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Adipose Tissue_Appetite_and Obesity

SUN-688 - Long-Term Reductions of Hyperphagia with Diazoxide Choline Extended Release (DCCR) in Participants with Prader-Willi Syndrome

Sunday, June 14, 2026
9:00 AM - 4:00 PM CT
Location: ENDOExpo; Poster Floor
Disclosure(s):
Ashley H. Shoemaker, MD, MSCI: .NovoNordisk: Clinical Trial Investigator (); Aardvark Therapeutics: Clinical Trial Investigator (); Eli Lilly & Company: Contracted Research (Principal Investigators must provide information, even if received by the institution) (); Rhythm Pharmaceuticals: Advisory Board (), Clinical Trial Investigator (), Consultant (), Employee (any industry) (); Soleno Therapeutics: Clinical Trial Investigator (), Consultant ()
Jennifer L. Miller, MD: Rhythm Therapeutics: Clinical Trial Investigator (); Soleno Therapeutics: Clinical Trial Investigator ()


Background:
Prader-Willi syndrome (PWS) is a rare genetic neuroendocrine disorder characterized by hyperphagia, excess body fat, and behavioral challenges. Diazoxide choline extended-release (DCCR), a potent activator of the adenosine triphosphate (ATP)-sensitive potassium channel, is an FDA-approved treatment for hyperphagia in individuals aged 4 years and older with PWS. The DCCR development program included a 13-week randomized, placebo-controlled study (C601), an open-label extension study (C602OLE) up to 3 years, a 16-week randomized withdrawal study (C602RWP), and an ongoing open-label study C614. Hyperphagia Questionnaire for Clinical Trials (HQ-CT) is a validated questionnaire used to assess hyperphagia associated food related behaviors in clinical trials. We present results for the HQ-CT for up to 3 years from participants who received DCCR in C601/C602OLE compared to real-world natural history data from the PATH for PWS study (PATH).

Methods:
Participants who completed C601 could enroll in C602OLE and receive long-term open-label DCCR. HQ-CT total score was evaluated from DCCR Baseline and compared to non-DCCR exposed participants from PATH who met C601 age, weight and baseline hyperphagia inclusion criteria. Difference in change from Baseline (95% CI) of HQ-CT total score is presented; p-values were obtained from linear mixed effects model with unstructured covariance adjusting for key variables. Reduced scores reflect improvement.

Results:
Participants (N=352) were included (DCCR:123; PATH:229). Differences in demographics and baseline characteristics included: mean (SD) age in years (13 ± 6, 18 ± 9), body mass index in kg/m2 (27 ± 9, 29 ± 10), and % of participants on growth-hormone (83, 57) for DCCR vs PATH. A majority were white (85% vs 81%) and deletion subtype (62% vs 58%) for DCCR and PATH, respectively. Baseline HQ-CT total scores were 21.6 ± 6.7 and 18.2 ± 5.0 for DCCR and PATH groups. Significant (p < 0.0001) reductions in hyperphagia occurred at all time points for DCCR compared to PATH cohort. Change in HQ-CT from Baseline between DCCR and PATH was -6.2 (95% CI: -7.8, -4.5; p< 0.0001) at year 1, -6.5 (95% CI: -8.3, -4.8; p< 0.0001) at year 2, and -6.2 (95% CI: -8.2, -4.1; p< 0.0001) at year 3 demonstrating persistence of hyperphagia reduction in the DCCR treated cohort.

Conclusion:
DCCR administration resulted in significant, sustained improvements up to 3 years in hyperphagia as demonstrated by reduction in HQ-CT total score compared to natural history controls who did not spontaneously improve. DCCR represents an effective, long-term intervention for hyperphagia in patients with PWS.

*Unless otherwise noted, all abstracts presented at ENDO must not be released to the press or the public until the date and time of presentation. For oral presentations, the abstracts are embargoed until the session begins. The Endocrine Society reserves the right to lift the embargo on specific abstracts that are selected for promotion prior to or during ENDO.*