| Title: |
Safety of home administration of cipaglucosidase alfa plus miglustat in late-onset Pompe disease:results from multiple clinical trials |
| Authors: |
Andersen, Henning; Díaz-Manera, Jordi; Goker-Alpan, Ozlem; Mozaffar, Tahseen; Sitaraman Das, Sheela; Fox, Brian; Amon, Farah; O’Brien-Prince, Kinesha; Goldman, Mitchell; Holdbrook, Fred; Jain, Vipul; Byrne, Barry J. |
| Source: |
Andersen, H, Díaz-Manera, J, Goker-Alpan, O, Mozaffar, T, Sitaraman Das, S, Fox, B, Amon, F, O’Brien-Prince, K, Goldman, M, Holdbrook, F, Jain, V & Byrne, B J 2026, 'Safety of home administration of cipaglucosidase alfa plus miglustat in late-onset Pompe disease : results from multiple clinical trials', Therapeutic Advances in Rare Disease, vol. 7. https://doi.org/10.1177/26330040261416943 |
| Publication Year: |
2026 |
| Collection: |
Aarhus University: Research |
| Subject Terms: |
cipaglucosidase alfa; enzyme replacement therapy; GAA deficiency; miglustat; Pompe disease |
| Description: |
Background: Late-onset Pompe disease (LOPD) is caused by a deficiency of the acid α-glucosidase enzyme. In LOPD treatment, enzyme replacement therapy is delivered via intravenous infusion, typically in clinical settings. Cipaglucosidase alfa is delivered with the oral enzyme stabilizer miglustat (cipa + mig). Objectives: Evaluate the safety of cipa + mig home infusions. Design: Post hoc analysis of pooled safety data from three clinical trials in adults with LOPD (NCT02675465, NCT03729362, NCT04138277). Methods: The frequency and severity of infusion-associated reactions (IARs) during cipa + mig home and clinic administration were analyzed. Results: In total, 65/151 patients (43.0%) received ⩾1 cipa + mig home treatment. Of 9185 treatments, 2024 (22.0%) were administered at home. IAR frequency was similar for home (1.3%, 26/2024) and clinic (1.8%, 129/7161). The most frequent IAR following home infusion was pyrexia (6.2% of patients). Two patients with ⩾1 home-based treatment experienced serious IARs. Conclusion: Analyses support the safety of home cipa + mig treatment in eligible adults with LOPD. |
| Document Type: |
article in journal/newspaper |
| Language: |
English |
| ISSN: |
2633-0040 |
| Relation: |
info:eu-repo/semantics/altIdentifier/pmid/41631150; info:eu-repo/semantics/altIdentifier/eissn/2633-0040 |
| DOI: |
10.1177/26330040261416943 |
| Availability: |
https://pure.au.dk/portal/en/publications/b40d0557-9b4c-498a-b04d-7f16dfe2420c; https://doi.org/10.1177/26330040261416943; https://www.scopus.com/pages/publications/105029923395 |
| Rights: |
info:eu-repo/semantics/openAccess ; http://creativecommons.org/licenses/by-nc/4.0/ |
| Accession Number: |
edsbas.48F325E7 |
| Database: |
BASE |