Researchers at Temple University Hospital are taking part in a large, global Phase 3 clinical trial to evaluate whether a novel, investigational drug for systemic lupus erythematosus (SLE) is more effective than previously approved medications.
This trial (NCT02446912), one of several pivotal and ongoing studies of anifrolumab to treat moderately to severely active SLE, also referred to as lupus, is recruiting patients at nearly 160 sites across the U.S., Europe, Latin America and elsewhere, including at Temple.
Anifrolumab is being developed by AstraZeneca, and in previous studies it demonstrated an ability to reduce disease symptoms in over 50% of patients after one year of treatment. Patients receiving the drug also needed fewer corticosteroids to treat inflammation, common in lupus.
“The data from the Phase 2 trial was so promising that the FDA has granted fast-track approval for this new medication,” Roberto Caricchio, MD, director of the Temple Lupus Clinic at the Lewis Katz School of Medicine at Temple and trial’s primary investigator there, said in a press release. “That’s excellent news because currently there are only four other drugs that have been approved to treat lupus, and only one of those was approved in the past 50 years. It is hoped that this treatment will help patients. However, this cannot be guaranteed.”
Anifrolumab targets interferon type-1, a protein involved in the inflammation known to play a key role in disease development.
“Interferon type-1 is a molecule called a cytokine, which activates a variety of immune cells in lupus and triggers flares,” said Caricchio. “Each immune cell has a ‘lock’ (receptor) that is opened by the ‘key’ interferon type-1. Once the lock is open the cell activates and so does the lupus. Previous lupus medications tried to block the interferon type-1 cytokine, but anifrolumab directly blocks the receptor (the lock) on the cell instead, making it more effective.”
Adults (18 to 70 years old) with moderate-to-severe SLE, who are currently being treated with prescription medication, are being enrolled in this double-blind, placebo-controlled study, evaluating the efficacy and safety of two different doses of anifrolumab, a high and low dose administered intravenously, versus placebo. Treatment will be given in addition to currently prescribed medication. The study’s primary goal is the number of treated patients who achieve an SLE Responder Index greater than or equal to 4 at week 52 (the index is a composite measure of disease activity). In total, the trial (NCT02446912) plans to enroll 450 people at its various sites; more information is available on the study’s clinical trials.gov webpage.
At Temple, researchers are looking to enroll between 10 and 20 SLE patients for this study, which will last one and a half years and require a total of 16 visits. Participants will be asked to fill regular questionnaires from home and submit them electronically. Those interested in enrolling at the Philadelphia-area site can contact the Temple Lupus Clinic directly at [email protected] or call 215-707-4479.
Two other Phase 3 trials assessing anifrolumab in lupus patients, one a long-term safety and tolerability study (NCT02794285) and the other an efficacy and safety study (NCT02446899), are also actively enrolling eligible patients. More information, including enrollment information, is available by clicking on each trial’s identification number.
Lupus is a complex autoimmune disease and every patient experiences different symptoms — these may include joint pain, muscle pain, and fever or fatigue. To manage their symptoms, patients often use medications that are prescribed off-label, meaning that many of them have not been tested in lupus-specific clinical trials. Such use could lead to unwanted side effects, especially when the drugs are taken for longer periods of time.
“Lupus causes such a spectrum of manifestations ranging from mild to life-threatening and everything in between, which is why it’s difficult to develop medications to treat it and to determine whether patients are responding to those medications,” Caricchio said.