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

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*

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*

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

Open, Enrolling

Therapy Approach

Blocking Antibody

Regeneron Pharmaceuticals

FAQs

Who is eligible to participate in Regeneron’s trial?
trial is open to men and women ages 18-60 who have a clinical diagnosis of the classic FOP mutation (R206H mutation). Participants need to have had FOP disease activity within one year of the screening visit. FOP disease activity is defined as pain, swelling, stiffness, and other signs and symptoms associated with FOP flare-ups; or worsening of joint function, or radiographic progression of heterotopic ossifications (increase in size or number of HO lesions) with/without being associated with flare-up episodes. Additional inclusion and exclusion criteria regarding medical history, medication use, allergies, and laboratory values are available at Clinicaltrials.gov.

Can children participate in the Regeneron trial?
The current trial is not open to individuals with FOP under the age of 18. Many treatments are first studied in adults to establish the therapy’s safety profile before studying in children.

How many people can participate in the trial?
Approximately 66 people will be enrolled.

How many will be on vs actual study drug?
Half or up to 33 people will randomly be given garetosmab and half will be given (i.e. no active drug) for the first 6 months. At the end of the first 6 months, all participants will receive garetosmab. So, half of the study participants will receive 12 months of garetosmab while the other half will receive 6 months of garetosmab (after 6 months of ).

Can I participate in LUMINA-1 if I am currently enrolled in the FOP Registry?
Yes. If you are currently in the IFOPA registry, you may be able to also participate in the LUMINA-1 trial, as long as you meet the trial’s eligibility criteria.

How long does the Regeneron’s trial last?
This trial is 56 weeks with an additional 12 month follow-up period.

What centers and locations will be involved in the REGN2477 trial?
Regeneron is currently in the process of identifying further centers to participate in the Phase 3 study. Please refer to ifopa.org/optima for the latest information on clinical sites.

How is REGN2477 given to people with FOP?
REGN2477 is taken by IV infusion (i.e. given through the vein) every 4 weeks.

What clinical data currently exists for REGN2477?
The safety of REGN2477 has been studied in healthy volunteers who do not have FOP. The trial will be the first study of REGN2477 in individuals with FOP.

What are the travel commitments and requirements to participate in the trial?
Regeneron has hired a specialty travel agency to assist with arrangements for patients and caregivers. Individuals who participate in the REGN2477 trial will receive further information about their travel commitments and study reimbursement.

Who do I contact if I’m interested in learning more about Regeneron’s trial?
Have your doctor connect with Regeneron through one of the resources below:
Medical Information Hotline:
United States: 1-844-MID-REGN (1-844-643-7346)
International: +1-510-496-3637
Email: [email protected]

*For the complete list of eligibility criteria and details on this study, visit ClinicalTrials.gov and enter Identifier NCT05394116.

Participating Clinical Sites (as of November 7, 2023)

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

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

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

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

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

University of Cape Town
South Africa
Principal Investigator: Christiaan Scott, MBChB, FCPaed (SA)

Seoul National University Hospital (New)
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

Mayo Clinic
Site is open to international participants -- contact Dr. Pignolo for additional information
United States
Principal Investigator: Robert Pignolo, MD, PhD

University of California Los Angeles (UCLA) Medical Center
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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