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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

調査の概要

状態

完了

条件

介入・治療

研究の種類

介入

入学 (実際)

173

段階

  • フェーズ2

連絡先と場所

このセクションには、調査を実施する担当者の連絡先の詳細と、この調査が実施されている場所に関する情報が記載されています。

研究場所

  • アメリカ
    • California
      • Orange、California、アメリカ、92868-3874
        • Children's Hospital of Orange County Onc Dept
    • Illinois
      • Chicago、Illinois、アメリカ、60611
        • Lurie Children's Hospital of Chicago Onc Dept
    • Massachusetts
      • Boston、Massachusetts、アメリカ、02115
        • Children's Hospital Boston Department of Hematology
    • New York
      • New York、New York、アメリカ、10021
        • Weill Cornell Medical College-Cornell University Onc Dept
    • Pennsylvania
      • Philadelphia、Pennsylvania、アメリカ、19104-4399
        • Children's Hospital of Philadelphia Onc. Dept
  • アラブ首長国連邦
      • Al Ain - Abu Dhabi、アラブ首長国連邦
        • Novartis Investigative Site
      • Dubai、アラブ首長国連邦、9115
        • Novartis Investigative Site
  • アルゼンチン
      • Buenos aires、アルゼンチン、C1221ADC
        • Novartis Investigative Site
  • イギリス
      • London、イギリス、N19 5NF
        • Novartis Investigative Site
      • London、イギリス、NW1 2PJ
        • Novartis Investigative Site
  • イタリア
      • Napoli、イタリア、80138
        • Novartis Investigative Site
    • BR
      • Brindisi、BR、イタリア、72100
        • Novartis Investigative Site
    • CT
      • Catania、CT、イタリア、95125
        • Novartis Investigative Site
    • FE
      • Cona、FE、イタリア、44100
        • Novartis Investigative Site
    • GE
      • Genova、GE、イタリア、16132
        • Novartis Investigative Site
      • Genova、GE、イタリア、16128
        • Novartis Investigative Site
    • ITA
      • Cagliari、ITA、イタリア、09121
        • Novartis Investigative Site
    • LE
      • Lecce、LE、イタリア、73100
        • Novartis Investigative Site
    • MI
      • Milano、MI、イタリア、20162
        • Novartis Investigative Site
      • Milano、MI、イタリア、20122
        • Novartis Investigative Site
    • PA
      • Palermo、PA、イタリア、90127
        • Novartis Investigative Site
      • Palermo、PA、イタリア、90146
        • Novartis Investigative Site
    • RC
      • Reggio Calabria、RC、イタリア、89100
        • Novartis Investigative Site
    • VR
      • Verona、VR、イタリア、37126
        • Novartis Investigative Site
  • オーストリア
      • Linz、オーストリア、A-4010
        • Novartis Investigative Site
      • Wien、オーストリア、1140
        • Novartis Investigative Site
  • ギリシャ
    • GR
      • Athens、GR、ギリシャ、GR-115 27
        • Novartis Investigative Site
      • Patra - RIO、GR、ギリシャ、265 04
        • Novartis Investigative Site
      • Thessaloniki、GR、ギリシャ、546 42
        • Novartis Investigative Site
  • サウジアラビア
      • Dammam、サウジアラビア、15215
        • Novartis Investigative Site
      • Dammam、サウジアラビア、40145
        • Novartis Investigative Site
      • Jeddah、サウジアラビア、21589
        • Novartis Investigative Site
      • Riyadh、サウジアラビア、11472
        • Novartis Investigative Site
  • スペイン
      • Barcelona、スペイン、08041
        • Novartis Investigative Site
      • Madrid、スペイン、28033
        • Novartis Investigative Site
  • タイ
      • Bangkok、タイ、10700
        • Novartis Investigative Site
      • Bangkok、タイ、10400
        • Novartis Investigative Site
  • ドイツ
      • Berlin、ドイツ、13353
        • Novartis Investigative Site
      • Dresden、ドイツ、01307
        • Novartis Investigative Site
      • Goslar、ドイツ、38642
        • Novartis Investigative Site
      • Hannover、ドイツ、30170
        • Novartis Investigative Site
      • Leipzig、ドイツ、04103
        • Novartis Investigative Site
      • Potsdam、ドイツ、14467
        • Novartis Investigative Site
    • Baden-Württemberg
      • Mannheim、Baden-Württemberg、ドイツ、68305
        • Novartis Investigative Site
  • フランス
      • Lille cedex、フランス、59020
        • Novartis Investigative Site
      • Paris、フランス、75010
        • Novartis Investigative Site
  • マレーシア
      • Kuala Lumpur、マレーシア、50589
        • Novartis Investigative Site
      • Pulau Pinang、マレーシア、10990
        • Novartis Investigative Site
  • メキシコ
    • Distrito Federal
      • Mexico、Distrito Federal、メキシコ、06726
        • Novartis Investigative Site
  • レバノン
    • Beirut
      • Hazmiyeh、Beirut、レバノン、PO BOX 213
        • Novartis Investigative Site
  • ロシア連邦
    • Russia
      • Moskow、Russia、ロシア連邦、117198
        • Novartis Investigative Site

