What next?
What next?
About this study
A new drug, called Efzofitimod, has shown potential to help people affected by severe inflammatory lung diseases. The drug works by dampening the immune responses, in the hope that it will resolve inflammation and prevent lung scarring from occurring. Small scale studies in patients with pulmonary sarcoidosis have shown a potential beneficial effect, however, this was only in a small number of people.
This study aims to investigate further in a larger group of people, to see if the drug could help people with pulmonary sarcoidosis, and if it has less severe side effects compared to other therapies.
There are many different types of research. These studies might ask participants to test different treatments, devices, or complete questionnaires. Some studies may not require any involvement from people affected by PF, but instead look at cells in a lab environment, animal models, or utilise computer modelling.
A study involving people can be:
- Interventional – participants are given an active treatment decided by researchers. This could be a change in medication or lifestyle (e.g. exercise, diet, complementary wellbeing therapy, or symptom management). These are commonly known as clinical trials.
- Observational – participants are assessed for specific changes over time, but researchers do not alter their standard treatment or care (e.g. questionnaires to understand the impact of symptom, or patient registries that track trends in how disease affects people or the services that are provided).
There are 3 main phases of clinical trials:
- Phase 1 is the earliest stage of research, where there is often a focus on understanding if the treatment is safe or if it causes any side effects.
- Phase 2 still focuses on safety and side effects, but also tries to find out more about what is the best dose.
- Phase 3 often compares the new treatment to existing treatments or lookalike medications.
As drugs or interventions move into the different phases, the number of people taking part will increase. Not all studies will make it to the later phases, especially if the intervention is not found to be safe or effective in phase 1 and 2.
The recruitment aim is the number of people that researcher want to take part in the research study.
What is involved?
You will be assigned to one of three groups.
- Group 1 and 2 will receive the study drug, Efzofitimod, through an intravenous infusion. This is a drip where the drug is provided directly into the bloodstream, in the form of fluids. The only difference between the two groups is the amount of drug that is given at each infusion. The infusions are every 4 weeks and there are a total of 12 doses in total.
- Group 3 will receive a placebo. This is something that looks exactly like the drug, but there are no active drug ingredients in the infusions.
You will not know which group you have been assigned to until the end of the study.
The researchers will measure your lung function at different points in the study, take blood tests and ask you to complete questionnaires to see if there are any benefits for taking the drug.
Summary of involvement
Duration up to 49 weeks
Infusions every 4 weeks, with a total of 12 doses
Infusions will either be with the study drug or a placebo
Blood tests
Questionnaires
Lung function tests
Can I take part?
You may be able to take part if you are aged between 18 and 75 years, and have a diagnosis of pulmonary sarcoidosis. You must have been receiving a stable dose of oral corticosteroids medication for at least 3 months. Your body weight must be between 40 and 160 kg.
In research studies, there are lots of different reasons why you may or may not be able to take part. These are known as inclusion and exclusion criteria (see list below). Only the clinical trial team will be able to fully determine whether you are able to take part in the study. You can withdraw at any point.
If you are not able to take part in this research study, there may be other opportunities for you to take part in different studies.
To find our more about other research opportunities visit our research finder main page.
Aged 18-75 years
Diagnosis of pulmonary sarcoidosis
Experiencing symptoms of disease
Stable dose of oral corticosteroids for at least 3 months
Body weight between 40 - 160 kg
You can't be taking more than 1 oral immunosuppressant therapy
Significant pulmonary fibrosis that has increased by more than 20% within the last 12 months
Severe pulmonary hypertension
There are other specific exclusion criteria points, but these will be explored by the research team
What difference could taking part make?
Taking part in this study will help researchers to understand if the new drug, Efzofitimod, has the potential to help people with pulmonary sarcoidosis, by reducing inflammation, symptom relief, subsequent scarring of the lung tissue, and stabilising the disease.
Taking part in research not only helps researcher now, but it helps those affected by the disease in the future. All research leads us to finding better and more effective treatment options, by figuring out what does and doesn't work.
Where does the study take place?
Study locations
Birmingham
Cambridge
Cottingham
Coventry
London
You may be able to take part in the study, if you do not live close to a research site, but are prepared to travel in order to take part. This will not affect you current standard treatment that you receive.
How to take part
If you would like to take part in this study or find out more information, please speak to your healthcare team to express your interest in research. You can also visit the Efzo-fit website to find out more and see if you Pre-Qualify to take part.
Further information
The summary on this page provides information about an opportunity to participate in research. More detailed information about the study can be found via the following the links and through contacting the research team.
If you have any questions about this research study, please speak to your medical team.
clinicaltrials@atyrpharma.comThis study is supported by
APF does not endorse or recommend any specific study. All responsibility for the study remains with the sponsors and investigators.
Every effort is made to keep these details up to date. If you are aware of any inaccuracies, please email research@actionpf.org