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


Frequently Asked Questions (FAQs) about this study:


What stage is this research?

  • This trial is actively recruiting participants.


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?


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 (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 52 weeks witht the possibility to add an additional 26 weeks of treatment with prior FibroGen medical monitor approval. All participants will be closely monitored for safety. Efficacy assessments will be performed routinely over 54 weeks and up to 80 weeks (for participants that qualify for additional treatemen); 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 approxmiately 52 weeks with the possibility to add an additional 26 weeks of treatment for up to a total of 78 weeks with FibroGen medical monitor approval.


Where does this study take place?

  • This study is open 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; University of Iowa in Iowa City, IA; 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.
  • Please refer to the study listing on (NCT02606136) for the most current list of open sites.


How many visits to the study site are necessary?

  • Approximately 34 visits over 56 weeks and up to 47 visits over 82 weeks  with FibroGen medical monitor approval.


Can any visits be done locally?

  • No, all study visits must be conducted at the participating clinical center.


Is there any funding to help pay for travel?

  • Yes, participants are eligible for reimbursement for some costs related to travel to/from study visits or overnight lodging. The travel reimbursement program for this study is being facilitated by NORD (National Organization for Rare Diseases) and information is available from each site’s study coordinator.


Why should I consider participating in this study?

  • While no personal benefit can ever be guaranteed by participation in a clinical trial, there are other benefits, including allowing you to play an active role in your own health care (or that of your child), gaining access to new investigational research treatments, having access to medical specialists that may not normally be available to you or your child, and helping others by contributing to the better understanding of Duchenne. While we do not yet know if there is any clinical benefit of pamrevlumab in Duchenne patients, it is possible that pamrevlumab treatment may slow the loss of muscle function.


Where can I learn more about this study?


Posted 5/4/2017