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Study of SRP-4045 and SRP-4053 in DMD Patients (ESSENCE)

This study is currently recruiting participants.
See Contacts and Locations
Verified September 2017 by Sarepta Therapeutics
Information provided by (Responsible Party):
Sarepta Therapeutics Identifier:
First received: July 14, 2015
Last updated: September 11, 2017
Last verified: September 2017
The main objective of this study is to evaluate the efficacy of SRP-4045 and SRP-4053 compared to placebo in Duchenne muscular dystrophy (DMD) patients with out-of-frame deletion mutations amenable to skipping exon 45 and exon 53 respectively. Additional objectives include evaluation of safety, pharmacokinetics and biomarkers.

Condition Intervention Phase
Duchenne Muscular Dystrophy DMD Muscular Dystrophy Drug: SRP-4045 Drug: SRP-4053 Drug: Placebo Phase 3

Study Type: Interventional
Study Design: Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
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

Resource links provided by NLM:

Further study details as provided by Sarepta Therapeutics:

Primary Outcome Measures:
  • Change in 6 Minute Walk Test (6MWT) from Baseline [ Time Frame: Baseline to Week 96 ]

Secondary Outcome Measures:
  • Dystrophin protein expression [ Time Frame: Baseline to Week 48 ]
  • Ability to rise independently from the floor [ Time Frame: Baseline to Week 96 ]
  • Functional status as measured by loss of ambulation (LOA) from Baseline [ Time Frame: Baseline to Week 96 ]
  • North Star Ambulatory Assessment (NSAA) from Baseline [ Time Frame: Baseline to Week 96 ]
  • Forced vital capacity (FVC)% predicted from Baseline [ Time Frame: Baseline to Week 96 ]
  • Frequency of falls from Baseline [ Time Frame: Baseline to Week 96 ]
  • Left ventricular ejection fraction (LVEF) from baseline [ Time Frame: Baseline to Week 96 ]

Estimated Enrollment: 99
Actual Study Start Date: August 2016
Estimated Study Completion Date: June 2021
Estimated Primary Completion Date: September 2019 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: SRP-4045
A planned minimum of 33 DMD patients with deletion mutations amenable to exon 45 skipping will be randomized to the active SRP-4045 group.
Drug: SRP-4045

SRP-4045 30 mg/kg will be administered as an IV infusion once a week for up to 96 weeks.

In the Open-Label Period, all patients will receive IV infusions of SRP-4045 at 30 mg/kg/week for up to 96 weeks.

Experimental: SRP-4053
A planned minimum of 33 DMD patients with deletion mutations amenable to exon 53 skipping will be randomized to the active SRP-4053 group.
Drug: SRP-4053

SRP-4053 30 mg/kg will be administered as an IV infusion once a week for up to 96 weeks.

In the Open-Label Period, all patients will receive IV infusions of SRP-4053 at 30 mg/kg/week for up to 96 weeks.

Placebo Comparator: Placebo
Approximately 33 DMD patients with deletion mutations amenable to exon 45 or exon 53 skipping will be randomized to the placebo group.
Drug: Placebo

Placebo will be administered as an IV infusion once a week for up to 96 weeks.

In the Open-Label Period, all patients will receive IV infusions of either SRP-4045 or SRP-4053 at 30 mg/kg/week, according to genotype, for up to 96 weeks.

Detailed Description:

This is a double-blind, placebo-controlled, multi-center study to evaluate the efficacy and safety of SRP-4045 and SRP-4053. Eligible patients 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 mg/kg SRP-4045 or 30 mg/kg SRP-4053 respectively (combined-active group, 66 patients) or placebo (33 patients) 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 patients will receive open-label active treatment for up to 96 weeks.

The study will enroll approximately 99 patients, with a planned minimum target of 45 patients amenable to exon 45 skipping and 45 patients amenable to exon 53 skipping.

Approximately 66 patients will be randomized to receive active treatment with either SRP-4045 or SRP-4053 (depending on deletion mutation), and 33 patients will be randomized to receive placebo. Twice as many patients will receive active treatment as will receive placebo (2:1 randomization).

When approximately 75 patients have been in the trial for 48 weeks a group of independent experts will review key study results and will make a determination on whether patients will roll into the open-label period of the study and receive active drug or continue in the placebo-controlled period out to Week 96 as planned.

Clinical efficacy will be assessed at regularly scheduled study visits, including functional tests such as the six-minute walk test (6MWT). All patients will undergo a muscle biopsy at baseline and a second muscle biopsy at Week 48.

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.


