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Phase II Study to Investigate the Benefits of an Improved Deferasirox Formulation (Film-coated Tablet)

2017年7月22日 更新者：Novartis Pharmaceuticals

A Randomized, Open-label, Multicenter, Two Arm, Phase II Study to Investigate the Benefits of an Improved Deferasirox Formulation (Film-coated Tablet)

Assessed the new film-coated tablet formulation to the currently approved dispersible tablet formulation with regards to overall safety, Gastrointestinal (GI) tolerability, palatability, satisfaction and compliance

研究概览

地位

完全的

条件

干预/治疗

研究类型

介入性

注册 (实际的)

173

阶段

  • 阶段2

联系人和位置

本节提供了进行研究的人员的详细联系信息，以及有关进行该研究的地点的信息。

学习地点

  • 俄罗斯联邦
    • Russia
      • Moskow、Russia、俄罗斯联邦、117198
        • Novartis Investigative Site
  • 墨西哥
    • Distrito Federal
      • Mexico、Distrito Federal、墨西哥、06726
        • Novartis Investigative Site
  • 奥地利
      • Linz、奥地利、A-4010
        • Novartis Investigative Site
      • Wien、奥地利、1140
        • Novartis Investigative Site
  • 希腊
    • GR
      • Athens、GR、希腊、GR-115 27
        • Novartis Investigative Site
      • Patra - RIO、GR、希腊、265 04
        • Novartis Investigative Site
      • Thessaloniki、GR、希腊、546 42
        • Novartis Investigative Site
  • 德国
      • Berlin、德国、13353
        • Novartis Investigative Site
      • Dresden、德国、01307
        • Novartis Investigative Site
      • Goslar、德国、38642
        • Novartis Investigative Site
      • Hannover、德国、30170
        • Novartis Investigative Site
      • Leipzig、德国、04103
        • Novartis Investigative Site
      • Potsdam、德国、14467
        • Novartis Investigative Site
    • Baden-Württemberg
      • Mannheim、Baden-Württemberg、德国、68305
        • Novartis Investigative Site
  • 意大利
      • Napoli、意大利、80138
        • Novartis Investigative Site
    • BR
      • Brindisi、BR、意大利、72100
        • Novartis Investigative Site
    • CT
      • Catania、CT、意大利、95125
        • Novartis Investigative Site
    • FE
      • Cona、FE、意大利、44100
        • Novartis Investigative Site
    • GE
      • Genova、GE、意大利、16132
        • Novartis Investigative Site
      • Genova、GE、意大利、16128
        • Novartis Investigative Site
    • ITA
      • Cagliari、ITA、意大利、09121
        • Novartis Investigative Site
    • LE
      • Lecce、LE、意大利、73100
        • Novartis Investigative Site
    • MI
      • Milano、MI、意大利、20162
        • Novartis Investigative Site
      • Milano、MI、意大利、20122
        • Novartis Investigative Site
    • PA
      • Palermo、PA、意大利、90127
        • Novartis Investigative Site
      • Palermo、PA、意大利、90146
        • Novartis Investigative Site
    • RC
      • Reggio Calabria、RC、意大利、89100
        • Novartis Investigative Site
    • VR
      • Verona、VR、意大利、37126
        • Novartis Investigative Site
  • 沙特阿拉伯
      • Dammam、沙特阿拉伯、15215
        • Novartis Investigative Site
      • Dammam、沙特阿拉伯、40145
        • Novartis Investigative Site
      • Jeddah、沙特阿拉伯、21589
        • Novartis Investigative Site
      • Riyadh、沙特阿拉伯、11472
        • Novartis Investigative Site
  • 法国
      • Lille cedex、法国、59020
        • Novartis Investigative Site
      • Paris、法国、75010
        • Novartis Investigative Site
  • 泰国
      • Bangkok、泰国、10700
        • Novartis Investigative Site
      • Bangkok、泰国、10400
        • Novartis Investigative Site
  • 美国
    • California
      • Orange、California、美国、92868-3874
        • Children's Hospital of Orange County Onc Dept
    • Illinois
      • Chicago、Illinois、美国、60611
        • Lurie Children's Hospital of Chicago Onc Dept
    • Massachusetts
      • Boston、Massachusetts、美国、02115
        • Children's Hospital Boston Department of Hematology
    • New York
      • New York、New York、美国、10021
        • Weill Cornell Medical College-Cornell University Onc Dept
    • Pennsylvania
      • Philadelphia、Pennsylvania、美国、19104-4399
        • Children's Hospital of Philadelphia Onc. Dept
  • 英国
      • London、英国、N19 5NF
        • Novartis Investigative Site
      • London、英国、NW1 2PJ
        • Novartis Investigative Site
  • 西班牙
      • Barcelona、西班牙、08041
        • Novartis Investigative Site
      • Madrid、西班牙、28033
        • Novartis Investigative Site
  • 阿拉伯联合酋长国
      • Al Ain - Abu Dhabi、阿拉伯联合酋长国
        • Novartis Investigative Site
      • Dubai、阿拉伯联合酋长国、9115
        • Novartis Investigative Site
  • 阿根廷
      • Buenos aires、阿根廷、C1221ADC
        • Novartis Investigative Site
  • 马来西亚
      • Kuala Lumpur、马来西亚、50589
        • Novartis Investigative Site
      • Pulau Pinang、马来西亚、10990
        • Novartis Investigative Site
  • 黎巴嫩
    • Beirut
      • Hazmiyeh、Beirut、黎巴嫩、PO BOX 213
        • Novartis Investigative Site

