A Study to Provide Expanded Access of (Exjade®) Deferasirox to Patients With Congenital Disorders of Red Blood Cells and Chronic Iron Overload From Blood Transfusions Who Cannot Adequately be Treated With Other Locally Approved Iron Chelators
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 入组人数
- 1,683
- 试验地点
- 138
- 主要终点
- Safety Profile of Deferasirox Based Upon Drug Administration and Reporting of Serious Adverse Events
研究概览
简要总结
This is an open-label, non-randomized, multi-center trial designed to provide expanded access of deferasirox to patients with congenital disorders of red blood cells and chronic iron overload from blood transfusions who cannot adequately be treated with locally approved iron chelators.
研究设计
- 研究类型
- Interventional
- 分配方式
- Non Randomized
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 2 Years 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Male or female patients greater than or equal to 2 years of age
- •Documented congenital disorder of red blood cells (e.g., β-thalassemia major, sickle cell anemia, diamond-blackfan anemia) requiring ongoing blood transfusions
- •Cannot be adequately treated with a locally approved iron chelator due to one of the following reasons:
- •Documented non-compliance, defined as having taken less than 50% of the prescribed chelation therapy doses in the 12 months prior to study entry
- •Contraindications, unacceptable toxicities and/or documented poor response to locally approved iron chelators despite proper compliance
- •History of at least 20 blood transfusions (equivalent to 100 mL/kg of packed red blood cells (PRBC])
- •Serum ferritin value greater than or equal to 1000 µg/L
- •Ability to comply with all study-related procedures, medications, and evaluations
排除标准
- •Ongoing treatment with another iron chelator (Any other iron chelation therapy must be discontinued at least 24 hours prior to study entry.)
- •Patients who meet the eligibility criteria for any other ongoing Novartis sponsored clinical study protocol with deferasirox and who have geographic access to these sites
- •Patients unable to tolerate (or who have unacceptable toxicities to) prior treatment with deferasirox
- •Serum creatinine above the upper limit of normal at screening.
- •Patients with ALT ≥ 500 U/L at screening.
- •Evidence of chelation-related cataracts or hearing loss within 4 weeks prior to baseline
- •Pregnancy (as indicated by serum β-HCG pregnancy test at screening for all female patients with the potential to become pregnant) and patients who are breastfeeding
- •Patients treated with systemic investigational drug within 4 weeks prior to or with topical investigational drug within 7 days prior to the baseline visit
- •Other protocol-defined inclusion/exclusion criteria may apply.
研究组 & 干预措施
Deferasirox
Deferasirox was administered orally once a day, 30 minutes prior to breakfast. Dosage was based on participant's body weight. Starting dose was determined by the frequency of blood transfusions and recommended initial daily dose of deferasirox is 20 mg/kg body weight for patients receiving blood transfusion, 10 mg/kg for patients receiving less frequent transfusion/exchange transfusion and 30 mg/kg for patients receiving more frequent blood transfusions.
干预措施: Deferasirox (Drug)
结局指标
主要结局
Safety Profile of Deferasirox Based Upon Drug Administration and Reporting of Serious Adverse Events
时间窗: Baseline to end of study (Median exposure time to drug was approximately 30 weeks; Maximum exposure was 104 weeks)
Safety as assessed by the number of participants with death, serious adverse events (SAE), and/or Adverse Events (AEs) leading to study drug interruption or discontinuation. Note: only treatment emergent AEs are summarized.
次要结局
- The Change in Serum Ferritin Values From Baseline Through Completion of the Study(Baseline to end of study (Median exposure time to drug was approximately 30 weeks; Maximum exposure was 104 weeks))