Study to Compare the Efficacy and Safety of Enzyme Replacement Therapies Avalglucosidase Alfa and Alglucosidase Alfa Administered Every Other Week in Patients With Late-onset Pompe Disease Who Have Not Been Previously Treated for Pompe Disease

Last updated: March 29, 2024
Sponsor: Genzyme, a Sanofi Company
Overall Status: Completed

Phase

3

Condition

Liver Disorders

Pompe Disease

Treatment

Avalglucosidase alfa (GZ402666)

Alglucosidase alfa (GZ419829)

Clinical Study ID

NCT02782741
EFC14028
U1111-1178-4806
2016-000942-77
  • Ages > 3
  • All Genders

Study Summary

Primary Objective:

To determine the effect of avalglucosidase alfa treatment on respiratory muscle strength measured by percent (%) predicted forced vital capacity (FVC) in the upright position, as compared to alglucosidase alfa.

Secondary Objective:

To determine the safety and effect of avalglucosidase alfa treatment on functional endurance (6-minute walk test, inspiratory muscle strength (maximum inspiratory pressure), expiratory muscle strength (maximum expiratory pressure), lower extremity muscle strength (hand-held dynamometry), motor function (Quick Motor Function Test), and health-related quality of life (Short Form-12).

Eligibility Criteria

Inclusion

Inclusion criteria :

  • The participant has confirmed acid alpha-glucosidase (GAA) enzyme deficiency from anytissue source and/or 2 confirmed GAA gene mutations.
  • The participant must provide signed, informed consent prior to performing any studyrelated procedures. Consent of a legally authorized guardian(s) is (are) required forlegally minor participant as defined by local regulation. If the participant islegally minor, signed written consent shall be obtained from parent(s)/legal guardianand assent obtained from participants, if applicable.

Exclusion

Exclusion criteria:

  • The participant is <3 years of age.
  • The participant has known Pompe specific cardiac hypertrophy.
  • The participant is wheelchair dependent.
  • The participant is not able to ambulate 40 meters (approximately 130 feet) withoutstopping and without an assistive device.
  • The participant requires invasive-ventilation (non-invasive ventilation is allowed).
  • The participant is not able to successfully perform repeated forced vital capacity (FVC) measurements in upright position of greater than or equal to 30% predicted andless than or equal to 85% predicted.
  • The participant (and participant's legal guardian if participant is legally minor asdefined by local regulation) is (are) not able to comply with the clinical protocol.
  • The participant has had previous treatment with alglucosidase alfa or anyinvestigational therapy for Pompe disease.
  • The participant has prior or current use of immune tolerance induction therapy.
  • The participant, if female and of childbearing potential, has a positive pregnancytest (beta-human chorionic gonadotropin) at baseline. The above information is not intended to contain all considerations relevant to aparticipant's potential participation in a clinical trial.

Study Design

Total Participants: 101
Treatment Group(s): 2
Primary Treatment: Avalglucosidase alfa (GZ402666)
Phase: 3
Study Start date:
November 02, 2016
Estimated Completion Date:
May 31, 2023

Study Description

The duration of the study per participant will be up to approximately 6 years that will consist of a 14-day screening period (may be extended up to 8 weeks in pre-specified situations), a 49-week blinded treatment period (except for the subgroup of pediatric patients aged 3 to less than (<) 18 years enrolling directly in the open-label long-term follow-up phase), a 240-week open-label treatment period, and a 4-week post-treatment observation period.

Connect with a study center

  • Investigational Site Number 0320001

    Buenos Aires,
    Argentina

    Site Not Available

  • Investigational Site Number 0320001

    Caba, C1181ACH
    Argentina

    Site Not Available

  • Investigational Site Number 0360001

    Auchenflower, 4066
    Australia

    Site Not Available

  • Investigational Site Number 0400001

    Wien, 1090
    Austria

    Site Not Available

  • Investigational Site Number 0560003

    Bruxelles, 1070
    Belgium

    Site Not Available

  • Investigational Site Number 0560001

    Leuven, 3000
    Belgium

    Site Not Available

  • Investigational Site Number 0760004

    Brasilia, 71625-009
    Brazil

    Site Not Available

  • Investigational Site Number 0760005

    Brasília, 72145-450
    Brazil

    Site Not Available

  • Investigational Site Number 0760001

    Sao Paulo, 04037-002
    Brazil

    Site Not Available

  • Investigational Site Number 1000001

    Sofia, 1431
    Bulgaria

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  • Investigational Site Number 1240003

