베커 근이영양증 환자의 EDG-5506 임상 2상 연구(CANYON)
베커 근이영양증이 있는 성인 및 청소년의 안전성, 바이오마커, 약동학 및 기능 측정에 대한 EDG-5506의 효과를 평가하기 위한 2상 무작위, 이중 맹검, 위약 대조 연구
A study of sevasemten (EDG-5506) in Becker muscular dystrophy (known as CANYON) and pivotal cohort (known as GRAND CANYON). The EDG-5506-201 CANYON study was expanded to include an additional 175 adult participants in a cohort called GRAND CANYON, that is a multicenter, randomized, double-blind, placebo-controlled study to evaluate the safety and efficacy of sevasemten in adults with Becker.
연구 개요
상태
상태
정황
정황
개입 / 치료
개입 / 치료
상세 설명
The EDG-5506-201 protocol was amended to include an additional cohort thus consists of two parts.
Part 1: CANYON is a double-blind, randomized, placebo-controlled design to investigate the effect of sevasemten on the safety, pharmacokinetics, biomarkers, and functional measures. Approximately 32 adults and 18 adolescents with Becker muscular dystrophy are planned to enroll in this study. This study will have up to a 4-week Screening period, a 12-month Treatment period, followed by a 4-week follow-up period.
Approximately 32 adult participants will randomize to Cohort 1 or Cohort 2 in a 1:1 ratio then each cohort will further randomize to sevasemten or placebo in a 3:1 ratio.
Approximately 9 adolescent participants will enroll in Cohort 4 and randomize in a 2:1 ratio to sevasemten or placebo. Cohort 5 will randomize an additional 9 participants in a 2:1 ratio to either sevasemten or placebo after Cohort 4.
Part 2: GRAND CANYON or Cohort 6 is a double-blind, randomized, placebo-controlled design to investigate the safety and efficacy of sevasemten in adults with Becker muscular dystrophy after 18 months of treatment. Approximately 175 adults with Becker muscular dystrophy are planned to enroll in this study. This study will have up to a 4-week Screening period, an 18-month Treatment period, followed by a 4-week follow-up period.
Approximately 175 adult participants will be randomized in Cohort 6 in a 2:1 ratio either to sevasemten or placebo.
Enrollment for CANYON and GRAND CANYON has been completed.
연구 유형
연구 유형
등록 (실제)
등록
단계
단계
- 2 단계
연락처 및 위치
연구 장소
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Leiden, 네덜란드, 2333 ZA
- Leids Universitair Medisch Centrum
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Auckland, 뉴질랜드, 1010
- Optimal Clinical Trials
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Copenhagen, 덴마크
- Rigshospitalet
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Munich, 독일, 80336
- Klinikum der Ludwig-Maximilians-Universitaet Muenchen
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Arkansas
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Little Rock, Arkansas, 미국, 72202
- Arkansas Children's Hospital
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California
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La Jolla, California, 미국, 92037
- UC San Diego
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Los Angeles, California, 미국, 90095
- UCLA Medical Center
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Orange, California, 미국, 92868
- UC Irvine Medical Center
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Palo Alto, California, 미국, 94304
- Stanford Neuroscience Health Center
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Sacramento, California, 미국, 95817
- UC Davis Medical Center
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Colorado
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Aurora, Colorado, 미국, 80045
- UC Denver
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Florida
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Gainesville, Florida, 미국, 32611
- University of Florida
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Georgia
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Atlanta, Georgia, 미국, 30329
- Rare Disease Research
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Illinois
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Chicago, Illinois, 미국, 60611
- Northwestern University
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Indiana
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Indianapolis, Indiana, 미국, 46202
- Indiana University School of Medicine
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Iowa
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Iowa City, Iowa, 미국, 52242
- University of Iowa
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Kansas
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Kansas City, Kansas, 미국, 66160
- University of Kansas Medical Center
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Maryland
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Baltimore, Maryland, 미국, 21205
- Kennedy Krieger Institute
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Massachusetts
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Worcester, Massachusetts, 미국, 01605
- University of Massachusetts Memorial Medical Center
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Minnesota
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Minneapolis, Minnesota, 미국, 55455
- University of Minnesota
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Missouri
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St Louis, Missouri, 미국, 63110
- Washington University School of Medicine
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New York
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Rochester, New York, 미국, 14642
- University of Rochester Medical Center
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North Carolina
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Hillsborough, North Carolina, 미국, 27278
- Rare Disease Research, LLC NC
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Ohio
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Cincinnati, Ohio, 미국, 45219
