Studio di fase 2 di EDG-5506 nella distrofia muscolare di Becker (CANYON)
Uno studio di fase 2 randomizzato, in doppio cieco, controllato con placebo per valutare l'effetto di EDG-5506 su sicurezza, biomarcatori, farmacocinetica e misure funzionali in adulti e adolescenti con distrofia muscolare di Becker
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.
Panoramica dello studio
Stato
Condizioni
Intervento / Trattamento
Descrizione dettagliata
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.
Tipo di studio
Iscrizione (Effettivo)
Fase
- Fase 2
Contatti e Sedi
Luoghi di studio
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Fitzroy, Australia, VIC, 3065
- St Vincent's Hospital Melbourne
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Leuven, Belgio
- Universitaire Ziekenhuizen Leuven
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Liège, Belgio, 4000
- Centre Hospitalier Régional de la Citadelle
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Belgium
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Ghent, Belgium, Belgio, 9000
- University Hospital Gent
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Copenhagen, Danimarca
- Rigshospitalet
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Marseille, Francia, 13005
- Centre de Reference des Maladies Neuromusculaires et de la SLA - AP-HM Hopital de La Timone
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Nantes, Francia, 44093
- CHU de Nantes
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Nice, Francia, 06001
- CHU de Nice - Hopital Pasteur 2 - Centre de reference des Maladies Neuromusculaires
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Paris, Francia, 75013
- AP-HP Hopital Pitie-Salpetriere
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Munich, Germania, 80336
- Klinikum der Ludwig-Maximilians-Universitaet Muenchen
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Jerusalem, Israele, 91240
- Hadassah University Hospital
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Petah Tikva, Israele, 49202
- Schneider Children's Hospital of Israel
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Milan, Italia, 20122
- Fondazione IRCCS Ca'Granda Ospedale Maggiore Policlinico di Milano
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Padova, Italia, 35128
- Azienda Ospedale - Università Padova
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Rome, Italia, 00168
- Fondazione Policlinico Universitario A. Gemelli IRCCS - Universita Cattolica del Sacro Cuore
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Auckland, Nuova Zelanda, 1010
- Optimal Clinical Trials
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Leiden, Olanda, 2333 ZA
- Leids Universitair Medisch Centrum
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London, Regno Unito, SW17 0QT
- St. George's University Hospitals NHS Foundation Trust
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London, Regno Unito, NW1 2PG
- University College London Hospital
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Newcastle, Regno Unito, NE7 7DN
- Newcastle Freeman Hospital
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Salford, Regno Unito, M68HD
- Salford Royal Hospital
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Barcelona, Spagna, 08035
- Hospital Universitario Vall d'Hebron
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Barcelona, Spagna, 08907
- Hospital Universitari de Bellvitge
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Donostia / San Sebastian, Spagna, 20014
- Hospital Universitario Donostia
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Valencia, Spagna, 46026
- Hospital Universitari i Politecnic La Fe
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Arkansas
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Little Rock, Arkansas, Stati Uniti, 72202
- Arkansas Children's Hospital
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California
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La Jolla, California, Stati Uniti, 92037
- UC San Diego
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Los Angeles, California, Stati Uniti, 90095
- UCLA Medical Center
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Orange, California, Stati Uniti, 92868
- UC Irvine Medical Center
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Palo Alto, California, Stati Uniti, 94304
- Stanford Neuroscience Health Center
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Sacramento, California, Stati Uniti, 95817
- UC Davis Medical Center
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Colorado
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Aurora, Colorado, Stati Uniti, 80045
- UC Denver
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Florida
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Gainesville, Florida, Stati Uniti, 32611
- University of Florida
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Georgia
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Atlanta, Georgia, Stati Uniti, 30329
- Rare Disease Research
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Illinois
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Chicago, Illinois, Stati Uniti, 60611
- Northwestern University
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Indiana
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Indianapolis, Indiana, Stati Uniti, 46202
- Indiana University School of Medicine
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Iowa
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Iowa City, Iowa, Stati Uniti, 52242
- University of Iowa
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Kansas
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Kansas City, Kansas, Stati Uniti, 66160
- University of Kansas Medical Center
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Maryland
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Baltimore, Maryland, Stati Uniti, 21205
- Kennedy Krieger Institute
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Massachusetts
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Worcester, Massachusetts, Stati Uniti, 01605
