NCT05935215招募中3 期
A Multicenter, Single Arm, Open-label Study to Evaluate Efficacy and Safety of Switching From Anti-C5 Antibody Treatment to Iptacopan Treatment in Study Participants With aHUS
适应症
干预措施
相关药物
试验速览
- 阶段
- 3 期
- 状态
- 招募中
- 入组人数
- 50
- 试验地点
- 31
- 主要终点
- Percentage of participants free of TMA manifestation
研究概览
简要总结
The purpose of this Phase 3 study is to evaluate the efficacy and safety of iptacopan upon switching from anti-C5 antibody to iptacopan treatment in study participants with aHUS.
详细描述
The study is designed as a multicenter, single-arm, open label study to evaluate the efficacy and safety of iptacopan upon switching from anti-C5 antibody to iptacopan treatment in participants with aHUS. It consists of a screening period of up to 14 weeks followed by a 12-Month Core Treatment period and 12-Month Extension Treatment period.
The study will assess the effects of iptacopan on a range of efficacy assessments relevant to aHUS.
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 100 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Male and female adult participants ≥ 18 years of age with diagnosis of aHUS for whom etiologies of other types of TMA and non-aHUS kidney disease have been excluded.
- •. Currently on the recommended (as per label) dosage regimen of anti-C5 antibody treatment, for at least 3 months prior to entering the screening period.
- •In the opinion of the investigator the participant has responded to anti-C5 antibodytreatment prior to screening and has clinical evidence of response (in absence of PE/PI) during the Screening period.
- •Clinical evidence of response to anti-C5 antibody treatment (in absence of PE/PI) confirmed during the Screening period by central laboratory at two visits 12 weeks apart. Clinical evidence of response is defined as:
- •Hematological normalization in platelet count ≥150 x 10^9/L and LDH below upper limit of normal [ULN], and
- •Stable kidney function as defined by serum creatinine values within ±15% during the Screening period
- •Vaccination against Neisseria meningitidis and Streptococcus pneumoniae infections is required prior to the start of treatment with iptacopan.
- •If not received previously or if a booster is required, vaccination against Haemophilus influenzae infection, should be given, if available and according to local regulations.
排除标准
- •History of aHUS disease relapse while on anti-C5 antibody treatment.
- •eGFR < 30 ml/min/1.73m^2
- •Active infection or history of recurrent invasive infections caused by encapsulated bacteria, i.e., meningococcus, pneumococcus (eg., N. meningitidis, S. pneumoniae) or H. influenzae.
- •Participants with sepsis or active systemic bacterial, viral (including COVID-19) or fungal infection within 14 days prior to study treatment administration.
- •Kidney, bone marrow transplant (BMT)/hematopoietic stem cell transplant (HSCT), heart, lung, small bowel, pancreas, liver transplantation or any other cell or solid organ transplantation
- •Female patients who are pregnant or breastfeeding, or intending to conceive during the course of the study
- •Any medical condition deemed likely to interfere with the patient's participation in the study
研究组 & 干预措施
iptacopan 200 mg b.i.d.
Experimental
open label arm of iptacopan 200 mg b.i.d.
干预措施: Iptacopan (Drug)
结局指标
主要结局
Percentage of participants free of TMA manifestation
时间窗: 12 months
Absence of thrombotic microangiopathy (TMA) manifestation, without use of anti-C5 antibody, during the 12 months of iptacopan treatment following the switch of treatment from an anti-C5 antibody to iptacopan treatment.
次要结局
- Time to TMA manifestation(12 months, 24 months)
- Percentage of participants with TMA related events.(month 12 and month 24)
- Number of participants who require dialysis(month 12 and month 24)
- Change from baseline in platelets(Baseline, month 12, month 24)
- Change from baseline in UPCR(Baseline, month 12, month 24)
- Change from baseline in eGFR(Baseline, month 12, month 24)
- Percentage of participants free of TMA manifestation(24 months)
- Change from baseline in LDH(Baseline, month 12, month 24)
- Change from baseline in serum creatinine(Baseline, month 12, month 24)
- Percentage of participants free of TMA manifestation in study participants with functionally significant mutations in complement genes or positive anti FH antibodies(12 months, 24 months)
- Change from baseline in hemoglobin(Baseline, month 12, month 24)
- Change from baseline in CKD stage(Baseline, month 12, month 24)
研究者
研究点 (31)
Loading locations...
相似试验
已完成
3 期
Study of Efficacy and Safety of Twice Daily Oral Iptacopan (LNP023) in Adult PNH Patients Who Are Naive to Complement Inhibitor TherapyParoxysmal Nocturnal Hemoglobinuria (PNH)NCT04820530Novartis Pharmaceuticals40
已完成
3 期
Efficacy and Safety of Iptacopan (LNP023) in Adult Patients With Atypical Hemolytic Uremic Syndrome Naive to Complement Inhibitor TherapyAtypical Hemolytic Uremic SyndromeNCT04889430Novartis Pharmaceuticals34
终止
2 期
Basket Study to Assess Efficacy, Safety and PK of Iptacopan (LNP023) in Autoimmune Benign Hematological DisordersImmune Thrombocytopenia (ITP)Cold Agglutinin Disease (CAD)NCT05086744Novartis Pharmaceuticals19
已完成
3 期
Single Arm, Open Label Trial With Iptacopan Treatment for 24 Weeks, in Patients on Stable Regimen of Anti-C5 Who Switch to Iptacopan.Paroxysmal Nocturnal HemoglobinuriaNCT05630001Novartis Pharmaceuticals52
进行中（未招募）
2 期
Iptacopan in Patients With ANCA Associated VasculitisAnti-Neutrophil Cytoplasm Antibodies (ANCA) Associated VasculitisNCT06388941Novartis Pharmaceuticals84