Clinical Trials

CLINICAL TRIALS:
THE BASICS

Before an investigational drug can be approved, it must be proven safe for people to use and effective for treating a medical condition. A clinical trial is a type of research study that can help provide that proof.

 
 
 
 

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

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)

For more information and enrollment status, please visit www.clinicaltrials.gov.
ESSENCE Study Brochure (PDF download)

4658-301 (PROMOVI): Phase III Study


Official Title: An Open-Label, Multi-Center, 48-Week 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 confirmatory 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 and enrollment status, 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 and enrollment status, 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 and enrollment status, please visit www.clinicaltrials.gov.

4658-us-202: Phase II Study


Official Title: An Open-Label, Multiple-Dose, Efficacy, Safety, and Tolerability Study of Eteplirsen in Subjects With Duchenne Muscular Dystrophy Who Participated in Study 4658-US-201

Purpose: The primary objective of this study is to assess the ongoing efficacy, safety, and tolerability of an additional 212 weeks of treatment with eteplirsen injection in Duchenne muscular dystrophy (DMD) subjects who have successfully completed the 28 week eteplirsen study: Study 4658-us-201. This study will also evaluate the correlation between biomarkers for DMD and the clinical status of participating DMD subjects.

Location: United States (multiple sites)

For more information and enrollment status, 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 and enrollment status, 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 and enrollment status, please visit www.clinicaltrials.gov.