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Fase 2-studie av EDG-5506 i Becker muskeldystrofi (CANYON)

16. september 2026 oppdatert av: Edgewise Therapeutics, Inc.

En fase 2 randomisert, dobbeltblind, placebokontrollert studie for å evaluere effekten av EDG-5506 på sikkerhet, biomarkører, farmakokinetikk og funksjonelle tiltak hos voksne og ungdom 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.

Studieoversikt

Status

Fullført

Forhold

Intervensjon / Behandling

Detaljert 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.

Studietype

Intervensjonell

Registrering (Faktiske)

244

Fase

  • Fase 2

Kontakter og plasseringer

Denne delen inneholder kontaktinformasjon for de som utfører studien, og informasjon om hvor denne studien blir utført.

Studiesteder

  • Australia
      • Fitzroy, Australia, VIC, 3065
        • St Vincent's Hospital Melbourne
  • Belgia
      • Leuven, Belgia
        • Universitaire Ziekenhuizen Leuven
      • Liège, Belgia, 4000
        • Centre Hospitalier Régional de la Citadelle
    • Belgium
      • Ghent, Belgium, Belgia, 9000
        • University Hospital Gent
  • Danmark
      • Copenhagen, Danmark
        • Rigshospitalet
  • Forente stater
    • Arkansas
      • Little Rock, Arkansas, Forente stater, 72202
        • Arkansas Children's Hospital
    • California
      • La Jolla, California, Forente stater, 92037
        • UC San Diego
      • Los Angeles, California, Forente stater, 90095
        • UCLA Medical Center
      • Orange, California, Forente stater, 92868
        • UC Irvine Medical Center
      • Palo Alto, California, Forente stater, 94304
        • Stanford Neuroscience Health Center
      • Sacramento, California, Forente stater, 95817
        • UC Davis Medical Center
    • Colorado
      • Aurora, Colorado, Forente stater, 80045
        • UC Denver
    • Florida
      • Gainesville, Florida, Forente stater, 32611
        • University of Florida
    • Georgia
      • Atlanta, Georgia, Forente stater, 30329
        • Rare Disease Research
    • Illinois
      • Chicago, Illinois, Forente stater, 60611
        • Northwestern University
    • Indiana
      • Indianapolis, Indiana, Forente stater, 46202
        • Indiana University School of Medicine
    • Iowa
      • Iowa City, Iowa, Forente stater, 52242
        • University of Iowa
    • Kansas
      • Kansas City, Kansas, Forente stater, 66160
        • University of Kansas Medical Center
    • Maryland
      • Baltimore, Maryland, Forente stater, 21205
        • Kennedy Krieger Institute
    • Massachusetts
      • Worcester, Massachusetts, Forente stater, 01605
        • University of Massachusetts Memorial Medical Center
    • Minnesota
      • Minneapolis, Minnesota, Forente stater, 55455
        • University of Minnesota
    • Missouri
      • St Louis, Missouri, Forente stater, 63110
        • Washington University School of Medicine
    • New York
      • Rochester, New York, Forente stater, 14642
        • University of Rochester Medical Center
    • North Carolina
      • Hillsborough, North Carolina, Forente stater, 27278
        • Rare Disease Research, LLC NC
    • Ohio
      • Cincinnati, Ohio, Forente stater, 45219
        • University of Cincinnati Gardner Neuroscience Institute
      • Columbus, Ohio, Forente stater, 43205
        • Nationwide Children's Hospital
    • Pennsylvania
      • Philadelphia, Pennsylvania, Forente stater, 19104
        • University of Pennsylvania
      • Pittsburgh, Pennsylvania, Forente stater, 15224
        • University of Pittsburgh
    • Texas
      • Austin, Texas, Forente stater, 78759
        • National Neuromuscular Research Institute
      • Denton, Texas, Forente stater, 76208
        • Neurology Rare Disease Center
    • Virginia
      • Richmond, Virginia, Forente stater, 23298
        • Virginia Commonwealth University Health
  • Frankrike
      • Marseille, Frankrike, 13005
        • Centre de Reference des Maladies Neuromusculaires et de la SLA - AP-HM Hopital de La Timone
      • Nantes, Frankrike, 44093
        • CHU de Nantes
      • Nice, Frankrike, 06001
        • CHU de Nice - Hopital Pasteur 2 - Centre de reference des Maladies Neuromusculaires
      • Paris, Frankrike, 75013
        • AP-HP Hopital Pitie-Salpetriere
  • Israel
      • Jerusalem, Israel, 91240
        • Hadassah University Hospital
      • Petah Tikva, Israel, 49202
        • Schneider Children's Hospital of Israel
  • Italia
      • Milan, Italia, 20122
        • Fondazione IRCCS Ca'Granda Ospedale Maggiore Policlinico di Milano
      • Padova, Italia, 35128
        • Azienda Ospedale - Università Padova
      • Rome, Italia, 00168
        • Fondazione Policlinico Universitario A. Gemelli IRCCS - Universita Cattolica del Sacro Cuore
  • Nederland
      • Leiden, Nederland, 2333 ZA
        • Leids Universitair Medisch Centrum
  • New Zealand
      • Auckland, New Zealand, 1010
        • Optimal Clinical Trials
  • Spania
      • Barcelona, Spania, 08035
        • Hospital Universitario Vall d'Hebron
      • Barcelona, Spania, 08907
        • Hospital Universitari de Bellvitge
      • Donostia / San Sebastian, Spania, 20014
        • Hospital Universitario Donostia
      • Valencia, Spania, 46026
        • Hospital Universitari i Politecnic La Fe
  • Storbritannia
      • London, Storbritannia, SW17 0QT
        • St. George's University Hospitals NHS Foundation Trust
      • London, Storbritannia, NW1 2PG
        • University College London Hospital
      • Newcastle, Storbritannia, NE7 7DN
        • Newcastle Freeman Hospital
      • Salford, Storbritannia, M68HD
        • Salford Royal Hospital
  • Tyskland
      • Munich, Tyskland, 80336
        • Klinikum der Ludwig-Maximilians-Universitaet Muenchen

