Fase 2 undersøgelse af EDG-5506 i Becker muskeldystrofi (CANYON)
Et fase 2 randomiseret, dobbeltblindt, placebokontrolleret studie for at evaluere effekten af EDG-5506 på sikkerhed, biomarkører, farmakokinetik og funktionelle mål hos voksne og unge med Becker muskeldystrofi
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.
Studieoversigt
Status
Betingelser
Intervention / Behandling
Detaljeret beskrivelse
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.
Undersøgelsestype
Tilmelding (Faktiske)
Fase
- Fase 2
Kontakter og lokationer
Studiesteder
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Fitzroy, Australien, VIC, 3065
- St Vincent's Hospital Melbourne
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Leuven, Belgien
- Universitaire Ziekenhuizen Leuven
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Liège, Belgien, 4000
- Centre Hospitalier Régional de la Citadelle
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Belgium
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Ghent, Belgium, Belgien, 9000
- University Hospital Gent
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Copenhagen, Danmark
- Rigshospitalet
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London, Det Forenede Kongerige, SW17 0QT
- St. George's University Hospitals NHS Foundation Trust
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London, Det Forenede Kongerige, NW1 2PG
- University College London Hospital
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Newcastle, Det Forenede Kongerige, NE7 7DN
- Newcastle Freeman Hospital
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Salford, Det Forenede Kongerige, M68HD
- Salford Royal Hospital
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Arkansas
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Little Rock, Arkansas, Forenede Stater, 72202
- Arkansas Children's Hospital
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California
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La Jolla, California, Forenede Stater, 92037
- UC San Diego
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Los Angeles, California, Forenede Stater, 90095
- UCLA Medical Center
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Orange, California, Forenede Stater, 92868
- UC Irvine Medical Center
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Palo Alto, California, Forenede Stater, 94304
- Stanford Neuroscience Health Center
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Sacramento, California, Forenede Stater, 95817
- UC Davis Medical Center
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Colorado
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Aurora, Colorado, Forenede Stater, 80045
- UC Denver
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Florida
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Gainesville, Florida, Forenede Stater, 32611
- University of Florida
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Georgia
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Atlanta, Georgia, Forenede Stater, 30329
- Rare Disease Research
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Illinois
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Chicago, Illinois, Forenede Stater, 60611
- Northwestern University
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Indiana
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Indianapolis, Indiana, Forenede Stater, 46202
- Indiana University School of Medicine
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Iowa
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Iowa City, Iowa, Forenede Stater, 52242
- University of Iowa
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Kansas
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Kansas City, Kansas, Forenede Stater, 66160
- University of Kansas Medical Center
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Maryland
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Baltimore, Maryland, Forenede Stater, 21205
- Kennedy Krieger Institute
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Massachusetts
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Worcester, Massachusetts, Forenede Stater, 01605
- University of Massachusetts Memorial Medical Center
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Minnesota
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Minneapolis, Minnesota, Forenede Stater, 55455
- University of Minnesota
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Missouri
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St Louis, Missouri, Forenede Stater, 63110
- Washington University School of Medicine
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New York
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Rochester, New York, Forenede Stater, 14642
- University of Rochester Medical Center
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North Carolina
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Hillsborough, North Carolina, Forenede Stater, 27278
- Rare Disease Research, LLC NC
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Ohio
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Cincinnati, Ohio, Forenede Stater, 45219
- University of Cincinnati Gardner Neuroscience Institute
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Columbus, Ohio, Forenede Stater, 43205
- Nationwide Children's Hospital
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Pennsylvania
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Philadelphia, Pennsylvania, Forenede Stater, 19104
- University of Pennsylvania
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Pittsburgh, Pennsylvania, Forenede Stater, 15224
- University of Pittsburgh
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Texas
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Austin, Texas, Forenede Stater, 78759
- National Neuromuscular Research Institute
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Denton, Texas, Forenede Stater, 76208
- Neurology Rare Disease Center
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Virginia
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Richmond, Virginia, Forenede Stater, 23298