- University of Massachusetts Memorial Medical Center
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Minnesota
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Minneapolis, Minnesota, Stati Uniti, 55455
- University of Minnesota
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Missouri
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St Louis, Missouri, Stati Uniti, 63110
- Washington University School of Medicine
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New York
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Rochester, New York, Stati Uniti, 14642
- University of Rochester Medical Center
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North Carolina
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Hillsborough, North Carolina, Stati Uniti, 27278
- Rare Disease Research, LLC NC
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Ohio
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Cincinnati, Ohio, Stati Uniti, 45219
- University of Cincinnati Gardner Neuroscience Institute
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Columbus, Ohio, Stati Uniti, 43205
- Nationwide Children's Hospital
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Pennsylvania
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Philadelphia, Pennsylvania, Stati Uniti, 19104
- University of Pennsylvania
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Pittsburgh, Pennsylvania, Stati Uniti, 15224
- University of Pittsburgh
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Texas
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Austin, Texas, Stati Uniti, 78759
- National Neuromuscular Research Institute
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Denton, Texas, Stati Uniti, 76208
- Neurology Rare Disease Center
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Virginia
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Richmond, Virginia, Stati Uniti, 23298
- Virginia Commonwealth University Health
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Criteri di partecipazione
Criteri di ammissibilità
Età idonea allo studio
Accetta volontari sani
Sessi ammissibili allo studio
Descrizione
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.
Piano di studio
Come è strutturato lo studio?
Dettagli di progettazione
- Scopo principale: Trattamento
- Assegnazione: Randomizzato
- Modello interventistico: Assegnazione sequenziale
- Mascheramento: Quadruplicare
Armi e interventi
Gruppo di partecipanti / Arm |
Intervento / Trattamento |
|---|---|
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Sperimentale: 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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Sperimentale: 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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Sperimentale: 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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Sperimentale: 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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Sperimentale: Adolescent Cohort 5
Drug: Sevasemten Drug: Placebo |
Placebo is administered orally once per day Sevasemten is administered orally once per day |
Cosa sta misurando lo studio?
Misure di risultato primarie
Misura del risultato |
Misura Descrizione |
Lasso di tempo |
|---|---|---|
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Number of treatment emergent adverse events in those treated with sevasemten or placebo
Lasso di tempo: 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
Lasso di tempo: 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
Lasso di tempo: 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
Lasso di tempo: 18 months (GRAND CANYON Cohort 6)
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Adult participants |
18 months (GRAND CANYON Cohort 6)
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Misure di risultato secondarie
Misura del risultato |
Misura Descrizione |
Lasso di tempo |
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Change from Baseline in total North Star Ambulatory Assessment score
Lasso di tempo: 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
Lasso di tempo: 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
Lasso di tempo: 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
Lasso di tempo: 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
Lasso di tempo: 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
Lasso di tempo: 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
Lasso di tempo: 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)
Lasso di tempo: 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
Lasso di tempo: 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
Lasso di tempo: 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
Lasso di tempo: 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)
Lasso di tempo: 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
Lasso di tempo: 18 months (GRAND CANYON Cohort 6)
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Adult participants |
18 months (GRAND CANYON Cohort 6)
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Collaboratori e investigatori
Sponsor
Investigatori
- Cattedra di studio: Joanne Donovan, MD, PhD, Edgewise Therapeutics, Inc.
- Cattedra di studio: Roxana D. Dreghici, Edgewise Therapeutics, Inc.
Pubblicazioni e link utili
Collegamenti utili
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Inizio studio (Effettivo)
Completamento primario (Effettivo)
Completamento dello studio (Effettivo)
Date di iscrizione allo studio
Primo inviato
Primo inviato che soddisfa i criteri di controllo qualità
Primo Inserito (Effettivo)
Aggiornamenti dei record di studio
Ultimo aggiornamento pubblicato (Effettivo)
Ultimo aggiornamento inviato che soddisfa i criteri QC
Ultimo verificato
Maggiori informazioni
Termini relativi a questo studio
Parole chiave
Termini MeSH pertinenti aggiuntivi
Altri numeri di identificazione dello studio
- EDG-5506-201
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