参与标准

研究人员寻找符合特定描述的人，称为资格标准。这些标准的一些例子是一个人的一般健康状况或先前的治疗。

资格标准

适合学习的年龄

10年 及以上 (孩子、成人、年长者)

接受健康志愿者

不

有资格学习的性别

全部

描述

Key Inclusion Criteria:

  • Male and female patients aged ≥ 10 years
  • Patients with transfusion-dependent thalassemia and iron overload, requiring deferasirox DT at doses of ≥ 30 mg/kg/day as per the investigator's decision OR Patients with very low, low or intermediate (int) risk myelodysplastic syndrome (MDS) and iron overload, requiring deferasirox DT at doses of ≥ 20 mg/kg/day as per the investigator's decision.
  • History of transfusion of at least 20 PRBC units and anticipated to be transfused with at least 8 units of PRBCs annually during the study
  • Serum ferritin > 1000 ng/mL, measured at screening Visit 1 and screening Visit 2 (the mean value will be used for eligibility criteria).

Key Exclusion Criteria:

  • Creatinine clearance below the contraindication limit in the locally approved prescribing information. Creatinine clearance will be estimated from serum creatinine at screening Visit 1 and screening Visit 2 and the mean value will be used for eligibility criteria.
  • Serum creatinine > 1.5 xULN at screening measured at screening Visit 1 and screening Visit 2 (the mean value will be used for eligibility criteria).
  • ALT (SGPT) > 5xULN, unless LIC confirmed as >10 mg Fe/dw within 6 months prior to screening visit 1.
  • Significant proteinuria as indicated by a urinary protein/creatinine ratio > 0.5 mg/mg in a non-first void urine sample at screening Visit 1 or screening Visit 2.
  • Patients with significant impaired gastrointestinal (GI) function or GI disease that may significantly alter the absorption of oral deferasirox (e.g. ulcerative diseases, uncontrolled nausea, vomiting, diarrhea, malabsorption syndrome, or small bowel resection).
  • Liver disease with severity of Child-Pugh Class B or C

学习计划

本节提供研究计划的详细信息，包括研究的设计方式和研究的衡量标准。

研究是如何设计的？

设计细节

  • 主要用途：治疗
  • 分配：随机化
  • 介入模型：并行分配
  • 屏蔽：无（打开标签）

武器和干预

参与者组/臂
干预/治疗
有源比较器：Deferasirox dispersible tablet (DFX-DT)
Iron chelation naïve participants received DFX-DT 20 mg/kg/day once daily orally from weeks 1 - 4. After week 4, the dose could be adjusted by +/- 5 to 10 mg/kg/day, with a maximum dose of 40 mg/kg/day. Iron chelation pre-treated participants were supposed to start on a dose that was equivalent to their pre-washout dose.
药品：Deferasirox dispersible tablet
Deferasirox DT was provided as 125 mg, 250 mg and 500 mg dispersible tablets for oral use.
其他名称：
  • ICL670, DT (dispersible tablet)
实验性的：Deferasirox film-coated tablet (DFX-FCT)
Participants received DFX-FCT 14 mg/kg/day once daily orally from weeks 1 - 4. After week 4, the dose could be adjusted by +/- 3.5 to 7 mg/kg/day, with a maximum dose of 28 mg/kg/day. Iron chelation pre-treated participants were supposed to start on a dose that was equivalent to their pre-washout dose
药品：Defearisox film-coated tablet
Deferasirox FCT was provided as 90 mg, 180 mg and 360 mg film-coated tablets for oral use.
其他名称：
  • ICL670, FCT (film-coated tablet)

