FibroGen's MissionDMD Trial Now Recruiting


This clinical trial is MissionDMD, as the sponsor company (FibroGen, Inc) is on a mission to develop safe and effective treatment options for those affected by Duchenne Muscular Dystrophy. This is a phase 2 trial designed to evaluate the safety, efficacy, tolerability, pharmacokinetics and pharmacodynamics of FG-3019 in Duchenne. FibroGen is fully supporting this trial and the clinical development of FG-3019.

Some of the enrollment criteria required for participating include:

  • Males at least 12 years of age diagnosed with Duchenne
  • Non-ambulatory; wheelchair dependent for less than 5 years
  • Corticosteroid use for at least 6 months prior to screening

Full eligibility requirements for this study will be assessed at participating study sites. For more information on additional enrollment criteria, please visit (NCT02606136).

Participants will receive FG-3019 intravenously every two weeks for up to 2 years. All participants will be closely monitored for safety on a continuous basis. Participants will undergo pulmonary function tests (Forced Vital Capacity, mean inspiratory flow, and peak expiratory flow), muscle function tests, and imaging studies (MRI) to evaluate changes in muscle and heart.

While FG-3019 has not been studied in Duchenne, it has been evaluated in other clinical trials with an acceptable safety profile. In a recent publication, FG-3019 was reported to show potentially beneficial effects in patients with idiopathic pulmonary fibrosis.

This study is taking place at several sites across the United States:

  • Boston Children's Hospital - Boston, MA
  • Cincinnati Children's Hospital Medical Center - Cincinnati, OH
  • Children's Hospital of Pittsburgh of UPMC - Pittsburgh, PA
  • UCSF Benioff's Children Hospital - San Francisco, CA
  • David Geffen School of Medicine at UCLA - Los Angeles, CA
  • Washington University in St. Louis School of Medicine - St. Louis, MO
  • Children's Hospital Colorado - Aurora, CO
  • University of Iowa - Iowa City, IA

Reasonable travel and accommodations will be provided or reimbursed.

If you wish to participate or have questions, please visit for site contact information. You may also contact Gustavo Lorente at FibroGen with questions at (415-978-1441) or by email at This email address is being protected from spambots. You need JavaScript enabled to view it..

In addition, if you missed the Parent Project Muscular Dystrophy webinar on the MissionDMD Trial (recorded February 1, 2017), the recording is now available. Click here to listen.

Posted 5/24/2016; Updated 2/3/2017