혈우병 A 환자의 efanesoctocog Alfa 예방 (SYNOVIIIUS)
혈우병 환자의 활막 비대에 대한 Efanesoctocog Alfa 예방의 효과에 대한 전향 적 개입 연구
The objective of the study is to assess the improvement of synovial hypertrophy during the 12 months of efanesoctocog alfa prophylaxis once per week (QW) in joints with existing evidence of synovial hypertrophy in participants with hemophilia A.
The study duration for each participant is approximately 12 months.
연구 개요
상태
상태
정황
정황
개입 / 치료
개입 / 치료
연구 유형
연구 유형
등록 (추정된)
등록
단계
단계
- 4단계
연락처 및 위치
연구 연락처
연구 연락처
- 이름: Trial Transparency email recommended (Toll free for US & Canada)
- 전화번호: option 6 800-633-1610
- 이메일: Contact-US@sanofi.com
연구 장소
-
-
-
Kaohsiung City, 대만, 80756
- 모병
- Investigational Site Number: 1580003
-
Taichung, 대만, 407
- 모병
- Investigational Site Number: 1580001
-
Taipei, 대만, 114
- 모병
- Investigational Site Number: 1580002
-
-
-
-
California
-
Los Angeles, California, 미국, 90007
- 완전한
- Orthopedic Institute for Children- Site Number : 8400009
-
Sacramento, California, 미국, 95817-1399
- 모병
- University of California Davis Medical Center-Site Number: 8400005
-
-
Indiana
-
Indianapolis, Indiana, 미국, 46260
- 모병
- Indiana Hemophilia and Thrombosis Center- Site Number : 8400006
-
-
Iowa
-
Iowa City, Iowa, 미국, 52242
- 모병
- University of Iowa- Site Number : 8400004
-
-
Ohio
-
Columbus, Ohio, 미국, 43203
- 완전한
- The Ohio State University Wexner Medical Center - CarePoint East- Site Number : 8400001
-
-
Oregon
-
Portland, Oregon, 미국, 97239-3011
- 모병
- Oregon Health and Science University-Site Number: 8400002
-
-
Washington
-
Seattle, Washington, 미국, 98101-3932
- 모병
- Washington Center for Bleeding Disorders- Site Number : 8400008
-
-
-
-
-
Hiroshima, 일본, 734-8551
- 완전한
- Investigational Site Number : 3920002
-
Saitama, 일본, 330-8777
- 모병
- Investigational Site Number : 3920001
-
Tokyo, 일본, 160-0023
- 완전한
- Investigational Site Number : 3920004
-
-
Kanagawa
-
Kawasaki, Kanagawa, 일본, 216-8511
- 모병
- Investigational Site Number : 3920003
-
-
-
-
Alberta
-
Calgary, Alberta, 캐나다, T2N 1N4
- 모병
- Investigational Site Number : 1240002
-
-
Newfoundland and Labrador
-
St. John's, Newfoundland and Labrador, 캐나다, A1B 3V6
- 모병
- Investigational Site Number : 1240001
-
-
Ontario
-
Hamilton, Ontario, 캐나다, L8N 3Z5
- 모병
- Investigational Site Number : 1240003
-
Hamilton, Ontario, 캐나다, L8N 3Z5
- 완전한
- Investigational Site Number : 1240004
-
-
-
-
Western Australia
-
Murdoch, Western Australia, 호주, 6150
- 완전한
- Investigational Site Number : 0360001
-
-
참여기준
자격 기준
자격 기준
공부할 수 있는 나이
건강한 자원 봉사자를 받아들입니다
연구 대상 성별
설명
Inclusion Criteria:
-
Participant is diagnosed with moderate to severe hemophilia A (defined as less than or equal to (≤)5 percent (%) of endogenous FVIII clotting activity) at the time of consent/assent.
-
Participant is more than or equal to (≥)12 years old at the time of consent/assent.
-
Participant has existing synovial hypertrophy, defined as at least 1 eligible* joint by the HEAD-US score (Synovitis score: 1 or 2) at the time of consent/assent.
