A multicenter, randomized, double-blind, parallel group, placebo-controlled studyto evaluate the efficacy and safety of iptacopan (LNP023) in complement 3glomerulopathy

Novartis Healthcare Pvt Ltd4 个研究点分布在 1 个国家目标入组 68 人开始时间: 2021年11月19日最近更新: 2022年5月12日

试验速览

阶段: 3 期
状态: 招募中
发起方
入组人数: 68
试验地点: 4
主要终点: The primary objective of the study is:

研究概览研究设计入排标准结局指标研究者研究点记录与标识符相似试验

研究概览

简要总结

The purpose of this Phase 3 study is to evaluate the efficacy and safety of iptacopan compared to placebo (and standard of care) in patients with native C3G. The study aims to demostrate a reduction in proteinuria and improvement in estimated Glomerular Filtration Rate (eGFR) in participants treated with iptacopan compared to placebo. Kidney biopsies will be performed to assess histopathological reductions in glomerular inflammation and Complement 3 (C3) deposition, and improvement in fatigue will be evaluated. Complement alternative pathway (AP) dysregulation is believed to underlie the clinical

manifestations and progression of C3G. Serum C3 and other complement pathway biomakers will be assessed to demonstrate that ipatacopan reduces AP activity and targets the underlying cause of disease.

研究设计

研究类型: Interventional
分配方式: Computer generated randomization
盲法: Participant and Investigator Blinded

入排标准

年龄范围: 18.00 Year(s) 至 60.00 Year(s)（—）
性别: All

入选标准

•Diagnosis of C3G as confirmed by renal biopsy within 12 months prior to enrollment (a biopsy report, review and confirmation by the Investigator is required).
•If such a biopsy is not available, confirmation may be obtained using tissue from the Day -45 biopsy for local assessment.
•Prior to randomization, all participants must have been on a maximally recommended or tolerated dose of an angiotensin converting enzyme inhibitor (ACEI) or angiotensin receptor blocker (ARB) for at least 90 days.
•The doses of other antiproteinuric medications including mycophenolic acids, corticosteroids and mineralocorticoid receptor antagonists should be stable for at least 90 days prior to randomization.
•Reduced serum C3 (defined as less than 0.85 x lower limit of the central laboratory normal range) at Screening.
•UPCR â‰¥ 1.0 g/g sampled from the first morning void urine sample at Day -75 and Day -
•Estimated GFR (using the CKD-EPI formula) or measured GFR â‰¥ 30 ml/min/1.73m2 at screening and Day -
•Vaccination against Neisseria meningitidis infection prior to the start of study treatment.
•If the patient has not been previously vaccinated, or if a booster is required, the vaccine should be given according to local regulations at least 2 weeks prior to the first administration of study treatment.
•If study treatment has to start earlier than 2 weeks post vaccination, prophylactic antibiotic treatment should be initiated.
•If not previously vaccinated, or if a booster is required, vaccination against Streptococcus pneumoniae and Haemophilus influenzae infections should be given, if available and according to local regulations, at least 2 weeks prior to the first study treatment administration.

排除标准

•Participants who have received any cell or organ transplantation, including kidney transplantation.
•Rapidly progressive crescentic glomerulonephritis defined as a 50% decline in the eGFR within 3 months) with renal biopsy findings of glomerular crescent formation seen in at least 50% of glomeruli.
•Renal biopsy showing interstitial fibrosis/tubular atrophy (IF/TA) of more than 50%.
•Monoclonal gammopathy of undetermined significance (MGUS) confirmed by the measurement of serum free light chains or other investigation as per local standard of care.
•Participants with an active systemic bacterial, viral or fungal infection within 14 days prior to study treatment administration or the presence of fever â‰¥ 38Â°C (100.4Â°F) within 7 days prior to study treatment administration.
•A history of recurrent invasive infections caused by encapsulated organisms, e.g., meningococcus or pneumococcus.
•The use of inhibitors of complement factors (e.g., Factor B, Factor D, C3 inhibitors, anti Complement 5 (C5) antibodies, C5a receptor antagonists) within 6 months prior to the Screening visit.
•The use of immunosuppressants (except mycophenolic acids), cyclophosphamide or systemic corticosteroids at a dose >7.5 mg/day (or equivalent for a similar medication) within 90 days of study drug administration.