NCT05897424进行中（未招募）2 期
A Phase 2, Single Arm, Open Label Extension Study, Evaluating the Long-Term Safety and Clinical Efficacy of SAR447537 (INBRX-101) in Adults With Alpha-1 Antitrypsin Deficiency (AATD) Emphysema
适应症
干预措施
相关药物
试验速览
- 阶段
- 2 期
- 状态
- 进行中（未招募）
- 发起方
- Sanofi
- 入组人数
- 185
- 试验地点
- 80
- 主要终点
- Long-term safety and tolerability
研究概览
简要总结
Phase 2 open label extension study to evaluate SAR447537 (INBRX-101) in adults with AATD emphysema
详细描述
This is a Phase 2, Single Arm, Open Label Extension Study, Evaluating the Long-Term Safety and Clinical Efficacy of SAR447537 (INBRX-101) in Adults With Alpha-1 Antitrypsin Deficiency (AATD) Emphysema.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 18 Years 至 80 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Males or females 18-80 years of age, inclusive, at the time of screening
- •Diagnosis of AATD
- •Evidence of emphysema secondary to AATD
- •FEV1 of ≥ 30% predicted at screening and post-bronchodilator FEV1/FVC<0.7
- •Current non-smoking status
排除标准
- •For newly identified participants
- •Receipt of A1PI augmentation therapy within 5 weeks prior to the first dose of study drug
- •Known or suspected allergy to components of SAR447537, A1PI or human IgG
- •Uncontrolled diabetes mellitus despite adequate antidiabetic pharmacologic treatment with a screening HbA1c value ≥9%
- •Received IV immunoglobulins, monoclonal antibodies and/or other biologic therapies within 30 days
- •On waiting list for lung or liver transplant
- •Acute respiratory tract infection or COPD exacerbation within 4 weeks prior to or during screening
- •Evidence of decompensated cirrhosis
- •Active cancers or has a history of malignancy within 5 years prior to screening
- •History of unstable cor pulmonale
- •The above information is not intended to contain all considerations relevant to a participant's potential participation in a clinical trial.
研究组 & 干预措施
SAR447537 (INBRX-101)
Experimental
A1PI, Recombinant, Bivalent Fc Fusion Protein, in a solution for intravenous injection
干预措施: SAR447537 (Drug)
结局指标
主要结局
Long-term safety and tolerability
时间窗: 3 years
Incidence of all treatment emergent adverse events (TEAEs), TEAEs ≥ Grade 3, serious adverse events (SAEs), TEAEs leading to discontinuation from SAR447537, and adverse events of special interest (AESIs) (including infusion-related reactions).
次要结局
- Change in lung density by quantitative computerized tomography (CT)(3 years)
- Trough SAR447537 concentration changes(3 years)
- Trough serum functional AAT (fAAT) concentration changes(3 years)
- Covariate Analysis: Biometric Values: Weight(3 years)
- Covariate Analysis: Biometric Values: Height(3 years)
- Covariate Analysis: Biometric Values: Age(3 years)
- Covariate Analysis: Biometric Values: Sex(3 years)
- Anti-drug antibodies(3 years)
研究者
研究点 (80)
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相关资讯
Sanofi's Efdoralprin Alfa Demonstrates Superior Efficacy Over Standard Care in Alpha-1 Antitrypsin Deficiency Phase 2 Trial- Sanofi's investigational efdoralprin alfa achieved mean increases in functional alpha-1 antitrypsin trough levels more than three times greater than plasma-derived therapy in the ElevAATe phase 2 study.
- The recombinant therapy maintained normal functional AAT levels for 100% of days during the 32-week study compared to 41% for standard-of-care treatment.
- Efdoralprin alfa represents the first potential therapy to sustain normal functional AAT levels with less frequent dosing, offering hope for patients with this rare genetic condition.4 months agoSanofi's Efdoralprin Alfa Achieves Primary Endpoints in Phase 2 Alpha-1 Antitrypsin Deficiency Study- Sanofi's efdoralprin alfa demonstrated statistically significant superiority over standard plasma-derived therapy in the ElevAATe phase 2 study, meeting all primary and key secondary endpoints for alpha-1 antitrypsin deficiency emphysema treatment.
- The recombinant therapy achieved consistently higher functional AAT levels with less frequent dosing (every 3-4 weeks) compared to weekly plasma-derived treatments, potentially offering significant convenience improvements for patients.
- Results support efdoralprin alfa's potential as the first restorative recombinant therapy that normalizes and maintains functional AAT levels, addressing a critical unmet need in AATD management.
- The therapy was well tolerated with a similar adverse event profile to current standard of care, with Sanofi planning to engage regulatory authorities on next steps following these positive results.10 months ago