welcome to DuchenneXchange- a positively charged Duchenne muscular dystrophy community.
- join today!
- log in
The Expanded Access Use of Viltolarsen in Duchenne Muscular Dystrophy With Confirmed Exon 53 Amenable Mutation
study id #: NCT04337112
condition: Muscular Dystrophy, Duchenne; DMD
status: approved for marketingpurpose:
This is an open label expanded access program for boys, 3 to 12 years old, for the treatment of Duchenne muscular dystrophy (DMD) with confirmed mutation(s) in the dystrophin gene that is amenable to skipping of exon 53.
mechanism of action: Exon-skipping to promote dystrophin production
last updated: August 27, 2020
phase of development: N/A
This expanded access program is designed to provide access to viltolarsen in patients with DMD with confirmed mutation(s) in the dystrophin gene amenable to skipping of exon 53, who in the opinion and clinical judgement of the treating physician, would benefit from treatment with viltolarsen.
• Eligible Sexes: male
• Clinical signs compatible with DMD
• Confirmed DMD mutation(s) in the dystrophin gene that is amenable to skipping of exon 53 to restore the dystrophin messenger ribonucleic acid (mRNA) reading frame
• Able to walk independently without assistive device
• Not able to participate in a Phase 3 trial
• Chronic systemic fungal or viral infections
• An acute illness within 4 weeks prior to the first dose of viltolarsen
• Symptomatic cardiomyopathy
• Patient has a previous or ongoing medical condition, medical history, physical findings, or laboratory abnormality that could affect participant safety in the opinion of the treating physician
• Surgery within the 3 months prior to the first anticipated administration of viltolarsen and in the opinion of the treating physician would impact weekly treatment schedule
• Positive test results for hepatitis B antigen, hepatitis C antibody, or human immunodeficiency virus (HIV) antibody at screening
• Currently taking any other investigational drug or has taken any other investigational drug within 3 months prior to the first dose of viltolarsen
• Previously enrollment in any viltolarsen study.
• Currently taking any other exon skipping agent or has taken any other exon skipping agent within 2 weeks prior to the first dose of viltolarsen (would need to be discontinued in order to be eligible)
• Any gene therapy for DMD
• Inadequate renal function as defined by a serum cystatin C > 1.5 x upper limit of normal (ULN). If the value is > 1.5 x ULN then the measurement can be repeated once. If repeat measurement is still > 1.5 x ULN then the patient should be excluded.
Study to Assess the Efficacy and Safety of Viltolarsen in Ambulant Boys With DMDThe main objective of this study is to e...
CRD007 for the Treatment of Duchenne Muscular Dystrophy, Becker Muscular Dystrophy and Symptomatic CarriersThis is an investigation of the efficacy...
Cardiac and Skeletal Muscle Effects in the Randomized HOPE-Duchenne TrialObjective: To assess the feasibility, s...
A Phase 3 Trial of Pamrevlumab or Placebo in Combination With Systemic Corticosteroids, in Subjects With Non-ambulat...To evaluate the efficacy and safety of p...
Transplantation of Myoblasts to Duchenne Muscular Dystrophy (DMD) PatientsThis Phase I/II of the clinical trial is...
Capricor to Meet with FDA under its RMAT Designation to Discuss HOPE-2 Clinical TrialCapricor Therapeutics announced today th...
A Phase IIa Study of TAS-205 for Duchenne Muscular DystrophyThe objective of this study is to evalua...
FDA grants RASRx1902 orphan drug status as potential therapy for Duchenne muscular dystrophyThe U.S. Food and Drug Administration ha...
FibroGen Receives Orphan Drug Designation from the U.S. FDA For Pamrevlumab for the Treatment of Duchenne Muscular D...FibroGen, Inc., a leading biopharmaceu...
Capricor Announces Positive Outcomes from Comprehensive Multidisciplinary Meeting with FDACapricor Therapeutics, a clinical-stag...