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血友病Aの参加者の長期的な関節の健康に対するエファネスソクトコグ アルファの影響を評価する研究

2026年9月24日 更新者：Sanofi

血友病A患者の長期的な関節の健康に対するエファネスソクトコグ アルファの有効性に関する前向き観察的多施設共同研究

This is a prospective, observational, multi-center longitudinal cohort study to describe the real-world effectiveness, safety and treatment usage of efanesoctocog alfa in patients with hemophilia A treated per standard of care in the US and Japan.

Patients will be enrolled in the study after the introduction of efanesoctocog alfa in the hemophilia treatment landscape in each study country. Decision to initiate treatment with commercially available efanesoctocog alfa will be made by the treating physician independently from the decision to include patients in the study. No study medication is provided. The data related to efanesoctocog alfa effectiveness, safety and usage will be collected prospectively during routine visits (expected annual/semi-annual visits) for up to 5 years following enrollment /treatment initiation.

調査の概要

状態

積極的、募集していない

条件

介入・治療

研究の種類

観察的

入学 (推定)

200

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
    • Arizona
      • Gilbert、Arizona、アメリカ、85234-2165
        • Banner MD Anderson Cancer Center- Site Number : 8400008
    • California
      • Loma Linda、California、アメリカ、92354-3450
        • Loma Linda University- Site Number : 8400015
      • Los Angeles、California、アメリカ、90007
        • Orthopedic Institute for Children- Site Number : 8400004
      • Orange、California、アメリカ、92868
        • The Center for Inherited Blood Disorders- Site Number : 8400001
    • Colorado
      • Aurora、Colorado、アメリカ、80045-7202
        • University of Colorado Hemophilia and Thrombosis Center- Site Number : 8400019
    • Florida
      • Gainesville、Florida、アメリカ、32610-3003
        • William A. Shands Hospital at the University of Florida- Site Number : 8400032
      • St. Petersburg、Florida、アメリカ、33701-4804
        • Johns Hopkins All Childrens Hospital - Outpatient Care Center - PIN- Site Number : 8400025
    • Illinois
      • Chicago、Illinois、アメリカ、60612-3863
        • Rush University Medical Center -1725 W Harrison St- Site Number : 8400014
      • Peoria、Illinois、アメリカ、61615
        • The Bleeding and Clotting Disorders Institute- Site Number : 8400005
    • Indiana
      • Indianapolis、Indiana、アメリカ、46260-1920
        • Indiana Hemophilia and Thrombosis Center- Site Number : 8400013
    • Iowa
      • Iowa City、Iowa、アメリカ、52242-1009
        • University Of Iowa Hospitals And Clinics- Site Number : 8400026
    • Massachusetts
      • Boston、Massachusetts、アメリカ、02114
        • ~Massachusetts General Hospital- Site Number : 8400017
      • Boston、Massachusetts、アメリカ、02215-5418
        • Dana Farber and Boston Children's Hospital- Site Number : 8400031
    • Michigan
      • Ann Arbor、Michigan、アメリカ、48109-5000
        • University of Michigan Hospital - 1500 E Medical Center Dr- Site Number : 8400023
    • Minnesota
      • Minneapolis、Minnesota、アメリカ、55455-4800
        • M Health Fairview- Masonic Cancer Clinic - Clinics- Site Number : 8400010
      • Rochester、Minnesota、アメリカ、55905-0001
        • Mayo Clinic - PPDS- Site Number : 8400035
    • Mississippi
      • Madison、Mississippi、アメリカ、39110-6115
        • Mississippi Center For Advanced Medicine - 7731 Old Canton Rd- Site Number : 8400016
    • Nebraska
      • Omaha、Nebraska、アメリカ、68114-4113
        • University of Nebraska Medical Center - 985400 Nebraska Medical Center- Site Number : 8400009
    • Nevada
      • Las Vegas、Nevada、アメリカ、89109
        • Children's Specialty Center - Las Vegas- Site Number : 8400007
    • New York
      • New York、New York、アメリカ、10065-4870
        • Weill Cornell Medicine-New York Presbyterian Hospital- Site Number : 8400021
    • Ohio
      • Cincinnati、Ohio、アメリカ、45229-3026
        • Cincinnati Children's Hospital Medical Center - PIN- Site Number : 8400006
    • Pennsylvania
      • Pittsburgh、Pennsylvania、アメリカ、15213-4306
        • Hemophilia Center of Western Pennsylvania- Site Number : 8400012
    • Tennessee
      • Nashville、Tennessee、アメリカ、37232-0011
        • Vanderbilt University Medical Center- Site Number : 8400003
    • Texas
      • Dallas、Texas、アメリカ、75390-7202
        • University of Texas Southwestern Medical Center-2001 Inwood Rd- Site Number : 8400020
      • Dallas、Texas、アメリカ、75390-8565
        • University of Texas Southwestern Medical Center- Site Number : 8400011
      • Houston、Texas、アメリカ、77030
        • Gulf States Hemophilia and Thrombophilia Center- Site Number : 8400029
    • Washington
      • Seattle、Washington、アメリカ、98101-3932
        • Washington Institute for Coagulation- Site Number : 8400022
    • Wisconsin
      • Milwaukee、Wisconsin、アメリカ、53233-2121
        • Blood Center of Wisconnsin- Site Number : 8400027

