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REGN2477

FOP is caused by a mutation in a gene called “ACVR1” which encodes for the receptor protein “ACVR1/ALK2”. In healthy individuals, a protein called Activin A usually turns this receptor “off”. However, in FOP, Activin A turns the mutant receptor “on”, resulting in the abnormal bone growth (Heterotopic Ossification, HO) that is characteristic of FOP. REGN2477 is an antibody that binds (ie. holds onto) to Activin A, and blocks its activity. By binding and blocking Activin A, REN2477 may prevent the formation and stop the growth of HO in FOP. REGN2477 has received Orphan Drug status for FOP from the U.S. Food and Drug Administration (FDA), and orphan status for the treatment of FOP in the EU.

About the Clinical Trial

The REGN2477
Phase 2
Phase 2: Studies that further test safety and begin to test the effectiveness of the drug, often at a variety of doses.
trial is a global, multi-center,
placebo
Placebo (control): An inactive product that resembles the test medicine or procedure, but without a treatment value. A placebo can be a pill, a powder, or a solution that resembles the test drug, but has no physical effect.  
-controlled trial. Up to 40 adults 18 years of age or older with the classic FOP mutation (R206H) will be enrolled. The trial is designed to evaluate the safety of REGN2477, and whether monthly dosing will reduce the formation of new HO and reduce the signs and symptoms of flare-ups.

Eligibility Criteria Icon
Eligibility Criteria*
  • AGE: ≥ 18
  • DISEASE ACTIVITY: Participants need to have had FOP disease activity within one year of the screening visit.
  • MUTATIONS: R206H
Study Design Icon
Study Design*
  • STUDY TYPE:  Interventional
  • RANDOMIZED STUDY:  Yes
  • PLACEBO-CONTROLLED:  Yes: for the first 6 months, participants will be given either REGN2477 (50% of participants) or
    Placebo
    Placebo (control): An inactive product that resembles the test medicine or procedure, but without a treatment value. A placebo can be a pill, a powder, or a solution that resembles the test drug, but has no physical effect.  
    (50% of participants). At the end of that period, all participants (100%) will receive REGN2477 for an additional 6 months.
  • LENGTH OF PARTICIPATION: 76 weeks
Status Icon
Status

Phase 2
Phase 2: Studies that further test safety and begin to test the effectiveness of the drug, often at a variety of doses.  
, Active, enrollment complete”

Therapy Approach Icon
Therapy Approach

Blocking Antibody

Study Sponsor Icon
Study Sponsor

Regeneron Pharmaceuticals

*For the complete list of eligibility criteria and details on this study, visit ClinicalTrials.gov.

Participating Clinical Sites

Mayo Clinic
Rochester, Minnesota, 55905
Contact: Robert Pignolo
Principal Investigator: Robert Pignolo, MD, PhD

Istituto Giannina, Department of Pediatrics
Genova, Liguria, Italy, 16148
Contact: Maja Di Rocco, MD
Principal Investigator: Maja Di Rocco, MD

VU University Medical Center
Amsterdam, The Netherlands, 1081 HV
Contact: Marelise Eekhoff, MD, PhD
Principal Investigator: Marelise Eekhoff, MD, PhD

Royal National Orthopaedic Hospital, Brockely Hill
Stanmore, United Kingdom, HA7 4LP
Contact: Richard Keen, MD
Contact: Jackie Vinton
Principal Investigator: Richard Keen, MD

University of Pennsylvania
Philadelphia, Pennsylvania, United States, 19104
Contact: Katherine S. Toder, CCRC
Principal Investigator: Mona Al Mukaddam, MD, MS

Vanderbilt University
Nashville, Tennessee, United States, 37240
Principal Investigator: Katherin Dahir, MD

Hospital Universitario San Ignacio
Bogotá, Cundinamarca, Colombia, 110231

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