-
Participant has at least 1 eligible* joint without planned future major orthopedic procedures (example, arthroscopic synovectomy, radioisotopic or chemical synoviorthesis), or major orthopedic procedures in the past 3 months prior to the screening visit (Visit 1).
*An eligible joint is a joint with existing synovial hypertrophy, as defined by a HEAD-US synovitis score of 1 or 2, considering hypertrophic synovium as an indication of the presence of synovitis
-
Participant has received prophylactic treatment with hemophilia prophylaxis prescriptions in the last 12 months prior to the baseline visit (Visit 2).
-
Participant is capable of understanding the written Informed Consent Form (ICF)/assent form, provides a signed and witnessed written ICF/assent form, and agrees to comply with the protocol requirements.
-
If male, no contraceptive measures are required for this study.
-
If female, is not pregnant or breastfeeding, and one of the following conditions applies:
-
Is a women of nonchildbearing potential (WONCBP)
-
Is a WOCBP and agrees to use a highly effective contraceptive method, with a failure rate of less than (<)1 percent during the study treatment period (at least until the Week 52/ End of Treatment [EoT] visit).
-
A WOCBP must have a negative serum pregnancy test at the screening visit (Visit 1)
Exclusion Criteria:
- Participants are excluded from the study if any of the following criteria apply:
- Has other associated clotting disorders at the time of consent/assent.
- Is already under efanesoctocog alfa treatment.
- Has a current diagnosis of an factor VIII (FVIII) inhibitor, defined as inhibitor titer ≥0.60 BU/mL.
- Has ITI within the last 2 years prior to the baseline visit (Visit 2).
- Has been enrolled in a concurrent clinical interventional study or exposed to other investigational drug(s) within 3 months prior to screening for this study.
- Is currently in an institution because of regulatory or legal order (that is, is a prisoner or a patient who is legally institutionalized).
- Is not suitable for participation, whatever the reason, as judged by the investigator, including medical or clinical conditions, or patients potentially at risk of noncompliance to study procedures.
- Is an employee or family member of the investigator or site personnel.
- Is involved in a specific situation during study implementation or the course of the study that may raise ethics considerations.
- Has hypersensitivity to efanesoctocog alfa or its components or any of its excipients that, in the opinion of the investigator, contraindicates participation in the study.
The above information is not intended to contain all considerations relevant to a patient's potential participation in a clinical trial.
공부 계획
연구는 어떻게 설계됩니까?
디자인 세부사항
- 주 목적: 치료
- 할당: 무작위화되지 않음
- 중재 모델: 단일 그룹 할당
- 마스킹: 없음(오픈 라벨)
팔의 수
무기와 개입
참가자 그룹 / 팔참가자 그룹 / 팔 |
개입 / 치료개입 / 치료 |
|---|---|
|
실험적: Efanesoctocog Alfa
Participants will receive Efanesoctocog Alfa at 50 international unit per kilogram (IU/kg) intravenous (IV) QW |
Pharmaceutical form:Lyophilized powder in a sterile vial that requires reconstitution with sterile water for injection (diluent)-Route of administration:Intravenous
다른 이름들:
|
연구는 무엇을 측정합니까?