- Virginia Commonwealth University Health
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Marseille, Frankrig, 13005
- Centre de Reference des Maladies Neuromusculaires et de la SLA - AP-HM Hopital de La Timone
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Nantes, Frankrig, 44093
- CHU de Nantes
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Nice, Frankrig, 06001
- CHU de Nice - Hopital Pasteur 2 - Centre de reference des Maladies Neuromusculaires
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Paris, Frankrig, 75013
- AP-HP Hopital Pitie-Salpetriere
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Leiden, Holland, 2333 ZA
- Leids Universitair Medisch Centrum
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Jerusalem, Israel, 91240
- Hadassah University Hospital
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Petah Tikva, Israel, 49202
- Schneider Children's Hospital of Israel
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Milan, Italien, 20122
- Fondazione IRCCS Ca'Granda Ospedale Maggiore Policlinico di Milano
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Padova, Italien, 35128
- Azienda Ospedale - Università Padova
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Rome, Italien, 00168
- Fondazione Policlinico Universitario A. Gemelli IRCCS - Universita Cattolica del Sacro Cuore
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Auckland, New Zealand, 1010
- Optimal Clinical Trials
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Barcelona, Spanien, 08035
- Hospital Universitario Vall d'Hebron
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Barcelona, Spanien, 08907
- Hospital Universitari de Bellvitge
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Donostia / San Sebastian, Spanien, 20014
- Hospital Universitario Donostia
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Valencia, Spanien, 46026
- Hospital Universitari i Politecnic La Fe
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Munich, Tyskland, 80336
- Klinikum der Ludwig-Maximilians-Universitaet Muenchen
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Deltagelseskriterier
Berettigelseskriterier
Aldre berettiget til at studere
Tager imod sunde frivillige
Køn, der er berettiget til at studere
Beskrivelse
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.
Studieplan
Hvordan er undersøgelsen tilrettelagt?
Design detaljer
- Primært formål: Behandling
- Tildeling: Randomiseret
- Interventionel model: Sekventiel tildeling
- Maskning: Firedobbelt
Våben og indgreb
Deltagergruppe / Arm |
Intervention / Behandling |
|---|---|
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Eksperimentel: 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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Eksperimentel: 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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Eksperimentel: 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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Eksperimentel: 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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Eksperimentel: Adolescent Cohort 5
Drug: Sevasemten Drug: Placebo |
Placebo is administered orally once per day Sevasemten is administered orally once per day |
Hvad måler undersøgelsen?
Primære resultatmål
Resultatmål |
Foranstaltningsbeskrivelse |
Tidsramme |
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Number of treatment emergent adverse events in those treated with sevasemten or placebo
Tidsramme: 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
Tidsramme: 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
Tidsramme: 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
Tidsramme: 18 months (GRAND CANYON Cohort 6)
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Adult participants |
18 months (GRAND CANYON Cohort 6)
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Sekundære resultatmål
Resultatmål |
Foranstaltningsbeskrivelse |
Tidsramme |
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Change from Baseline in total North Star Ambulatory Assessment score
Tidsramme: 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
Tidsramme: 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
Tidsramme: 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
Tidsramme: 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
Tidsramme: 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
Tidsramme: 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
Tidsramme: 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)
Tidsramme: 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
Tidsramme: 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
Tidsramme: 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
Tidsramme: 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)
Tidsramme: 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
Tidsramme: 18 months (GRAND CANYON Cohort 6)
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Adult participants |
18 months (GRAND CANYON Cohort 6)
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Samarbejdspartnere og efterforskere
Sponsor
Efterforskere
- Studiestol: Joanne Donovan, MD, PhD, Edgewise Therapeutics, Inc.
- Studiestol: Roxana D. Dreghici, Edgewise Therapeutics, Inc.
Publikationer og nyttige links
Hjælpsomme links
Datoer for undersøgelser
Studer store datoer
Studiestart (Faktiske)
Primær færdiggørelse (Faktiske)
Studieafslutning (Faktiske)
Datoer for studieregistrering
Først indsendt
Først indsendt, der opfyldte QC-kriterier
Først opslået (Faktiske)
Opdateringer af undersøgelsesjournaler
Sidste opdatering sendt (Faktiske)
Sidste opdatering indsendt, der opfyldte kvalitetskontrolkriterier
Sidst verificeret
Mere information
Begreber relateret til denne undersøgelse
Nøgleord
Yderligere relevante MeSH-vilkår
Andre undersøgelses-id-numre
- EDG-5506-201
Plan for individuelle deltagerdata (IPD)
Planlægger du at dele individuelle deltagerdata (IPD)?
Lægemiddel- og udstyrsoplysninger, undersøgelsesdokumenter
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