Vertex Pharmaceuticals is “highly confident” that it can continue to supply its approved cystic fibrosis (CF) therapies to patients worldwide, but expects at least some of its ongoing and planned clinical trials to be affected by the COVID-19 pandemic.
Among likely changes to ongoing trials well into the dosing of patients — including a Phase 3 study of the recently approved Trikafta triple combination in children with CF — will be “virtual” (secure online) clinical visits and at-home treatment.
“[A] number of our clinical trials, such as our study of TRIKAFTA in children ages 6-11, are either fully enrolled or nearing full enrollment, and we are working with sites to enable virtual clinic visits and to ensure home delivery of the study drug,” a company spokeswoman said in an email exchange.
Trials in earlier stages may temporarily stop enrolling people, and the start of others may be postponed.
“Despite the widespread impact of the COVID-19 pandemic, the outlook for our business remains unchanged, and we continue to be highly confident in our supply chain and our ability to supply all of our approved medicines to patients who need them,” Jeffrey Leiden, MD, PhD, Vertex chairman, president and CEO, said in a press release.
“The COVID-19 pandemic is a rapidly evolving global health crisis that will require unprecedented utilization of healthcare resources that we expect will have an impact on our clinical trials,” Leiden added.
“In order to ensure patient safety and the appropriate use of healthcare resources, and to maintain study integrity, we have made proactive changes to some of our ongoing and planned clinical trials,” although “it is too early to precisely determine the long-term effects of the outbreak on trial timelines.”
Vertex, known for its approved CF treatments, continues to run a number of trials in this disease, as well as disorders that include sickle cell to a genetic kidney disease most common among African-Americans. All are at varying stages, from early and proof-of-concept studies to pivotal late-stage and post-marketing trials.
Virtual clinical visits, also known as e-Visits, use secure online communication systems to allow patients and treating physician-researchers to interact. These visits, along with home delivery of medications and their instructed use, would help to ensure study continuity and patient monitoring.
The Phase 3 study (NCT03691779) is evaluating oral Trikafta’s safety and efficacy (as a fixed 100 mg dose tablet taken each morning, plus ivacaftor each night) in more than 60 pediatric patients (ages 6 to 11) with CF. It opened in 2018 at sites across the U.S., and in Australia, Ireland, and the U.K., and is scheduled to conclude in August.
Trikafta is approved to treat patients ages 12 and older with the most common CF-causing mutation, that of at least one F508del defect in the CFTR gene, found in an estimated 90% of all people with this disease.
It was developed to benefit people with one or two F508del mutations in CFTR who don’t respond or respond poorly to Vertex’s other approved CF therapies for CF — Kalydeco (ivacaftor), a CFTR potentiator, and the potentiator-corrector combinations of Orkambi (lumacaftor/ivacaftor), and Symdeko (tezacaftor/ivacaftor, ivacaftor).
In reaffirming its confidence in its ability to continue to supply all its commercialized therapies to patients needing them, Vertex said its expects its manufacturing facilities to remain operational during the global COVID-19 pandemic.
It also said it has a large safety stock to cushion any potential disruptions. As a precaution, the company also identified other possible source suppliers in various regions worldwide.
A list of ongoing Vertex trials in CF patients can be found here.
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