Clinical trials are designed to add medical knowledge related to the treatment of diseases and medical conditions, and are a critical part of the drug development process. Typically, three phases of clinical trials are needed to develop a new treatment. Each study is designed to answer specific questions about an investigational drug, including:

  • How does it work in the body?
  • What happens to it in the body?
  • What are the side effects?
  • What is a safe and appropriate dose?
  • Does it change how patients feel, function, or survive?

OUR CLINICAL TRIALS

 

5051-101: Phase 1 Study of SRP-5051 (PPMO for Exon 51)

Official Title:  A Phase 1 Trial to Evaluate the Safety, Tolerability, and Pharmacokinetics of a Single Dose of SRP-5051 in Patients with Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping Treatment

Purpose: This is a first-in-human, open-label study to evaluate the safety, tolerability and pharmacokinetics (PK) of 5 escalating doses of SRP-5051 administered as a single dose to patients with DMD amenable to exon 51 skipping treatment.

Location: United States (multiple sites)

For more information, please visit www.clinicaltrials.gov

 

4045-301 (ESSENCE): Phase III Study

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

Purpose: 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. 

Location: United States (multiple sites), Europe (multiple sites), Canada (multiple sites) , Israel

For more information, please visit www.clinicaltrials.gov or www.essencetrial.com
ESSENCE Study Brochure (PDF download) 

4658-102: Phase II Study in Young DMD Patients

Official Title: An Open-Label Safety, Tolerability, and Pharmacokinetics Study of Eteplirsen in Young Patients with Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping

Purpose: This is a multicenter, open-label, dose-escalation study to evaluate the safety, tolerability, PK, and efficacy of once-weekly IV infusions of eteplirsen in approximately 12 male patients, ages 6 months to 48 months (inclusive), who have genotypically confirmed DMD with a deletion mutation amenable to exon 51 skipping. 

Location: Europe (multiple sites) 

For more information please visit www.clinicaltrials.gov 

4658-301 (PROMOVI): Phase III Study

Official Title: An Open-Label, Multi-Center Study With a Concurrent Untreated Control Arm to Evaluate the Efficacy and Safety of Eteplirsen in Duchenne Muscular Dystrophy

Purpose: The main objective of this study is to provide evidence of efficacy of eteplirsen (AVI-4658) in Duchenne muscular dystrophy (DMD) patients that are amenable to skipping exon 51. Additional objectives include evaluation of safety and biomarkers. 

Location: United States (multiple sites) 

For more information, please visit www.clinicaltrials.gov. 
 

4053-101: Phase I/II Study

Official Title: A 2-Part, Randomized, Double-Blind, Placebo-Controlled, Dose-Titration, Safety, Tolerability, and Pharmacokinetics Study (Part 1) Followed by an Open-Label Efficacy and Safety Evaluation (Part 2) of SRP-4053 in Patients With Duchenne Muscular Dystrophy Amenable to Exon 53 Skipping

Purpose: This is a first-in-human, multiple-dose 2-part study to assess the safety, tolerability, efficacy, and pharmacokinetics of SRP-4053 in Duchenne muscular dystrophy (DMD) patients with deletions amenable to exon 53 skipping. 

Location: Europe (multiple sites) 

For more information, please visit www.clinicaltrials.gov.

4045-101: Phase I/II Study

Official Title: A Randomized, Double-Blind, Placebo-Controlled, Dose-Titration, Safety, Tolerability, and Pharmacokinetics Study Followed by an Open-Label Safety and Efficacy Evaluation of SRP-4045 in Advanced-Stage Patients With Duchenne Muscular Dystrophy Amenable to Exon 45 Skipping

Purpose: The main objective of this first-in-human, dose-titration, and open-label extension study is to assess safety, tolerability, and pharmacokinetics of SRP-4045 in advanced-stage Duchenne muscular dystrophy (DMD) patients with deletions amenable to exon 45 skipping.

Location: United States (multiple sites) 

For more information, please visit www.clinicaltrials.gov. 

4658-203: Phase II Study

Official Title: An Open-Label, Multi-Center Study to Evaluate the Safety, Efficacy and Tolerability of Eteplirsen in Early Stage Duchenne Muscular Dystrophy

Purpose: This is an open-label study to assess the safety, tolerability, efficacy and pharmacokinetics of eteplirsen in patients with early stage Duchenne muscular dystrophy (DMD) who are amenable to exon 51 skipping. 

Location: United States (multiple sites) 

For more information, please visit www.clinicaltrials.gov. 

4658-204: Phase II Study

Official Title: An Open-Label, Multi-Center Study to Evaluate the Safety and Tolerability of Eteplirsen in Patients With Advanced Stage Duchenne Muscular Dystrophy

Purpose: The primary objective of this study is to explore safety and tolerability of eteplirsen in patients with advanced stage Duchenne muscular dystrophy (DMD) who are amenable to exon 51 skipping. The exploratory objectives are to evaluate the effect of eteplirsen on pulmonary function tests (PFTs) and other functional clinical measures. 

Location: United States (multiple sites) 

For more information, please visit www.clinicaltrials.gov.