JPRN-jRCT2031230013尚未招募3 期
An Open-Label Extension Study to Evaluate the Long-Term Safety, Tolerability, and Efficacy of Pozelimab and Cemdisiran Combination Therapy in Patients with Paroxysmal Nocturnal Hemoglobinuria - ACCESS-EXT
Aurand Lisa0 个研究点目标入组 11 人开始时间: 2023年4月9日最近更新:
适应症
试验速览
- 阶段
- 3 期
- 状态
- 尚未招募
- 发起方
- 入组人数
- 11
研究概览
简要总结
暂无简介。
研究设计
- 研究类型
- Interventional
入排标准
- 年龄范围
- >= 18age old 至 ot applicable（—）
- 性别
- All
入选标准
- •Patients Entering from the Parent Study
- •1. Patients with PNH who have completed, without permanent discontinuation, study treatment in the parent study (R3918-PNH-2021 [NCT05133531]), including the post-Open-label treatment period (OLTP) transition period, if applicable.
- •Patients Entering with C5 polymorphism
- •1. Patients with PNH who have a documented C5 polymorphism rendering them refractory to eculizumab or ravulizumab (eg, p.Arg885His, p.Arg885Cys), as described in the protocol
- •2. Diagnosis of PNH confirmed by high-sensitivity flow cytometry testing with PNH granulocytes or monocytes
- •3. Active disease, as defined by the presence of 1 or more PNH-related sign or symptom as described in the protocol
- •4. LDH level >= 2 x upper limit of normal (ULN) at the screening visit
- •Note: Other protocol-defined Inclusion Criteria apply.
排除标准
- •Patients Entering from the Parent Study
- •1. Significant protocol deviation(s) in the parent study based on the investigators judgment and to the extent that these would (if continued) impact the study objectives and/or safety of the patient
- •2. Any new condition or worsening of an existing condition which, in the opinion of the investigator, would make the patient unsuitable for enrollment or could interfere with the patient participating in or completing the study
- •Patients Entering with C5 polymorphism
- •1. Prior treatment with complement inhibitors within 5 half-lives of the respective agent prior to screening, except for prior eculizumab or ravulizumab which are not exclusionary
- •2. Receipt of an organ transplant, history of bone marrow transplantation or other hematologic transplant
- •3. No documentation of meningococcal vaccination within 5 years prior to enrollment
- •4. Positive hepatitis B surface antigen or hepatitis C virus Ribonucleic acid (RNA) during screening
- •5. Patients with known HIV with history of opportunistic infections in the last 1 year as described in the protocol
- •6. Known hereditary complement deficiency
- •7. Documented history of active, uncontrolled, ongoing systemic autoimmune diseases
- •8. Documented history of liver cirrhosis or patients with liver disease with evidence of current impaired liver function or patients with elevations in Alanine aminotransferase (ALT) or Aspartate aminotransferase (AST) (unrelated to PNH or its complications) as described in the protocol
- •Note: Other protocol-defined Exclusion Criteria apply.
研究者
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