Presentation (CTI)

OBJECTIVES: Congenital adrenal hyperplasia (CAH) is a rare autosomal recessive disorder in which an enzyme deficiency leads to insufficient cortisol and aldosterone production, increased adrenocorticotropic hormone (ACTH) production, and hyperandrogenemia. Symptoms are associated with impaired quality of life and long-term supraphysiologic glucocorticoid treatment with increased morbidity. The novel anti-ACTH antibody asedebart is being investigated in CAH. Here we present patient experience data from qualitative exit interviews in a first-in-human Phase I trial of asedebart in classic CAH.
METHODS: Semi-structured exit interviews were conducted with adult patients with classic CAH who participated in the multisite, open-label, multiple-ascending-dose trial (NCT05669950), within 3 weeks of the completion or withdrawal visit. Key interview objectives were to understand symptom burden and perceptions of change during the trial.
RESULTS: Eleven participants (7 women and 4 men; median age 34 years) received monthly asedebart infusions for up to 6 months and were interviewed. All participants reported fatigue before trial enrollment; most also reported body weight issues (72.7%), depression (72.7%), impact on romantic relationships (63.6%), sleep issues (63.6%), and adrenal crises (54.5%). Almost half of participants (45.5%) spontaneously reported that the most bothersome aspect of CAH was medication concerns; 81.8% reported that the most meaningful change in their daily life with CAH would be to medications. All 9 participants who completed the trial reported stability (88.9%) or improvement (33.3%) in ≥1 of their most bothersome aspects of CAH. The most common improvements were reported in sleep (33.3%) and body weight issues (33.3%), and the most common decline was in fatigue (44.4%).
CONCLUSIONS: These qualitative patient experience data from a first-in-human, multiple-ascending-dose trial reveal the symptom burden of classic CAH and perceived impact of asedebart. These results help contextualize unmet needs in CAH, guide future asedebart clinical development, and inform subsequent patient-focused research.