    Hamilton, L8N 3Z5
    Canada

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  • Investigational Site Number 1240002

    Montreal, H3A 2B4
    Canada

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  • Investigational Site Number 1240002

    Montréal, H3A 2B4
    Canada

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  • Investigational Site Number 1700001

    Bogota,
    Colombia

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  • Investigational Site Number 1700001

    Bogotá,
    Colombia

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  • Investigational Site Number 2030001

    Praha, 12100
    Czech Republic

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  • Investigational Site Number 2030001

    Praha 2, 12808
    Czechia

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  • Investigational Site Number 2080002

    Aarhus C, 8000
    Denmark

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  • Investigational Site Number 2080003

    Copenhagen, 2100
    Denmark

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  • Investigational Site Number 2080003

    København Ø, 2100
    Denmark

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  • Investigational Site Number 2500008

    Angers, 49933
    France

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  • Investigational Site Number 2500007

    Bordeaux,
    France

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  • Investigational Site Number 2500011

    Brest Cedex 2, 29609
    France

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  • Investigational Site Number 2500004

    Bron, 69677
    France

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  • Investigational Site Number 2500003

    Bron Cedex, 69677
    France

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  • Investigational Site Number 2500010

    Clermont Ferrand, 63003
    France

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  • Investigational Site Number 2500005

    Lille, 59037
    France

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  • Investigational Site Number 2500006

    Marseille, 13385
    France

    Site Not Available

  • Investigational Site Number 2500006

    Marseille Cedex 5, 13385
    France

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  • Investigational Site Number 2500002

    Nice Cedex 1, 06002
    France

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  • Investigational Site Number 2500001

    Paris, 75013
    France

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  • Investigational Site Number 2500001

    Paris Cedex 13, 75013
    France

    Site Not Available

  • Investigational Site Number 2760006

    Bochum, 44789
    Germany

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  • Investigational Site Number 2760004

    Hamburg, 20246
    Germany

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  • Investigational Site Number 2760001

    Mainz, 55131
    Germany

    Site Not Available

  • Investigational Site Number 2760002

    Munster, 48149
    Germany

    Site Not Available

  • Investigational Site Number 2760003

    München, 80336
    Germany

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  • Investigational Site Number 2760002

    Münster, 48149
    Germany

    Site Not Available

  • Investigational Site Number 3480001

    Budapest, 1083
    Hungary

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  • Investigational Site Number 3800006

    Brescia, 25123
    Italy

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  • Investigational Site Number 3800001

    Messina, 98125
    Italy

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  • Investigational Site Number 3800002

    Milano, 20122
    Italy

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  • Investigational Site Number 3800007

    Napoli, 80131
    Italy

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  • Investigational Site Number 3800004

    Padova, 35128
    Italy

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  • Investigational Site Number 3800007

    Roma, 00168
    Italy

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    Torino, 10126
    Italy

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  • Investigational Site Number 3920003

    Izumi-Shi,
    Japan

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  • Investigational Site Number 3920002

    Kodaira-Shi,
    Japan

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  • Investigational Site Number 3920003

    Osaka, 594-0071
    Japan

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  • Investigational Site Number 3920002

    Tokyo, 187-8551
    Japan

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  • Investigational Site Number 4100004

    Daejeon, 35015
    Korea, Republic of

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  • Investigational Site Number 4100001

    Seoul, 03080
    Korea, Republic of

    Site Not Available

  • Investigational Site Number 4100002

    Seoul, 06273
    Korea, Republic of

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  • Investigational Site Number 4100003

    Yangsan-Si,
    Korea, Republic of

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  • Investigational Site Number 4840001

    Guadalajara,
    Mexico

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  • Investigational Site Number 4840001

    Jalisco, 45030
    Mexico

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  • Investigational Site Number 4840001

    Mexico,
    Mexico

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  • Investigational Site Number 5280001

    Rotterdam, 3015 GE
    Netherlands

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  • Investigational Site Number 6160001

    Warszawa, 02-097
    Poland

    Site Not Available

  • Investigational Site Number 6200001

    Braga, 4710-243
    Portugal

    Site Not Available

  • Investigational Site Number 6430001

    Moscow, 125367
    Russian Federation

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  • Investigational Site Number 7240002

