DOMAGROZUMAB (PF-06252616) - Development of a myostatin inhibitor as a potential therapy for Duchenne Muscular Dystrophy


Frequently Asked Questions (FAQs) about this research:


What stage is this research?

  • This Phase 2 randomized, double-blind, placebo-controlled, multiple ascending dose study to evaluate the safety, efficacy, pharmacokinetics and pharmacodynamics of Domagrozumab (PF-06252616) administered to ambulatory boys diagnosed with Duchenne muscular dystrophy. Enrollment of participants has completed.  
  • Additional details about this study can be found at (NCT#02310763) or
  • Participants completing the Phase 2 study of PF-06252616 may be eligible to enroll in the open-label extension study of Domagrozumab (PF-06252616).  Information about the open-label extension study may be found at  (NCT#02907619).


What is Domagrozumab?

  • Domagrozumab is an investigational drug (not approved for commercial use) designed to block the activity of myostatin. Myostatin is a Glossary Link protein that acts in the body to prevent muscle from growing too large. This drug is being tested to see if blocking myostatin will help to increase muscle strength and function in boys with DMD.


  What are the inclusion (enrollment) criteria for this trial?

  • Participants must be ambulatory boys with Duchenne ages 6 to <16 years old.
  • Eligible participants must also able to perform the 4 stair climb in ≥  0.33 stairs/second ≤1.6 stairs/second (with or without the use of handrails).
  • The diagnosis of Duchenne must be confirmed in the subject's medical history and by genetic testing obtained during routine medical care. Please note: The investigational therapy is not Glossary Link mutation-specific, so boys with any DMD Glossary Link gene mutation may participate.
  • Eligible participants must be on glucocorticosteroids for a minimum of 6 months prior to signing informed consent to join the study.
  • Participants must be able to tolerate MRI scanning.
  • Additional screening evaluations will be conducted at the study site to confirm eligibility.


What happens during the course of the study?

  • Participants receive monthly IV infused doses for up to 2 years at their study site.
  • Based on the design of the study, all participants will receive the investigational drug during half or all of the duration of the study.
  • Participants undergo safety evaluations at regular intervals (including monthly clinical exams and blood tests).
  • Participants undergo functional evaluations (including the 4 stair climb and 6 minute walk test every 2 months and MRI scans to evaluate the leg muscles). 


Where is the trial taking place?

  • Study sites exist in the United States, Canada, Italy, United Kingdom, Poland, Bulgaria, Australia and Japan.  Please check for details regarding the trial sites.


What are the potential benefits and risks of participating in this study?

  • Participation in a clinical study may not provide any personal benefit but may potentially help others by contributing to a better understanding of DMD and to the collective scientific knowledge driving possible therapeutic approaches.


Where can I learn more about this study?


  Is Pfizer committed to pursuing possible treatments for DMD?

  • Our investment in DMD currently extends beyond Domagrozumab. Pfizer is also advancing pre-clinical work with Bamboo Therapeutics on PF-06939926, a mini-dystrophin gene therapy for DMD.
  • Pfizer is an active participant in advocacy/academic/industry consortia


Updated 5/11/2017