Study of SRP-4045 and SRP-4053 in Participants With Duchenne Muscular Dystrophy (DMD) (ESSENCE)
![]() |
The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details. |
ClinicalTrials.gov Identifier: NCT02500381 |
Recruitment Status :
Recruiting
First Posted : July 16, 2015
Last Update Posted : March 15, 2022
|
- Study Details
- Tabular View
- No Results Posted
- Disclaimer
- How to Read a Study Record
Condition or disease | Intervention/treatment | Phase |
---|---|---|
Duchenne Muscular Dystrophy | Drug: SRP-4045 Drug: SRP-4053 Drug: Placebo | Phase 3 |
This is a double-blind, placebo-controlled, multi-center study to evaluate the efficacy and safety of SRP-4045 and SRP-4053. Eligible participants with out-of-frame deletion mutations amenable to exon 45 or 53 skipping will be randomized to receive once weekly intravenous (IV) infusions of 30 milligrams/kilograms (mg/kg) SRP-4045 or 30 mg/kg SRP-4053 respectively (combined-active group) or placebo for up to 96 weeks (the placebo-controlled period of the trial). This will be followed by an open-label extension period in which all participants will receive open-label active treatment for 48 weeks (up to Week 144 of study).
The study will enroll approximately 222 participants. Twice as many participants will be randomized to receive active treatment as will receive placebo (2:1 randomization).
Clinical efficacy will be assessed at regularly scheduled study visits, including functional tests, such as the 6-minute walk test (6MWT). All participants will undergo a muscle biopsy at baseline and a second muscle biopsy either at Week 48 or Week 96.
Safety will be assessed through the collection of adverse events (AEs), laboratory tests, electrocardiograms (ECGs), echocardiograms (ECHOs), vital signs, and physical examinations throughout the study.
Blood samples will be taken periodically throughout the study to assess the pharmacokinetics of both drugs.
Study Type : | Interventional (Clinical Trial) |
Estimated Enrollment : | 222 participants |
Allocation: | Randomized |
Intervention Model: | Parallel Assignment |
Masking: | Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor) |
Masking Description: | Part 1 is double-blind and randomized; Part 2 is open-label. |
Primary Purpose: | Treatment |
Official Title: | A Double-Blind, Placebo-Controlled, Multi-Center Study With an Open-Label Extension to Evaluate the Efficacy and Safety of SRP-4045 and SRP-4053 in Patients With Duchenne Muscular Dystrophy |
Actual Study Start Date : | September 28, 2016 |
Estimated Primary Completion Date : | April 30, 2024 |
Estimated Study Completion Date : | April 30, 2024 |

Arm | Intervention/treatment |
---|---|
Experimental: SRP-4045
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).
|
Drug: SRP-4045
SRP-4045 solution for IV infusion
Other Names:
|
Experimental: SRP-4053
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).
|
Drug: SRP-4053
SRP-4053 solution for IV infusion
Other Names:
|
Placebo Comparator: Placebo followed by SRP-4045 or SRP-4053
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).
|
Drug: SRP-4045
SRP-4045 solution for IV infusion
Other Names:
Drug: SRP-4053 SRP-4053 solution for IV infusion
Other Names:
Drug: Placebo SRP-4045 or SRP-4053 placebo-matching solution for IV infusion |
- Change From Baseline in the Total Distance Walked During 6MWT at Week 96 [ Time Frame: Baseline, Week 96 ]
- Change from Baseline in the Total Distance Walked During 6MWT at Week 144 (Week 48 of the Open-Label Extension Period) [ Time Frame: Baseline, Week 144 ]
- Change from Baseline in Dystrophin Protein Levels Determined by Western Blot at Weeks 48 or 96 [ Time Frame: Baseline, Week 48 or Week 96 ]
- Change from Baseline in Dystrophin Intensity Levels Determined by Immunohistochemistry (IHC) at Weeks 48 or 96 [ Time Frame: Baseline, Week 48 or Week 96 ]
- Participant's Ability to Rise Independently From the Floor, as indicated by a North Star Ambulatory Assessment (NSAA) Subscore [ Time Frame: Week 96, Week 144 ]The NSAA is a clinician administered scale that rates the participant's performance on various functional activities. During this assessment, the participant's ability to rise independently from the floor (without external support) will be reported as an NSAA subscore of "2" (without modification) or "1" (Gower's maneuver).