Deltakelseskriterier

Forskere ser etter personer som passer til en bestemt beskrivelse, kalt kvalifikasjonskriterier. Noen eksempler på disse kriteriene er en persons generelle helsetilstand eller tidligere behandlinger.

Kvalifikasjonskriterier

Alder som er kvalifisert for studier

12 år til 50 år (Barn, Voksen)

Tar imot friske frivillige

Nei

Kjønn som er kvalifisert for studier

Mann

Beskrivelse

GRAND CANYON eligibility is listed below.

Key Inclusion Criteria:

  1. 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.
  2. Able to complete the 100-meter timed test in < 200 seconds with or without use of mobility aid devices.
  3. Able to perform the North Star Ambulatory Assessment scale and achieve a score of 5 to 32, inclusive.

Key Exclusion Criteria:

  1. 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.
  2. Cardiac echocardiogram ejection fraction < 40%
  3. Forced vital capacity predicted <60% or using daytime ventilatory support
  4. Receipt of oral corticosteroids for the treatment of BMD in the previous 6 months.
  5. Receipt of an investigational drug within 30 days or 5 half-lives (whichever is longer) of the screening visit in the present study.

Studieplan

Denne delen gir detaljer om studieplanen, inkludert hvordan studien er utformet og hva studien måler.

Hvordan er studiet utformet?

Designdetaljer

  • Primært formål: Behandling
  • Tildeling: Randomisert
  • Intervensjonsmodell: Sekvensiell tildeling
  • Masking: Firemannsrom

Våpen og intervensjoner

Deltakergruppe / Arm
Intervensjon / Behandling
Eksperimentell: Adult Cohort 1

Drug: Sevasemten Drug: Placebo
Legemiddel: Sevasemten 10 mg

Sevasemten is administered orally once per day

Legemiddel: Placebo

Placebo is administered orally once per day
Eksperimentell: Adult Cohort 2

Drug: Sevasemten Drug: Placebo
Legemiddel: Sevasemten 10 mg

Sevasemten is administered orally once per day

Legemiddel: Placebo

Placebo is administered orally once per day
Eksperimentell: Adult Cohort 6

Drug: Sevasemten Drug: Placebo
Legemiddel: Sevasemten 10 mg

Sevasemten is administered orally once per day

Legemiddel: Placebo

Placebo is administered orally once per day
Eksperimentell: Adolescent Cohort 4

Drug: Sevasemten Drug: Placebo
Legemiddel: Placebo

Placebo is administered orally once per day

Legemiddel: Sevasemten 5 mg

Sevasemten is administered orally once per day
Eksperimentell: Adolescent Cohort 5

Drug: Sevasemten Drug: Placebo
Legemiddel: Placebo

Placebo is administered orally once per day

Legemiddel: Sevasemten 12.5 mg

Sevasemten is administered orally once per day

Hva måler studien?

Primære resultatmål

Resultatmål
Tiltaksbeskrivelse
Tidsramme
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)

All participants
12 months (CANYON Cohorts 1, 2, 4, 5), 18 months (GRAND CANYON Cohort 6)
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)

All participants
12 months (CANYON Cohorts 1, 2, 4, 5), 18 months (GRAND CANYON Cohort 6)
Change from Baseline in serum Creatine Kinase
Tidsramme: Averaged across Months 6, 9, 12 Months (CANYON Cohorts 1, 2)

Adult participants
Averaged across Months 6, 9, 12 Months (CANYON Cohorts 1, 2)
Change from Baseline in total North Star Ambulatory Assessment score
Tidsramme: 18 months (GRAND CANYON Cohort 6)

Adult participants
18 months (GRAND CANYON Cohort 6)

Sekundære resultatmål

Resultatmål
Tiltaksbeskrivelse
Tidsramme
Change from Baseline in total North Star Ambulatory Assessment score
Tidsramme: 12 Months (CANYON Cohorts 1, 2)

