Open-label extension study following the Late-Onset Treatment Study (LOTS) of alglucosidase alfa

Ans T. Van der Ploeg, Richard Barohn, Lisa Carlson, Joel Charrow, Paula R. Clemens, Robert J. Hopkin, Priya S. Kishnani, Pascal Laforêt, Claire Morgan, Sharon Nations, Alan Pestronk, Horacio Plotkin, Barry E. Rosenbloom, Katherine B. Sims, Elisa Tsao

Research output: Contribution to journalArticle

59 Scopus citations

Abstract

Objective: Late-onset Pompe disease is a progressive, debilitating, and often fatal neuromuscular disorder resulting from the deficiency of a lysosomal enzyme, acid α-glucosidase. This extension study was conducted to determine the durability of the efficacy and safety of alglucosidase alfa observed over a period of 78. weeks in the Late-Onset Treatment Study (LOTS). Methods: Patients who completed the LOTS study were eligible for this open-label extension study and received alglucosidase alfa 20. mg/kg biweekly for an additional 26. weeks. The primary efficacy assessments were the distance walked during a 6-minute walk test and the percentage of predicted forced vital capacity in the upright position. Data are reported as change from patient's original LOTS baseline for each measure. Results: The benefit of alglucosidase alfa treatment observed in LOTS at Week 78 was, in general, maintained at Week 104. The mean increase in distance walked measured 28.2 ± 66.5. m from LOTS baseline to Week 78 and 21.3 ± 78.0. m from LOTS baseline to Week 104. The mean change from baseline in percentage of predicted forced vital capacity was 1.3% ± 5.7% from LOTS baseline to Week 78 and 0.8% ± 6.7% from LOTS baseline to Week 104. Treatment-related adverse events were mainly infusion-associated reactions observed in 35% of patients. No deaths or anaphylactic reactions were observed during the extension study. Conclusions: The LOTS Extension study showed that patients treated with alglucosidase alfa for up to 104. weeks maintained the improved walking distance and stabilization in pulmonary function observed in the first 78 weeks of alglucosidase alfa therapy.

Original languageEnglish (US)
Pages (from-to)456-461
Number of pages6
JournalMolecular genetics and metabolism
Volume107
Issue number3
DOIs
StatePublished - Nov 2012

Keywords

  • Genetics
  • Glycogenoses
  • Inherited metabolic disease
  • Neuromuscular disease
  • Pompe disease

ASJC Scopus subject areas

  • Endocrinology, Diabetes and Metabolism
  • Biochemistry
  • Molecular Biology
  • Genetics
  • Endocrinology

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    Van der Ploeg, A. T., Barohn, R., Carlson, L., Charrow, J., Clemens, P. R., Hopkin, R. J., Kishnani, P. S., Laforêt, P., Morgan, C., Nations, S., Pestronk, A., Plotkin, H., Rosenbloom, B. E., Sims, K. B., & Tsao, E. (2012). Open-label extension study following the Late-Onset Treatment Study (LOTS) of alglucosidase alfa. Molecular genetics and metabolism, 107(3), 456-461. https://doi.org/10.1016/j.ymgme.2012.09.015