- Time to Loss of Ambulation (LOA) [ Time Frame: Baseline, Week 96, and Week 144 ]
- Change From Baseline in the NSAA Total Score at Week 96 and Week 144 [ Time Frame: Baseline, Week 96 and Week 144 ]The NSAA is a clinician administered scale that rates the participant's performance on various functional activities. During this assessment, participants will be asked to perform 17 different functional activities, including a 10 meter walk/run, rising from a sit to standing, standing on 1 leg, climbing a box step, descending a box step, rising from lying to sitting, rising from the floor, lifting the head, standing on heels, and jumping. Participants will be graded as follows: 2 = achieves goal without any assistance; 1 = modified method but achieves goal independent of physical assistance from another person; and 0 = unable to achieve goal independently. NSAA Total Score ranges from 0 to 34, with a score of 34 implying normal function.
- Change From Baseline in Forced Vital Capacity Percent (FVC%) Predicted at Week 96 and Week 144 [ Time Frame: Baseline, Week 96 and Week 144 ]

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.
Ages Eligible for Study: | 6 Years to 13 Years (Child) |
Sexes Eligible for Study: | Male |
Accepts Healthy Volunteers: | No |
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.

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02500381
Contact: Medical Information | +1 800-690-2003 | clinicaltrials@sarepta.com |

United States, Arizona | |
Neuromuscular Research Center | Active, not recruiting |
Phoenix, Arizona, United States, 85028 | |
United States, California | |
Children's Hospital Los Angeles | Active, not recruiting |
Los Angeles, California, United States, 90027 | |
David Geffen School of Medicine, UCLA | Active, not recruiting |
Los Angeles, California, United States, 90095 | |
UC Davis Medical Center | Withdrawn |
Sacramento, California, United States, 95817 | |
Rady Children's Hospital San Diego/ UCSD | Active, not recruiting |
San Diego, California, United States, 92123 | |
Stanford University School of Medicine/Medical Center | Active, not recruiting |
Stanford, California, United States, 94305 | |
United States, Connecticut | |
Connecticut Children's Medical Center | Withdrawn |
Hartford, Connecticut, United States, 06106 | |
United States, Florida | |
University of Florida | Active, not recruiting |
Gainesville, Florida, United States, 32610 | |
NW Florida Clinical Research Group, LLC | Active, not recruiting |
Gulf Breeze, Florida, United States, 32561 | |
United States, Georgia | |
Center for Integrative Rare Disease Research (CIRDR) | Active, not recruiting |
Atlanta, Georgia, United States, 30318 | |
United States, Illinois | |
Ann and Robert H. Lurie Children's Hospital of Chicago | Active, not recruiting |
Chicago, Illinois, United States, 60611 | |
United States, Iowa | |
University of Iowa Children's Hospital | Active, not recruiting |
Iowa City, Iowa, United States, 52242 | |
United States, Kansas | |
University of Kansas, Medical Center | Active, not recruiting |
Kansas City, Kansas, United States, 66160 | |
United States, Massachusetts | |
Boston Children's Hospital | Active, not recruiting |
Boston, Massachusetts, United States, 02115 | |
United States, Missouri | |
St. Louis Children's Hospital | Active, not recruiting |
Saint Louis, Missouri, United States, 63110 | |
United States, Nevada | |
Las Vegas Clinic | Active, not recruiting |
Las Vegas, Nevada, United States, 89145 | |
United States, New York | |