研究衡量的是什么？

主要结果指标

结果测量
措施说明
大体时间
Overall Safety as Measured by Frequency of Adverse Events
大体时间：28 weeks
The percentage of participants with adverse events, serious adverse events and deaths was assessed.
28 weeks
Overall Safety as Measured by Changes in Laboratory Values From Baseline
大体时间：baseline (BL), 30 weeks
The percentage of participants with post-baseline laboratory values meeting specified criteria for notable/extended range was assessed. The following laboratory parameters were measured: platelet count, absolute neutrophils, serum creatinine , creatinine clearance, urinary protein/urinary creatinine ratio, alanine aminotransferase (ALT) and aspartate aminotransferase (AST). Note that within data categories, creat = creatinine, cons = consecutive, ULN = upper limit of normal and urin = urinary.
baseline (BL), 30 weeks

次要结果测量

结果测量
措施说明
大体时间
Frequency of Selected Gastro-intestinal (GI) Adverse Events
大体时间：28 weeks
The percentage of participants with any GI adverse event, diarrhea, constipation, nausea, vomiting, abdominal pain was assessed.
28 weeks
Mean Domain Scores of the Modified Satisfaction With Iron Chelation Therapy (Modified SICT)
大体时间：weeks 2, 3, 13 and 24 (end of treatment or within 7 days of last dose)
The modified SICT consisted of 13 items that represent 3 domains: adherence, satisfaction and concerns. The adherence domain consisted of 7 items, 6 which were measured using a 5-point response scale and was calculated by summing the 6 items. The score range from 6 to 30 and higher scores indicated worse adherence. The satisfaction domain consisted of 3 items, 2 which were measured using a 5-point response scale and was calculated by summing the 2 items. The score range from 2 to 10 and higher scores indicated worse satisfaction. The concerns domain consisted of 3 items to address any concerns or worries with his/her medication. All 3 items were measured on a 5-point response scale and were calculated by summing the 3 items. The score range from 3 to 15 and higher scores indicated fewer concerns. For all three domains, the meaningful difference between two treatment arms was determined to be 1 point.
weeks 2, 3, 13 and 24 (end of treatment or within 7 days of last dose)
Palatability Questionnaire Score
大体时间：weeks 2, 3, 13 and 24 (end of treatment or within 7 days of last dose)
The palatability questionnaire consisted of 4 items. The first item measured the taste and aftertaste of the medication and were scored a on a 5-point response scale. The second item offered an additional response option of "no aftertaste". The last 2 items referred to whether the medication was taken, i.e. swallowed or vomited, and how the participant perceived the amount of medication to be taken. The palatability summary score was calculated using a scoring matrix from items 1, 3 and 4 scores and the score ranges from 0 - 11. Higher scores indicated the best palatability. A meaningful difference between two treatment arms was determined to be 1 point.
weeks 2, 3, 13 and 24 (end of treatment or within 7 days of last dose)
Weekly Average of Daily Scores of the Gastrointestinal (GI) Symptom Diary
大体时间：weeks -1, 4, 8, 12, 16, 20, 24
The GI symptom diary consisted of 6 items, five which were scored using a 0 - 10 rating scale with item appropriate anchors to rate the symptom, for example, Pain in your belly: 0 = no pain and 10 = worst pain. The GI diary summary score was created using the 10 point response scale for the 5 items. The GI symptom daily diary had a minimum score of 0 and a maximum score of 50. The weekly average score for the 7 days was calculated for each individual item and the GI summary score was created from these weekly averages. Higher scores indicated worse symptoms. A meaningful difference between two treatment arms was determined to be 0.3 point.
weeks -1, 4, 8, 12, 16, 20, 24
Number of Participants With Weekly Average Compliance of Medication Consumption
大体时间：Weeks 1, 2, 3, 4, 5, 6, 7, 8, 9, 10, 11, 12, 13, 14, 15, 16, 17, 18, 19, 20, 21, 22, 23, 24
A compliance questionnaire assessed whether the medication was taken. Weekly average compliance was calculated when there were at least four non-missing daily responses.
Weeks 1, 2, 3, 4, 5, 6, 7, 8, 9, 10, 11, 12, 13, 14, 15, 16, 17, 18, 19, 20, 21, 22, 23, 24
Weekly Dose Violation Rate
大体时间：weeks 1, 4, 8, 12, 16, 20, 24
The dose violation is defined as a dose either missed completely or not taken in accordance with the timing instruction (no later than 12:00 pm. The rate was calculated as [number of dose violations/drug exposure (days)] x 100.
weeks 1, 4, 8, 12, 16, 20, 24
Area Under the Plasma Concentration-time Curve From Time Zero to the Last Quantifiable Concentration (AUClast)
大体时间：week 1, day 1: pre-dose (0 hour) and 1, 2, 3, 4, 8 and 24 hours post dose; week 3, day 1: pre-dose (0 hour) and 1, 2, 3, 4, 8 and 24 hours post dose
Blood samples were collected to assess AUClast.
week 1, day 1: pre-dose (0 hour) and 1, 2, 3, 4, 8 and 24 hours post dose; week 3, day 1: pre-dose (0 hour) and 1, 2, 3, 4, 8 and 24 hours post dose
Observed Maximum Plasma Concentration Following Drug Administration (Cmax)
大体时间：week 1, day 1: pre-dose (0 hour) and 1, 2, 3, 4, 8 and 24 hours post dose; week 3, day 1: pre-dose (0 hour) and 1, 2, 3, 4, 8 and 24 hours post dose
Blood samples were collected to assess Cmax.
week 1, day 1: pre-dose (0 hour) and 1, 2, 3, 4, 8 and 24 hours post dose; week 3, day 1: pre-dose (0 hour) and 1, 2, 3, 4, 8 and 24 hours post dose
Time to Reach the Maximum Plasma Concentration After Drug Administration (Tmax)
大体时间：week 1, day 1: pre-dose (0 hour) and 1, 2, 3, 4, 8 and 24 hours post dose; week 3, day 1: pre-dose (0 hour) and 1, 2, 3, 4, 8 and 24 hours post dose
Blood samples were collected to assess Tmax.
week 1, day 1: pre-dose (0 hour) and 1, 2, 3, 4, 8 and 24 hours post dose; week 3, day 1: pre-dose (0 hour) and 1, 2, 3, 4, 8 and 24 hours post dose
Dererasirox Plasma Concentration
大体时间：Week 3, day 1, pre-dose (0 hour (h)) and 2 h post-dose; week 13, day 1, pre-dose (0 hour (h)) and 2 h post-dose; and week 21, day 1, pre-dose (0 hour (h)) and 2 h post-dose
Blood samples were collected to assess deferasirox concentration. Dose-adjusted calculations are presented: (concentration/actual dose)*20 for participants on DFX-DT and (concentration/actual dose)*14 for participants on DFX-FCT.
Week 3, day 1, pre-dose (0 hour (h)) and 2 h post-dose; week 13, day 1, pre-dose (0 hour (h)) and 2 h post-dose; and week 21, day 1, pre-dose (0 hour (h)) and 2 h post-dose

