2022-502965-34-00招募中3 期
A multicenter, single arm, open-label extension study to evaluate the long-term safety, tolerability and efficacy of iptacopan in participants with atypical hemolytic uremic syndrome (aHUS) who have completed a preceding iptacopan phase 3 study in aHUS
试验速览
- 阶段
- 3 期
- 状态
- 招募中
- 入组人数
- 6
- 试验地点
- 1
- 主要终点
- Safety evaluations including adverse events (AE) /serious adverse events (SAE), safety laboratory parameters, vital signs and electrocardiograms (ECGs) through study duration.
研究概览
简要总结
To evaluate the long-term safety and tolerability of iptacopan in study participants with aHUS
入排标准
- 年龄范围
- 18 years 至 65+ years（65+ Years, 18-64 Years）
- 接受健康志愿者
- 是
入选标准
- •Signed informed consent must be obtained prior to participation in the open label extension study
- •Willing and able to comply with the study Schedule of Activities (Section 1.3 of the protocol)
- •Participants who have completed the full study treatment period of any prior "Novartis sponsored" iptacopan Phase 3 clinical trial in aHUS (e.g. CLNP023F12301, CLNP023F12302) are still on iptacopan study treatment and derive benefit from it as per Investigator's judgement
- •Prior vaccinations against Neisseria meningitidis, Streptococcus pneumoniae and Haemophilus influenzae infections should be up to date (i.e. any boosters required should be administered according to local guidelines)
排除标准
- •Concomitant treatment with any complement inhibitor as well as concomitant treatment with any of the drugs listed in Section 6.8.2
- •Any comorbidity or medical condition (including but not limited to any active systemic bacterial, viral or fungal infection or malignancy) that, in the opinion of the Investigator could put the participant at risk
- •Active infection or history of recurrent invasive infections caused by encapsulated bacteria such as Neisseria meningitidis, Streptococcus pneumoniae or Haemophilus influenzae
- •History of hypersensitivity to iptacopan or its excipients or to drugs of similar chemical classes
- •Pregnant or nursing (lactating) women
- •Women of child-bearing potential, defined as all women physiologically capable of becoming pregnant, unless they are using effective methods of contraception during dosing of investigational drug and for 1 week after stopping of investigational drug.
结局指标
主要结局
Safety evaluations including adverse events (AE) /serious adverse events (SAE), safety laboratory parameters, vital signs and electrocardiograms (ECGs) through study duration.
Safety evaluations including adverse events (AE) /serious adverse events (SAE), safety laboratory parameters, vital signs and electrocardiograms (ECGs) through study duration.
次要结局
- Absence of TMA manifestation without use of anti-C5 antibody throughout the study. TMA manifestation defined by the coexistence of min. two of the three criteria at the same visit attributable to aHUS: ● thrombocytopenia (platelet count decrease of ≥ 25% compared to baseline and < LLN), ● microangiopathic hemolytic anemia (hemoglobin ≤ LLN for age and gender and LDH ≥ 1.5 x ULN), ● worsening kidney function (serum creatinine increase of >25% compared to baseline levels)
- Complete TMA response status without the use of anti-C5 antibody therapy through study duration. Complete TMA Response is defined as: hematological normalization in platelet count (platelet count ≥150 x 109 /L) and LDH (below ULN), and improvement in kidney function (≥ 25% serum creatinine reduction from baseline or ≥ 25% serum creatinine reduction compared to serum creatinine values prior to initiation of anti-C5 antibody therapy)
- Observed value and change from baseline in eGFR and CKD stage (1-5) based on eGFR categories through study duration
- Dialysis requirement status through study duration
- TMA related events during the study defined as any of the following: ● Irreversible (>3 months) reduction in eGFR rate by ≥20%, not attributable to another cause ● An episode of acute kidney injury (AKI) attributed to a TMA that requires renal replacement therapy ● A non-renal manifestation of a TMA that requires hospitalization or causes irreversible organ damage or death
研究者
Novartis Pharma Arzneimittel GmbH
Scientific
Novartis Pharma AG
研究点 (1)
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