- University of Cincinnati Gardner Neuroscience Institute
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Columbus, Ohio, 미국, 43205
- Nationwide Children's Hospital
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Pennsylvania
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Philadelphia, Pennsylvania, 미국, 19104
- University of Pennsylvania
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Pittsburgh, Pennsylvania, 미국, 15224
- University of Pittsburgh
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Texas
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Austin, Texas, 미국, 78759
- National Neuromuscular Research Institute
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Denton, Texas, 미국, 76208
- Neurology Rare Disease Center
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Virginia
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Richmond, Virginia, 미국, 23298
- Virginia Commonwealth University Health
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Leuven, 벨기에
- Universitaire Ziekenhuizen Leuven
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Liège, 벨기에, 4000
- Centre Hospitalier Régional de la Citadelle
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Belgium
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Ghent, Belgium, 벨기에, 9000
- University Hospital Gent
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Barcelona, 스페인, 08035
- Hospital Universitario Vall d'Hebron
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Barcelona, 스페인, 08907
- Hospital Universitari de Bellvitge
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Donostia / San Sebastian, 스페인, 20014
- Hospital Universitario Donostia
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Valencia, 스페인, 46026
- Hospital Universitari i Politecnic La Fe
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London, 영국, SW17 0QT
- St. George's University Hospitals NHS Foundation Trust
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London, 영국, NW1 2PG
- University College London Hospital
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Newcastle, 영국, NE7 7DN
- Newcastle Freeman Hospital
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Salford, 영국, M68HD
- Salford Royal Hospital
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Jerusalem, 이스라엘, 91240
- Hadassah University Hospital
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Petah Tikva, 이스라엘, 49202
- Schneider Children's Hospital of Israel
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Milan, 이탈리아, 20122
- Fondazione IRCCS Ca'Granda Ospedale Maggiore Policlinico di Milano
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Padova, 이탈리아, 35128
- Azienda Ospedale - Università Padova
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Rome, 이탈리아, 00168
- Fondazione Policlinico Universitario A. Gemelli IRCCS - Universita Cattolica del Sacro Cuore
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Marseille, 프랑스, 13005
- Centre de Reference des Maladies Neuromusculaires et de la SLA - AP-HM Hopital de La Timone
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Nantes, 프랑스, 44093
- CHU de Nantes
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Nice, 프랑스, 06001
- CHU de Nice - Hopital Pasteur 2 - Centre de reference des Maladies Neuromusculaires
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Paris, 프랑스, 75013
- AP-HP Hopital Pitie-Salpetriere
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Fitzroy, 호주, VIC, 3065
- St Vincent's Hospital Melbourne
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참여기준
자격 기준
자격 기준
공부할 수 있는 나이
건강한 자원 봉사자를 받아들입니다
연구 대상 성별
설명
GRAND CANYON eligibility is listed below.
Key Inclusion Criteria:
- Adults (aged 18 to 50 years, inclusive) with a documented dystrophin mutation and phenotype consistent with Becker muscular dystrophy, and history of being ambulatory beyond 16 years of age without steroids; history of being ambulatory beyond 18 years of age with steroids.
- Able to complete the 100-meter timed test in < 200 seconds with or without use of mobility aid devices.
- Able to perform the North Star Ambulatory Assessment scale and achieve a score of 5 to 32, inclusive.
Key Exclusion Criteria:
- Medical history or clinically significant physical examination/laboratory result that, in the opinion of the investigator, would render the participant unsuitable for the study. This includes contraindications to magnetic resonance imaging such as non-compatible implanted medical devices or severe claustrophobia.
- Cardiac echocardiogram ejection fraction < 40%
- Forced vital capacity predicted <60% or using daytime ventilatory support
- Receipt of oral corticosteroids for the treatment of BMD in the previous 6 months.
- Receipt of an investigational drug within 30 days or 5 half-lives (whichever is longer) of the screening visit in the present study.
공부 계획
연구는 어떻게 설계됩니까?
디자인 세부사항
- 주 목적: 치료
- 할당: 무작위
- 중재 모델: 순차적 할당
- 마스킹: 네 배로
팔의 수
무기와 개입
참가자 그룹 / 팔참가자 그룹 / 팔 |
개입 / 치료개입 / 치료 |
|---|---|
|
실험적: Adult Cohort 1
Drug: Sevasemten Drug: Placebo |
Sevasemten is administered orally once per day Placebo is administered orally once per day |
|
실험적: Adult Cohort 2
Drug: Sevasemten Drug: Placebo |
Sevasemten is administered orally once per day Placebo is administered orally once per day |
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실험적: Adult Cohort 6
Drug: Sevasemten Drug: Placebo |
Sevasemten is administered orally once per day Placebo is administered orally once per day |
|
실험적: Adolescent Cohort 4
Drug: Sevasemten Drug: Placebo |
Placebo is administered orally once per day Sevasemten is administered orally once per day |
|
실험적: Adolescent Cohort 5
Drug: Sevasemten Drug: Placebo |
Placebo is administered orally once per day Sevasemten is administered orally once per day |
연구는 무엇을 측정합니까?
