Fase 2-studie van EDG-5506 bij Becker spierdystrofie (CANYON)
Een gerandomiseerde, dubbelblinde, placebogecontroleerde fase 2-studie om het effect van EDG-5506 op veiligheid, biomarkers, farmacokinetiek en functionele metingen bij volwassenen en adolescenten met Becker-spierdystrofie te evalueren
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.
Studie Overzicht
Toestand
Conditie
Interventie / Behandeling
Gedetailleerde beschrijving
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.
Studietype
Inschrijving (Werkelijk)
Fase
- Fase 2
Contacten en locaties
Studie Locaties
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Fitzroy, Australië, VIC, 3065
- St Vincent's Hospital Melbourne
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Leuven, België
- Universitaire Ziekenhuizen Leuven
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Liège, België, 4000
- Centre Hospitalier Régional de la Citadelle
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Belgium
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Ghent, Belgium, België, 9000
- University Hospital Gent
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Copenhagen, Denemarken
- Rigshospitalet
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Munich, Duitsland, 80336
- Klinikum der Ludwig-Maximilians-Universitaet Muenchen
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Marseille, Frankrijk, 13005
- Centre de Reference des Maladies Neuromusculaires et de la SLA - AP-HM Hopital de La Timone
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Nantes, Frankrijk, 44093
- CHU de Nantes
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Nice, Frankrijk, 06001
- CHU de Nice - Hopital Pasteur 2 - Centre de reference des Maladies Neuromusculaires
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Paris, Frankrijk, 75013
- AP-HP Hopital Pitie-Salpetriere
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Jerusalem, Israël, 91240
- Hadassah University Hospital
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Petah Tikva, Israël, 49202
- Schneider Children's Hospital of Israel
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Milan, Italië, 20122
- Fondazione IRCCS Ca'Granda Ospedale Maggiore Policlinico di Milano
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Padova, Italië, 35128
- Azienda Ospedale - Università Padova
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Rome, Italië, 00168
- Fondazione Policlinico Universitario A. Gemelli IRCCS - Universita Cattolica del Sacro Cuore
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Leiden, Nederland, 2333 ZA
- Leids Universitair Medisch Centrum
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Auckland, Nieuw-Zeeland, 1010
- Optimal Clinical Trials
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Barcelona, Spanje, 08035
- Hospital Universitario Vall d'Hebron
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Barcelona, Spanje, 08907
- Hospital Universitari de Bellvitge
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Donostia / San Sebastian, Spanje, 20014
- Hospital Universitario Donostia
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Valencia, Spanje, 46026
- Hospital Universitari i Politecnic La Fe
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London, Verenigd Koninkrijk, SW17 0QT
- St. George's University Hospitals NHS Foundation Trust
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London, Verenigd Koninkrijk, NW1 2PG
- University College London Hospital
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Newcastle, Verenigd Koninkrijk, NE7 7DN
- Newcastle Freeman Hospital
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Salford, Verenigd Koninkrijk, M68HD
- Salford Royal Hospital
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Arkansas
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Little Rock, Arkansas, Verenigde Staten, 72202
- Arkansas Children's Hospital
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California
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La Jolla, California, Verenigde Staten, 92037
- UC San Diego
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Los Angeles, California, Verenigde Staten, 90095
- UCLA Medical Center
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Orange, California, Verenigde Staten, 92868
- UC Irvine Medical Center
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Palo Alto, California, Verenigde Staten, 94304
- Stanford Neuroscience Health Center
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Sacramento, California, Verenigde Staten, 95817
- UC Davis Medical Center
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Colorado
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Aurora, Colorado, Verenigde Staten, 80045
- UC Denver
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Florida
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Gainesville, Florida, Verenigde Staten, 32611
- University of Florida
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Georgia
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Atlanta, Georgia, Verenigde Staten, 30329
- Rare Disease Research
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Illinois
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Chicago, Illinois, Verenigde Staten, 60611
- Northwestern University
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Indiana
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Indianapolis, Indiana, Verenigde Staten, 46202
- Indiana University School of Medicine
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Iowa
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Iowa City, Iowa, Verenigde Staten, 52242
- University of Iowa
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Kansas
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Kansas City, Kansas, Verenigde Staten, 66160
- University of Kansas Medical Center
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Maryland
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Baltimore, Maryland, Verenigde Staten, 21205
- Kennedy Krieger Institute
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Massachusetts
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Worcester, Massachusetts, Verenigde Staten, 01605
- University of Massachusetts Memorial Medical Center
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Minnesota
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Minneapolis, Minnesota, Verenigde Staten, 55455
- University of Minnesota
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Missouri
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St Louis, Missouri, Verenigde Staten, 63110
- Washington University School of Medicine
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New York
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Rochester, New York, Verenigde Staten, 14642
- University of Rochester Medical Center
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North Carolina
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Hillsborough, North Carolina, Verenigde Staten, 27278
- Rare Disease Research, LLC NC
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Ohio
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Cincinnati, Ohio, Verenigde Staten, 45219
- University of Cincinnati Gardner Neuroscience Institute
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Columbus, Ohio, Verenigde Staten, 43205
- Nationwide Children's Hospital
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Pennsylvania
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Philadelphia, Pennsylvania, Verenigde Staten, 19104
- University of Pennsylvania
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Pittsburgh, Pennsylvania, Verenigde Staten, 15224
- University of Pittsburgh
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Texas
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Austin, Texas, Verenigde Staten, 78759
- National Neuromuscular Research Institute
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Denton, Texas, Verenigde Staten, 76208
- Neurology Rare Disease Center
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Virginia
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Richmond, Virginia, Verenigde Staten, 23298
- Virginia Commonwealth University Health
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Deelname Criteria
Geschiktheidscriteria
Leeftijden die in aanmerking komen voor studie
Accepteert gezonde vrijwilligers
Geslachten die in aanmerking komen voor studie
Beschrijving
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.
