Nonalcoholic Steatohepatitis (NASH)

TARGET-NASH

TARGET-NASH is critical for the scientific and regulatory community as we prepare for the plethora of new agents for the treatment of NASH.  In the short term, it gives us a critical understanding of NASH diagnosis and management in the real world- beyond the ivory tower, across multiple populations. In the longer term, it's the perfect platform to have a deep understanding of the safety and effectiveness of these new agents across populations not included or underrepresented in Phase 3 trials. Arun Sanyal, MD

According to recent studies, between 30 and 40% of adults in the United States have nonalcoholic fatty liver (NAFL) disease, of which 3% to 12% have NASH. Given the lack of available therapeutics and limited information on this disease, the TARGET-NASH study was launched with the goal of providing insight to regulatory bodies and pharmaceutical partners who are focused on developing new approved drugs.

TARGET-NASH is a longitudinal observational study of patients being managed for NAFL or NASH in usual clinical practice, within academic and community real-world practices. The dataset is critical to providing a baseline and measure the impact of current practice guidelines, management and new therapies utilized on patients with medical co-morbidities, hepatic, cardiovascular, and/or other endocrine-associated outcomes. The unique study design allows for a better understanding of the natural history of the disease with its 3 years of retrospective analysis of NASH patients and subsequent prospective enrollment of at least 5 years. Furthermore, pediatric and endocrinology TARGET-NASH working groups have been formed, in addition to the steering committee.

TARGET-NASH is comprised of a robust and meticulously documented cohort. The platform aggregates data from medical records and patient-reported outcome measures and collects biospecimen samples. Partners can query the longitudinal dataset and analyses biospecimens linked to validated clinical data to assess the safety and effectiveness of current and future therapies and support their drug development programs and RWE initiatives.

 

Study Goals:

  • Establish an understanding of the current natural history of NAFLD at academic and community medical centers
  • Evaluate NAFLD treatment regimens being used in clinical practice
  • Examine populations underrepresented in phase II-III clinical trials
  • Evaluate optimal duration and combination of NAFLD therapies to achieve clinical response and clinical remission
  • Examine liver histology
  • Estimate adverse event frequency and severity and describe management practices
  • Evaluate the impact of NAFLD therapies on medical co-morbidities

Launched:

August 2016

 

ENROLLMENT GOAL:

Up to 15,000

TARGET-NASH Steering Committee

Kenneth Cusi, MD (Co-Chair)
Chief of the Division of Endocrinology, Diabetes and Metabolism, Gainesville, FL

Arun Sanyal, MD (Co-Chair)
Chair of the Division of Gastroenterology, Hepatology and Nutrition, Richmond, VA

Brent Tetri, MD (Co-Chair)
Director, Division of Gastroenterology & Hepatology, St. Louis, MO

Miriam Vos, MD, MSPH (Pediatrics Chair)
Assistant Professor of Pediatrics, Atlanta, GA

Michael Roden, MD
Chair of Endocrinology and Metabolic Diseases, Düsseldorf, Germany

A. Sidney Barritt, MD, MSCR
Associate Professor of Medicine, Division of Gastroenterology & Hepatology, Chapel Hill, NC

Rohit Loomba, MD
Professor of Medicine, Division of Gastroenterology, San Diego, CA

Philip Newsome, PhD, FRCPE
Director, Centre for Liver Research, Birmingham, UK

Samuel Klein, MD
William H. Danforth Professor of Medicine and Nutritional Science, St. Louis, MO

Vincent Wong, MBChB (Hons), MD, FRCP, FHKCP, FHKAM
Professor, Department of Medicine and Therapeutics, Hong Kong

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