Active not recruitingPHASE3INTERVENTIONAL

A Study to Assess Safety, Tolerability and Efficacy of Garetosmab Versus Placebo Administered Intravenously (IV) in Adult Participants With Fibrodysplasia Ossificans Progressiva (FOP)

This research study is testing a new medication called garetosmab for adults who have a rare condition called Fibrodysplasia Ossificans Progressiva, or FOP. FOP causes soft tissues in the body to turn into bone, which can cause pain and limit movement. The main goal is to see if garetosmab is safe to use and if it can help improve the symptoms of FOP. We'll also be looking at any potential side effects, how the body handles the drug, and if the body creates antibodies against it. This is a Phase 3 study, which means it's a key step before the drug might become widely available. Participants will either receive garetosmab or a placebo (a dummy drug).

At a glance

Status

Active not recruiting

Phase

PHASE3

Sponsor

Regeneron Pharmaceuticals

Enrolment target

Start

21 Nov 2022

Estimated completion

27 Feb 2029

Fibrodysplasia Ossificans Progressiva

What is this study about?

This study is designed to explore a new drug called garetosmab for adults living with Fibrodysplasia Ossificans Progressiva (FOP). FOP is a very rare condition that causes extra bone to form in soft tissues like muscles, tendons, and ligaments. This extra bone can cause a lot of pain and severely limit movement, making everyday activities difficult.

The main purpose of this research is to see if garetosmab is a safe and effective treatment for FOP. We want to understand if it can help reduce the symptoms of FOP and improve the lives of those affected. To do this, some participants will receive the active drug, garetosmab, while others will receive a placebo, which looks like the drug but doesn't contain any active medicine. This allows researchers to fairly compare the effects of the new drug.

Throughout the study, the research team will carefully monitor participants. They will be looking for any side effects from the drug, how the body processes garetosmab, and whether the body's immune system reacts to it. All this information is crucial for understanding how the drug works and deciding if it could become a new treatment option for FOP in the future.

Key takeaways

This study is for adults with Fibrodysplasia Ossificans Progressiva (FOP).
It's testing a new drug called garetosmab against a placebo.
The main goals are to check the drug's safety and how well it works.
Participation involves regular clinic visits, blood tests, and scans.
You might get the active drug or a dummy drug.
You can stop participating at any time.

Who may be eligible?

To be considered for this study, you must be an adult aged 18 or older and have a confirmed diagnosis of Fibrodysplasia Ossificans Progressiva (FOP). This diagnosis needs to be based on specific findings, like typical changes in your big toes, episodes of swelling in soft tissues, or extra bone growth. We'll also need proof from a genetic test showing you have the specific gene change linked to FOP.

Crucially, you must have experienced some FOP activity within the last year before joining the study. This means you've had pain, swelling, stiffness, or other symptoms of FOP flare-ups, or that your joint movement has worsened, or X-rays have shown new or growing areas of extra bone.

There are also some reasons why you might not be able to join. For example, if your FOP is very advanced, if you have other serious health problems like severe heart, kidney, or liver conditions, or a history of cancer, you might not be eligible. Also, certain uncontrolled conditions like very high blood pressure or diabetes could prevent you from taking part, as these might make the study results unclear or put you at higher risk.

Quick self-check

Are you 18 years old or older?
Do you have a confirmed diagnosis of FOP, including a genetic test?
Have you had FOP symptoms or progression in the last year?
Are you able to have CT scans and other required medical procedures?
Do you have any serious health conditions like severe heart disease or cancer?
Is your diabetes or blood pressure well-controlled?

This is a guide only — the research team will confirm whether you can take part.

What does participation involve?

If you join this study, you'll be randomly assigned to receive either the study drug, garetosmab, or a placebo (a dummy drug) through an IV drip. You won't know which one you're getting. Regular visits to the clinic will be needed, where the study team will perform various assessments. These will likely include physical examinations, blood and urine tests, and detailed imaging scans, such as CT scans, to monitor your FOP and see how the medication is affecting you. You'll also be asked about any symptoms you're experiencing and any side effects. The total duration of your participation in the study, including the treatment period and follow-up, will be explained in full detail by the study team, but typically, Phase 3 trials can last for a year or more. You are free to withdraw from the study at any time.

Potential risks and benefits

Potential benefits of taking part could include gaining access to a new treatment that might help with your FOP symptoms, and you'll be closely monitored by a specialist medical team. However, there are potential risks, including side effects from the study drug, discomfort from blood tests or scans, and the possibility that the drug may not help you or may even lead to unexpected problems. Since you might receive a placebo, there's also a chance you won't get the active drug. You have the right to leave the study at any time, for any reason, without it affecting your usual medical care.

Locations (22)

University of California Los Angeles (UCLA) Medical Center
Los Angeles, United States
Vanderbilt University Medical Center
Nashville, United States
Royal North Shore Hospital
St Leonards, Australia
Hospital Israelita Albert Einstein
São Paulo, Brazil
Universidad de Concepcion
Concepción, Chile
Tongji Hospital of Tongji University
Shanghai, China
Clinica Universidad de La Sabana
Chía, Colombia
HUS Children and Adolescents Park Hospital Clinical Trial Unit
Helsinki, Finland
Hôpital Lapeyronie
Montpellier, France
Hopital Lariboisiere
Paris, France
Queen Mary Hospital
Hong Kong, Hong Kong
IRCCS Istituto Giannina Gaslini
Genoa, Italy

+10 more sites — see the official record for the full list.

Common questions

What is FOP?

FOP (Fibrodysplasia Ossificans Progressiva) is a rare condition where soft tissues, like muscles, turn into bone over time, causing pain and limiting movement.

What is garetosmab?

Garetosmab is a new investigational drug being tested to see if it can help treat FOP.

What is a placebo?

A placebo is a substance that looks exactly like the study drug but doesn't contain any active medication. It's used to compare against the real drug.

Will I know if I'm getting the drug or the placebo?

No, this study is 'blinded,' meaning neither you nor your study doctor will know whether you are receiving garetosmab or the placebo.

Who is funding this study?

The information provided doesn't state the specific funder, but typically such studies are funded by pharmaceutical companies or research organisations.