OPTIMA Trial (garetosmab)

OPTIMA Trial (garetosmab)

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 or HO) that is characteristic of FOP.

REGN2477 (also known as garetosmab) is an antibody that binds (i,e. holds onto) to Activin A, and blocks its activity. By binding and blocking Activin A, garetosmab may prevent the formation and stop the growth of HO in FOP. Garetosmab 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 garetosmab

Phase 3
Phase 3: 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-controlled trial. Approximately 66 adults 18 years of age or older with the classic FOP mutation (R206H) or non-classic mutations will be enrolled. The trial is designed to evaluate the safety of garetosmab, 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 and other mutations of the ACVR1 gene resulting in heterotopic ossification
Study Design Icon
Study Design*
  • STUDY TYPE:  Interventional
  • RANDOMIZED STUDY:  Yes
  • PLACEBO-CONTROLLED:  Yes, participants will be randomized to high-dose garetosmab, low-dose garetosmab, 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.  
  • LENGTH OF PARTICIPATION: 56 weeks
Status Icon
Status

Phase 3,
Phase 3: Studies that further test safety and begin to test the effectiveness of the drug, often at a variety of doses.  
Open, Enrolling

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 and enter Identifier NCT05394116.

Participating Clinical Sites (as of June 4, 2024)

Northern Sydney Local Health District-Royal North Shore Hospital 
Australia
Principal Investigator: Roderick Clifton-Bligh, BSC MB FRACP PhD FFSc

Hospital Israelita Albert Einstein
Brazil
Principal Investigator: Patricia Delai, MD or +55-11-99394-5848

Inmunomedica Concepción 
Chile
Principal Investigator: Erik Iván Quevedo Langenegger, MD

Clinica Universidad de La Sabana (New)
Colombia

Tongji University – Tongji Hospital 
China
Principal Investigator: 
Keqin Zhang, MD, PhD

New Children's Hospital, Helsinki University Hospital
Finland
Principal Investigator: Matti Hero, MD, PhD

Hôpital Lapeyronie
France
Principal Investigator: Christian Jorgensen, MD, PhD

Lariboisière Hospital AP-HP
France
Principal Investigator: Thomas Funck-Brentano, MD, PhD

Queen Mary Hospital 
Hong Kong
Principal Investigator: Evelyn Kuong, MBBS, FHKCOS, FHKAM, FRCSEd

Istituto Giannina Gaslini
Italy
Principal Investigator: Marco Gattorno, MD
Sub Investigator: Riccardo Papa, MD

Kyushu University Hospital 
Japan
Principal Investigator: Dr. Toshifumi Fujiwara

Nagoya University Hospital
Japan
Principal Investigator: Kenichi Mishima, MD

Oita University 
Japan
Principal Investigator: Naoto Uemura, MD, PhD 

Kuala Lumpur Hospital
Malaysia
Principal Investigator: Kavitha Rethnavelu

Amsterdam University Medical Center 
Netherlands
Principal Investigator: Elisabeth Eekhoff, MD, PhD
Contact: FOP Expertise Center Amsterdam or +31 20 444 0588 or Dr. Rosenberg, Sub-PI at +31 20 444 4588

Medyk Medical Center Hospital 
Poland
Principal Investigator: Jacek Tabarkiewicz, MD, PhD

University of Cape Town 
South Africa
Principal Investigator: Dr. Raphaella Stander

Seoul National University Hospital 
South Korea
Principal Investigator: Tae-Joon Cho, MD

Ramon y Cajal University Hospital
Spain
Principal Investigator: Javier Bachiller Corral, MD

Royal National Orthopaedic Hospital NHS Trust
Site is open to international participants -- contact Dr. Keen for additional information
United Kingdom
Principal Investigator: Richard Keen, MD

University of California Los Angeles (UCLA) Medical Center 
Site is open to international participants -- contact Dr. Krakow for additional information

United States
Principal Investigator: Deb Krakow, MD
Contact: 
Sarah Gaunt

Vanderbilt University 
Site is open to international participants -- contact Margo Black for additional information
United States
Principal Investigator: Kathryn Dahir, MD
Contact: Margo Black, BSN, MSN


OPTIMA Trial Website

More information is available at optimatrial.com
This website is only visible to those living in countries where there is an active clinical trial site.

The IFOPA does not endorse nor recommend specific clinical trials. Please speak with your doctor if you are interested in participating in a clinical trial.

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