Adult participants
12 Months (CANYON Cohorts 1, 2)
Pharmacokinetics as measured by steady state plasma concentration
Tidsramme: 12 Months (CANYON Cohorts 1, 2, 4, 5), 18 months (GRAND CANYON Cohort 6)

All participants
12 Months (CANYON Cohorts 1, 2, 4, 5), 18 months (GRAND CANYON Cohort 6)
Change from Baseline in growth as assessed by height centile on World Health Organization growth charts
Tidsramme: 12 months (CANYON Cohorts 4, 5)

Adolescent participants
12 months (CANYON Cohorts 4, 5)
Incidence of laboratory test-related treatment emergent adverse events
Tidsramme: 18 months (GRAND CANYON Cohort 6)

Adult participants
18 months (GRAND CANYON Cohort 6)
Change from Baseline in serum Creatine Kinase
Tidsramme: 18 months (GRAND CANYON Cohort 6)

Adult participants
18 months (GRAND CANYON Cohort 6)
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)

Adult participants
Averaged across Months 6 and 12 (CANYON Cohorts 1, 2); 18 months (GRAND CANYON Cohort 6)
Change from Baseline in serum myoglobin
Tidsramme: Averaged across Months 6, 9, and 12 (CANYON Cohorts 1,2); 18 months (GRAND CANYON Cohort 6)

Adult participants
Averaged across Months 6, 9, and 12 (CANYON Cohorts 1,2); 18 months (GRAND CANYON Cohort 6)
Change from Baseline in total NSAD score (CANYON Cohorts 1,2)
Tidsramme: 12 Months (CANYON Cohorts 1, 2)

Adult participants
12 Months (CANYON Cohorts 1, 2)
Change from Baseline in 4-stair climb test
Tidsramme: 18 months (GRAND CANYON Cohort 6)

Adult participants
18 months (GRAND CANYON Cohort 6)
Change from Baseline in the 10-meter walk/run test
Tidsramme: 12 Months (CANYON Cohorts 1, 2), 18 Months (GRAND CANYON Cohort 6)

Adult participants
12 Months (CANYON Cohorts 1, 2), 18 Months (GRAND CANYON Cohort 6)
Change from Baseline in 100-meter timed test
Tidsramme: 12 Months (CANYON Cohorts 1, 2), 18 Months (GRAND CANYON Cohort 6)

Adult participants
12 Months (CANYON Cohorts 1, 2), 18 Months (GRAND CANYON Cohort 6)
Change from Baseline in stride velocity (95th percentile)
Tidsramme: 18 Months (GRAND CANYON Cohort 6)

Adult participants
18 Months (GRAND CANYON Cohort 6)
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)

Adult participants
18 months (GRAND CANYON Cohort 6)

Samarbeidspartnere og etterforskere

Det er her du vil finne personer og organisasjoner som er involvert i denne studien.

Sponsor

Edgewise Therapeutics, Inc.

Samarbeidspartnere

Medpace, Inc.
SYSNAV
ImagingNMD

Etterforskere

  • Studiestol: Joanne Donovan, MD, PhD, Edgewise Therapeutics, Inc.
  • Studiestol: Roxana D. Dreghici, Edgewise Therapeutics, Inc.

Publikasjoner og nyttige lenker

Den som er ansvarlig for å legge inn informasjon om studien leverer frivillig disse publikasjonene. Disse kan handle om alt relatert til studiet.

Hjelpsomme linker

Studierekorddatoer

Disse datoene sporer fremdriften for innsending av studieposter og sammendragsresultater til ClinicalTrials.gov. Studieposter og rapporterte resultater gjennomgås av National Library of Medicine (NLM) for å sikre at de oppfyller spesifikke kvalitetskontrollstandarder før de legges ut på det offentlige nettstedet.

Studer hoveddatoer

Studiestart (Faktiske)

10. november 2022

Primær fullføring (Faktiske)

7. september 2026

Studiet fullført (Faktiske)

7. september 2026

Datoer for studieregistrering

Først innsendt

14. mars 2022

Først innsendt som oppfylte QC-kriteriene

14. mars 2022

Først lagt ut (Faktiske)

22. mars 2022

Oppdateringer av studieposter

Sist oppdatering lagt ut (Faktiske)

21. september 2026

Siste oppdatering sendt inn som oppfylte QC-kriteriene

16. september 2026

Sist bekreftet

1. september 2026

Mer informasjon

Begreper knyttet til denne studien

Nøkkelord

Ytterligere relevante MeSH-vilkår

Andre studie-ID-numre

  • EDG-5506-201

Plan for individuelle deltakerdata (IPD)

Planlegger du å dele individuelle deltakerdata (IPD)?

NEI

Legemiddel- og utstyrsinformasjon, studiedokumenter

Studerer et amerikansk FDA-regulert medikamentprodukt

Ja

Studerer et amerikansk FDA-regulert enhetsprodukt

Nei

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