Efficacy and safety of PEGylated uricase in healthy adults and patients with hyperuricaemia: A phase I, randomized, double‐blind, dose‐escalation, placebo‐controlled trial
Authors' conclusion
PEG-UOX demonstrated favourable pharmacokinetics, urate-lowering effects, and tolerability over a 21-day observation period in both healthy adults and patients with hyperuricaemia. Its uricolytic mechanism offers a promising therapeutic alternative for managing hyperuricaemia, particularly in cases refractory to conventional urate-lowering therapies.
Study context
- Population
- Healthy adults and patients with hyperuricaemia24 healthy, 24 hyperuricaemic
- Participants
- 48 adults
- Comparison
- Single IV dose of PEG-UOX vs placebo
- Primary endpoint
- Tolerability, PK/PD and immunogenicity of PEG-UOX
- Follow-up
- 21-day follow-up
Methodological detailsphase I, double-blind, placebo-controlled, dose-escalation, sentinel dosing design
- Phase I
- Double-blind
- Placebo-controlled
- Dose-escalation
- Sentinel dosing design
Primary result
As reported in the abstract
Serum uric acid
- Marked reductions in serum uric acid in both healthy and hyperuricaemic subjects; greatest efficacy in hyperuricaemic patients receiving 8 mg
Further findings
Urinary uric acid and allantoin
- Urinary uric acid excretion decreased while allantoin levels increased, confirming uricolytic activity
Immunogenicity
- Higher incidence of anti-PEG antibodies in healthy subjects than in patients with hyperuricaemia
Safety
- Serious adverse events
- 0
- Most treatment-emergent AEs mild, incl. transient asymptomatic ST-T changes and Ventricular Premature Contractions (VPCs) (Grade 1)
Original publication
Efficacy and safety of PEGylated uricase in healthy adults and patients with hyperuricaemia: A phase I, randomized, double‐blind, dose‐escalation, placebo‐controlled trial. Diabetes Obes Metab. Published 25 June 2025. doi:10.
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