Ages Eligible for Study:   7 Years to 13 Years   (Child)
Sexes Eligible for Study:   Male
Accepts Healthy Volunteers:   No

Inclusion Criteria:

  • Male 7-13 years old
  • Genotypically confirmed DMD, with genetic deletion amenable to exon 45 or exon 53 skipping
  • Stable dose of corticosteroids for at least 6 months
  • Intact right and left biceps or 2 alternative upper muscle groups
  • Mean 6MWT greater than or equal 300 meters and less than or equal to 450 meters
  • Stable pulmonary and cardiac function: forced vital capacity (FVC) equal to or greater than 50% predicted and left ventricular ejection fraction (LVEF) greater than 50%

Exclusion Criteria:

  • Previous treatment with the following experimental compounds: SMT C1100 (BMN-195) at any time
  • Previous treatment with PRO045 or PRO053 within 6 months prior to Week 1
  • Current or previous treatment with any other experimental treatment (other than deflazacort) within 12 weeks prior to Week 1
  • Participation in any other DMD interventional clinical study within 12 weeks prior to Week 1
  • Major surgery within 3 months prior to Week 1
  • Presence of other clinically significant illness
  • Major change in physical therapy regimen within 3 months prior to Week 1
  Contacts and Locations
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, see Learn About Clinical Studies.

