NCT03372083已完成4 期
A Single-arm Interventional Phase IV, Post-authorisation Study Evaluating the Safety of Pediatric Patients With Transfusional Hemosiderosis Treated With Deferasirox Crushed Film Coated Tablets
适应症
干预措施
相关药物
试验速览
- 阶段
- 4 期
- 状态
- 已完成
- 入组人数
- 44
- 试验地点
- 1
- 主要终点
- Number of Participants With Selected Gastrointestinal Disorders up to 24 Weeks
研究概览
简要总结
This study employed a prospective, single-arm, global multi-center interventional open-label, non-randomized design to identify and assess safety profile of the crushed deferasirox FCT when administered up to 24 weeks in pediatric patients aged ≥2 to <6 years with transfusional hemosiderosis. The study was designed to enroll a minimum of 40 patients. Forty-four patients were treated and analyzed.
详细描述
The study included a screening period (from Day 0-14) with two visits at least 7 days apart to assess eligibility of patients that were chelation naïve or on a prior iron chelator treatment other than DFX. For Patients on DFX treatment prior to study entry only one screening visit (screening visit 1) were to occur to determine eligibility. Any current chelation therapy except deferasirox were to be discontinued to undergo a 5-day washout period prior to commencing a 24 week treatment period with crushed deferasirox FCT.
All patients were to have weekly visits for the first month to monitor renal function. Hepatic function were to be assessed biweekly during the first month. Thereafter, monthly safety assessments were to be performed, including the monitoring of serum ferritin values and trends in order to adapt patient treatment.
Eligibility, application of dosing standards and adjustments, as well as safety and serum ferritin assessments as specified in the protocol.
The planned duration of treatment was 24 weeks followed by a 30-day safety follow up.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 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 6 Years（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Patients ≥2 to <6 years old diagnosed with transfusional hemosiderosis
- •Documented history of red blood cell transfusions
- •Written informed consent/assent before any study-specific procedures. The consent will be obtained from caregiver(s) or patient's legal representative. Investigators will also obtain assent of patients according to local, regional, or national regulations.
- •For patients on prior DFX: Serum ferritin (SF) >500 ng/mL, measured at screening visit 1 and requiring a DFX daily dose equivalent to FCT ≥ 7mg/kg/day.
- •For patients on a prior chelator other than DFX (e.g. deferiprone or deferoxamine) or chelation naive: Serum ferritin (SF) >1000 ng/mL measured at screening visits 1 and
排除标准
- •Patients that receive more than one iron chelator at the same time as current iron chelation treatment. (Patients who have received combination therapy in their medical history but are currently being treated with a single ICT agent are eligible.)
- •Patients continuing on deferoxamine or deferiprone in addition to study treatment. (Patients switching to or continuing on deferasirox are eligible).
- •Unresolved adverse events if the patient was previously treated with deferiprone or deferoxamine or deferasirox.
- •Significant proteinuria as indicated by a urinary protein/creatinine ratio > 0.5 mg/mg in a non-first void sample urine measured at screening visit
- •Serum creatinine > age adjusted ULN measured at any screening visit
- •Creatinine clearance below 90 mL/minute measured at any screening visit. Creatinine clearance using the Schwartz formula will be estimated from serum creatinine measured at each respective visit.
- •ALT and/or AST > 2.5 x ULN measured at screening visit
- •Total bilirubin (TBIL) >1.5 x ULN measured at screening visit
- •Patients with significant impaired GI function or GI disease that may significantly alter the absorption of oral deferasirox FCT (e.g. ulcerative diseases, uncontrolled nausea, vomiting, diarrhea, malabsorption syndrome, or small bowel resection).
- •History of and/or laboratory evidence of active Hepatitis B or Hepatitis C (HBsAg in the absence of HBsAb OR HCV Ab positive with HCV RNA positive.
- •Liver disease with severity of Child-Pugh Class B or C.
- •History of hypersensitivity to any of the study drug or excipients.
- •Patients participating in another clinical trial or receiving an investigational drug.
- •Patients with a known history of HIV seropositivity.
- •Patients unwilling or unable to comply with the protocol.
- •History of malignancy of any organ system, treated or untreated, within the past 5 years whether or not there is evidence of local recurrence or metastases, with the exception of localized basal cell carcinoma of the skin.
- •Significant medical condition interfering with the ability to partake in this study (e.g. uncontrolled hypertension, unstable cardiac disease not controlled by standard medical therapy, systemic disease: cardiovascular, renal, hepatic, etc.).
- •Female patients who reach menarche and they or their caregivers refuse pregnancy testing and/or if there is a positive pregnancy test result.
研究组 & 干预措施
Deferasirox
Experimental
Crushed deferasirox (ICL670) FCT for oral use daily. Deferasirox FCT dosing was based on subject's weight.
干预措施: Deferasirox (Drug)
结局指标
主要结局
Number of Participants With Selected Gastrointestinal Disorders up to 24 Weeks
时间窗: Baseline (Week 1 Day 1) up to Week 24, plus 30 day safety follow-up.
To assess the safety of crushed deferasirox FCT with respect to selected gastrointestinal (GI) disorders (esophagitis, stomatitis, mouth ulceration, gastric ulcers, haemorrhage, abdominal pain, diarrhea, nausea, and vomiting). Only descriptive analysis performed.
