A Phase 3 Evaluation of Sutacimig in Glanzmann Thrombasthenia (SOLARA)
SOLARA: A Phase 3 Study of the Efficacy and Safety of Sutacimig in Participants With Glanzmann Thrombasthenia
Glanzmann thrombasthenia (GT) is a serious inherited bleeding disorder. In GT, platelets, the blood cells that normally stick together at the site of an injury to stop bleeding, are unable to properly do so. As a result, bleeding does not stop as it should. Bleeding in GT often starts without warning, can happen frequently, and can be severe. It most commonly affects the nose, mouth and gums, gastrointestinal tract, and skin, and many women and girls with GT experience heavy menstrual bleeding. Over time, bleeding can lead to iron deficiency and anemia. Some bleeding episodes require emergency care, hospitalization, transfusion, or surgery, and some can be life-threatening.
Sutacimig is an investigational medicine given as an injection under the skin (subcutaneous injection). It is designed to help platelets work at the site of bleeding despite the underlying defect in GT. It is intended to be given regularly to help prevent bleeding (prophylaxis), rather than only to treat bleeding after it starts.
The purpose of this study is to find out whether sutacimig, given as prophylaxis, reduces the number of treated bleeding episodes in people with GT compared with no prophylaxis, and to evaluate its safety and tolerability.
연구 개요
연구 유형
등록 (추정된)
단계
- 3단계
연락처 및 위치
연구 연락처
- 이름: Clinical Trials
- 전화번호: 1 833 328 7111
- 이메일: clinicaltrials@hemab.com
연구 장소
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California
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La Jolla, California, 미국, 92037
- 모병
- University of California San Diego (UC San Diego) Health - Jacobs Medical Center
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Los Angeles, California, 미국, 90027
- 모병
- Children's Hospital Los Angeles
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Sacramento, California, 미국, 95817
- 모병
- University of California Davis (UC Davis) Medical Center
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San Francisco, California, 미국, 94143
- 모병
- University of California San Francisco (UCSF) Medical Center
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Indiana
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Indianapolis, Indiana, 미국, 46260
- 모병
- Innovative Hematology - Indiana Hemophilia & Thrombosis Center
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Minnesota
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Minneapolis, Minnesota, 미국, 55455
- 모병
- M Health Fairview - University of Minnesota Medical Center
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New York
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New York, New York, 미국, 10065
- 모병
- New York-Presbyterian - Weill Cornell Medical Center
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North Carolina
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Durham, North Carolina, 미국, 27709
- 모병
- Science 37
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Texas
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Houston, Texas, 미국, 77030
- 모병
- Gulf States Hemophilia and Thrombophilia Center - Texas Medical Center
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참여기준
자격 기준
공부할 수 있는 나이
건강한 자원 봉사자를 받아들입니다
연구 대상 성별
설명
Inclusion Criteria:
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Has the ability to provide informed consent to participate in the clinical study before completing any study-related procedures, and has an understanding, ability, and willingness to comply with clinical study procedures and restrictions.
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Adults 18 to 70 years.
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Adolescents 12 to <18 years, dependent on geographical location.
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Has clinical bleeding phenotype consistent with Glanzmann thrombasthenia (GT) and documentation of either diagnostic laboratory criteria (a) or (b) below:
- Abnormal platelet aggregometry AND flow cytometry
- A confirmed genetic mutation in ITGA2B or ITGB3.
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Participants have experienced at least four treated bleeding events related to GT within the 12 months prior to Screening.
Exclusion Criteria:
- Prior exposure to sutacimig.
- History of clinically significant hypersensitivity associated with monoclonal antibody therapies.
- Congenital or acquired bleeding disorders other than GT.
- Medical condition (other than GT) that constitutes an increased risk of having uncontrolled bleeding events during the study or other conditions per Investigator discretion.
- History of venous or arterial thromboembolic (TE) event or TE disease except for catheter-associated vein thrombosis.