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

  • 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

サンプリング方法

非確率サンプル

調査対象母集団

Approximately 200 patients from sites in the US and Japan will be enrolled in the study after the introduction of efanesoctocog alfa in the hemophilia treatment landscape in each of these two countries. To generate data for the broad real-world population of individuals with hemophilia A, eligible patients will include all ages, sexes and hemophilia A severities, whether on prophylactic or on-demand therapy or if efanesoctocog alfa is taken perioperatively.

説明

Inclusion Criteria:

  • Have a diagnosis of hemophilia A
  • Patients starting efanesoctocog alfa treatment as per standard of care no more than one month prior to the enrollment date, for either on demand or prophylaxis. Patients starting efanesoctocog alfa treatment for a surgery event may also be enrolled only if the treatment is prescribed at enrollment.
  • Physician's decision to treat the patient with efanesoctocog alfa is made prior to and independently of participation in the study.
  • Signed and dated informed consent provided by the patient, or by the patient's legally acceptable representative for patients under the legal age before any study-related activities are undertaken. Assent should be obtained for pediatric patients according to local regulations.

Exclusion Criteria:

Diagnosed with other known bleeding disorder

  • Participation in an investigational medicinal product trial at enrollment visit, or intake of an Investigational Medicinal Product within 3 months prior to inclusion in this study
  • Current diagnosis of a FVIII inhibitor, defined as inhibitor titer ≥0.60 BU/mL

"The above information is not intended to contain all considerations relevant to a potential participation in a clinical trial."

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

  • 観測モデル：COHORT
  • 時間の展望：PROSPECTIVE

コホートと介入

グループ/コホート
介入・治療
Cohort A (Prophylactic treatment)

All participants on efanesoctocog alfa prophylactic treatment fulfilling the overall study inclusion/exclusion criteria. The prophylactic cohort will include the following sub-cohorts:

Sub-cohort A1 (Joint imaging): Participants with severe hemophilia A and joint imaging by Hemophilia Early Arthropathy Detection with Ultrasound (HEAD-US) or Joint Tissue Activity and Damage Exam (JADE) protocol performed within 6 months of initiating treatment with efanesoctocog alfa or within 3 months after initiating treatment with efanesoctocog alfa available.

Sub-cohort A2 (Children with no prior joint damage):Participants with severe hemophilia A who have no prior joint damage
薬：Efanesoctocog Alfa BIVV001

Given per investigator's discretion

他の名前：
  • アルトゥヴィオ
Cohort B (On-Demand treatment)

Participants receiving on-demand treatment with efanesoctocog alfa who fulfil the overall study inclusion/exclusion criteria
薬：Efanesoctocog Alfa BIVV001

Given per investigator's discretion

他の名前：
  • アルトゥヴィオ

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
Change from baseline in Annualized joint bleeding rate (AjBR) for treated bleeds
時間枠：Up to 5 years

Data will be reported for the effectiveness of efanesoctocog alfa prophylaxis on clinical joint status over 5-years for prophylactic cohort
Up to 5 years
Change from baseline in Annualized joint bleeding rate (AjBR) for all (treated and untreated) bleeds
時間枠：Up to 5 years

Data will be reported for the effectiveness of efanesoctocog alfa prophylaxis on clinical joint status over 5-years for prophylactic cohort
Up to 5 years
Number of Target joint development, resolution and/or recurrence
時間枠：Up to 5 years

Data will be reported for the effectiveness of efanesoctocog alfa prophylaxis on clinical joint status over 5-years for prophylactic coho
Up to 5 years

二次結果の測定

結果測定
メジャーの説明
時間枠
Change from baseline in the Hemophilia Joint Health Score (HJHS v2.1) total/domains scores
時間枠：At 1, 2, 3, 4, 5 years

HJHS total/domain scores will be reported for prophylactic cohorts (A1 ad A2)
At 1, 2, 3, 4, 5 years
Annual Bleeding Rate (ABR) by type for treated bleeds
時間枠：At 1, 2, 3, 4, 5 years