주요 결과 측정
주요 결과 측정
결과 측정 |
측정값 설명 |
기간 |
|---|---|---|
|
Proportion of joints with improvement in the Hemophilia Early Arthropathy Detection with Ultrasound (HEAD-US) synovitis domain score
기간: From Baseline up to Week 52
|
HEAD-US scoring system is a simplified ultrasound scanning procedure to image the elbow, knee, and ankle (index joints) and a scoring system based on an additive scale to define the joint status from disease activity and disease damage parameters to evaluate disease progression and monitor the results of treatment. |
From Baseline up to Week 52
|
2차 결과 측정
2차 결과 측정
결과 측정 |
측정값 설명 |
기간 |
|---|---|---|
|
Change from baseline in the distribution of joint HEAD-US synovitis score
기간: From Baseline up to Week 52
|
HEAD-US scoring system is a simplified ultrasound scanning procedure to image the elbow, knee, and ankle (index joints) and a scoring system based on an additive scale to define the joint status from disease activity and disease damage parameters to evaluate disease progression and monitor the results of treatment. |
From Baseline up to Week 52
|
|
Number of joints with the HEAD-US synovitis domain score that has remained unchanged or worsened
기간: From Baseline up to Week 52
|
HEAD-US scoring system is a simplified ultrasound scanning procedure to image the elbow, knee, and ankle (index joints) and a scoring system based on an additive scale to define the joint status from disease activity and disease damage parameters to evaluate disease progression and monitor the results of treatment. |
From Baseline up to Week 52
|
|
Percentage of joints with the HEAD-US synovitis domain score that has remained unchanged or worsened
기간: From Baseline up to Week 52
|
HEAD-US scoring system is a simplified ultrasound scanning procedure to image the elbow, knee, and ankle (index joints) and a scoring system based on an additive scale to define the joint status from disease activity and disease damage parameters to evaluate disease progression and monitor the results of treatment. |
From Baseline up to Week 52
|
|
Number of index joint type with the HEAD-US synovitis domain score that has improved, remained unchanged, or worsened
기간: From Baseline up to Week 52
|
HEAD-US scoring system is a simplified ultrasound scanning procedure to image the elbow, knee, and ankle (index joints) and a scoring system based on an additive scale to define the joint status from disease activity and disease damage parameters to evaluate disease progression and monitor the results of treatment. |
From Baseline up to Week 52
|
|
Percentage of index joint type with the HEAD-US synovitis domain score that has improved, remained unchanged, or worsened
기간: From Baseline up to Week 52
|
HEAD-US scoring system is a simplified ultrasound scanning procedure to image the elbow, knee, and ankle (index joints) and a scoring system based on an additive scale to define the joint status from disease activity and disease damage parameters to evaluate disease progression and monitor the results of treatment. |
From Baseline up to Week 52
|
|
Number of participants with at least 1 joint with the HEAD-US synovitis domain score that has improved, remained unchanged, or worsened
기간: From Baseline up to Week 52
|
HEAD-US scoring system is a simplified ultrasound scanning procedure to image the elbow, knee, and ankle (index joints) and a scoring system based on an additive scale to define the joint status from disease activity and disease damage parameters to evaluate disease progression and monitor the results of treatment. |
From Baseline up to Week 52
|
|
Percentage of participants with at least 1 joint with the HEAD-US synovitis domain score that has improved, remained unchanged, or worsened
기간: From Baseline up to Week 52
|
HEAD-US scoring system is a simplified ultrasound scanning procedure to image the elbow, knee, and ankle (index joints) and a scoring system based on an additive scale to define the joint status from disease activity and disease damage parameters to evaluate disease progression and monitor the results of treatment. |
From Baseline up to Week 52
|
|
Change from baseline in average HEAD-US synovitis domain score across all index joints
기간: From Baseline up to Week 52
|
HEAD-US scoring system is a simplified ultrasound scanning procedure to image the elbow, knee, and ankle (index joints) and a scoring system based on an additive scale to define the joint status from disease activity and disease damage parameters to evaluate disease progression and monitor the results of treatment. |
