Safety, Efficacy, and Pharmacokinetics of Agalsidase Alfa in Treatment-Naive Chinese Patients with Fabry Disease: 52-Week Phase 3, Open-Label, Single-Arm Study.
Enzyme replacement therapy remained well tolerated in Chinese Fabry disease patients, with kidney and heart function stable over a year.
This phase 3 open-label single-arm multicenter study evaluated We evaluated the safety, efficacy, and pharmacokinetics of agalsidase alfa in Chinese patients who are treatment-naive over 52 weeks. The authors report that in 20 treatment-naive Chinese patients with Fabry disease, agalsidase alfa was generally well tolerated and renal and cardiac measures remained stable over 52 weeks.
What the study was
- Study design
- Phase 3 open-label single-arm multicenter study
- Sample size
- 20
- Category
- Treatment Innovation
- Maturity
- Validated
- Journal
- Advances in therapy
Why it surfaced
8/10: Phase 3 open-label single-arm multicenter study with direct relevance to Rare diseases with high unmet need; scoring remains conservative because triage used the PubMed abstract without full-text review.
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