From Research to Reality: Understanding the Clinical Trial Process
Every family in our community brings a unique perspective to the world of research. Some of you may already be familiar with the clinical trial landscape, while others may find the scientific language a bit daunting or may not be quite sure where to begin. No matter where you are in your journey, please know that your experience is valid, and you are not alone.
We have created these pages to be a welcoming home for information. Our goal is to provide clear information for anyone considering, preparing for, or currently navigating a clinical trial.
We have gathered resources from the most trusted organisations to help explain the “how” and “why” of research, ensuring you have the tools you need to make the best decisions for your family.
Resources for You and Your Family
These materials are designed for you to explore whenever you feel ready. You don’t need to be an expert to get started; these guides are here to provide context, clarity and reassurance:
* Simple Explainer Videos: To help you visualise how research works.
* Genetic Primers: Easy-to-follow articles that explain the science behind FOP.
* A Helpful Glossary: A guide to the terms you may hear during doctor visits and trial appointments.
* Webinars: To help you understand and navigate the procesess, as well as ask questions to the experts.
Our hope is to take the mystery out of FOP drug development.
By providing these resources, we aim to help you feel more prepared for conversations with clinicians and more confident in your role as an advocate for yourself or your child.
This collection will grow over time. If you come across a video, article or resources what you find particularly helpful, please share it with us so we can pass that help along to others.
Visit our Learning Hub, register for a Webinar, or read on to find out other ways you can get involved in progressing the understanding of FOP.
Before we begin, we would like to acknowledge the dedication and hard work of the many individuals who, over the years, have helped bring us to where we are today – patients, parents, family members, friends, doctors, nurses, researchers, pharmaceutical companies, and many others.
We are ultra rare! And within the rare disease space we are most definitely punching above our weight! This is thanks to the hard work and dedication of so many people over the years, from the researchers who discovered the gene to those working on treatments today. We are lucky to have researchers around the world all trying to better understand FOP and ultimately to find a treatment and a cure.
One of the reasons they have been able to make such progress is thanks to the dedication and commitment of families over the years. From the parents who set up the first patient organisations and pen-pal groups – both here and around the world – to our community today. We have always been a loud and determined group who have raised awareness of the challenges of living with FOP fought to have our voices heard!
If you have taken part in the Natural History study, participated in a clinical trial, are signed up to the IFOPA’s FOP Registry, completed surveys, raised money to support the research, shared awareness day posts, told your story… then you have made a difference. You have helped us to get to where we are today.
We still have an incredibly long way to go, but today we want to thank you for getting us this far. With your continued support, we can do this. We are filled with hope for the journey ahead.
The FOP Registry is independently operated by the IFOPA.
The Registry is the largest and most detailed global collection of clinical and medical information about FOP, contributed by individuals living with the condition. Its primary purpose is to help clinicians and researchers gain a deeper understanding of how FOP affects people, aiming to improve patient care and accelerate the development of new treatments and a cure. The Registry is open to anyone with FOP, with information securely provided online by the person living with FOP or their caregiver, ensuring only anonymous data is shared with researchers.
Participants also receive a $25 gift card every time they complete a survey.
Learn more here: The FOP Registry
Download the brochure: Here