From Baseline up to Week 52
|
|
Change from baseline in total/ domain scores of the HEAD-US
기간: From Baseline up to Week 26
|
HEAD-US scoring system is a simplified ultrasound scanning procedure to image the elbow, knee, and ankle (index joints) and a scoring system based on an additive scale to define the joint status from disease activity and disease damage parameters to evaluate disease progression and monitor the results of treatment. |
From Baseline up to Week 26
|
|
Change from baseline in total/domain scores of the HJHS
기간: From Baseline up to Week 26
|
HJHS comprises an assessment of specific features, or items, of the 6 index joints and an assessment of global gait. For each of the 6 joints, the following items are scored: swelling (0 to 3), duration of swelling (0 to 1), muscle atrophy (0 to 2), crepitus on motion (0 to 2), flexion loss (0 to 3), extension loss (0 to 3), joint pain (0 to 2), and strength (0 to 4). The maximum score for an individual index joint is 20. Gait is scored from 0 to 4. The maximum HJHS total score is 124, with a higher score indicating worse joint health. |
From Baseline up to Week 26
|
|
Change from baseline in total/domain scores of the HJHS
기간: From Baseline up to Week 52
|
HJHS comprises an assessment of specific features, or items, of the 6 index joints and an assessment of global gait. For each of the 6 joints, the following items are scored: swelling (0 to 3), duration of swelling (0 to 1), muscle atrophy (0 to 2), crepitus on motion (0 to 2), flexion loss (0 to 3), extension loss (0 to 3), joint pain (0 to 2), and strength (0 to 4). The maximum score for an individual index joint is 20. Gait is scored from 0 to 4. The maximum HJHS total score is 124, with a higher score indicating worse joint health. |
From Baseline up to Week 52
|
|
Change from baseline in total/ domain scores of magnetic resonance imaging (MRI)
기간: From Baseline up to Week 52
|
MRI will be used to detect changes in synovial hypertrophy in index joints of participants. |
From Baseline up to Week 52
|
|
Change from baseline in patient-reported outcome (PRO)
기간: From Baseline up to Week 52
|
Change from baseline in PRO will be assessed by the EQ-5D 5-level (EQ-5D-5L), Patient-Reported Outcomes Measurement Information System - Short Form (PROMIS-SF) version 2.0 Pain Intensity 3a, PROMIS-SF version 1.1 Pain Interference 6a, and PROMIS-SF version 2.0 Physical Function 6b. |
From Baseline up to Week 52
|
|
Patient reported treatment preference and satisfaction
기간: At Week 52
|
Patient reported treatment preference and satisfaction will be assessed through surveys and exit interviews. |
At Week 52
|
|
Change from baseline in annualized bleeding rate (ABR) (spontaneous, traumatic)
기간: At Week 52
|
The ABR will be defined as (number of bleeding episodes occurring during the treatment period)/(duration of the treatment period [days]) × 365.25. |
At Week 52
|
|
Change from baseline in annualized joint bleeding rate (AjBR) (spontaneous, traumatic)
기간: At Week 52
|
The AjBR will be defined as (number of joint bleeding episodes occurring during the treatment period)/(duration of the treatment period [days]) × 365.25. |
At Week 52
|
|
Change from baseline in target joint resolution, recurrence, or development
기간: At Week 52
|
Change from baseline in target joint resolution, recurrence, or development will be assessed. |
At Week 52
|
|
The occurrence of Adverse Events (AEs), all AEs leading to treatment discontinuation, Serious Adverse Events (SAEs), and Adverse Event of Special Interest (AESIs)
기간: Up to Week 52
|
The safety and tolerability of efanesoctocog alfa evaluated in all participants |
Up to Week 52
|
공동 작업자 및 조사자
간행물 및 유용한 링크
연구 기록 날짜
연구 주요 날짜
연구 시작 (실제)
연구 시작
기본 완료 (추정된)
기본 완료
연구 완료 (추정된)
연구 완료
연구 등록 날짜
최초 제출
최초 제출
QC 기준을 충족하는 최초 제출
QC 기준을 충족하는 최초 제출
처음 게시됨 (실제)
처음 게시됨
연구 기록 업데이트
마지막 업데이트 게시됨 (실제)
마지막 업데이트 게시됨
QC 기준을 충족하는 마지막 업데이트 제출
QC 기준을 충족하는 마지막 업데이트 제출
마지막으로 확인됨
마지막으로 확인됨
추가 정보
이 연구와 관련된 용어
추가 관련 MeSH 약관
기타 연구 ID 번호
기타 연구 ID 번호
- LPS18168
- U1111-1311-1701 (레지스트리 식별자: ICTRP)
개별 참가자 데이터(IPD) 계획
개별 참가자 데이터(IPD)를 공유할 계획입니까?
IPD 계획 설명
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
약물 및 장치 정보, 연구 문서
미국 FDA 규제 의약품 연구
미국 FDA 규제 기기 제품 연구
이 정보는 변경 없이 clinicaltrials.gov 웹사이트에서 직접 가져온 것입니다. 귀하의 연구 세부 정보를 변경, 제거 또는 업데이트하도록 요청하는 경우 register@clinicaltrials.gov. 문의하십시오. 변경 사항이 clinicaltrials.gov에 구현되는 즉시 저희 웹사이트에도 자동으로 업데이트됩니다. .