Please refer to this study by its identifier: NCT02500381

Contact: Medical Information

  Hide Study Locations
United States, Arizona
Neuromuscular Research Center Recruiting
Phoenix, Arizona, United States, 85028
Contact: Kristy Osgood    480-314-1007   
Principal Investigator: Kumaraswamy Sivakumar, MD         
United States, California
Children's Hospital Los Angeles Recruiting
Los Angeles, California, United States, 90027
Contact: Claudia Dozal    323-361-5825   
Principal Investigator: Leigh Maria Ramos-Platt, MD         
David Geffen School of Medicine, UCLA Recruiting
Los Angeles, California, United States, 90095
Contact: Melody Badii    310-825-3264   
Principal Investigator: Perry Shieh, MD, PhD         
UC Davis Medical Center Recruiting
Sacramento, California, United States, 95817
Contact: Erica Goude    916-734-0968   
Principal Investigator: Craig McDonald, MD         
Stanford University School of Medicine/Medical Center Recruiting
Stanford, California, United States, 94305
Contact: Carolyn Mclaughlin    650-206-3178   
Principal Investigator: John Day, MD         
United States, Florida
University of Florida Recruiting
Gainesville, Florida, United States, 32610
Contact: Stephanie Salabarria    352-273-6582   
Principal Investigator: Barry Byrne, MD, PhD         
NW Florida Clinical Research Group, LLC Recruiting
Gulf Breeze, Florida, United States, 32561
Contact: Shae Lancelin   
Principal Investigator: Ben Renfroe, MD         
Miami, Florida, United States
United States, Georgia
Center for Integrative Rare Disease Research (CIRDR) Recruiting
Atlanta, Georgia, United States, 30318
Contact: Krystal Reese    404-829-2380   
Principal Investigator: Han Phan, MD         
United States, Illinois
Ann and Robert H. Lurie Children's Hospital of Chicago Recruiting
Chicago, Illinois, United States, 60611
Contact: Theresa Oswald    312-227-4483   
Principal Investigator: Nancy Kuntz, MD         
United States, Iowa
University of Iowa Children's Hospital Recruiting
Iowa City, Iowa, United States, 52242
Contact: Carrie Stephan    319-356-2673   
Principal Investigator: Katherine Mathews, MD         
United States, Kansas
University of Kansas, Medical Center Recruiting
Kansas City, Kansas, United States, 66160
Contact: Kiley Sims   
Principal Investigator: Jeffery Statland, MD         
United States, Massachusetts
Boston Children's Hospital Recruiting
Boston, Massachusetts, United States, 02115
Contact: Timothy Harrington    857-218-4677   
Principal Investigator: Basil Darras, MD         
United States, Missouri
St. Louis Children's Hospital Recruiting
Saint Louis, Missouri, United States, 63110
Contact: Traci Christenson    314-362-6991   
Principal Investigator: Ann M Connolly, MD         
United States, Nevada
Las Vegas Clinic Recruiting
Las Vegas, Nevada, United States, 89145
Contact: Kaitlyn McKenna    702-505-4230   
Principal Investigator: Jonathan McKinnon, MD         
United States, New York
Not yet recruiting
New York, New York, United States
University of Rochester Clinical Research Center Recruiting
Rochester, New York, United States, 14642
Contact: Patricia Smith    585-275-4339   
Principal Investigator: Emma Ciafaloni, MD         
United States, Ohio
Cincinnati Children's Hospital Medical Center (CCHMC) Recruiting
Cincinnati, Ohio, United States, 45229
Contact: Paula Casson    513-803-9003   
Principal Investigator: John Jefferies, MD         
Nationwide Children's Hospital Recruiting
Columbus, Ohio, United States, 43205
Contact: Lisa Moffitt    614-722-8528   
Principal Investigator: Jerry R Mendell, MD         
United States, Oregon
Shriners Hospital for Children Recruiting
Portland, Oregon, United States, 97239
Contact: Cathleen Buckon, OTR/L, MS    503-221-3471   
Principal Investigator: Erika Finanger, MD, MS         
United States, Pennsylvania
Children's Hospital of Pittsburgh of UPMC Recruiting
Pittsburgh, Pennsylvania, United States, 15224
Contact: Jennifer M Monahan    412-692-5176   
Principal Investigator: Hoda Z Abdel-Hamid, MD         
United States, Texas
Children's Medical Center Dallas Recruiting
Dallas, Texas, United States, 75235
Contact: Holly Lawrence    214-456-2463   
Principal Investigator: Susan Iannaccone, MD         
United States, Utah
University of Utah Recruiting
Salt Lake City, Utah, United States, 84132
Contact: Bria Jensen    801-585-9399   
Principal Investigator: Russell Butterfield, MD, PhD         
United States, Virginia
Children's Hospital of the King's Daughters Recruiting
Norfolk, Virginia, United States, 23507
Contact: Terrie Conklin    757-668-9356   
Principal Investigator: Crystal Proud, MD         
Universitair Ziekenhuis Gent Recruiting
Ghent, Belgium, 9000
Contact: Elke De Vos    +3293321954   
Principal Investigator: Nicolas Deconinck, MD         
Universitair Ziekenhuis Leuven Recruiting
Leuven, Belgium, 3000
Contact: Anne Vanden Eynden    +3216343991   
Principal Investigator: Nathalie Goemans, MD         
Not yet recruiting
TBD, Canada
Not yet recruiting
Nantes, France
Hôpital Armand Trousseau Recruiting
Paris, France, 75012
Contact: Dominique Duchêne    +33171738313   
Principal Investigator: Laurent Servais, MD, PhD         
Not yet recruiting
Toulouse, France
Universitätsklinikum Essen Recruiting
Essen, Germany, 45122
Contact: Corinna Seifert    +4920172385170   
Principal Investigator: Ulrike Schara, MD         
University Hospital Freiburg Recruiting
Freiburg, Germany, 79106
Contact: Sabine Wider    +4976127043440   
Principal Investigator: Janbernd Kirschner, MD         
LMU Klinikum der Universität München Recruiting
Munich, Germany, 80337
Contact: Astrid Blaschek    +4989440055110   
Principal Investigator: Wolfgang Müller-Felber, MD         
Azienda Ospedaliero-Universitaria di Ferrara - Arcispedale Sant' Anna Recruiting
Ferrara, Italy, 44124
Contact: Fernanda Fortunato    +393465374262   
Principal Investigator: Alessanda Ferlini, MD, PhD         
Istituto Giannina Gaslini Recruiting
Genoa, Italy, 16147
Contact: Marina Pedemonte, MD    +3901056362675   
Principal Investigator: Claudio Bruno, MD         
Az Ospedaliera Universitaria Policlinico G Martino Recruiting
Messina, Italy, 98125
Contact: Daniela Quattrocchi    +393493500123   
Principal Investigator: Giuseppe Vita, MD         
Policlinico Universitario A Gemelli Recruiting
Rome, Italy, 00168
Contact: Mariarosaria Vizzino    +390630158581   
Principal Investigator: Eugenio Mercuri, MD         
Hospital de La Santa Creu i Sant Pau Recruiting
Barcelona, Spain, 08041
Contact: Nuria Vidal    +34935537115   
Principal Investigator: Jorge Diaz Manera, MD         
Hospital Sant Joan de Deu Recruiting
Barcelona, Spain, 08950
Contact: Marina Garcia    +34936009733   
Principal Investigator: Andres Nascimento, MD         
Drottning Silvias Barn Och Ungdomssjukhus Recruiting
Göteborg, Sweden, SE-41685
Contact: Anna-Lena Tulinius    +46313436069   
Principal Investigator: Mar Tulinius, MD         
Not yet recruiting
Stockholm, Sweden
United Kingdom
Leeds Teaching Hospitals NHS Trust Recruiting
Leeds, United Kingdom, LS1 3EX
Contact: Heather Rostron    +441133922344   
Principal Investigator: Ann-Marie Childs, MD         
Alder Hey Childrens Hospital Recruiting
Liverpool, United Kingdom, L12 2AP
Contact: Timothy Henderson    +441512525164   
Principal Investigator: Stefan Spinty, MD         
Great Ormond Street Hospital (GOSH) Recruiting
London, United Kingdom, WC1N 1EH
Contact: Hinal Patel    020 7905 2639 ext 2639   
Principal Investigator: Francesco Muntoni, MD         
Royal Victoria Infirmary Recruiting
Newcastle upon Tyne, United Kingdom, NE1 4LP
Contact: Valerie Corrall    +4401912084569   
Principal Investigator: Volker Straub, MD         
Sponsors and Collaborators
Sarepta Therapeutics
Study Director: Jon Lu, MD, PhD Sarepta Therapeutics
Study Director: Catherine Stehman-Breen, MD, MS Sarepta Therapeutics
  More Information

Responsible Party: Sarepta Therapeutics Identifier: NCT02500381     History of Changes
Other Study ID Numbers: 4045-301
Study First Received: July 14, 2015
Last Updated: September 11, 2017
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Undecided

Additional relevant MeSH terms:
Muscular Dystrophies
Muscular Dystrophy, Duchenne
Muscular Disorders, Atrophic
Muscular Diseases
Musculoskeletal Diseases
Neuromuscular Diseases
Nervous System Diseases
Genetic Diseases, Inborn
Genetic Diseases, X-Linked processed this record on September 21, 2017