PATIENT DESCRIPTIONS OF IMPROVED ABILITY TO SELF-REGULATE BLOOD SUGAR LEVELS IN A PHASE 3 TRIAL OF DTX401 FOR GLYCOGEN STORAGE DISEASE TYPE IA
Author(s)
Diane M. Turner-Bowker, PhD1, Shayna Egan, MPH1, Blaise Cureg, MPH2, Adriana Voci, BS2, Monica Boyer, CPNP3, Jessica Butler, BA1, Richard Collis, MD1, Elizabeth Dorn, PhD1, Kristin Voorhees, MA1, Martha Gauthier, MA2.
1Ultragenyx Pharmaceutical Inc., Novato, CA, USA, 2Lumanity, Boston, MA, USA, 3Rady Children’s Health, Orange, CA, USA.
1Ultragenyx Pharmaceutical Inc., Novato, CA, USA, 2Lumanity, Boston, MA, USA, 3Rady Children’s Health, Orange, CA, USA.
OBJECTIVES: GSDIa is a rare, inherited, autosomal recessive disease with deficiency of glucose-6-phosphatase requiring the frequent consumption of exogenous glucose (e.g., uncooked cornstarch) for patient survival. Qualitative in-trial interviews explored patient treatment experiences in a Phase 3, double-blind, randomized, placebo-controlled crossover study of DTX401, an investigational gene therapy for GSDIa in patients ≥8 years (NCT05139316). This research describes trial participant reports of an improved ability to self-regulate blood sugar levels following DTX401 treatment.
METHODS: Following ethics approval, 60-minute web-based telephone interviews were conducted at trial Week 96 (W96) using semi-structured interview guides. Interviews were audio-recorded, transcribed, and coded. Interview questions included reflections on treatment experience, overall satisfaction, treatment preferences, and post-DTX401 GSDIa management burden and symptoms. A subset of patients also self-reported an improved ability to self-regulate blood sugar levels; this content was analyzed, and thematic results were summarized.
RESULTS: At W96, 42% (n=16/38) of participants spontaneously described an improved ability to self-regulate their blood sugar. This finding was reported by a higher proportion of participants in the Crossover DTX401 (Placebo --> DTX401) group (50%, n=10/20) than the DTX401 group (33%, n=6/18). Participant descriptors for improved self-regulation of blood sugar following DTX401 treatment included slower drops in blood sugar, less severe hypoglycemia, more stable blood sugars, fewer crashes or sudden drops in blood sugar and increased symptomatic hypoglycemia, which led to feelings of a “safety net,” i.e., less danger if a cornstarch dose was missed, less worry about experiencing hypoglycemic events that would require emergency treatment or hospitalization, and easier recovery from hypoglycemic events.
CONCLUSIONS: This research summarizes the lived experiences of symptom improvements in a trial from the perspective of individuals with GSDIa. Allowing trial participants to directly share their experiences, in addition to standard trial assessments, provides a unique opportunity to better understand the impact of DTX401 treatment.
METHODS: Following ethics approval, 60-minute web-based telephone interviews were conducted at trial Week 96 (W96) using semi-structured interview guides. Interviews were audio-recorded, transcribed, and coded. Interview questions included reflections on treatment experience, overall satisfaction, treatment preferences, and post-DTX401 GSDIa management burden and symptoms. A subset of patients also self-reported an improved ability to self-regulate blood sugar levels; this content was analyzed, and thematic results were summarized.
RESULTS: At W96, 42% (n=16/38) of participants spontaneously described an improved ability to self-regulate their blood sugar. This finding was reported by a higher proportion of participants in the Crossover DTX401 (Placebo --> DTX401) group (50%, n=10/20) than the DTX401 group (33%, n=6/18). Participant descriptors for improved self-regulation of blood sugar following DTX401 treatment included slower drops in blood sugar, less severe hypoglycemia, more stable blood sugars, fewer crashes or sudden drops in blood sugar and increased symptomatic hypoglycemia, which led to feelings of a “safety net,” i.e., less danger if a cornstarch dose was missed, less worry about experiencing hypoglycemic events that would require emergency treatment or hospitalization, and easier recovery from hypoglycemic events.
CONCLUSIONS: This research summarizes the lived experiences of symptom improvements in a trial from the perspective of individuals with GSDIa. Allowing trial participants to directly share their experiences, in addition to standard trial assessments, provides a unique opportunity to better understand the impact of DTX401 treatment.
Conference/Value in Health Info
2026-05, ISPOR 2026, Philadelphia, PA, USA
Value in Health, Volume 29, Issue S6
Code
PCR24
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