SLN12140 in Adult Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) in China

Официальное название

A Phase II Clinical Study Evaluating SLN12140 in Complement Inhibitor-Naïve Adult Subjects With Paroxysmal Nocturnal Hemoglobinuria

Обзор

The goal of this clinical trial is to learn if drug SLN12140 works to treat Complement Inhibitor-Naïve Subjects with Paroxysmal Nocturnal Hemoglobinuria in adults. It will also learn about the safety, pharmacokinetic characteristics, and dosing of drug SLN12140. The study is divided into four phases: screening period, core treatment period, extended dosing period, and follow-up period, and includes two cohorts (Cohorts 1-2), with each cohort enrolling at least 5 treatment-naïve adult PNH subjects for complement inhibitor therapy.

Вмешательства

• Препарат SLN12140
  5 participants will receive SLN12140 100mg QW for 4 weeks, then 300mg QW for 8 weeks, then 200mg QW for 52 weeks. 5 participants will receive SLN12140 200mg QW for 4 weeks, then 600mg Q4W for 60 weeks

Первичные конечные точки

• During the 12-week treatment period, the proportion of participants whose Lactate Dehydrogenase (LDH) decreased by 60% or more from baseline or whose LDH was below the upper limit [Срок оценки: 12weeks after baseline]

Вторичные конечные точки (12)

• Percentage change of LDH from baseline [Срок оценки: Baseline through Week 64]
• Proportion of participants achieving hemolysis control (LDH ≤ 1.5×ULN) [Срок оценки: Baseline through Week 64]
• Change in hemoglobin (Hb) levels from baseline [Срок оценки: Baseline through week 64]
• The proportion of participants whose hemoglobin (Hb) increased by ≥2 g/dL from baseline and who avoided blood transfusion [Срок оценки: Baseline through Week 64]
• Proportion of participants who avoided blood transfusion [Срок оценки: Baseline through Week 64]
• Incidence(%) of Breakthrough Hemolysis (BTH) [Срок оценки: Baseline through Week 64]
• Changes from baseline in intravascular and extravascular hemolysis indicators (including but not limited to reticulocytes, bilirubin, red blood cell count, platelet count, ferritin, etc.) [Срок оценки: baseline through week 64]
• Changes in thrombus formation risk markers from baseline (including but not limited to fibrinogen, prothrombin time, activated partial thromboplastin time, thrombin time, fibrin D-dimer, etc.); [Срок оценки: Baseline through week 64]
• Change in functional assessment of Functional Assessment of Chronic Illness Therapy (FACIT) [Срок оценки: Baseline through Week 64]
• Number(%) of participants with Adverse Events (AEs) , Treatment-Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs) [Срок оценки: Baseline through Week 64]
• Pharmacokinetics (PK)parameters of SLN12140: Area Under The Plasma Concentration-time Curve [Срок оценки: Baseline through week 64 (predose and postdose)]
• Immunegenicity in Paraxysmal Nocturnal Hemoglobinuria [Срок оценки: Baseline through Week 64]

Критерии участия

Критерии включения

• Adult complement inhibitor naïve PNH patients (age>=18), which is confirmed by flow cytometry evaluation
• Must be vaccinated against meningococcal vaccine and pneumococcal vaccine

Критерии исключения

• Significant bone marrow failure
• Meningitidis infection or unresolved meningococcal disease
• Other significant systemic diseases that might have impact on efficacy and safety assessment

Критерии приведены из реестра в оригинале (на английском). Окончательную оценку соответствия проводит исследовательский центр.

Здоровые добровольцы: Нет

Дизайн исследования

Распределение

Не применимо

Модель

Одна группа

Маскирование

Открытое

Основная цель

Лечение

Центры проведения

Список центров уточняется — проверьте первичный протокол.

Идентификаторы

NCT: NCT07387302 · LIN2102-CN201

Первоисточники (государственные реестры)

• ClinicalTrials.gov (NIH, США)

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