Study of Safety And Efficacy Of ReFacto AF In Previously Untreated Hemophilia A Patients In The Usual Care Setting
2019年5月10日 更新者：Pfizer
AN OPEN-LABEL STUDY OF THE SAFETY AND EFFICACY OF REFACTO AF IN PREVIOUSLY UNTREATED PATIENTS IN USUAL CARE SETTINGS
Study to evaluate the safety and effectiveness of ReFacto AF for the treatment of severe hemophilia A in patients who have not yet received treatment for their hemophilia.
Study subjects will be males less than 6 years old who have not taken any clotting factor or other blood products before the study.
The safety and effectiveness of ReFacto AF will be determined in this study by tests and procedures done at the doctor's office.
調査の概要
詳細な説明
Regulatory Commitment
研究の種類
介入
入学 (実際)
23
段階
- フェーズ 4
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
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Firenze、イタリア、50134
- Agenzia per L'emofilia e Centro Regionale Riferimento Coagulopatie Congenite
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Lviv、ウクライナ、79044
- Derzhavna ustanova "Instytut patolohii krovi ta transfuziinoi medytsyny Natsionalnoi akademii
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Groningen、オランダ、9713 GZ
- University Medical Center Groningen
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Madrid、スペイン、28046
- Hospital Universitario La Paz
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Valencia、スペイン、46009
- Hospital Universitario La Fe
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Bremen、ドイツ、28177
- Klinikum Bremen-Mitte gGmbH, Professor Hess Kinderklinik
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Niedersachsen
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Hannover、Niedersachsen、ドイツ、30625
- Medizinische Hochschule, Hannover
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Nantes、フランス、44093
- CHU de Nantes
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Adana、七面鳥、01330
- Cukurova Universitesi Tip Fakultesi
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Istanbul、七面鳥、34098
- Istanbul Universitesi Cerrahpasa Tip Fakultesi
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Izmir
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Bornova、Izmir、七面鳥、35100
- Ege Universitesi Tip Fakultesi
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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
6年歳未満 (子)
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
男
説明
Inclusion Criteria:
- Male subjects <6 years of age with severe hemophilia A (FVIII:C <1%) based on clinical records, including newborns.
- No prior exposure to factor products or any blood products.
Exclusion Criteria:
- Presence of any bleeding disorder in addition to hemophilia A.
- Treatment with any investigational agent or device within the past 30 days.
- Any condition(s) that compromises the ability to collect study-related observations, or that poses a contraindication to study participation (these conditions include, but are not limited to, inadequate medical history to assure study eligibility; and expectation of poor adherence to study requirements).
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：他の
- 割り当て：なし
- 介入モデル：単一グループの割り当て
- 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 |
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実験的：1
The investigator treats subjects with ReFacto AF in the usual care setting.
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Blood draws to determine the level of factor VIII activity before and after dosing at appropriate visits, and blood draws to determine levels of factor VIII inhibitor (antibody to factor VIII).
他の名前：
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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 |
|---|---|---|
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Percentage of Participants Who Developed Clinically Significant Factor VIII (FVIII) Inhibitors During the Course of the Study
時間枠：2 years
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Percentage of participants who developed clinically significant FVIII inhibitors: those persistent over a defined period with clinically impactful effects like breakthrough bleed, low recovery, etc., during the course of the study.
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2 years
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二次結果の測定
結果測定 |
メジャーの説明 |
時間枠 |
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Annualized Bleeding Rate (ABR)
時間枠：2 years
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Annualized bleeding rate was calculated as the number of bleeds divided by the treatment interval duration (enrollment visit to final visit) and then multiplied by 365.25.
If there was more than 1 bleed location (like ankle and joint) with identical bleed start date and time, it was treated as 1 bleed occurrence.
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2 years
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Total Number of Infusions to Treat a New Bleed Classified on Basis of Response to First On-Demand Treatment With Refacto AF
時間枠：2 years
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Number of infusions of Refacto AF required to treat a new bleed were classified on basis of the response to at 4-point response scale of assessment (excellent, good, moderate and no response).
Assessment was completed each time a participant experienced a new bleed requiring an 'on-demand' IV infusion.
Excellent: definite pain relief and/or improvement in bleeding signs within 8 hours (hr) after infusion, no additional infusion administered; Good: definite pain relief and/or improvement in bleeding signs within 8 hr after infusion, at least 1 additional infusion administered for complete resolution or with no additional infusion administered; Moderate: probable or slight improvement starting after 8 hr following infusion, at least 1 additional infusion administered for complete resolution; No Response: no improvement at all between infusions or during 24 hr interval following infusion or condition worsen.
Bleeds for which response not recorded, reported as: Data Not Recorded.
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2 years
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Total Number of Infusions Needed for Resolution of Bleeding Episodes Classified on Basis of Response to Study Drug Infusion
時間枠：Within 48 hours after infusion, up to 2 years treatment duration
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Number of infusions of Refacto AF required for resolution of a bleeding episodes were classified on basis of the response at 4-point response scale of assessment (excellent, good, moderate and no response).
Excellent: definite pain relief and/or improvement in bleeding signs within 8 hours (hr) after infusion, no additional infusion administered; Good: definite pain relief and/or improvement in bleeding signs within 8 hr after infusion, at least 1 additional infusion administered for complete resolution or with no additional infusion administered; Moderate: probable or slight improvement starting after 8 hr following infusion, at least 1 additional infusion administered for complete resolution; No Response: no improvement at all between infusions or during 24 hr interval following infusion or condition worsen.
Bleeds for which response not recorded, reported as: Data Not Recorded.
