Trial reportOrphanet journal of rare diseases2025
Safety and efficacy of laronidase in Chinese patients with mucopolysaccharidosis type I: a phase IV, single-arm, open-label, multicenter study.
Trial report in Orphanet journal of rare diseases, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT05134571 (A Phase 4, Single-arm, Open-label Safety and Efficacy Study of Aldurazyme®), which is not on this map. Not yet cited in PubMed.
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The trial behind it
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A Phase 4, Single-arm, Open-label Safety and Efficacy Study of Aldurazyme® (Laronidase) as Enzyme Replacement Therapy in Participants With Mucopolysaccharidosis I (MPS I) in China
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Authors and funding
8 authors.
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Abstract
backgroundMucopolysaccharidosis type I (MPS I) is a lysosomal storage disorder caused by deficiency of the enzyme α-L-iduronidase. Laronidase (Aldurazyme
methodsFrom October 2021 to July 2023, 12 MPS I patients at four institutions in China received weekly intravenous injections of laronidase at a dose of 100 U/kg of body weight once a week for 26 weeks. The primary efficacy endpoint was the percentage change in urinary glycosaminoglycans (uGAGs) levels at week 26 relative to baseline. Safety endpoints included the incidence of adverse events (AEs), serious adverse events (SAEs), and adverse events of special interest (AESIs, including infusion-related reactions) during the treatment period (TE).
resultsLaronidase consistently reduced uGAGs levels from baseline to week 26, with a percentage change of -64.61% ± 26.90% (95% CI: -81.70% to -47.52%). There was a revealed reduction following laronidase treatment. The percentage reductions in uGAGs from baseline to weeks 2, 4, 8, 12, and 20 were decreased. The decreases in absolute change of uGAGs were observed at weeks 2, 4, 8, 12, 20, and 26. The percentage reduction in liver volume from baseline to week 26 was - 13.24% ± 7.86% (95% CI: -18.24% to -8.25%). Nine participants (75%) achieved an overall treatment compliance rate ≥80%. Eleven participants (91.7%) experienced treatment-emergent adverse events (TEAEs). Four participants (33.3%) experienced AESIs.
conclusionsIn Chinese patients with MPS I, laronidase as an enzyme replacement therapy effectively reduces glycosaminoglycan storage and liver volume while demonstrating a favorable safety profile. CLINICAL TRIAL REGISTRATION NUMBER: NCT05134571. CLINICAL TRIAL REGISTRATION DATE: 2021-10-21. NAME OF THE REGISTRY: China Post-marketing Surveillance (PMS) Study of Aldurazyme
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