UX053 / Ultragenyx, Arcturus Therap - LARVOL DELTA

A Phase 1/2 first-in-human study to evaluate the safety, tolerability, and pharmacokinetics of UX053 in patients with glycogen storage disease type III (SIMD 2024) - P1/2 | "Single doses of UX053 were well tolerated with an acceptable safety profile. Pharmacodynamic effects on blood glucose and urinary Glc4 were observed after a single dose, indicating that UX053 triggered physiologically-relevant expression of GDE, even at the lowest dose tested. Caution should be taken when administering UX053 to patients with high anti-PEG antibody titers."

Clinical • P1/2 data • PK/PD data • Back Pain • Hepatology • Hypoglycemia • Metabolic Disorders • Musculoskeletal Pain • Pain • Pompe Disease

Safety, Tolerability, and Pharmacokinetics of UX053 in Patients With Glycogen Storage Disease Type III (GSD III) (clinicaltrials.gov) - P1/2 | N=8 | Terminated | Sponsor: Ultragenyx Pharmaceutical Inc | Active, not recruiting ➔ Terminated; Sponsor decision not related to safety concerns

Trial termination • Metabolic Disorders • Pompe Disease

Safety, Tolerability, and Pharmacokinetics of UX053 in Patients With Glycogen Storage Disease Type III (GSD III) (clinicaltrials.gov) - P1/2 | N=8 | Active, not recruiting | Sponsor: Ultragenyx Pharmaceutical Inc | Recruiting ➔ Active, not recruiting | N=18 ➔ 8 | Trial completion date: Jun 2024 ➔ Mar 2023 | Trial primary completion date: Jun 2024 ➔ Mar 2023

Enrollment change • Enrollment closed • Trial completion date • Trial primary completion date • Metabolic Disorders • Pompe Disease

Safety, Tolerability, and Pharmacokinetics of UX053 in Patients With Glycogen Storage Disease Type III (GSD III) (clinicaltrials.gov) - P1/2 | N=18 | Recruiting | Sponsor: Ultragenyx Pharmaceutical Inc | N=30 ➔ 18 | Trial completion date: Sep 2023 ➔ Jun 2024 | Trial primary completion date: Sep 2023 ➔ Jun 2024

Enrollment change • Trial completion date • Trial primary completion date • Metabolic Disorders • Pompe Disease