A Study of SNH-119014 in Adult Participants With Non-transfusion-dependent Thalassemia (NTDT)

Официальное название

A Phase 2 Study to Determine the Safety, Efficacy, Pharmacokinetics, and Pharmacodynamics of SNH-119014 in Adult Subjects With Non-transfusion-dependent Thalassemia

Обзор

This study is a multicenter study to evaluate the safety, efficacy, pharmacokinetics, and pharmacodynamics of treatment with SNH-119014 in adult participants with non-transfusion-dependent thalassemia. 30 participants with non-transfusion-dependent thalassemia were enrolled.

Вмешательства

• Препарат SNH-119014 dose 1
  Study Drug
• Препарат SNH-119014 dose 2
  Study Drug
• Препарат Placebo
  Placebo

Первичные конечные точки

• Safety evaluation [Срок оценки: From signing the informed consent to 4 weeks after the last dose]

Критерии участия

Критерии включения

• Male or Female;

-≥18 years;

• Documented diagnosis of thalassemia;
• Non-transfusion-dependent;
• Hb concentration ≤10.0 grams per deciliter (g/dL) during the screening period;
• For women of reproductive potential: negative serum pregnancy test during the screening period;
• Agreement to use approved contraceptive measures;
• Informed consent;

Критерии исключения

• Hemoglobin S forms of thalassemia;
• History of Congestive Heart Failure Within 6 months prior to signed informed consent;
• Myocardial Infarction/unstable angina Within 6 months prior to signed informed consent;
• Deep Vein Thrombosis/stroke/thromboembolic events Within 6 months prior to signed informed consent;
• Poorly controlled hypertension or diabetes;
• Markedly abnormal QTcF interval;
• Severe arrhythmia requiring treatment as assessed by the investigator;
• Markedly abnormal left ventricular ejection fraction;
• Diagnosis of any other congenital or acquired hematologic disorder, or any other hemolytic process;
• Active infection requiring intravenous antibiotics or of Grade ≥3 severity within 4 weeks prior to the first dose;
• History of primary malignancy, except for: cured non-melanoma skin cancer; curatively treated cervical or breast carcinoma in situ; or other primary tumors treated with curative intent with no known active disease and no treatment in the past 5 years;
• History of neurological or psychiatric disorders that, in the investigator's assessment, may affect the participant's ability to participate in the clinical study;
• Post-splenectomy sepsis;
• Severe pulmonary disease or hepatobiliary disease;
• Abnormal laboratory tests during screening;
• Use of luspatercept, hydroxyurea, erythropoietin, strong CYP3A4 inhibitors, or strong CYP3A4 inducers within the prohibited period specified in the protocol;
• Receipt of anticoagulant therapy prior to screening, or requirement for anticoagulant therapy during the study period;
• Prior bone marrow or stem cell transplant;
• Pregnant or lactating women;
• Major surgery within 6 months prior to signing informed consent or planned during the study period;
• Splenectomy scheduled during the study treatment period;
• Participation in another clinical trial and receipt of investigational treatment within 3 months;
• History of severe allergy, or allergy to SNH-119014 or its excipients;
• Any other condition that, in the investigator's opinion, makes the participant unsuitable for enrollment.

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

Китай · 8 центров

• Foshan First People's Hospital — Foshan
• Nanfang Hospital, Southern Medical University — Гуанчжоу
• Maoming People's Hospital — Maoming
• Liuzhou People's Hospital — Liuzhou
• 923rd Hospital of the Joint Logistics Support Force of the People's Liberation Army of Chi — Nanning
• The First Affiliated Hospital of Guangxi Medical University — Nanning
• The First Affiliated Hospital of Hainan Medical University — Haikou
• Sichuan Academy of Medical Sciences & Sichuan Provincial People's Hospital — Чэнду

Идентификаторы

NCT: NCT07579949 · SNH-119014-II-01

Первоисточники (государственные реестры)

• ClinicalTrials.gov (NIH, США)

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