Data will be reported for prophylactic cohorts (A1 and A2)
At 1, 2, 3, 4, 5 years
ABR by type all (treated and untreated) bleeds
時間枠：At 1, 2, 3, 4, 5 years

Data will be reported for prophylactic cohorts (A1 and A2)
At 1, 2, 3, 4, 5 years
ABR by location for treated bleeds
時間枠：At 1, 2, 3, 4, 5 years

Data will be reported for prophylactic cohorts (A1 and A2)
At 1, 2, 3, 4, 5 years
ABR by location for all (treated and untreated) bleeds
時間枠：At 1, 2, 3, 4, 5 years

Data will be reported for prophylactic cohorts (A1 and A2)
At 1, 2, 3, 4, 5 years
ABR for all bleeding episodes (including untreated bleeding episodes)
時間枠：At 1, 2, 3, 4, 5 years

Data will be reported for prophylactic cohorts (A1 and A2)
At 1, 2, 3, 4, 5 years
Percentage of patients with zero joint bleeds
時間枠：At 1, 2, 3, 4, 5 years

Data will be reported for prophylactic cohorts (A1 and A2)
At 1, 2, 3, 4, 5 years
Annualized factor consumption per participant (IU/kg) assessed by prescription during the follow-up period.
時間枠：At 1, 2, 3, 4, 5 years

Data will be reported for prophylactic cohorts (A1 and A2)
At 1, 2, 3, 4, 5 years
Annualized injection frequency per participant (assessed by prescription) during the follow- up period
時間枠：At 1, 2, 3, 4, 5 years

Data will be reported for prophylactic cohorts (A1 and A2)
At 1, 2, 3, 4, 5 years
Treatment adherence (%) as judged by the physician during the follow-up period
時間枠：At 1, 2, 3, 4, 5 years

Data will be reported for prophylactic cohorts (A1 and A2)
At 1, 2, 3, 4, 5 years
Number of injections of efanesoctocog alfa to treat a bleeding episode.
時間枠：At 1, 2, 3, 4, 5 years

Data will be reported for prophylactic cohorts (Sub Cohorts A1 and A2)
At 1, 2, 3, 4, 5 years
Total dose of efanesoctocog alfa to treat a bleeding episode.
時間枠：At 1, 2, 3, 4, 5 years

Data will be reported for prophylactic cohorts (Sub Cohorts A1 and A2)
At 1, 2, 3, 4, 5 years
Number of injections of efanesoctocog alfa to treat a bleeding episode
時間枠：At 1, 2, 3, 4, 5 years

Data will be reported for On-demand cohort
At 1, 2, 3, 4, 5 years
Total dose of efanesoctocog alfa to treat a bleeding episode
時間枠：At 1, 2, 3, 4, 5 years

Data will be reported for On-demand cohort
At 1, 2, 3, 4, 5 years
Percentage of bleeding episodes treated with a single injection of efanesoctocog alfa.
時間枠：At 1, 2, 3, 4, 5 years

Data will be reported for On-demand cohort
At 1, 2, 3, 4, 5 years
Occurrence of a change in treatment regimen (on-demand to prophylactic or prophylactic to on-demand) at baseline and follow-up.
時間枠：At 1, 2, 3, 4, 5 years

Data will be reported for On-demand cohort
At 1, 2, 3, 4, 5 years
Change from baseline in Hemophilia Early Arthropathy Detection with Ultrasound (HEAD-US) total/domain scores
時間枠：At 1, 2, 3, 4 and 5 years

HEAD-US total/domain scores will be reported for joint imaging sub cohort
At 1, 2, 3, 4 and 5 years
Change from baseline in Hemophilia Early Arthropathy Detection with Joint Tissue Activity and Damage exam (JADE) musculoskeletal ultrasound (MSKUS) (JADE MSKUS)
時間枠：At 1, 2, 3, 4 and 5 years

Data will be reported for consenting participants from the joint imaging sub-cohort
At 1, 2, 3, 4 and 5 years
Change from baseline in synovial hypertrophy by change in mm thickness AND/OR HEAD-US synovitis domain, AND/OR by JADE MSKUS synovial hypertrophy +/- power doppler signal
時間枠：At 6 months, 1, 2, 3, 4 and 5 years

Data will be reported from the consenting participants from the joint imaging sub-cohort
At 6 months, 1, 2, 3, 4 and 5 years
Occurrence of adverse events (AEs) and serious adverse events (SAEs)
時間枠：Over 5 year period

The safety and tolerability of efanesoctocog alfa over 5 years evaluated in all participants
Over 5 year period
Development of inhibitors (neutralizing antibodies directed against factor FVIII as determined via the Nijmegen modified Bethesda assay.
時間枠：Over 5 year period

