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Open-label extension study following the Late-Onset Treatment Study (LOTS) of alglucosidase alfa.
Mol Genet Metab. 2012 Nov; 107(3):456-61.MG

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 20mg/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 ± 66.5m from LOTS baseline to Week 78 and 21.3 ± 78.0m 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.

Authors+Show Affiliations

Department of Pediatrics, Division of Metabolic Diseases and Genetics, Center for Lysosomal and Metabolic Diseases, Erasmus MC University Medical Center-University Hospital Sophia Children's Hospital, Dr Molewaterplein 60, 3015 GJ Rotterdam, The Netherlands. a.vanderploeg@erasmusmc.nlNo affiliation info availableNo affiliation info availableNo affiliation info availableNo affiliation info availableNo affiliation info availableNo affiliation info availableNo affiliation info availableNo affiliation info availableNo affiliation info availableNo affiliation info availableNo affiliation info availableNo affiliation info availableNo affiliation info availableNo affiliation info available

Pub Type(s)

Journal Article
Multicenter Study
Randomized Controlled Trial
Research Support, Non-U.S. Gov't

Language

eng

PubMed ID

23031366

Citation

van der Ploeg, Ans T., et al. "Open-label Extension Study Following the Late-Onset Treatment Study (LOTS) of Alglucosidase Alfa." Molecular Genetics and Metabolism, vol. 107, no. 3, 2012, pp. 456-61.
van der Ploeg AT, Barohn R, Carlson L, et al. Open-label extension study following the Late-Onset Treatment Study (LOTS) of alglucosidase alfa. Mol Genet Metab. 2012;107(3):456-61.
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), 456-61. https://doi.org/10.1016/j.ymgme.2012.09.015
van der Ploeg AT, et al. Open-label Extension Study Following the Late-Onset Treatment Study (LOTS) of Alglucosidase Alfa. Mol Genet Metab. 2012;107(3):456-61. PubMed PMID: 23031366.
* Article titles in AMA citation format should be in sentence-case
TY - JOUR T1 - Open-label extension study following the Late-Onset Treatment Study (LOTS) of alglucosidase alfa. AU - van der Ploeg,Ans T, AU - Barohn,Richard, AU - Carlson,Lisa, AU - Charrow,Joel, AU - Clemens,Paula R, AU - Hopkin,Robert J, AU - Kishnani,Priya S, AU - Laforêt,Pascal, AU - Morgan,Claire, AU - Nations,Sharon, AU - Pestronk,Alan, AU - Plotkin,Horacio, AU - Rosenbloom,Barry E, AU - Sims,Katherine B, AU - Tsao,Elisa, Y1 - 2012/09/17/ PY - 2012/07/14/received PY - 2012/09/12/revised PY - 2012/09/12/accepted PY - 2012/10/4/entrez PY - 2012/10/4/pubmed PY - 2013/3/28/medline SP - 456 EP - 61 JF - Molecular genetics and metabolism JO - Mol. Genet. Metab. VL - 107 IS - 3 N2 - 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 20mg/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 ± 66.5m from LOTS baseline to Week 78 and 21.3 ± 78.0m 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. SN - 1096-7206 UR - https://www.unboundmedicine.com/medline/citation/23031366/Open_label_extension_study_following_the_Late_Onset_Treatment_Study__LOTS__of_alglucosidase_alfa_ L2 - https://linkinghub.elsevier.com/retrieve/pii/S1096-7192(12)00356-3 DB - PRIME DP - Unbound Medicine ER -