주요 결과 측정
주요 결과 측정
결과 측정 |
측정값 설명 |
기간 |
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Number of treatment emergent adverse events in those treated with sevasemten or placebo
기간: 12 months (CANYON Cohorts 1, 2, 4, 5), 18 months (GRAND CANYON Cohort 6)
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All participants |
12 months (CANYON Cohorts 1, 2, 4, 5), 18 months (GRAND CANYON Cohort 6)
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Severity of treatment emergent adverse events in those treated with sevasemten or placebo
기간: 12 months (CANYON Cohorts 1, 2, 4, 5), 18 months (GRAND CANYON Cohort 6)
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All participants |
12 months (CANYON Cohorts 1, 2, 4, 5), 18 months (GRAND CANYON Cohort 6)
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Change from Baseline in serum Creatine Kinase
기간: Averaged across Months 6, 9, 12 Months (CANYON Cohorts 1, 2)
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Adult participants |
Averaged across Months 6, 9, 12 Months (CANYON Cohorts 1, 2)
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Change from Baseline in total North Star Ambulatory Assessment score
기간: 18 months (GRAND CANYON Cohort 6)
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Adult participants |
18 months (GRAND CANYON Cohort 6)
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2차 결과 측정
2차 결과 측정
결과 측정 |
측정값 설명 |
기간 |
|---|---|---|
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Change from Baseline in total North Star Ambulatory Assessment score
기간: 12 Months (CANYON Cohorts 1, 2)
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Adult participants |
12 Months (CANYON Cohorts 1, 2)
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Pharmacokinetics as measured by steady state plasma concentration
기간: 12 Months (CANYON Cohorts 1, 2, 4, 5), 18 months (GRAND CANYON Cohort 6)
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All participants |
12 Months (CANYON Cohorts 1, 2, 4, 5), 18 months (GRAND CANYON Cohort 6)
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|
Change from Baseline in growth as assessed by height centile on World Health Organization growth charts
기간: 12 months (CANYON Cohorts 4, 5)
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Adolescent participants |
12 months (CANYON Cohorts 4, 5)
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Incidence of laboratory test-related treatment emergent adverse events
기간: 18 months (GRAND CANYON Cohort 6)
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Adult participants |
18 months (GRAND CANYON Cohort 6)
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Change from Baseline in serum Creatine Kinase
기간: 18 months (GRAND CANYON Cohort 6)
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Adult participants |
18 months (GRAND CANYON Cohort 6)
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Change from Baseline in plasma fast skeletal muscle Troponin I
기간: Averaged across Months 6 and 12 (CANYON Cohorts 1, 2); 18 months (GRAND CANYON Cohort 6)
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Adult participants |
Averaged across Months 6 and 12 (CANYON Cohorts 1, 2); 18 months (GRAND CANYON Cohort 6)
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Change from Baseline in serum myoglobin
기간: Averaged across Months 6, 9, and 12 (CANYON Cohorts 1,2); 18 months (GRAND CANYON Cohort 6)
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Adult participants |
Averaged across Months 6, 9, and 12 (CANYON Cohorts 1,2); 18 months (GRAND CANYON Cohort 6)
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Change from Baseline in total NSAD score (CANYON Cohorts 1,2)
기간: 12 Months (CANYON Cohorts 1, 2)
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Adult participants |
12 Months (CANYON Cohorts 1, 2)
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Change from Baseline in 4-stair climb test
기간: 18 months (GRAND CANYON Cohort 6)
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Adult participants |
18 months (GRAND CANYON Cohort 6)
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Change from Baseline in the 10-meter walk/run test
기간: 12 Months (CANYON Cohorts 1, 2), 18 Months (GRAND CANYON Cohort 6)
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Adult participants |
12 Months (CANYON Cohorts 1, 2), 18 Months (GRAND CANYON Cohort 6)
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Change from Baseline in 100-meter timed test
기간: 12 Months (CANYON Cohorts 1, 2), 18 Months (GRAND CANYON Cohort 6)
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Adult participants |
12 Months (CANYON Cohorts 1, 2), 18 Months (GRAND CANYON Cohort 6)
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Change from Baseline in stride velocity (95th percentile)
기간: 18 Months (GRAND CANYON Cohort 6)
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Adult participants |
18 Months (GRAND CANYON Cohort 6)
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Month 18 change from Baseline in fat fraction of upper leg muscles as assessed by Magnetic Resonance Imaging
기간: 18 months (GRAND CANYON Cohort 6)
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Adult participants |
18 months (GRAND CANYON Cohort 6)
|
공동 작업자 및 조사자
협력자
협력자
수사관
수사관
- 연구 의자: Joanne Donovan, MD, PhD, Edgewise Therapeutics, Inc.
- 연구 의자: Roxana D. Dreghici, Edgewise Therapeutics, Inc.
간행물 및 유용한 링크
유용한 링크
연구 기록 날짜
연구 주요 날짜
연구 시작 (실제)
연구 시작
기본 완료 (실제)
기본 완료
연구 완료 (실제)
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
키워드
추가 관련 MeSH 약관
기타 연구 ID 번호
기타 연구 ID 번호
- EDG-5506-201
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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