Pinnacle Clinical Research, PLLC Participating in RESPONSE, a Global Phase 3 Clinical Research Study Evaluating an Investigational Therapy for the Treatment of Primary Biliary Cholangitis (PBC)

Novel oral medication could provide a new treatment option for people with PBC who have not achieved the recommended treatment goal or who cannot tolerate first-line therapy that prevents them from continuing with treatment

SAN ANTONIO, Nov. 02, 2021 (GLOBE NEWSWIRE) — Pinnacle Clinical Research, PLLC is enrolling participants into RESPONSE, a global phase 3 clinical research study evaluating the safety and efficacy of seladelpar, an investigational drug for people already diagnosed with PBC who have been using ursodeoxycholic acid (also known as UDCA or ursodiol) but have not achieved the recommended treatment goal or cannot tolerate UDCA. PBC is a chronic, serious and potentially life-threatening liver disease.

Seladelpar (study drug) is a potent, selective, orally active peroxisome proliferator-activated receptor delta (PPARδ) agonist in development for the treatment of people with PBC and other liver diseases. It controls metabolic and inflammatory pathways in the liver, inflammatory cells, muscle, fat, and intestinal tissues and has the potential to improve cholestasis, the key mechanism in play in PBC.

PBC is characterized by impaired bile flow (known as cholestasis) and the accumulation of toxic bile acids in the liver, leading to inflammation and destruction of the bile ducts within the liver and causing increased levels of alkaline phosphatase (ALP) and total bilirubin. The most common early symptoms of PBC are itching (pruritus) and fatigue,¹ which can be very debilitating for some people. Other early symptoms include abdominal pain, nausea, poor appetite, weight loss and arthritis.¹ Symptoms that may occur as PBC progresses include weakness, diarrhea, swelling in the legs, ankles or feet, dark urine, jaundice and raised patches on the skin.¹ Progression of PBC is associated with an increased risk of liver cancer and liver-related mortality.^2,3,4

RESPONSE is being conducted to learn more about the effectiveness and safety of seladelpar in people who have an inadequate response or are intolerant to UDCA. In this study, researchers want to find out if seladelpar can improve PBC-related blood laboratory tests and can improve symptoms of PBC, such as itching. To date, more than 500 individuals have received seladelpar in clinical trials, with some being exposed to seladelpar beyond one year. Seladelpar has received Breakthrough Therapy Designation from the U.S. Food and Drug Administration (FDA) and Priority Medicine Designation from the European Medicines Agency (EMA) because it has the potential to serve key unmet needs for people with PBC. In preclinical and phase 2 clinical studies, seladelpar has demonstrated the ability to decrease bile acid synthesis and inflammation, which are detrimental consequences of PBC.

The study is open to adults 18 to 75 years old who have been diagnosed with PBC; have been on a stable and recommended dose of UDCA for the past 12 months or cannot tolerate UDCA; and have ALP levels at least 1.67 times the upper limit of normal (ULN) and bilirubin less than two times the ULN. Participants who qualify for and participate in RESPONSE will receive seladelpar (study drug), or placebo, and UDCA (if applicable), study-related care from a physician with significant experience treating people with PBC, and study-related medical tests. All study medication and study-related care will be at no cost to participants in RESPONSE.

The FDA has cleared the RESPONSE protocol and enrollment in the study has also been approved by the Pinnacle Clinical Research, PLLC Institutional Review Board (IRB).

If you or someone you know is interested in joining the study, please call 210-982-0320.

About Pinnacle Clinical Research

Pinnacle Clinical Research is a leading provider of late phase clinical trials in the areas of hepatology and gastroenterology in the San Antonio and Austin area. Led by our world-renowned Medical Director, Dr. Stephen Harrison, we pride ourselves on conducting high quality research as a complement to the medical care that our volunteers receive from their routine care center as we search for new cures for future generations.

Contacts
Jessie Fernandez, Study Coordinator
[email protected]
210-982-0320 x 1458

Note to Editors: Images and additional information are available upon request.

¹ National Institute of Diabetes and Digestive and Kidney Diseases. Symptoms & Causes of Primary Biliary Cholangitis. Updated March 2017. Available at National Institute of Diabetes and Digestive and Kidney Diseases. Definition & Facts of Primary Biliary Cholangitis. Updated March 2017. Available at https://www.niddk.nih.gov/health-information/liver-disease/primary-biliary-cholangitis/definition-facts. Accessed June 4, 2019.
² Kaplan MM, Gershwin ME. Primary Biliary Cirrhosis. N Engl J Med. 2005;353(12):1261-1273.
³ Kumagi T, Heathcote EJ. Primary biliary cirrhosis. Orphanet J Rare Dis. 2008;3:1-17.
⁴ Lindor KD. Primary biliary cirrhosis. Hepatology. 2009;50(1):291-308.

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