University of Rochester Clinical Research Center | Active, not recruiting |
Rochester, New York, United States, 14642 | |
United States, Ohio | |
Cincinnati Children's Hospital Medical Center (CCHMC) | Active, not recruiting |
Cincinnati, Ohio, United States, 45229 | |
Nationwide Children's Hospital | Active, not recruiting |
Columbus, Ohio, United States, 43205 | |
United States, Oregon | |
Shriners Hospital for Children | Active, not recruiting |
Portland, Oregon, United States, 97239 | |
United States, Pennsylvania | |
Childrens Hospital of Pennsylvania | Withdrawn |
Philadelphia, Pennsylvania, United States, 19104 | |
Children's Hospital of Pittsburgh of UPMC | Active, not recruiting |
Pittsburgh, Pennsylvania, United States, 15224 | |
United States, Texas | |
Children's Medical Center Dallas | Active, not recruiting |
Dallas, Texas, United States, 75235 | |
United States, Utah | |
University of Utah | Active, not recruiting |
Salt Lake City, Utah, United States, 84132 | |
United States, Virginia | |
Children's Hospital of the King's Daughters | Active, not recruiting |
Norfolk, Virginia, United States, 23507 | |
United States, Wisconsin | |
Children's Hospital of Wisconsin | Active, not recruiting |
Milwaukee, Wisconsin, United States, 53226 | |
Australia, Victoria | |
Royal Children's Hospital Melbourne | Active, not recruiting |
Parkville, Victoria, Australia, 3052 | |
Australia | |
Queensland Children's Hospital | Recruiting |
South Brisbane, Australia, 4101 | |
Principal Investigator: Anita Cairns, MD | |
Children's Hospital at Westmead | Recruiting |
Westmead, Australia, 2145 | |
Contact: Kaitlyn Griffin +61298450495 kaitlyn.griffin@health.nsw.gov.au | |
Principal Investigator: Michelle Lorentzos, MD | |
Belgium | |
Universitair Ziekenhuis Gent | Recruiting |
Ghent, Belgium, 9000 | |
Contact: Elke De Vos +3293321954 elke.devos@uzgent.be | |
Principal Investigator: Nicolas Deconinck, MD | |
Universitair Ziekenhuis Leuven | Recruiting |
Leuven, Belgium, 3000 | |
Contact: Anne Vanden Eynden +3216343991 anne.vandeneynden@uzleuven.be | |
Principal Investigator: Liesbeth De Waele, MD, PHD | |
CHR de la Citadelle | Recruiting |
Liège, Belgium, 4000 | |
Contact: Laura Buscemi +3243218322 laura.buscemi@chuliege.be | |
Principal Investigator: Laurent Servais, MD | |
Bulgaria | |
University Multiprofile Hospital for Active Treatment Aleksandrovska EAD | Recruiting |
Sofia, Sofia-Grad, Bulgaria, 1431 | |
Contact: Tanya Dimitrova +359889320421 taniadimi@abv.bg | |
Principal Investigator: Ivaylo Tarnev, MD, PhD | |
Canada, Alberta | |
Alberta Childrens Hospital | Active, not recruiting |
Calgary, Alberta, Canada, T3B 6A8 | |
Canada, British Columbia | |
Children's and Women's Health Centre of British Columbia | Active, not recruiting |
Vancouver, British Columbia, Canada, V6H 3V4 | |
Canada, Ontario | |
London Health Sciences Centre | Recruiting |
London, Ontario, Canada, N6A 5W9 | |
Contact: Gina Bhullar (519) 685-8500 ext 55058 gina.bhullar@lhsc.on.ca | |
Principal Investigator: Craig Campbell, MD | |
Children's Hospital of Eastern Ontario | Recruiting |
Ottawa, Ontario, Canada, K1H 8L1 | |
Contact: Rosa Ramos rramos@cheo.on.ca | |
Principal Investigator: Hanns Lochmuller, MD | |
Czechia | |
University Hospital Brno | Recruiting |
Brno, Czechia, 61300 | |
Principal Investigator: Lenka Jurikova, MD | |
Fakultni nemocnice v Motole | Recruiting |
Praha, Czechia, 15008 | |
Contact: Marcela Hlozankova +420224433367 m.hlozankova@centrum.cz | |
Principal Investigator: Jana Haberlová, MD | |
Denmark | |
Rigshospitalet Copenhagen University Hospital | Recruiting |
