CLINICAL TRIALS IN SARCOIDOSIS
For many years there has been little interest and research into sarcoidosis by pharmaceutical companies. That is now changing. New clinical trials specifically designed for sarcoidosis are taking place in the UK as well as USA and other countries.
What are clinical trials?
Clinical trials are research studies that test new medical treatments in patients to see if they are safe and effective. They are important because they can help us to find new and better ways to treat diseases.
Clinical trials are tightly regulated by laws, which were made to protect participants, while ensuring that the drug in development will not be released for use as a patient treatment until it is safe and effective.
These trials are advanced in steps, each of which has to be successful in order to move on to the next phase – see phases I to IV below:
- Phase I trials determine how safe a compound is in healthy volunteers (looking out for potential undesired side effects).
- Phase II trials determine safety and the optimal dose in patients (the amount of the compound and how often it should be taken).
- Phase III trials determine the compound’s effectiveness in patients.
- Phase IV trials may be performed after the drug is already licensed and usually look for the effect of the drug outside the limited specifications of earlier clinical phase trials.
Each phase trial must show that it was successful in order to move on to the next phase trial.
Due to the complexity of these processes, it typically takes years from early development to marketing of a drug. In this process, many compounds fail to demonstrate effectiveness or for other reasons their development may be stopped.
Further details of clinical trials taking part in the UK can be found on the National Institute of Health Research clinical trial site https://bepartofresearch.nihr.ac.uk/
What are the potential benefits and risks to taking part in clinical trials?
Taking part in a clinical trial needs careful consideration. Read the NHS information and guidance about taking part in clinical trials for more information about the risks and benefits. It’s important to carefully consider these factors and discuss them with your specialist before deciding to join a trial.
These clinical trials are now recruiting. Follow the links if you are interested in finding out more.
Xentria
The Xentria trial is a Phase 1b/2 study of XTMAB-16 in patients with pulmonary sarcoidosis.
The trial will test an investigational drug called XTMAB-16, a monoclonal antibody targeting the inflammatory response known to occur in patients living with sarcoidosis.
For more information about this trial, visit https://xentria.com/clinical/xtmab16-phase-2
For information about the Principal Investigators and trial sites, click here.
KITE
The KITE Study is a Phase II clinical trial. The trial is testing OATD-01, an experimental potential alternative to standard of care medicine, for the first time in patients with active pulmonary sarcoidosis. The study goal is to evaluate this medicine in the reduction of inflammation, slowing of disease progress, and to assess its safety.
OATD-01 is a novel small molecule, which affects the activity of CHIT1. CHIT1 is an enzyme which has been shown to be involved in several lung diseases including pulmonary sarcoidosis.
For more information about this trial, visit https://www.thekitestudy.com/.
For information about the Principal Investigators and trial sites, click here.
These clinical trials have finished recruitment and are ongoing. Follow the links if you are interested in finding out more.
EFZO-FIT
The EFZO-FIT study is a Phase III clinical trial. It is evaluating the safety and effectiveness of the investigational drug efzofitimod for the treatment of pulmonary sarcoidosis.
Efzofitimod is a protein which may help to reduce inflammation in the lungs. By targeting chronic lung inflammation, efzofitimod may help improve symptoms, including fatigue, prevent permanent lung damage, and preserve lung function in people with pulmonary sarcoidosis.
This study will test whether efzofitimod will allow people to reduce the dose of steroids they are taking to treat their disease, while maintaining symptom control and preserving lung function.
For more information about this trial, visit here.
For information about the Principal Investigators and trial sites, click here.
The EFZO-FIT Study has now finished enrollment. You can find their press release here.
These clinical trials have now finished. Follow the links if you are interested in finding out more.
RESOLVE-Lung
The RESOLVE-Lung study was a Phase II clinical trial. It evaluated the safety and effectiveness of the investigational medicine namilumab for the treatment of pulmonary sarcoidosis.
Primary Endpoint Details: Namilumab failed to meet the primary endpoint of proportion of subjects with a Rescue Event during the double-blind period.
Secondary Endpoints Details: Secondary efficacy endpoints, including change in percent predicted forced vital capacity, corticosteroid tapering success, and change in the patient reported King’s Sarcoidosis Questionnaire failed to show a treatment benefit consistent with the primary endpoint.
Safety Details: The safety profile of namilumab in RESOLVE-Lung remains similar to previous studies.
“Although RESOLVE-Lung failed to show a treatment benefit for namilumab, the significant amount of information produced by the study will be tremendously helpful to those in the field who are committed to improving the lives of sarcoidosis patients,” said Bill Gerhart, CEO of Kinevant.
Find out more here.
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Sarcoidosis is a misunderstood disease with limited treatment options, low levels of support and no cure. It does not receive the attention it deserves from medical researchers.
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