- Co-existing thrombophilia disorder.
공부 계획
연구는 어떻게 설계됩니까?
디자인 세부사항
- 주 목적: 치료
- 할당: 해당 없음
- 중재 모델: 단일 그룹 할당
- 마스킹: 없음(오픈 라벨)
무기와 개입
참가자 그룹 / 팔 |
개입 / 치료 |
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실험적: Sutacimig
Participants will receive sutacimig 0.2 milligrams per kilogram (mg/kg) by subcutaneous injection once weekly for 48 weeks. Upon completion of the 48-week treatment period, participants will be offered the opportunity to transition to a separate open-label extension (OLE) study for continued assessment of the prophylactic treatment with sutacimig. |
Sutacimig will be administered by subcutaneous injection. |
연구는 무엇을 측정합니까?
주요 결과 측정
결과 측정 |
기간 |
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Annualized Treated Bleed Rate (ATBR)
기간: From the first dose through Week 48
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From the first dose through Week 48
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2차 결과 측정
결과 측정 |
기간 |
|---|---|
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High-Intensity Annualized Treated Bleed Rate (Hi-ATBR)
기간: From the first dose through Week 48
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From the first dose through Week 48
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Annualized Treated Bleed Days (ATBD)
기간: From the first dose through Week 48
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From the first dose through Week 48
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Annualized Treated Bleed Rate (ATBR) by Bleeding Event Type
기간: From the first dose through Week 48
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From the first dose through Week 48
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Annualized Treated Bleed Rate (ATBR) by Bleeding Event Cause
기간: From the first dose through Week 48
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From the first dose through Week 48
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Percentage of Participants With Reductions in Annualized Treated Bleed Rate (ATBR)
기간: From the first dose through Week 48
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From the first dose through Week 48
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Percentage of Participants With Reductions in High-Intensity Annualized Treated Bleed Rate (Hi-ATBR)
기간: From the first dose through Week 48
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From the first dose through Week 48
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Number of Participants With Treatment-Emergent Adverse Events (TEAEs)
기간: From the first dose through Week 56
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From the first dose through Week 56
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Number of Participants With Physical Examination Abnormalities
기간: From the first dose through Week 56
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From the first dose through Week 56
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Number of Participants With Vital Sign Abnormalities
기간: From the first dose through Week 56
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From the first dose through Week 56
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Number of Participants With Clinical Laboratory Test Abnormalities
기간: From the first dose through Week 56
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From the first dose through Week 56
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Number of Participants With Anti-Drug Antibody (ADA) Development
기간: From the first dose through Week 56
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From the first dose through Week 56
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Number of Participants With Changes in Electrocardiogram (ECG) Parameters
기간: From the first dose through Week 56
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From the first dose through Week 56
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Plasma Concentration for Sutacimig
기간: Baseline through Week 56
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Baseline through Week 56
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공동 작업자 및 조사자
스폰서
수사관
- 연구 책임자: Medical Director, Hemab
연구 기록 날짜
연구 주요 날짜
연구 시작 (실제)
기본 완료 (추정된)
연구 완료 (추정된)
연구 등록 날짜
최초 제출
QC 기준을 충족하는 최초 제출
처음 게시됨 (실제)
연구 기록 업데이트
마지막 업데이트 게시됨 (실제)
QC 기준을 충족하는 마지막 업데이트 제출
마지막으로 확인됨
추가 정보
이 연구와 관련된 용어
키워드
추가 관련 MeSH 약관
기타 연구 ID 번호
- HMB-001-103
- U1111-1342-3293 (기타 식별자: Universal Trial Number)
개별 참가자 데이터(IPD) 계획
개별 참가자 데이터(IPD)를 공유할 계획입니까?
약물 및 장치 정보, 연구 문서
미국 FDA 규제 의약품 연구
미국 FDA 규제 기기 제품 연구
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