次要结局
- Number of Participants With Notable Changes in ECG Values From Baseline(Baseline (Week 1 Day 1) up to Week 24, plus 30 day safety follow-up.)
- Number of Participants With Clinically Significant Ocular Assessments Changes From Baseline(Baseline (Week 1 Day 1) up to Week 24, plus 30 day safety follow-up.)
- Absolute Change From Baseline in Systolic and Diastolic Blood Pressures (mmHg)(Baseline (BL), Week 2, Week 3, Week 4, Week 8, Week 12, Week 16, Week 20, EOT (Week 24))
- Adverse Events Profile(Baseline (Week 1 Day 1) up to Week 24, plus 30 day safety follow-up.)
- Absolute Change From Baseline in Serum Ferritin (SF)(Baseline (BL), Week 4, Week 8, Week 12, Week 16, EOT (Week 24))
- Number of Participants With Worst Post-baseline Values in Selected Chemistry Parameters(Baseline (Week 1 Day 1) up to Week 24, plus 30 day safety follow-up.)
- Number of Participants With Clinically Significant Auditory Assessments Changes From Baseline(Baseline (Week 1 Day 1) up to Week 24, plus 30 day safety follow-up.)
- Absolute Change From Baseline in Body Weight (kg)(Baseline (BL), Week 4, Week 8, Week 12, Week 16, Week 20, EOT (Week 24))
- Absolute Change From Baseline in Body Temperature (°C)(Baseline (BL), Week 2, Week 3, Week 4, Week 8, Week 12, Week 16, Week 20, EOT (Week 24))
- Modified Satisfaction With Iron Chelation Therapy (Modified SICT) in Participants Pre-treated With Deferasirox: Mean Change From Baseline in Adherence(Week 4, Week 12, EOT (Week 24))
- Modified SICT in Participants Pre-treated With Deferasirox: Number of Participants With Type of Medicine Child Like Scoring(Baseline (BL), Week 4, Week 12, EOT (Week 24))
- Modified Satisfaction With Iron Chelation Therapy (Modified SICT) in Chelation Naive Participants: Mean Change From Baseline in Adherence(Week 4, Week 12, EOT (Week 24))
- Modified Satisfaction With Iron Chelation Therapy (Modified SICT) in Chelation Naive Participants: Number of Participants With Type of Medicine Child Like Scoring(Week 4, Week 12, EOT (Week 24))
- Modified Satisfaction With Iron Chelation Therapy (Modified SICT) in Chelation Naive Participants: Number of Participants With Rank Based on Child's Preference Scoring(Week 4, Week 12, EOT (Week 24))
- Modified Satisfaction With Iron Chelation Therapy (Modified SICT) in Chelation Naive Participants: Mean Change From Baseline in Concerns(Week 4, Week 12, EOT (Week 24))
- Absolute Change From Baseline in Pulse Rate (Bpm)(Baseline (BL), Week 2, Week 3, Week 4, Week 8, Week 12, Week 16, Week 20, EOT (Week 24))
- Modified SICT in Participants Pre-treated With Deferasirox: Number of Participants With Reasons Child Preferred Crushed Medicine Scoring(Baseline (BL), Week 4, Week 12, EOT (Week 24))
- Modified SICT in Participants Pre-treated With Deferasirox: Number of Participants With Rank Based on Child's Preference Scoring(Baseline (BL), Week 4, Week 12, EOT (Week 24))
- Modified Satisfaction With Iron Chelation Therapy (Modified SICT) in Participants Pre-treated With Deferasirox: Mean Change From Baseline in Concerns(Week 4, Week 12, EOT (Week 24))
- Modified Satisfaction With Iron Chelation Therapy (Modified SICT) in Chelation Naive Participants: Number of Participants With Reasons Child Preferred Crushed Medicine Scoring(Week 4, Week 12, EOT (Week 24))
- Palatability Score in Participants Pre-treated With Deferasirox(Week 4, Week 12, EOT (Week 24))
- GI Symptom Score in Chelation Naive Participants(Week 2, Week 3, Week 4, Week 8, Week 12, Week 16, Week 20, EOT (Week 24))
- Number of Participants With GI Bowel Movements Item Scoring in Chelation Naive Participants(Week 2, Week 3, Week 4, Week 8, Week 12, Week 16, Week 20, EOT (Week 24))
- Number of Participants Pre-treated With Deferasirox With Palatability After Taste Item Scoring(Baseline, Week 4, Week 12, EOT (Week 24))
- Number of Participants With GI Bowel Movements Item Scoring in Participants Pre-treated With Deferasirox(Baseline, Week 2, Week 3, Week 4, Week 8, Week 12, Week 16, Week 20, EOT (Week 24))
- Palatability Score in Chelation Naive Participants(Week 4, Week 12, EOT (Week 24))
- Number of Chelation Naive Participants With Palatability After Taste Item Scoring(Week 4, Week 12, EOT (Week 24))
- GI Symptom Score in Participants Pre-treated With Deferasirox(Week 2, Week 3, Week 4, Week 8, Week 12, Week 16, Week 20, EOT (Week 24))
研究者
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