NCT01090323已完成2 期
A One Year Open Label, Non-comparative Extension to a Randomized, Multicenter, Phase II Study to Evaluate the Safety, Tolerability, Pharmacokinetics (PK) and Effects on Liver Iron Concentration (LIC) of Repeated Doses of 5-30mg/kg/Day ICL670 Relative to Deferoxamine (DFO) in Sickle Cell Disease (SCD) Patients With Transfusional Hemosideresis (THS) [Amendment 3: Extension Prolonged to 4-years]
适应症
干预措施
相关药物
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 入组人数
- 185
- 试验地点
- 32
- 主要终点
- Number of Participants With Adverse Events After Start of ICL670
研究概览
简要总结
The safety, tolerability, effects on liver iron concentration and pharmacokinetics of ICL670 is studied in sickle cell disease patients with transfusional hemosiderosis.
详细描述
The treatment period started once the patient completed the core study and signed informed consent. It is continued for up to 4 years. Safety parameters were assessed every 4 weeks. Eye and Ear examinations were performed on a yearly basis. To further investigate the extent of iron overload, serum ferritin, iron, and transferrin were monitored every four weeks. The Program Safety Board monitored the safety of ICL670 during the study to evaluate and categorize any serious case reported in association with ICL670.
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
- •Patients were included who met the following criteria:
- •Completion of the core [Study 0109]
- •Serum ferritin greater than or equal to 500 µg/L
- •Ability to comply with all study-related procedures, medications, and evaluations
- •Sexually active post-menarche female patients must use double-barrier contraception, oral contraceptive plus barrier contraceptive, or must have undergone clinically documented total hysterectomy and/or oophorectomy, tubal ligation or be postmenopausal defined by amenorrhea for at least 12 months.
- •Written informed consent and assent by the patient and or their parents or legal guardian.
- •Additional inclusion criteria for pediatric patients The definition of the term 'pediatric' for enrollment and study conduct was in accordance with local law. Parents or the legal guardians were fully informed by the investigator as to the requirements of the study. The pediatric patients themselves were informed according to their capabilities in a language and terms that they were able to understand. Written informed consent was obtained from their legal guardian on the patient's behalf in accordance with national legislation. If capable, all patients had to also personally sign their written informed consent.
排除标准
- •Patients who met the following criteria were to be excluded:
- •History of non-compliance to medical regimens and patients who are considered potentially unreliable and/or not cooperative
- •Serum creatinine above the age-appropriate upper limit of normal within one week prior to entry
- •Patients with ALT ≥ 500 U/L within one week prior to entry
- •Evidence of chelation-related cataracts or hearing loss within 4 weeks prior to baseline
- •Pregnancy (as indicated by serum β-HCG pregnancy test 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
ICL670
Experimental
干预措施: ICL670 (Drug)
结局指标
主要结局
Number of Participants With Adverse Events After Start of ICL670
时间窗: 0 - 60 months
Safety as assessed by the number of participants with adverse event or death after the start of ICL670.
次要结局
- Change in Serum Ferritin From Start of ICL670 to End of Study(0 - 60 months)
研究者
研究点 (32)
Loading locations...
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