København Ø, Denmark, 2100 | |
Principal Investigator: Christina Hoi-Hansen, MD | |
France | |
Hôpital Des Enfants | Recruiting |
Toulouse, Haute-Garonne, France, 31059 | |
Principal Investigator: Claude Cances, MD | |
Reference Centre for Neuromuscular Diseases | Recruiting |
Nantes, France, 44093 | |
Principal Investigator: Yann Péréon, MD | |
Hôpital Armand Trousseau | Recruiting |
Paris, France, 75012 | |
Contact: Dominique Duchêne +33171738313 d.duchene@institut-myologie.org | |
Principal Investigator: Andrea Sefarian, MD, PhD | |
Germany | |
Charité - Universitätsmedizin Berlin | Recruiting |
Berlin, Germany, 13353 | |
Contact: Regina Rybka Golm +49 30 450 66 10 44 regina.rybka-golm@charite.de | |
Principal Investigator: Claudia Weiss, MD | |
Universitätsklinikum Essen | Recruiting |
Essen, Germany, 45122 | |
Contact: Corinna Seifert +4920172385170 corinna.seifert@uk-essen.de | |
Principal Investigator: Ulrike Schara-Schmidt, MD | |
University Hospital Freiburg | Recruiting |
Freiburg, Germany, 79106 | |
Contact: Sabine Wider +4976127043440 sabine.wider@uniklinik-freiburg.de | |
Principal Investigator: David Schorling, MD | |
Greece | |
IASO Children's Hospital | Recruiting |
Maroussi, Greece, 15123 | |
Contact: Maria Konstantinou +306931838751 | |
Principal Investigator: Antigone Papavasiliou, MD | |
Ippokratio General Hospital of Thessaloniki | Recruiting |
Thessaloniki, Greece, 54642 | |
Principal Investigator: Dimitrios Zafeiriou, MD | |
Hungary | |
Semmelweis Egyetem Genomikai Medicina és Ritka Betegsegek Intezete | Recruiting |
Budapest, Hungary, 1083 | |
Contact: Noemi Toreki +3614591492 tnoemy@gmail.com | |
Principal Investigator: Maria Judith Molnar, MD | |
Ireland | |
The Children's University Hospital Temple Street | Recruiting |
Dublin, Ireland, D1 | |
Contact: Declan O'Rourke 353 (1) 8784200 declan.orourke@cuh.ie | |
Principal Investigator: Declan O'Rourke, MD | |
Israel | |
Schneider Children's Medical Center of Israel | Recruiting |
Petah Tikvah, Israel, 49102 | |
Contact: Ori Raz (972) 525-1860 x44 orira1@clalit.org.il | |
Principal Investigator: Yoram Nevo, MD | |
Italy | |
Azienda Ospedaliero-Universitaria di Ferrara - Arcispedale Sant' Anna | Recruiting |
Ferrara, Italy, 44124 | |
Contact: Fernanda Fortunato +393465374262 frtfnn@unife.it | |
Principal Investigator: Alessanda Ferlini, MD, PhD | |
Istituto Giannina Gaslini | Recruiting |
Genoa, Italy, 16147 | |
Contact: Marina Pedemonte, MD +3901056362675 marinapedemonte@gaslini.org | |
Principal Investigator: Claudio Bruno, MD | |
Az Ospedaliera Universitaria Policlinico G Martino | Recruiting |
Messina, Italy, 98125 | |
Principal Investigator: Giuseppe Vita, MD | |
Fondazione IRCCS Istituto Neurologico Carlo Besta | Recruiting |
Milano, Italy, 20133 | |
Contact: Angela Pasquariello 390223942967 angela.pasquariello@istituto-besta.it | |
Principal Investigator: Riccardo Masson, MD | |
Policlinico Universitario A Gemelli | Recruiting |
Rome, Italy, 00168 | |
Contact: Mariarosaria Vizzino +390630158581 npi.clinicaltrials@rm.unicatt.it | |
Principal Investigator: Eugenio Mercuri, MD | |
Mexico | |
Neurociencias Estudios Clínicos S.C | Recruiting |
Culiacán, Mexico, 80020 | |
Principal Investigator: Elmer Lopez, MD | |
Instituto de Investigaciones Clínicas para la Salud A.C | Recruiting |
Durango, Mexico, 34000 | |
Principal Investigator: Marco Martinez, MD | |
Poland | |
Samodzielny Publiczny Centralny Szpital Kliniczny | Recruiting |
Warsaw, Mazowieckie, Poland, 02-097 | |
Contact: Anna Kostera-Pruszczyk, MD, PhD (+48 22) 599 28 57 neurologia1-sekretariat@wum.edu.pl | |
