A CROSS-SECTIONAL SURVEY ON CRINECERFONT AND QUALITY OF LIFE OF ADULTS WITH CLASSIC CONGENITAL ADRENAL HYPERPLASIA (CAH) IN THE UNITED STATES (US) PARTICIPATING IN CAHTALYST ADULT OPEN-LABEL EXTENSION (OLE) STUDY
Author(s)
Sonal Vaid, MD, BS BME1, Jessica Abramowitz, MD2, Irina Bancos, MD, MS3, Henry Kim Cheng, MA, MS, PhD4, Jean Chan, MD4, Robert Farber, PhD4, Nandini Hadker, MA5, Sanjay Keswani, MD4, Hyunwoo Kim, MS, PharmD4, Vivian Lin, MD4, Natalie Nokoff, MD6, Eiry Roberts, MD4, Maamoun Salam, MD7, Brittany Carletha Smith, MBA, MPH5, Oksana Hamidi, DO, MSCS2.
1National Institutes of Health, Bethesda, MD, USA, 2UT Southwestern Medical Center, Dallas, TX, USA, 3Mayo Clinic, Rochester, MN, USA, 4Neurocrine Biosciences, Inc., San Diego, CA, USA, 5Trinity Life Sciences, Waltham, MA, USA, 6Children's Hospital Colorado, Aurora, CO, USA, 7WashU Medicine, St. Louis, MO, USA.
1National Institutes of Health, Bethesda, MD, USA, 2UT Southwestern Medical Center, Dallas, TX, USA, 3Mayo Clinic, Rochester, MN, USA, 4Neurocrine Biosciences, Inc., San Diego, CA, USA, 5Trinity Life Sciences, Waltham, MA, USA, 6Children's Hospital Colorado, Aurora, CO, USA, 7WashU Medicine, St. Louis, MO, USA.
OBJECTIVES: Crinecerfont is a novel corticotropin releasing factor type 1 receptor (CRF1) antagonist, FDA-approved as an adjunct to glucocorticoid (GC) replacement to control androgens in patients (≥4 years) with classic congenital adrenal hyperplasia (CAH). By decreasing ACTH-driven adrenal steroidogenesis, crinecerfont, as shown in the CAHtalyst Adult trial (NCT04490915), enables treatment with more physiologic GC doses while maintaining or improving ACTH and adrenal androgen levels. The study aims to assess the impact of crinecerfont on quality of life and treatment satisfaction.
METHODS: The CAHtalyst Adult trial evaluated the efficacy and safety of crinecerfont in adults with CAH. Participants who completed Month 12 of CAHtalyst Adult continued taking crinecerfont in an open-label extension (OLE). The OLE was completed for United States (US) participants in 2025. At their final OLE visit, US participants completed a custom survey comparing their most recent month on crinecerfont with their pre-trial experience, prior to starting crinecerfont.
RESULTS: Of 182 randomized adult participants in CAHtalyst, 69 were in the US, with 60 continuing into the OLE. On their last visit, 78% (47/60) participants completed the survey and were eligible for analysis. Most participants (96%) were moderately or very satisfied with crinecerfont; none reported dissatisfaction. The majority reported meaningful improvements in feeling optimistic about reducing long-term impacts of high-dose GCs (96%) and high ACTH or androgens (89%), hopeful about their future living with CAH (94%), and more in control of their CAH (91%) because of crinecerfont. Additionally, participants reported meaningful improvements in calmness (72%), happiness (70%), daytime energy (68%), and sleep (68%). Lastly, 96% agreed or strongly agreed that they wish they could have started crinecerfont at a younger age.
CONCLUSIONS: While possible attrition, recall, and nonresponse bias should be acknowledged, these findings suggest crinecerfont as an adjunct treatment to manage CAH may lead to meaningful improvements in patients’ perceived lived experiences.
METHODS: The CAHtalyst Adult trial evaluated the efficacy and safety of crinecerfont in adults with CAH. Participants who completed Month 12 of CAHtalyst Adult continued taking crinecerfont in an open-label extension (OLE). The OLE was completed for United States (US) participants in 2025. At their final OLE visit, US participants completed a custom survey comparing their most recent month on crinecerfont with their pre-trial experience, prior to starting crinecerfont.
RESULTS: Of 182 randomized adult participants in CAHtalyst, 69 were in the US, with 60 continuing into the OLE. On their last visit, 78% (47/60) participants completed the survey and were eligible for analysis. Most participants (96%) were moderately or very satisfied with crinecerfont; none reported dissatisfaction. The majority reported meaningful improvements in feeling optimistic about reducing long-term impacts of high-dose GCs (96%) and high ACTH or androgens (89%), hopeful about their future living with CAH (94%), and more in control of their CAH (91%) because of crinecerfont. Additionally, participants reported meaningful improvements in calmness (72%), happiness (70%), daytime energy (68%), and sleep (68%). Lastly, 96% agreed or strongly agreed that they wish they could have started crinecerfont at a younger age.
CONCLUSIONS: While possible attrition, recall, and nonresponse bias should be acknowledged, these findings suggest crinecerfont as an adjunct treatment to manage CAH may lead to meaningful improvements in patients’ perceived lived experiences.
Conference/Value in Health Info
2026-05, ISPOR 2026, Philadelphia, PA, USA
Value in Health, Volume 29, Issue S6
Code
PCR67
Topic
Patient-Centered Research
Topic Subcategory
Patient-reported Outcomes & Quality of Life Outcomes
Disease
SDC: Diabetes/Endocrine/Metabolic Disorders (including obesity), SDC: Rare & Orphan Diseases