2024-515926-10-00招募中3 期
An open-label, single-arm, multicenter, phase 3 study to assess pharmacokinetics, safety and tolerability of iptacopan in pediatric PNH patients aged 2 to <18 years of age
试验速览
- 阶段
- 3 期
- 状态
- 招募中
- 入组人数
- 5
- 试验地点
- 7
- 主要终点
- Safety evaluations, including AEs, SAEs, laboratory parameters, vital signs, and cardiovascular parameters.
研究概览
简要总结
To evaluate the safety and tolerability of iptacopan during the 26-week treatment period. To characterize the PK of iptacopan.
入排标准
- 年龄范围
- 0 years 至 17 years（0-17 Years）
- 接受健康志愿者
- 是
入选标准
- •Male and female participants 2 to < 18 years of age with a diagnosis of PNH confirmed by high-sensitivity flow cytometry with red blood cells (RBCs) and with white blood cells granulocytes/monocytes clone size ≥ 10%. The minimum body weight for patients in Cohort 1 is 35 kg.
- •Participants being treated with anti-C5 therapy and who have been on a stable regimen (dose and interval) for at least 6 months prior to enrollment, may be screened and enrolled in the study and switched to iptacopan irrespective of their anemia and hemolysis status, at the discretion of the Principal Investigator.
- •Participants who are anti-C5 treatment naive: mean hemoglobin level < 10 g/dL confirmed by central laboratory assessment during screening.
- •Participants who are anti-C5 treatment naive: lactate dehydrogenase (LDH) > 1.5 × upper limit of normal (ULN) documented by at least 2 laboratory measurements 2 to 6 weeks apart during the screening period, one of which is to be done by the central lab.
- •Vaccination against Neisseria meningitidis and Streptococcus pneumoniae infection is required prior to the start of study treatment. If the participant has not been previously vaccinated, or if a booster is required, vaccine should be given according to local guidelines at least 2 weeks prior to first study drug administration. If study treatment has to start earlier than 2 weeks post-vaccination, prophylactic antibiotic treatment should be initiated.
- •Vaccination against Haemophilus influenzae is recommended, according to local guidelines, at least 2 weeks before iptacopan.
排除标准
- •History of hypersensitivity to the study drug or its excipients or to drugs of similar chemical classes.
- •Known or suspected hereditary complement deficiency at screening.
- •History of hematopoietic stem cell transplantation (HSCT) or scheduled for HSCT within 52 weeks from enrollment into the study (Day 1).
- •Participants with laboratory evidence of bone marrow failure (reticulocytes < 100 × 10^9/L, platelets < 30 × 10^9/L, neutrophils < 0.5 × 10^9/L).
- •Active systemic bacterial, viral (including COVID-19), or fungal infection within 14 days prior to study drug administration.
- •Presence of fever ≥ 38 °C (100.4 °F) within 7 days prior to study drug administration.
结局指标
主要结局
Safety evaluations, including AEs, SAEs, laboratory parameters, vital signs, and cardiovascular parameters.
Safety evaluations, including AEs, SAEs, laboratory parameters, vital signs, and cardiovascular parameters.
PK parameters Cmax, AUClast, AUCtau (and other PK parameters in plasma, as appropriate), and Ctrough concentrations.
PK parameters Cmax, AUClast, AUCtau (and other PK parameters in plasma, as appropriate), and Ctrough concentrations.
次要结局
- Change in hemoglobin (Hb) from baseline ≥ 1 g/dL at Week 26 (in the absence of red blood cell (RBC) transfusions from Day 14). Change in Hb from baseline ≥ 1 g/dL at Week 52 (in the absence of RBC transfusions from Day 14). Change in Hb from baseline ≥ 2 g/dL at Week 26 (in the absence of RBC transfusions from Day 14). Change in Hb from baseline ≥ 2 g/dL at Week 52 (in the absence of RBC transfusions from Day 14).
- Normal Hb at Weeks 26 in the absence of RBC transfusions from Day 14. Normal Hb at Week 52 in the absence of RBC transfusions from Day 14.
- Absence of packed-RBC transfusions and not meeting transfusion criteria from Day 14 at Week 26. Absence of packed-RBC transfusions and not meeting transfusion criteria from Day 14 at Week 52.
- Change from baseline in Hb and LDH at Week 26. Change from baseline in Hb and LDH at Week 52.
- Safety evaluations (including AEs, SAEs, lab parameters, vital signs, and cardiovascular parameters).
研究者
Novartis Pharma Arzneimittel GmbH
Scientific
Novartis Pharma AG
研究点 (7)
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