2023-510336-36-00招募中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
Regeneron Pharmaceuticals Inc.12 个研究点 分布在 6 个国家目标入组 17 人开始时间: 2024年11月27日最近更新:
相关药物
试验速览
- 阶段
- 3 期
- 状态
- 招募中
- 发起方
- 入组人数
- 17
- 试验地点
- 12
- 主要终点
- Incidence of treatment-emergent serious adverse events (SAEs)
研究概览
简要总结
The primary objective of the study is to describe the long-term safety, tolerability, and efficacy of pozelimab + cemdisiran combination therapy in patients with PNH.
入排标准
- 年龄范围
- 18 years 至 65+ years（18-64 Years, 65+ Years）
- 接受健康志愿者
- 是
入选标准
- •Patients Entering from the Parent Study: 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 from the Parent Study: Willing and able to comply with clinic visits and study-related procedures, including meningococcal vaccinations required per protocol
- •Patients Entering with C5 polymorphism: 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
- •Patients Entering with C5 polymorphism: Diagnosis of PNH confirmed by high-sensitivity flow cytometry testing with PNH granulocytes or monocytes
- •Patients Entering with C5 polymorphism: Active disease, as defined by the presence of 1 or more PNH-related sign or symptom as described in the protocol
- •Patients Entering with C5 polymorphism: LDH level ≥2 × upper limit of normal (ULN) at the screening visit
- •Patients Entering with C5 polymorphism: Willing and able to comply with clinic visits and study-related procedures, including meningococcal vaccinations required per protocol
排除标准
- •Patients Entering from the Parent Study: Significant protocol deviation(s) in the parent study based on the investigator’s judgment and to the extent that these would (if continued) impact the study objectives and/or safety of the patient
- •Patients Entering with C5 polymorphism: 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 Inclusion/ Exclusion Criteria apply
- •Patients Entering from the Parent Study: 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: 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
- •Patients Entering with C5 polymorphism: Receipt of an organ transplant, history of bone marrow transplantation or other hematologic transplant
- •Patients Entering with C5 polymorphism: Not meeting meningococcal vaccination requirements and, at a minimum, documentation of quadrivalent meningococcal vaccination within 5 years prior to enrollment and serotype B vaccine within 3 years prior to enrollment as described in the protocol
- •Patients Entering with C5 polymorphism: Positive hepatitis B surface antigen or hepatitis C virus Ribonucleic acid (RNA) during screening
- •Patients Entering with C5 polymorphism: Patients with known HIV with history of opportunistic infections in the last 1 year as described in the protocol
- •Patients Entering with C5 polymorphism: Known hereditary complement deficiency
- •Patients Entering with C5 polymorphism: Documented history of active, uncontrolled, ongoing systemic autoimmune diseases
结局指标
主要结局
Incidence of treatment-emergent serious adverse events (SAEs)
Incidence of treatment-emergent serious adverse events (SAEs)
Severity of treatment-emergent SAEs
Severity of treatment-emergent SAEs
Incidence of treatment emergent adverse events of special interest (AESIs)
Incidence of treatment emergent adverse events of special interest (AESIs)
Severity of treatment emergent AESIs
Severity of treatment emergent AESIs
Incidence of adverse events (AEs) leading to permanent treatment discontinuation
Incidence of adverse events (AEs) leading to permanent treatment discontinuation
Severity of adverse events (AEs) leading to permanent treatment discontinuation
Severity of adverse events (AEs) leading to permanent treatment discontinuation
Percent change from baseline in lactate dehydrogenase (LDH)
Percent change from baseline in lactate dehydrogenase (LDH)
次要结局
- Maintenance of adequate control of hemolysis (LDH ≤1.5 × ULN)
- Adequate control of hemolysis (LDH ≤1.5 × ULN)
- Transfusion avoidance
- Breakthrough hemolysis (defined as LDH ≥2 × ULN [subsequent to initial achievement of LDH ≤1.5 × ULN] concomitant with signs or symptoms associated with hemolysis)
- Hemoglobin stabilization
- Percent change in LDH
- Change in fatigue
- Change in physical function (PF) scores on the EORTC QLQ-C30
- Change in GHS/quality of life (QOL) scale on the EORTC QLQ-C30
- Normalization of LDH
- Rate of red blood cell (RBC) transfusion
- Number of units of RBC transfusion
- Percentage of days with LDH ≤1.5x upper limit of normal (ULN)
- Change in hemoglobin levels
- Change in total complement hemolytic activity assay (CH50)
- Percent change in CH50
- Concentrations of total pozelimab in serum
- Concentrations of cemdisiran in plasma
- Incidence of treatment-emergent anti-drug antibodies to pozelimab
- Incidence of treatment-emergent anti-drug antibodies to cemdisiran
- Concentration of total complement component 5 (C5) in plasma
- Percent change of concentration of total C5 in plasma
研究者
Clinical Trial Information
Scientific
Regeneron Pharmaceuticals Inc.
研究点 (12)
Loading locations...
相似试验
终止
3 期
REGN3918 in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) to Evaluate Its Long Term Safety, Efficacy and Tolerability.Paroxysmal Nocturnal HemoglobinuriaNCT04162470Regeneron Pharmaceuticals24
已完成
3 期
An Extension Study of Maralixibat in Patients With Progressive Familial Intrahepatic Cholestasis (PFIC)Progressive Familial Intrahepatic Cholestasis (PFIC)NCT04185363Mirum Pharmaceuticals, Inc.84
已完成
3 期
Study of Etanercept in the Treatment of Psoriasis in Adult SubjectsInflammationPsoriasisNCT00121615Amgen391
Enrolling By Invitation
2 期
A Study of Long-term Safety and Efficacy of VX-670 in Participants With Myotonic Dystrophy Type INCT06926621Vertex Pharmaceuticals Incorporated44
已完成
3 期
A Study to Evaluate the Long-term Use of Valsartan in Children 6 Months to 5 Years Old With HypertensionHypertensionNCT00457626Novartis Pharmaceuticals66