Lupus Foundation Publishes Recommendations to Improve Clinical Trials

Clinical trials with lupus patients should have a longer duration and a careful selection of patients based on certain parameters such as disease severity and treatment, recommends the Lupus Foundation of America (LFA), according to a news release.

These recommendations are a result of the most recent study of LFA’s Collective Data Analysis Initiative (LFA CDAI), which aims to improve the design of clinical trials and research in lupus, and speed up the development of novel treatments.

The study, “Impact Of Standard Of Care Treatments And Disease Variables On Outcomes In Systemic Lupus Erythematosus Trials: Analysis From The Lupus Foundation Of America Collective Data Analysis Initiative,” was published in the European Journal of Rheumatology.

The objective of a clinical trial is often to investigate whether a potential therapy improves disease symptoms in patients compared to standard-of-care treatments (therapies that are widely used by doctors).

But each lupus patient is different, making it difficult to draw general conclusions from trials with groups of patients. Also, analysis of the specific benefits of a given therapy with standard-of-care treatments becomes harder when patients are also receiving steroid treatments or more than one therapy at the same time.

In the new study, researchers followed 981 lupus patients to evaluate the long-term benefits of a potential treatment on their health outcomes.

They found that Phase 2 trials that have a short duration may not properly predict patient outcomes in longer trials.

They also found that patients’ responses to standard of care treatment is influenced by disease severity and the aggressiveness of immunosuppressive treatments, such as mycophenolate mofetil (CellCept), methotrexate (MTX), azathioprine (Imuran), and intravenous cyclophosphamide (Cytoxan). Lupus patients use immunosuppressants to decrease the activation of the immune system and, as a result, inflammation.

These findings have important implications for the selection of patients and the design of future clinical studies, so that proper comparisons and conclusions can be made.

“The growing CDAI database may support adequate power to detect clinically meaningful data from rare subsets of trial participants and facilitate the development of new definitions of response and/or flare to moderate the impact of [standard of care] background medications on outcomes,” the team wrote in their report.

Paola Daly, the study’s author and LFA Outcomes & Health Senior Manager, said in an LFA interview: “We found how important it is to examine how many times individuals flare over the course of a clinical trial … Most studies are set up to look at the level of lupus disease activity improvement overall.”

“However, many individuals have flares between those times that may be overlooked,” she said. “In addition, when designing a trial, it will be equally important to take into account the level of disease severity at the beginning of the trial and make firmer guidelines around what kinds of and what amounts of different treatments people can take when starting a new trial.”