Farmacon Global, a strategic CRO of Medical Consultants with a specialty in Rare Disease, patient recruitment and medical trials in Latin America, has assisted Mirum Pharmaceuticals in completing their MARCH-PFIC double-blind placebo-controlled Phase 3 study to evaluate the efficacy and safety of maralixibat in the treatment of patients with Progressive Familial Intrahepatic Cholestasis (PFIC), a rare liver disease impacting children.
“Sometimes ‘less is more,’”said Sara Tylosky, CEO Farmacon Global. “This particular enrollment for Mirum needed thirty participants for their pivotal Phase 3 global clinical trial for PFIC, during the COVID-19 pandemic of 2020-2021. It was not a case of recruiting a vast population for this study, but a case of realizing the intricacies, sensitivities, and interconnections needed to find each participant, who were children, and then implementing the special organization needed for their caregivers.
“Farmacon Global is an extremely patient-centric organization, so through our Latin American medical team and strategic lab partner, Mendelics (Brazil), along with our referring network, KOLs and Principal Investigators, we were able to significantly contribute to this critical enrollment.
“PFIC or Progressive Familial Intrahepatic Cholestasis is a rare inherited condition for children who are not able to drain bile from the liver even though the large bile ducts are open (cholestasis). This gets worse over time, and some children may succumb to their disease due to extensive liver damage. Thus, our team members are highly motivated to give many patients an opportunity to participate in a clinical study that could help reduce the burdensome symptoms related to their disease,” concluded Tylosky.
Dr. Tiago Nunes, MD, PhD, Executive Director, Clinical Development of Mirum Pharmaceuticals said, “Latin America was key for the enrollment completion in our MARCH-PFIC study. Latin America, through Farmacon Global, was the top recruiter location with sites in Argentina, Brazil, Colombia, and Mexico. Their hands-on coordination with sites was key to the enrollment completion of our PFIC global clinical trial.”
For more information on Global Clinical Trial Enrollment and Strategic Support, contact Sara Tylosky, CEO Farmacon Global 561-232-3441