Principal Investigator: Anna Kostera-Pruszczyk, MD, PhD | |
Uniwersyteckie Centrum Kliniczne | Recruiting |
Gdansk, Poland, 80-952 | |
Principal Investigator: Maria Mazurkiewicz-Beldzinska, MD | |
Russian Federation | |
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 | Active, not recruiting |
Moscow, Russian Federation, 125412 | |
State Autonomous Healthcare Institution of Sverdlovsk Region Children's Clinical Hospital No. 9 City of Ekaterinburg | Active, not recruiting |
Yekaterinburg, Russian Federation | |
Serbia | |
Clinic for Neurology and Psychiatry for Children and Youth | Recruiting |
Belgrade, Serbia, 11000 | |
Contact: Biljana Milich fax +381 11 2645064 biljana.milich@gmail.com | |
Principal Investigator: Vedrana Milic Rasic, MD | |
Spain | |
Hospital de La Santa Creu i Sant Pau | Recruiting |
Barcelona, Spain, 08041 | |
Principal Investigator: Jordi Diaz Manera, MD | |
Hospital Sant Joan de Deu | Recruiting |
Barcelona, Spain, 08950 | |
Contact: Marina Garcia +34936009733 mgarciago@fsjd.org | |
Principal Investigator: Andres Nascimento, MD | |
Hospital Universitari i Politecnic La Fe de Valencia | Recruiting |
Valencia, Spain | |
Contact: Pilar Marti +34961244000 mapifres@gmail.com | |
Principal Investigator: Nuria Muelas, MD | |
Sweden | |
Drottning Silvias Barn Och Ungdomssjukhus | Recruiting |
Göteborg, Sweden, SE-41685 | |
Contact: Anna-Lena Tulinius +46313436069 anna-lena.tulinius@vgregion.se | |
Principal Investigator: Eva Micheal, MD | |
United Kingdom | |
Royal Hospital for Children (Glasgow) | Recruiting |
Glasgow, United Kingdom, G51 4TF | |
Contact: Iain Horrocks 44 (0)141 451 6527 iain.horrocks@ggc.scot.nhs.uk | |
Principal Investigator: Iain Horrocks, MD | |
Leeds Teaching Hospitals NHS Trust | Recruiting |
Leeds, United Kingdom, LS1 3EX | |
Contact: Heather Rostron | |
Principal Investigator: Ann-Marie Childs, MD | |
Alder Hey Childrens Hospital | Recruiting |
Liverpool, United Kingdom, L12 2AP | |
Contact: Timothy Henderson +441512525164 Timothy.Henderson@alderhey.nhs.uk | |
Principal Investigator: Stefan Spinty, MD | |
Great Ormond Street Hospital (GOSH) | Recruiting |
London, United Kingdom, WC1N 1EH | |
Contact: Hinal Patel 020 7905 2639 ext 2639 hinal.patel@ucl.ac.uk | |
Principal Investigator: Francesco Muntoni, MD | |
Royal Victoria Infirmary | Recruiting |
Newcastle upon Tyne, United Kingdom, NE1 4LP | |
Contact: Valerie Corrall +4401912084569 valerie.corrall@ncl.ac.uk | |
Principal Investigator: Volker Straub, MD | |
John Radcliffe Hospital | Recruiting |
Oxford, United Kingdom, OX3 9DU | |
Contact: Sithara Ramdas 44 (0) 1865234188 sithara.ramdas@ouh.nhs.uk | |
Principal Investigator: Sithara Ramdas, MD |
Study Director: | Medical Director | Sarepta Therapeutics, Inc. |
Responsible Party: | Sarepta Therapeutics, Inc. |
ClinicalTrials.gov Identifier: | NCT02500381 |
Other Study ID Numbers: |
4045-301 |
First Posted: | July 16, 2015 Key Record Dates |
Last Update Posted: | March 15, 2022 |
Last Verified: | March 2022 |
Individual Participant Data (IPD) Sharing Statement: | |
Plan to Share IPD: | Undecided |
Studies a U.S. FDA-regulated Drug Product: | Yes |
Studies a U.S. FDA-regulated Device Product: | No |
Duchenne muscular dystrophy Exon Skipping DMD Exon 53 |
Exon 45 Ambulatory Pediatric Duchenne |
Muscular Dystrophies Muscular Dystrophy, Duchenne Muscular Disorders, Atrophic Muscular Diseases Musculoskeletal Diseases |
Neuromuscular Diseases Nervous System Diseases Genetic Diseases, Inborn Genetic Diseases, X-Linked |