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Within 48 hours after infusion, up to 2 years treatment duration
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Total Number of Breakthrough Bleeding Episodes Occurring Within 48 Hours After a Prophylaxis Infusion of ReFacto AF
時間枠：2 years
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The number of breakthrough bleeds (spontaneous or traumatic) within 48 hours following a prophylaxis dose of ReFacto AF are summarized.
If there was more than 1 bleed location (like ankle and joint) with identical bleed start date and time, it was treated as 1 bleed occurrence.
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2 years
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Consumption of Total International Units of Factor VIII
時間枠：2 years
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2 years
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Consumption of Total International Units of Factor VIII Per Year
時間枠：2 years
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Consumption of total international units of Factor VIII per year was calculated for a participant: dividing the total consumption of factor VIII by participant's treatment interval duration (in days), then multiplying by 365.25.
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2 years
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Mean Dose (IU) of Study Drug Consumed Per Infusion
時間枠：2 years
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Mean dose for each participant was calculated as participant's total factor VIII consumption (in IU) divided by the number of infusions administered.
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2 years
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Consumption of Total International Units of Factor VIII by Weight
時間枠：2 years
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Consumption of total international units of Factor VIII by weight was calculated for a participant: dividing the total consumption of factor VIII by participant's weight (the most recently recorded).
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2 years
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Consumption of Total International Units of Factor VIII Per Year by Weight
時間枠：2 years
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Consumption of total international units of Factor VIII per year by weight was calculated for a participant: the total consumption of factor VIII divided by participant's treatment interval duration (in days), then multiplying by 365.25 and then dividing by participant's weight (the most recently recorded).
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2 years
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Mean Dose (IU) of Study Drug Consumed Per Infusion by Weight
時間枠：2 years
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Mean dose for each participant was calculated as participant's total factor consumption (in IU) divided by the number of infusions administered and then dividing by participant's weight (the most recently recorded).
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2 years
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Mean of Total Number of Infusions of Study Drug Received
時間枠：2 years
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2 years
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Mean of Total Number of Days Participants Exposed to Study Drug
時間枠：2 years
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2 years
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Number of Participants Who Required Dose Escalation of Their Prescribed Prophylaxis Regimen During Their Participation in This Study
時間枠：2 years
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The number of participants who met the dose escalation criteria were prescribed a higher dose and/or were prescribed more frequent doses.
When dose escalation was required, the specific dose and dosing schedule was at the investigator's discretion.
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2 years
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Percentage of Bleeding Episodes With Less-Than-Expected Therapeutic Effect (LETE) in On-Demand (OD) Setting
時間枠：2 years
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LETE occurs in OD setting if participant recorded 2 successive "No Response" (no improvement at all between infusions, or condition worsens) ratings after 2 successive infusions of study drug.
Infusions must have been given within 24 hours (hr) of each other for treatment of same bleeding event in absence of confounding factors (known presence or subsequent identification of a FVIII inhibitor, known inadequate dose for type and/or severity of bleed in opinion of investigator, delay of greater than (>) 4 hr between onset of bleed to infusion, delay of >24 hr before administration of a follow-up infusion, known compromised study drug, faulty administration of study drug, participant had an underlying, predisposing condition responsible for bleed in opinion of investigator, ongoing trauma responsible for continued bleeding.
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2 years
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Percentage of Bleeding Episodes With Less-Than-Expected Therapeutic Effect (LETE) in the Prophylaxis Setting
時間枠：2 years
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LETE in prophylaxis setting if there was a spontaneous bleed within 48 hours after a regularly scheduled prophylactic dose of study drug (which was not used to treat a bleed) in the absence of confounding factors (known presence or subsequent identification of a FVIII inhibitor, known inadequate prophylactic dose [a dose less than that prescribed in participant's regimen], known lack of adherence to the prescribed prophylaxis regimen, known compromised study drug, faulty administration of study drug, participant had an underlying, predisposing condition responsible for the bleed in the opinion of the investigator, traumatic injury responsible for bleeding.
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2 years
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Total Number of Events of Potential Less-Than-Expected Therapeutic Effect (LETE) in the Low Recovery Setting
時間枠：2 years
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LETE was lower than expected recovery of FVIII in the opinion of the investigator following infusion of study drug in the absence of confounding factors (known presence or subsequent identification of a FVIII inhibitor, known compromised study drug, faulty administration of study drug including inadequate dosing).
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2 years
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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
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。
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)
2010年2月10日
一次修了 (実際)
2016年11月24日
研究の完了 (実際)
2016年11月24日
試験登録日
最初に提出
2009年7月29日
QC基準を満たした最初の提出物
2009年7月30日
最初の投稿 (見積もり)
2009年7月31日
学習記録の更新
投稿された最後の更新 (実際)
2019年7月19日
QC基準を満たした最後の更新が送信されました
2019年5月10日
最終確認日
2019年5月1日
詳しくは
本研究に関する用語
その他の研究ID番号
- 3082B2-4434
- B1831006 (その他の識別子：Alias Study Number)
- 2008-008436-93 (EudraCT番号)
個々の参加者データ (IPD) の計画
個々の参加者データ (IPD) を共有する予定はありますか?
はい
IPD プランの説明
Pfizer will provide access to individual de-identified participant data and related study documents (e.g.
protocol, Statistical Analysis Plan (SAP), Clinical Study Report (CSR)) upon request from qualified researchers, and subject to certain criteria, conditions, and exceptions.
Further details on Pfizer's data sharing criteria and process for requesting access can be found at: https://www.pfizer.com/science/clinical_trials/trial_data_and_results/data_requests.
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。