The safety and tolerability of efanesoctocog alfa over 5 years assessed in all participants
Over 5 year period
Change from baseline in Patient-Reported Outcomes Measurement Information System (PROMIS) pain intensity 3a questionnaire (adults)
時間枠：At 3 months, 6 months, 1, 2, 3, 4 and 5 years

Effectiveness of efanesoctocog alfa assessed per participants' PROs in all participants
At 3 months, 6 months, 1, 2, 3, 4 and 5 years
Change from baseline in Canadian Hemophilia Outcomes-Kids' Life Assessment Tool (CHO-KLAT) 3.0 (boys ≤18 years old) questionnaire (including Parent Proxy)
時間枠：At 1, 2, 3, 4 and 5 years

Effectiveness of efanesoctocog alfa assessed per participants' PROs in all participants
At 1, 2, 3, 4 and 5 years
Change from baseline in the occurrence of inpatient and outpatient visits, and length of hospital stay related to hemophilia A
時間枠：At 1, 2, 3, 4 and 5 years

Healthcare resource use (HCRU) in efanesoctocog alfa related to hemophilia A over a 5-year period
At 1, 2, 3, 4 and 5 years
Hemostatic response/physician reported during peri-operative period for surgery (major, minor) with efanesoctocog alfa.
時間枠：Over a 5 year period

Data reported to describe the usage and effectiveness of efanesoctocog alfa during the perioperative period
Over a 5 year period
Number of injections required to maintain hemostasis during perioperative period for surgery (major, minor)
時間枠：Over 5 year period

Data reported to describe the usage and effectiveness of efanesoctocog alfa during the perioperative period
Over 5 year period
Dose per injection required to maintain hemostasis during perioperative period for surgery (major, minor)
時間枠：Over 5 year period

Data reported to describe the usage and effectiveness of efanesoctocog alfa during the perioperative period
Over 5 year period
Total efanesoctocog alfa consumption (IU) during perioperative period for surgery (major, minor)
時間枠：Over 5 year period

Data reported to describe the usage and effectiveness of efanesoctocog alfa during the perioperative period
Over 5 year period
Number of blood component transfusions used during perioperative period for surgery
時間枠：Over 5 year Period

Data reported to describe the usage and effectiveness of efanesoctocog alfa during the perioperative period
Over 5 year Period
Type of blood component transfusions used during perioperative period for surgery
時間枠：Over 5 year period

Data reported to describe the usage and effectiveness of efanesoctocog alfa during the perioperative period
Over 5 year period
Estimated blood loss (ml) (intraoperative and post-operative period) for major surgery
時間枠：Over 5 year period

Data reported to describe the usage and effectiveness of efanesoctocog alfa during the perioperative period
Over 5 year period
Number of transfusions required for surgery (intraoperative and post-operative period)
時間枠：Over 5 year period

Data reported to describe the usage and effectiveness of efanesoctocog alfa during the perioperative period
Over 5 year period
Duration of hospitalization (major, minor)
時間枠：Over 5 years period

Data reported to describe the usage and effectiveness of efanesoctocog alfa during the perioperative period.
Over 5 years period
Dose per injection required to maintain hemostasis during perioperative period for surgery (major, minor)
時間枠：Over a 5 year period

Data reported to describe the usage and effectiveness of efanesoctocog alfa during the perioperative period for On-demand Cohort
Over a 5 year period

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

Sanofi

捜査官

  • スタディディレクター：Clinical Sciences & Operations、Sanofi

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)

2023年6月30日

一次修了 (推定)

2028年6月6日

研究の完了 (推定)

2030年9月17日

試験登録日

最初に提出

2023年5月12日

QC基準を満たした最初の提出物

2023年6月19日

最初の投稿 (実際)

2023年6月22日

学習記録の更新

投稿された最後の更新 (実際)

2026年9月29日

QC基準を満たした最後の更新が送信されました

2026年9月24日

最終確認日

2026年9月1日

詳しくは

本研究に関する用語

追加の関連 MeSH 用語

その他の研究ID番号

  • OBS17523
  • U1111-1281-8840 (レジストリ識別子：ICTRP)

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

Qualified researchers may request access to patient level data and related study documents including the clinical study report, study protocol with any amendments, blank case report form, statistical analysis plan, and dataset specifications. Patient level data will be anonymized and study documents will be redacted to protect the privacy of trial participants. Further details on Sanofi's data sharing criteria, eligible studies, and process for requesting access can be found at: https://vivli.org

医薬品およびデバイス情報、研究文書

米国FDA規制医薬品の研究

はい

米国FDA規制機器製品の研究

いいえ

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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