BridgeMD
A Study to Assess Safety, Tolerability and Efficacy of Garetosmab versus Placebo Administered Intravenously (IV) in Adult Participants With Fibrodysplasia Ossificans Progressiva (FOP) (OPTIMA)
EU CTIS ID: 2023-508350-26-00
What this study is testing
The primary efficacy objective of the study is to assess the effect of garetosmab versus placebo on the formation of new HO lesions, as determined by low-dose computerized tomography (CT). The primary safety objective of the study is to assess the safety and tolerability of garetosmab versus placebo.
- Therapeutic confirmatory (Phase III)
A plain-language read of the study's public EU CTIS listing. The study team confirms the details.
Who can take part
You may be able to join if
- Clinical diagnosis of Fibrodysplasia Ossificans Progressiva (FOP) [(based on findings of congenital malformation of the great toes, episodic soft tissue swelling, and/or progressive Heterotopic Ossification (HO)].
- Confirmation of FOP diagnosis with documentation of Type I activin A receptor (ACVR1) FOP causing mutation.
- FOP disease activity within 1 year of screening visit. FOP disease activity is defined as pain, swelling, stiffness, or other signs and symptoms associated with FOP flare-ups; or worsening of joint function, or radiographic progression of HO lesions (increase in size or number of HO lesions) with/without being associated with flare-up episodes.
- Willing and able to undergo CT imaging procedures and other procedures as defined in the protocol.
- Note: Other protocol defined Inclusion Criteria apply
You likely can't join if
- Cumulative Analog Joint Involvement Scale (CAJIS) score at screening >19.
- Prior use in the past year and concomitant use of bisphosphonates.
- Concurrent participation in another interventional clinical study or a non-interventional study with radiographic measures or invasive procedures (eg, collection of blood or tissue samples).
- Treatment with another investigational drug, denosumab, imatinib or isotretinoin in the last 30 days or within 5 half-lives of the investigational drug, whichever is longer.
- Pregnant or breastfeeding women.
- Women of childbearing potential (WOCBP) who are unwilling to practice highly effective contraception, as defined in the protocol.
See the full eligibility criteria
- Clinical diagnosis of Fibrodysplasia Ossificans Progressiva (FOP) [(based on findings of congenital malformation of the great toes, episodic soft tissue swelling, and/or progressive Heterotopic Ossification (HO)].
- Confirmation of FOP diagnosis with documentation of Type I activin A receptor (ACVR1) FOP causing mutation.
- FOP disease activity within 1 year of screening visit. FOP disease activity is defined as pain, swelling, stiffness, or other signs and symptoms associated with FOP flare-ups; or worsening of joint function, or radiographic progression of HO lesions (increase in size or number of HO lesions) with/without being associated with flare-up episodes.
- Willing and able to undergo CT imaging procedures and other procedures as defined in the protocol.
- Note: Other protocol defined Inclusion Criteria apply
- Cumulative Analog Joint Involvement Scale (CAJIS) score at screening >19.
- Prior use in the past year and concomitant use of bisphosphonates.
- Concurrent participation in another interventional clinical study or a non-interventional study with radiographic measures or invasive procedures (eg, collection of blood or tissue samples).
- Treatment with another investigational drug, denosumab, imatinib or isotretinoin in the last 30 days or within 5 half-lives of the investigational drug, whichever is longer.
- Pregnant or breastfeeding women.
- Women of childbearing potential (WOCBP) who are unwilling to practice highly effective contraception, as defined in the protocol.
- Male patients with WOCBP partners who are not willing to use condoms with WOCBP partners to prevent potential fetal exposure, as defined in the protocol.
- Note: Other protocol defined Exclusion Criteria apply
- Participant has significant concomitant illness or history of significant illness such as but not limited to cardiac, renal, rheumatologic, neurologic, psychiatric, endocrine, metabolic, or lymphatic disease, that in the opinion of the study investigator might confound the results of the study or pose additional risk to the patient by their participation in the study.
- Previous history or diagnosis of cancer.
- Severely impaired renal function defined as estimated glomerular filtration rate <30 milliliter per minute (mL/min) (/1.73 m^2 calculated by the Modification of Diet in Renal Disease equation.
- Uncontrolled diabetes defined as hemoglobin A1C (HbA1c) >9% at screening.
- History of poorly controlled hypertension, as defined by: a. Systolic blood pressure ≥180 mm Hg or diastolic blood pressure ≥110 mm Hg at the screening visit b. Systolic blood pressure of 160 mm Hg to 179 mm Hg or diastolic blood pressure of 100 mm Hg to 109 mm Hg at the screening visit, AND a history of end-organ damage (including history of left-ventricular hypertrophy, heart failure, angina, myocardial infarction, stroke, transient ischemic attack, peripheral arterial disease, end-stage renal disease, and moderate-to-advanced retinopathy.
- Known history of cerebral vascular malformation.
- Cardiovascular conditions such as New York Heart Association class III or IV heart failure, cardiomyopathy, intermittent claudication, myocardial infarction, or acute coronary syndrome within 6 months prior to screening; symptomatic ventricular cardiac arrhythmia.
- History of severe respiratory compromise requiring oxygen, respiratory support (eg, bilevel positive airway pressure [biPAP] or continuous positive airway pressure [CPAP]), or a history of aspiration pneumonia requiring hospitalization.
The study team makes the final eligibility decision.
Where it's taking place
- Colombia
- China
- Hong Kong
- Australia
- United States
- United Kingdom
- Malaysia
- Chile
- Mexico
- Korea, Republic of
- South Africa
- Japan
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 65+ years, 18-64 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Colombia; China; Hong Kong; Australia; United States; United Kingdom and 6 more location(s). Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from EU CTIS; the study team decides eligibility.