Skip to main content Accessibility help
×
Home
Hostname: page-component-59b7f5684b-j4fss Total loading time: 0.412 Render date: 2022-10-05T15:42:05.569Z Has data issue: true Feature Flags: { "shouldUseShareProductTool": true, "shouldUseHypothesis": true, "isUnsiloEnabled": true, "useRatesEcommerce": false, "displayNetworkTab": true, "displayNetworkMapGraph": true, "useSa": true } hasContentIssue true

P.025 Efficacy and safety results of the avalglucosidase alfa phase 3 COMET trial in participants with late-onset Pompe disease (LOPD)

Published online by Cambridge University Press:  05 January 2022

M Tarnopolsky
Affiliation:
(Hamilton)*
S Attarian
Affiliation:
(Marseille)
J Borges
Affiliation:
(Brasilia)
F Bouhour
Affiliation:
(Lyon-Bron) Y Chien (Taipei)
Y Choi
Affiliation:
(Seoul)
P Clemens
Affiliation:
(Pittsburgh)
J Day
Affiliation:
(Stanford)
J Díaz-Manera
Affiliation:
(Newcastle upon Tyne)
S Erdem-Ozdamar
Affiliation:
(Ankara)
O Goker-Alpan
Affiliation:
(Fairfax)
S Illarioshkin
Affiliation:
(Moscow)
PS Kishnani
Affiliation:
(Durham)
A Kostera-Pruszczyk
Affiliation:
(Warsaw)
H Kushlaf
Affiliation:
(Cincinnati)
S Ladha
Affiliation:
(Phoenix)
T Mozaffar
Affiliation:
(Irvine, Orange)
M Roberts
Affiliation:
(Salford)
V Straub
Affiliation:
(Newcastle upon Tyne)
A Toscano
Affiliation:
(Messina)
AT van der Ploeg
Affiliation:
(Rotterdam)
K An Haack
Affiliation:
(Shanghai)
C Hug
Affiliation:
(Cambridge)
O Huynh-Ba
Affiliation:
(Chilly-Mazarin)
J Johnson
Affiliation:
(Cambridge)
T Zhou
Affiliation:
(Cambridge)
MM Dimachkie
Affiliation:
(Kansas City)
B Schoser
Affiliation:
(München)
Rights & Permissions[Opens in a new window]

Abstract

HTML view is not available for this content. However, as you have access to this content, a full PDF is available via the ‘Save PDF’ action button.

Background: Phase 3 COMET trial (NCT02782741) compares avalglucosidase alfa (n=51) with alglucosidase alfa (n=49) in treatment-naïve LOPD. Methods: Primary objective: determine avalglucosidase alfa effect on respiratory muscle function. Secondary/other objectives include: avalglucosidase alfa effect on functional endurance, inspiratory/expiratory muscle strength, lower/upper extremity muscle strength, motor function, health-related quality of life, safety. Results: At Week 49, change (LSmean±SE) from baseline in upright forced vital capacity %predicted was greater with avalglucosidase alfa (2.89%±0.88%) versus alglucosidase alfa (0.46%±0.93%)(absolute difference+2.43%). The primary objective, achieving statistical non-inferiority (p=0.0074), was met. Superiority testing was borderline significant (p=0.0626). Week 49 change from baseline in 6-minute walk test was 30.01-meters greater for avalglucosidase alfa (32.21±9.93m) versus alglucosidase alfa (2.19±10.40m). Positive results for avalglucosidase alfa were seen for all secondary/other efficacy endpoints. Treatment-emergent adverse events (AEs) occurred in 86.3% of avalglucosidase alfa-treated and 91.8% of alglucosidase alfa-treated participants. Five participants withdrew, 4 for AEs, all on alglucosidase alfa. Serious AEs occurred in 8 avalglucosidase alfa-treated and 12 alglucosidase alfa-treated participants. IgG antidrug antibody responses were similar in both. High titers and neutralizing antibodies were more common for alglucosidase alfa. Conclusions: Results demonstrate improvements in clinically meaningful outcome measures and a more favorable safety profile with avalglucosidase alfa versus alglucosidase alfa. Funding: Sanofi Genzyme

Type
Poster Presentations
Copyright
© The Author(s), 2021. Published by Cambridge University Press on behalf of Canadian Neurological Sciences Federation
You have Access

Save article to Kindle

To save this article to your Kindle, first ensure coreplatform@cambridge.org is added to your Approved Personal Document E-mail List under your Personal Document Settings on the Manage Your Content and Devices page of your Amazon account. Then enter the ‘name’ part of your Kindle email address below. Find out more about saving to your Kindle.

Note you can select to save to either the @free.kindle.com or @kindle.com variations. ‘@free.kindle.com’ emails are free but can only be saved to your device when it is connected to wi-fi. ‘@kindle.com’ emails can be delivered even when you are not connected to wi-fi, but note that service fees apply.

Find out more about the Kindle Personal Document Service.

P.025 Efficacy and safety results of the avalglucosidase alfa phase 3 COMET trial in participants with late-onset Pompe disease (LOPD)
Available formats
×

Save article to Dropbox

To save this article to your Dropbox account, please select one or more formats and confirm that you agree to abide by our usage policies. If this is the first time you used this feature, you will be asked to authorise Cambridge Core to connect with your Dropbox account. Find out more about saving content to Dropbox.

P.025 Efficacy and safety results of the avalglucosidase alfa phase 3 COMET trial in participants with late-onset Pompe disease (LOPD)
Available formats
×

Save article to Google Drive

To save this article to your Google Drive account, please select one or more formats and confirm that you agree to abide by our usage policies. If this is the first time you used this feature, you will be asked to authorise Cambridge Core to connect with your Google Drive account. Find out more about saving content to Google Drive.

P.025 Efficacy and safety results of the avalglucosidase alfa phase 3 COMET trial in participants with late-onset Pompe disease (LOPD)
Available formats
×
×

Reply to: Submit a response

Please enter your response.

Your details

Please enter a valid email address.

Conflicting interests

Do you have any conflicting interests? *