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Идёт набор NCT07471516

Zoledronic Acid Treatment in Patients With Congenital Dyserythropoietic Anemia

Фаза I / Фаза II С лечением Congenital Dyserythropoietic Anemia (CDA)

Ориентир для пациента и семьи

Простыми словами

Автоматическая сводка по структурированным данным реестра. Она помогает сориентироваться, но не заменяет официальный протокол или оценку врача.

Что изучают
В протоколе указаны: zoledronic acid.
Кому может быть актуально
Состояния в реестре: Congenital Dyserythropoietic Anemia (CDA). Базовые параметры: от 18 лет · Все.
Что важно проверить
Возраст, диагноз и пол — только базовые ориентиры. Предыдущее лечение, анализы и другие обязательные условия указаны ниже в критериях участия.
Где проводится
Китай
Следующий шаг
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Официальное название

Zoledronic Acid Treatment in Patients With Congenital Dyserythropoietic Anemia: An Exploratory Study

Обзор

Background: Congenital Dyserythropoietic Anemia (CDA) is a group of rare hereditary blood disorders characterized by ineffective erythropoiesis, leading to chronic anemia and organ damage. Current treatment options are very limited, mainly relying on regular blood transfusions, which can cause severe complications over time. Our laboratory research and animal models suggest that Zoledronic acid, a medication commonly used for bone health, may improve ineffective erythropoiesis. Purpose: The purpose of this exploratory study is to evaluate the efficacy and safety of Zoledronic acid in adult patients with CDA who do not have other effective treatment options. The primary goal is to see if this treatment can increase hemoglobin levels and reduce the need for blood transfusions. Study Design: This is a prospective, single-center, single-arm study. Participants will receive an initial intravenous dose (4 mg) of Zoledronic acid. After a 4-week observation period to ensure safety, participants will receive additional doses every 4 weeks for a total of 4 doses. Researchers will monitor hemoglobin levels, transfusion frequency, spleen size, and overall quality of life over a period of 12 to 24 weeks.

Вмешательства

  • Препарат zoledronic acid
    Zoledronic acid will be administered at a dose of 4 mg via a standardized intravenous (IV) infusion lasting no less than 15 minutes. To ensure safety, the initial dose is followed by a 4-week rigorous observation period. If the treatment is well-tolerated without significant adverse events, subsequent doses will be given every 28 days for a total of 4 cycles. Patients will be closely monitored for serum calcium levels and renal function (eGFR) throughout the intervention period to mitigate poten

Первичные конечные точки

  • Change in Hemoglobin Level From Baseline [Срок оценки: 12 weeks after the first dose]
Вторичные конечные точки (4)
  • Change in Hemoglobin Level at 4 Weeks [Срок оценки: 4 weeks after the first dose]
  • Change in Red Blood Cell Transfusion Interval [Срок оценки: Up to 24 weeks]
  • Change in Spleen Size [Срок оценки: Up to 24 weeks]
  • Change in Quality of Life Assessed by the 36-Item Short Form Health Survey (SF-36) [Срок оценки: Up to 24 weeks]

Критерии участия

Критерии включения

  • Age ≥ 18 years, regardless of gender.
  • Diagnosis of Congenital Dyserythropoietic Anemia (CDA) based on clinical presentation, laboratory tests, and family investigation, with the presence of RBM28 mutation and/or increased vacuolization within nucleated red blood cells under light microscopy of bone marrow.
  • Presence of anemia (Hemoglobin < 100 g/L at screening) or transfusion dependence (defined as an average transfusion interval of < 8 weeks within the past 3 months).
  • Performance status is acceptable (ECOG score 0-2).
  • Normal renal function (estimated glomerular filtration rate, eGFR ≥ 60 mL/min/1.73m²).
  • Serum calcium levels within the normal range.
  • Female patients of childbearing potential must have a negative pregnancy test before enrollment and agree to use effective contraception during the study and for 3 months after completion.
  • The patient and/or guardian voluntarily sign the informed consent form.

Критерии исключения

  • Known hypersensitivity to bisphosphonates or any of their excipients.
  • Severe periodontal disease or a recent history (within 6 months) of osteonecrosis of the jaw.
  • Hypocalcemia.
  • Pregnant or lactating women.
  • Currently receiving other experimental drug treatments that may affect erythropoiesis (e.g., Luspatercept).
  • Active, uncontrolled systemic infection.
  • Severe cardiac, pulmonary, or hepatic dysfunction, as judged by the investigator to be unsuitable for participation in the study

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

Китай · 1 центр
  • Institute of Hematology & Blood Diseases Hospital — Тяньцзинь

Идентификаторы

NCT: NCT07471516 · IIT2025152

Первоисточники (государственные реестры)

Открыть это исследование на ClinicalTrials.gov ↗