Safety and Efficacy Study of QL0911 to Treat Immune Thrombocytopenia (ITP) in Pediatric Patients

Официальное название

A Phase III, Randomized, Double-Blind, Placebo-Controlled Study of QL0911 in Pediatric Patients With Primary Immune Thrombocytopenia.

Обзор

The purpose of this study is to evaluate efficacy and of safety QL0911 in the treatment of thrombocytopenia in pediatric patients with previously treated chronic ITP.

Вмешательства

• Препарат QL0911
  The starting dose of QL0911 is 1 µg/kg administered weekly by subcutaneous injection. Participants will return to the clinic weekly to provide platelet counts and undergo dose titrations under the supervision of the treating physician. Weekly dose increases will continue in increments of 1 µg/kg up to a maximum dose of 10 µg/kg in an attempt to reach a target platelet count of ≥ 50 x 10\^9/L. Dose adjustment will be allowed during the treatment period to maintain a platelet count between ≥ 50 x
• Препарат Placebo
  Matching placebo administered by subcutaneous injection.

Первичные конечные точки

• Percentage of Participants With a Durable Platelet Response [Срок оценки: Week 18 to week 25]

Вторичные конечные точки (6)

• Percentage of Participants With an Overall Platelet Response [Срок оценки: 24 weeks]
• The proportion of subjects with a weekly platelet count ≥ 30 × 10^9/Land at least twice the baseline platelet count without bleeding during the double-blind period of 24 weeks. [Срок оценки: 24 weeks]
• Number of Weeks With Platelet Response [Срок оценки: 24 weeks]
• Percentage of Participants Who Received Rescue Medication During the Treatment Period. [Срок оценки: 24 weeks]
• Number of Participants With Treatment-emergent Adverse Events (TEAEs) [Срок оценки: Up to Week 38]
• Number of Participants With Antidrug Antibodies (ADAs) , Anti endogenous TPO antibody and Neutralizing Antibodies (Nab)； [Срок оценки: Up to Week 38]

Критерии участия

Критерии включения

• Age ≥ 1 years old and < 18 years old.
• Diagnosed primary ITP for at least 12 months;
• Had received at least one first-line ITP treatment with no response or recurrence after treatment;
• Had a platelet count <30×10\^9/L within 48 hours before the first dose;
• Eastern Cooperative Oncology Group (ECOG) performance status of 0, 1, or 2;
• Patients of child-bearing potential must agree to use effective contraception during the study and for 3 months following the last dose of study treatment.

Критерии исключения

• Had a history of bone marrow stem cell abnormalities or myelodysplastic syndrome other than ITP-specific changes.
• Underwent splenectomy within 12 weeks before the first dose;
• Had received ITP treatments (including rescue treatment) within 2 weeks before the first dose;
• Had received romiplostim (Nplate®) or eltrombopag (Revolade®), rhTPO or other agents that stimulate TPO receptors (also known as c-Mpl)within 4 weeks before the first dose;
• Had received antibody-based therapies within 14 weeks before the first dose.
• Patients with concurrent or past malignant disease.
• Serum creatinine or total bilirubin >1.5\*ULN) alanine transaminase (ALT) or aspartate transaminase (AST) >3\* ULN.
• Had received antibody-based therapies within 14 weeks before the first dose.
• Had prothrombin time (PT) or prothrombin time-international normalized ratio (PT-INR) or activated partial thromboplastin time (APTT) exceeded 20% of the reference range of normal values.

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

Здоровые добровольцы: Нет

Дизайн исследования

Распределение

Рандомизированное

Модель

Параллельные группы

Маскирование

Четверное слепое

Основная цель

Лечение

Центры проведения

Список центров уточняется — проверьте первичный протокол.

Идентификаторы

NCT: NCT07455006 · QL0911-303

Первоисточники (государственные реестры)

• ClinicalTrials.gov (NIH, США)

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