.

IF 3.9 3区 工程技术 Q2 BIOCHEMISTRY & MOLECULAR BIOLOGY Biomedicines Pub Date : 2024-10-16 DOI:10.3390/biomedicines12102367
Bruce D Forrest, Namita A Goyal, Thomas R Fleming, Paige M Bracci, Neil R Brett, Zaeem Khan, Michelle Robinson, Ari Azhir, Michael McGrath
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Abstract

Background/objectives: The aim of this study was to estimate the effect of a 6 months' treatment course of the innate immune modulator NP001 (a pH-adjusted intravenous formulation of purified sodium chlorite), on disease progression, as measured by overall survival (OS) in patients with amyotrophic lateral sclerosis.

Methods: Blinded survival data were retrospectively collected for 268 of the 273 patients who had participated in two phase 2 placebo-controlled clinical trials of NP001 (ClinicalTrials.gov: NCT01281631 and NCT02794857) and received at least one dose of either 1 mg/kg or 2 mg/kg of NP001 as chlorite based on actual body weight, or placebo. Kaplan-Meier methods were used on the intent-to-treat population to estimate survival probabilities.

Results: In the overall population, the median OS was 4.8 months (2.7 years [95% CI: 2.3, 3.5] in the 2 mg/kg NP001group and 2.3 years [95% CI: 1.8, 2.9] in the placebo group). Hazard ratio (HR): 0.77 (95% CI: 0.57, 1.03), p = 0.073. Among patients aged ≤ 65 years, the median OS for the 2 mg/kg NP001 group was 10.8 months (3.3 years [95% CI: 2.4, 3.8] in the 2 mg/kg NP001 group and 2.4 years [95% CI: 1.7, 3.3] in the placebo group). HR: 0.69 (95% CI: 0.50, 0.95). No differences were observed in the 1 mg/kg NP001 group or in patients aged > 65 years.

Conclusions: The findings from this study suggest that a 6 months' treatment course of NP001 resulted in a 4.8-month increase in overall survival in patients with ALS. The findings from this study indicate that targeting inflammation associated with the innate immune system may provide a pathway for new therapeutic options for the treatment of ALS.

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NP001 对肌萎缩侧索硬化症患者长期生存的影响
背景/目的:本研究旨在估算先天性免疫调节剂NP001(一种经pH值调整的纯化亚氯酸钠静脉注射制剂)6个月疗程对肌萎缩侧索硬化症患者疾病进展的影响(以总生存期(OS)衡量):回顾性收集了273名患者中268人的盲法生存数据,这些患者参加了NP001的两项安慰剂对照2期临床试验(ClinicalTrials.gov:NCT01281631和NCT02794857),并根据实际体重接受了至少一次剂量的1毫克/千克或2毫克/千克亚氯酸盐NP001或安慰剂。对意向治疗人群采用卡普兰-梅耶法估算生存概率:在总体人群中,中位生存期为4.8个月(2毫克/千克NP001组为2.7年[95% CI:2.3,3.5],安慰剂组为2.3年[95% CI:1.8,2.9])。危险比(HR):0.77(95% CI:0.57,1.03),p = 0.073。在年龄≤65岁的患者中,2 mg/kg NP001组的中位OS为10.8个月(2 mg/kg NP001组为3.3年[95% CI:2.4,3.8],安慰剂组为2.4年[95% CI:1.7,3.3])。HR:0.69(95% CI:0.50,0.95)。1毫克/千克NP001组和年龄大于65岁的患者中未观察到差异:本研究结果表明,NP001 6 个月的疗程可使 ALS 患者的总生存期延长 4.8 个月。这项研究的结果表明,针对与先天性免疫系统相关的炎症可能会为治疗 ALS 提供新的治疗方案。
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来源期刊
Biomedicines
Biomedicines Biochemistry, Genetics and Molecular Biology-General Biochemistry,Genetics and Molecular Biology
CiteScore
5.20
自引率
8.50%
发文量
2823
审稿时长
8 weeks
期刊介绍: Biomedicines (ISSN 2227-9059; CODEN: BIOMID) is an international, scientific, open access journal on biomedicines published quarterly online by MDPI.
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