Studie plan
Hoe is de studie opgezet?
Ontwerpdetails
- Primair doel: Behandeling
- Toewijzing: Gerandomiseerd
- Interventioneel model: Sequentiële toewijzing
- Masker: Verviervoudigen
Wapens en interventies
Deelnemersgroep / Arm |
Interventie / Behandeling |
|---|---|
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Experimenteel: Adult Cohort 1
Drug: Sevasemten Drug: Placebo |
Sevasemten is administered orally once per day Placebo is administered orally once per day |
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Experimenteel: 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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Experimenteel: Adult Cohort 6
Drug: Sevasemten Drug: Placebo |
Sevasemten is administered orally once per day Placebo is administered orally once per day |
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Experimenteel: Adolescent Cohort 4
Drug: Sevasemten Drug: Placebo |
Placebo is administered orally once per day Sevasemten is administered orally once per day |
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Experimenteel: Adolescent Cohort 5
Drug: Sevasemten Drug: Placebo |
Placebo is administered orally once per day Sevasemten is administered orally once per day |
Wat meet het onderzoek?
Primaire uitkomstmaten
Uitkomstmaat |
Maatregel Beschrijving |
Tijdsspanne |
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Number of treatment emergent adverse events in those treated with sevasemten or placebo
Tijdsspanne: 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
Tijdsspanne: 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
Tijdsspanne: 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
Tijdsspanne: 18 months (GRAND CANYON Cohort 6)
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Adult participants |
18 months (GRAND CANYON Cohort 6)
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Secundaire uitkomstmaten
Uitkomstmaat |
Maatregel Beschrijving |
Tijdsspanne |
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Change from Baseline in total North Star Ambulatory Assessment score
Tijdsspanne: 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
Tijdsspanne: 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
Tijdsspanne: 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
Tijdsspanne: 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
Tijdsspanne: 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
Tijdsspanne: 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
Tijdsspanne: 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)
Tijdsspanne: 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
Tijdsspanne: 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
Tijdsspanne: 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
Tijdsspanne: 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)
Tijdsspanne: 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
Tijdsspanne: 18 months (GRAND CANYON Cohort 6)
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Adult participants |
18 months (GRAND CANYON Cohort 6)
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Medewerkers en onderzoekers
Sponsor
Onderzoekers
- Studie stoel: Joanne Donovan, MD, PhD, Edgewise Therapeutics, Inc.
- Studie stoel: Roxana D. Dreghici, Edgewise Therapeutics, Inc.
Publicaties en nuttige links
Nuttige links
Studie record data
Bestudeer belangrijke data
Studie start (Werkelijk)
Primaire voltooiing (Werkelijk)
Studie voltooiing (Werkelijk)
Studieregistratiedata
Eerst ingediend
Eerst ingediend dat voldeed aan de QC-criteria
Eerst geplaatst (Werkelijk)
Updates van studierecords
Laatste update geplaatst (Werkelijk)
Laatste update ingediend die voldeed aan QC-criteria
Laatst geverifieerd
Meer informatie
Termen gerelateerd aan deze studie
Trefwoorden
Aanvullende relevante MeSH-voorwaarden
Andere studie-ID-nummers
- EDG-5506-201
Plan Individuele Deelnemersgegevens (IPD)
Bent u van plan om gegevens van individuele deelnemers (IPD) te delen?
Informatie over medicijnen en apparaten, studiedocumenten
Bestudeert een door de Amerikaanse FDA gereguleerd geneesmiddel
Bestudeert een door de Amerikaanse FDA gereguleerd apparaatproduct
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