A Study to Compare Safety and Efficacy of a High Dose of Eteplirsen in Participants With Duchenne Muscular Dystrophy (DMD) (MIS51ON)
Muscular Dystrophy, Duchenne
About this trial
This is an interventional treatment trial for Muscular Dystrophy, Duchenne focused on measuring Duchenne muscular dystrophy, Exon Skipping, Exon 51, North Star Ambulatory Assessment, Ambulatory, DMD, Pediatric, Duchenne, EXONDYS, MIS51ON
Eligibility Criteria
Inclusion Criteria:
- Be a male with an established clinical diagnosis of DMD and an out-of-frame deletion mutation of the DMD gene amenable to exon 51 skipping.
- Ambulatory participant, able to perform TTRISE in 10 seconds or less at the time of screening visit.
- Able to walk independently without assistive devices.
- Have intact right and left biceps muscles or an alternative upper arm muscle group.
- Have been on a stable dose or dose equivalent of oral corticosteroids for at least 12 weeks prior to randomization and the dose is expected to remain constant (except for modifications to accommodate changes in weight and stress-related needs as per the recently published guidelines throughout the study.
- For ages 7 years and older, has stable pulmonary function (forced vital capacity ≥50 percent (%) of predicted and no requirement for nocturnal ventilation). For ages 4 to 6 years, does not require support from ventilator or non-invasive ventilation at time of screening.
Exclusion Criteria:
- Use of any pharmacologic treatment (other than corticosteroids) within 12 weeks prior to randomization.
- Current or previous treatment with any other experimental pharmacologic treatment for DMD or any prior exposure to antisense oligonucleotide, gene therapy or gene editing; except the following: Ezutromid in the last 12 weeks prior to first dose; Drisapersen in the last 36 weeks prior to first dose; Suvodirsen in the last 12 weeks prior to first dose; Vamorolone in the last 12 weeks prior to first dose; and Eteplirsen (previous or current use).
- Major surgery within 3 months prior to randomization.
- Presence of any other significant neuromuscular or genetic disease other than DMD.
- Presence of any known impairment of renal function and/or other clinically significant illness.
- Has evidence of cardiomyopathy, as defined by left ventricular ejection fraction less than <50% on the screening echocardiogram or Fridericia's correction formula (QTcF) ≥450 millisecond based on the screening electrocardiograms (ECGs).
Other inclusion/exclusion criteria apply.
Sites / Locations
- University of Alabama at BirminghamRecruiting
- University of FloridaRecruiting
- Rare Disease Research, LLCRecruiting
- Children's Hospital - London Health Sciences Centre
- Children's Hospital of Eastern Ontario
- Hospital Universitario San IgnacioRecruiting
- Instituto Neurologico de Colombia (INDEC)Recruiting
- Hospital Pablo Tobón UribeRecruiting
- Brno Klinika detske neurologieRecruiting
- Fakultni nemocnice v MotoleRecruiting
- Rigshospitalet Copenhagen University HospitalRecruiting
- Hopital Femme Mere EnfantRecruiting
- Hopital Armand TrousseauRecruiting
- CHRU de StrasbourgRecruiting
- Charité Universitätsmedizin Berlin CVKRecruiting
- Universitätsklinikum EssenRecruiting
- Universitätsklinikum FreiburgRecruiting
- IASO Children's HospitalRecruiting
- Semmelweis Egyetem Genomikai Medicina és Ritka Betegsegek IntezeteRecruiting
- Royal Institute of Child NeurosciencesRecruiting
- Aster RV HospitalRecruiting
- Nizam's Institute of Medical SciencesRecruiting
- Jaicare Hospital (A Unit of Sarvee Integra Pvt Ltd.)Recruiting
- All India Institute of Medical SciencesRecruiting
- Sir Ganga Ram HospitalRecruiting
- Deenanath Mangeshkar Hospital & Research CentreRecruiting
- Christian Medical CollegeRecruiting
- Children's Health Ireland (CHI) at Temple StreetRecruiting
- IRCCS Instituto Gianna GasliniRecruiting
- The Specialty Hospital (TSH)/Advanced Clinical CenterRecruiting
- Istiklal Hosptial (IST)Recruiting
- Irbid Specialty HospitalRecruiting
- Pharmaceutical Research Center/Jordan University of Science and TechnologyRecruiting
- Kyungpook National University Chilgok HospitalRecruiting
- Seoul National University HospitalRecruiting
- Samsung Medical CenterRecruiting
- Pusan National University Yangsan HospitalRecruiting
- Neurociencias Estudios Clínicos S.C.Recruiting
- Instituto de Investigaciones Clinicas para la Salud A.CRecruiting
- Leids Universitair Medisch CentrumRecruiting
- Radboud University Nijmegen Medical CentreRecruiting
- New Zealand Clinical Research - AucklandRecruiting
- Oslo Universitetssykehus HF RikshospitaletRecruiting
- Children's Department and Department for Children's Habilitation at Stavanger University HospitalRecruiting
- Klinika Neurologii RozwojowejRecruiting
- National Clinical Hospital for Children Neurorehabilitation "Dr. Nicolae Robănescu"Recruiting
- Clinic for Neurology and Psychiatry for Children and YouthRecruiting
- University Children's HospitalRecruiting
- Mother and Child Health Care Institute of Serbia "Dr Vukan Cupic"Recruiting
- University Medical Centre LjubljanaRecruiting
- Hospital Sant Joan de DeuRecruiting
- Hospital Universitari i Politecnic La Fe de ValenciaRecruiting
- Universitätsspital BaselRecruiting
- Kaohsiung Medical UniversityRecruiting
- National Taiwan University HospitalRecruiting
- Akdeniz Universitesi Tip FakultesiRecruiting
- Mersin University Medical FacultyRecruiting
- Leeds Teaching Hospitals NHS TrustRecruiting
- Birmingham Heartlands HospitalRecruiting
- UCL Institute of Child Health Great Ormond StreetRecruiting
Arms of the Study
Arm 1
Arm 2
Arm 3
Arm 4
Experimental
Active Comparator
Experimental
Experimental
Part 1: Eteplirsen
Part 2: Eteplirsen 30 mg/kg
Part 2: Eteplirsen 100 mg/kg
Part 2: Eteplirsen 200 mg/kg
Participants will receive eteplirsen 100 mg/kg once weekly for at least 4 weeks, followed by eteplirsen 200 mg/kg once weekly for at least 4 weeks.
Randomized participants will receive eteplirsen 30 mg/kg once weekly for up to 144 weeks.
Randomized participants will receive eteplirsen 100 mg/kg once weekly before the selection of the high dose occurs and then will receive the selected high dose once weekly for up to 144 weeks.
Randomized participants will receive eteplirsen 200 mg/kg once weekly before the selection of the high dose occurs and then will receive the selected high dose once weekly for up to 144 weeks.