Phase 2 study of Pamrevlumab (FG-3019) in people with Duchenne

An Investigational Therapeutic Monoclonal Antibody to inhibit the activity of Connective Tissue Growth Factor (CTGF)

Questions and Answers

What stage is this research?

This trial is active, but no longer recruiting.

What is the goal or purpose of this study?

This is a Phase 2, open-label, single arm trial of pamrevlumab (FG-3019) to estimate its efficacy in non-ambulatory patients with Duchenne muscular dystrophy. The rationale for using pamrevlumab in patients with Duchenne is based on data that show that CTGF promotes muscle fibrosis and reduces the ability of damaged muscle cells to repair. Pamrevlumab binds to CTGF and may prevent this cascade. In a preclinical study using an mdx mouse model, pamrevlumab reduced muscle fibrosis and improved muscle function. These data suggest that treatment with pamrevlumab may slow the loss of muscle function. The use of pamrevlumab is investigational in this study. Pamrevlumab is not an FDA-approved drug for any indication and its efficacy and safety have not been demonstrated yet.

Who is sponsoring this study?

This study is sponsored by FibroGen, Inc., a clinical stage biopharmaceutical company (www.fibrogen.com).

Who is eligible to participate in this study?

To participate in this study you must be age 12 years or older with Duchenne, and non-ambulatory (wheelchair dependent). Please see ClinicalTrials.gov (NCT 02606136) for additional inclusion and exclusion criteria.

What do I have to do if I decide to participate in this study?

Each eligible participant will receive pamrevlumab every two weeks by intravenous infusion for up to 3 years. All participants will be closely monitored for safety. Efficacy assessments will be performed routinely; pulmonary and muscle function tests approximately every 3 months, MRIs approximately once a year. This study also includes collection of quality of life data in a questionnaire.

How long will this study last?

The anticipated length of the study is approximately 3 years.

Where does this study take place?

This study is ongoing at several sites across the United States: Cincinnati Children’s in Cincinnati, OH; Washington University in St. Louis, MO; UCSF Benioff Children’s Hospital in San Francisco, CA; Children’s Hospital Colorado in Aurora, CO; Rare Disease Research in Atlanta, GA; Children’s Hospital of Philadelphia in Philadelphia, PA; Children’s Hospital Boston in Boston, MA; Shriner’s Hospital for Children in Portland, OR; University of California, Los Angeles in Los Angeles, CA; Children’s Medical Center Dallas in Dallas, TX.

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