参加基準

研究者は、適格基準と呼ばれる特定の説明に適合する人を探します。これらの基準のいくつかの例は、人の一般的な健康状態または以前の治療です。

適格基準

就学可能な年齢

10年歳以上 (子、大人、高齢者)

健康ボランティアの受け入れ

いいえ

受講資格のある性別

全て

説明

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

研究計画

このセクションでは、研究がどのように設計され、研究が何を測定しているかなど、研究計画の詳細を提供します。

研究はどのように設計されていますか？

デザインの詳細

  • 主な目的：処理
  • 割り当て：ランダム化
  • 介入モデル：並列代入
  • マスキング：なし（オープンラベル）

武器と介入

参加者グループ / アーム
介入・治療
アクティブコンパレータ：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.
薬：Deferasirox dispersible tablet
Deferasirox DT was provided as 125 mg, 250 mg and 500 mg dispersible tablets for oral use.
他の名前：
  • ICL670, DT (dispersible tablet)
実験的：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
薬：Defearisox film-coated tablet
Deferasirox FCT was provided as 90 mg, 180 mg and 360 mg film-coated tablets for oral use.
他の名前：
  • ICL670, FCT (film-coated tablet)

この研究は何を測定していますか？

主要な結果の測定

結果測定
メジャーの説明
時間枠
Overall Safety as Measured by Frequency of Adverse Events
時間枠：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
時間枠：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

二次結果の測定

結果測定
メジャーの説明
時間枠
Frequency of Selected Gastro-intestinal (GI) Adverse Events
時間枠：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)
時間枠：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
時間枠：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
時間枠：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
時間枠：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
時間枠：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)
時間枠：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)
時間枠：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)
時間枠：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
時間枠：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

協力者と研究者

ここでは、この調査に関係する人々や組織を見つけることができます。

スポンサー

Novartis Pharmaceuticals

出版物と役立つリンク

研究に関する情報を入力する責任者は、自発的にこれらの出版物を提供します。これらは、研究に関連するあらゆるものに関するものである可能性があります。

一般刊行物

研究記録日

これらの日付は、ClinicalTrials.gov への研究記録と要約結果の提出の進捗状況を追跡します。研究記録と報告された結果は、国立医学図書館 (NLM) によって審査され、公開 Web サイトに掲載される前に、特定の品質管理基準を満たしていることが確認されます。

主要日程の研究

研究開始 (実際)

2014年7月8日

一次修了 (実際)

2016年2月24日

研究の完了 (実際)

2016年2月24日

試験登録日

最初に提出

2014年4月26日

QC基準を満たした最初の提出物

2014年4月26日

最初の投稿 (見積もり)

2014年4月29日

学習記録の更新

投稿された最後の更新 (実際)

2017年7月25日

QC基準を満たした最後の更新が送信されました

2017年7月22日

最終確認日

2017年7月1日

詳しくは

本研究に関する用語

キーワード

追加の関連 MeSH 用語

その他の研究ID番号

  • CICL670F2201

この情報は、Web サイト clinicaltrials.gov から変更なしで直接取得したものです。研究の詳細を変更、削除、または更新するリクエストがある場合は、register@clinicaltrials.gov。 までご連絡ください。 clinicaltrials.gov に変更が加えられるとすぐに、ウェブサイトでも自動的に更新されます。

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