合作者和调查者

在这里您可以找到参与这项研究的人员和组织。

赞助

Novartis Pharmaceuticals

出版物和有用的链接

负责输入研究信息的人员自愿提供这些出版物。这些可能与研究有关。

一般刊物

研究记录日期

这些日期跟踪向 ClinicalTrials.gov 提交研究记录和摘要结果的进度。研究记录和报告的结果由国家医学图书馆 (NLM) 审查，以确保它们在发布到公共网站之前符合特定的质量控制标准。

研究主要日期

学习开始 (实际的)

2014年7月8日

初级完成 (实际的)

2016年2月24日

研究完成 (实际的)

2016年2月24日

研究注册日期

首次提交

2014年4月26日

首先提交符合 QC 标准的

2014年4月26日

首次发布 (估计)

2014年4月29日

研究记录更新

最后更新发布 (实际的)

2017年7月25日

上次提交的符合 QC 标准的更新

2017年7月22日

最后验证

2017年7月1日

更多信息

与本研究相关的术语

关键字

其他相关的 MeSH 术语

其他研究编号

  • CICL670F2201

此信息直接从 clinicaltrials.gov 网站检索，没有任何更改。如果您有任何更改、删除或更新研究详细信息的请求，请联系 register@clinicaltrials.gov. clinicaltrials.gov 上实施更改，我们的网站上也会自动更新.

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