Study of SRP-4045 (Casimersen) and SRP-4053 (Golodirsen) in Participants With Duchenne Muscular Dystrophy (DMD) (ESSENCE)
Duchenne Muscular Dystrophy
About this trial
This is an interventional treatment trial for Duchenne Muscular Dystrophy focused on measuring Duchenne muscular dystrophy, Exon Skipping, DMD, Exon 53, Exon 45, Ambulatory, Pediatric, Duchenne
Eligibility Criteria
Inclusion Criteria:
- Genotypically confirmed DMD, with genetic deletion amenable to exon 45 or exon 53 skipping
- Stable dose of oral corticosteroids for at least 24 weeks prior to Week 1, and the dose is expected to remain constant throughout the study (except for modifications to accommodate changes in weight).
- Intact right and left biceps or 2 alternative upper muscle groups
- Mean 6MWT ≥300 meters and ≤450 meters
- Stable pulmonary function: forced vital capacity (FVC) ≥50% predicted
Exclusion Criteria:
- Treatment with gene therapy at any time
- Previous treatment with SMT C1100 within 1 week prior to Week 1 and previous treatment with PRO045 (BMN 045), PRO053 (BMN 053), or PRO051 (BMN 051) within 24 weeks prior to Week 1
- Current or previous treatment with any other experimental treatment within 12 weeks prior to Week 1
- Major surgery within 3 months prior to Week 1
- Presence of other clinically significant illness
Other inclusion/exclusion criteria may apply.
Sites / Locations
- Neuromuscular Research Center
- Children's Hospital Los Angeles
- David Geffen School of Medicine, UCLA
- Rady Children's Hospital San Diego/ UCSD
- Stanford University School of Medicine/Medical Center
- University of Florida
- NW Florida Clinical Research Group, LLC
- Center for Integrative Rare Disease Research (CIRDR)
- Ann and Robert H. Lurie Children's Hospital of Chicago
- University of Iowa Children's Hospital
- University of Kansas, Medical Center
- Boston Children's Hospital
- St. Louis Children's Hospital
- Las Vegas Clinic
- University of Rochester Clinical Research Center
- Cincinnati Children's Hospital Medical Center (CCHMC)
- Nationwide Children's Hospital
- Shriners Hospital for Children
- Children's Hospital of Pittsburgh of UPMC
- Children's Medical Center Dallas
- University of Utah
- Children's Hospital of the King's Daughters
- Children's Hospital of Wisconsin
- DOM Centro de Reumatologia
- Royal Children's Hospital Melbourne
- Queensland Children's Hospital
- Children's Hospital at Westmead
- Universitair Ziekenhuis Gent
- Universitair Ziekenhuis Leuven
- CHR de la Citadelle
- University Multiprofile Hospital for Active Treatment Aleksandrovska EAD
- Alberta Childrens Hospital
- Children's and Women's Health Centre of British Columbia
- London Health Sciences Centre
- Children's Hospital of Eastern Ontario
- University Hospital Brno
- Fakultni nemocnice v Motole
- Rigshospitalet Copenhagen University Hospital
- Hôpital Des Enfants
- Reference Centre for Neuromuscular Diseases
- Hôpital Armand Trousseau
- Charité - Universitätsmedizin Berlin
- Universitätsklinikum Essen
- University Hospital Freiburg
- IASO Children's Hospital
- Ippokratio General Hospital of Thessaloniki
- Semmelweis Egyetem Genomikai Medicina és Ritka Betegsegek Intezete
- Royal Instituite of Child Neurosciences
- Deenanth Mangeshkar Hospital
- The Children's University Hospital
- Schneider Children's Medical Center of Israel
- Azienda Ospedaliero-Universitaria di Ferrara - Arcispedale Sant' Anna
- Istituto Giannina Gaslini
- Az Ospedaliera Universitaria Policlinico G Martino
- Fondazione IRCCS Istituto Neurologico Carlo Besta
- Policlinico Universitario A Gemelli
- Seoul National University Hospital
- Pusan National University Yangsan Hospital
- Neurociencias Estudios Clínicos S.C
- Instituto de Investigaciones Clínicas para la Salud A.C
- Samodzielny Publiczny Centralny Szpital Kliniczny
- Uniwersyteckie Centrum Kliniczne
- Federal state budget educational institution of higher education "Russian national research medical university n.a. N.I. Pirogov" of Ministry of healthcare of Russian Federation
- State Autonomous Healthcare Institution of Sverdlovsk Region Children's Clinical Hospital No. 9 City of Ekaterinburg
- Clinic for Neurology and Psychiatry for Children and Youth
- Hospital de La Santa Creu i Sant Pau
- Hospital Sant Joan de Deu
- Hospital Universitari i Politecnic La Fe de Valencia
- Drottning Silvias Barn Och Ungdomssjukhus
- Royal Hospital for Children (Glasgow)
- Leeds Teaching Hospitals NHS Trust
- Alder Hey Childrens Hospital
- Great Ormond Street Hospital (GOSH)
- Royal Victoria Infirmary
- John Radcliffe Hospital
Arms of the Study
Arm 1
Arm 2
Arm 3
Experimental
Experimental
Placebo Comparator
SRP-4045
SRP-4053
Placebo followed by SRP-4045 or SRP-4053
Participants amenable to exon 45 skipping will receive SRP-4045 IV infusions, weekly, at 30 mg/kg for up to 96 weeks in the double-blinded period. This will be followed by an open-label extension period in which all participants will receive open-label active treatment of SRP-4045 at 30 mg/kg/week IV infusions for 48 weeks (up to Week 144 in the study).
Participants amenable to exon 53 skipping will receive SRP-4053 IV infusions, weekly, at 30 mg/kg for up to 96 weeks in the double-blinded period. This will be followed by an open-label extension period in which all participants will receive open-label active treatment of SRP-4053 at 30 mg/kg/week IV infusions for 48 weeks (up to Week 144 in the study).
Participants amenable to exon 45 or 53 skipping will receive SRP-4045 or SRP-4053 placebo-matching IV infusions, weekly, at 30 mg/kg for up to 96 weeks in the double-blinded period. This will be followed by an open-label extension period in which all participants will receive open-label active treatment of SRP-4045 or SRP-4053 at 30 mg/kg/week IV infusions for 48 weeks (up to Week 144 in the study).