    Barcelona, 08025
    Spain

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  • Investigational Site Number 7240003

    Barcelona, 08950
    Spain

    Site Not Available

  • Investigational Site Number 7240003

    Barcelona / Esplugues De Llobregat, 08950
    Spain

    Site Not Available

  • Investigational Site Number 7240004

    Cadiz, 11009
    Spain

    Site Not Available

  • Investigational Site Number 7240003

    Esplugues De Llobregat, 08950
    Spain

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  • Investigational Site Number 7240001

    Madrid, 28046
    Spain

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  • Investigational Site Number 7240005

    Valencia, 46026
    Spain

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  • Investigational Site Number 7520001

    Uppsala, 751 85
    Sweden

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  • Investigational Site Number 7560002

    Zurich, 8091
    Switzerland

    Site Not Available

  • Investigational Site Number 7560002

    Zürich, 8091
    Switzerland

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  • Investigational Site Number 1580001

    Taipai, 10043
    Taiwan

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  • Investigational Site Number 1580001

    Taipei, 10043
    Taiwan

    Site Not Available

  • Investigational Site Number 1580002

    Taoyuan County, 33305
    Taiwan

    Site Not Available

  • Investigational Site Number 7920001

    Ankara, 06100
    Turkey

    Site Not Available

  • Investigational Site Number 7920002

    Istanbul, 34390
    Turkey

    Site Not Available

  • Investigational Site Number 8260005

    Birmingham, B15 2GW
    United Kingdom

    Site Not Available

  • Investigational Site Number 8260002

    Cambridge, CB2 OQQ
    United Kingdom

    Site Not Available

  • Investigational Site Number 8260001

    London, NW3 2QG
    United Kingdom

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  • Investigational Site Number 8260004

    Newcastle Upon Tyne, NE1 4LP
    United Kingdom

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  • Investigational Site Number 8260003

    Salford, M6 8HD
    United Kingdom

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  • Investigational Site Number 8400015

    Phoenix, Arizona 85013
    United States

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  • Investigational Site Number 8400020

    Los Angeles, California 90095
    United States

    Site Not Available

  • Investigational Site Number 8400011

    Orange, California 92868
    United States

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  • Investigational Site Number 84000017

    Palo Alto, California 94304
    United States

    Site Not Available

  • Investigational Site Number 8400017

    Stanford, California 94305
    United States

    Site Not Available

  • Investigational Site Number 8400016

    Gainesville, Florida 32610
    United States

    Site Not Available

  • Investigational Site Number 8400016

    Gainsville, Florida 32610
    United States

    Site Not Available

  • Investigational Site Number 8400007

    Decatur, Georgia 30033
    United States

    Site Not Available

  • Investigational Site Number 8400023

    Chicago, Illinois 60611
    United States

    Site Not Available

  • Investigational Site Number 8400002

    Iowa City, Iowa 52242
    United States

    Site Not Available

  • Investigational Site Number 8400012

    Kansas City, Kansas 66160-7321
    United States

    Site Not Available

  • Investigational Site Number 8400010

    Boston, Massachusetts 02114
    United States

    Site Not Available

  • Investigational Site Number 8400001

    Detroit, Michigan 48201
    United States

    Site Not Available

  • Investigational Site Number 8400019

    Minneapolis, Minnesota 55455
    United States

    Site Not Available

  • Investigational Site Number 8400013

    Saint Louis, Missouri 63110
    United States

    Site Not Available

  • Investigational Site Number 8400026

    Great Neck, New York 11020
    United States

    Site Not Available

  • Investigational Site Number 8400021

    New York, New York 10016
    United States

    Site Not Available

  • Investigational Site Number 8400008

    Valhalla, New York 10595
    United States

    Site Not Available

  • Investigational Site Number 8400006

    Durham, North Carolina 27710
    United States

    Site Not Available

  • Investigational Site Number 8400009

    Cincinnati, Ohio 45267-0542
    United States

    Site Not Available

  • Investigational Site Number 8400014

    Portland, Oregon 97239
    United States

    Site Not Available

  • Investigational Site Number 8400004

    Hershey, Pennsylvania 17033
    United States

    Site Not Available

  • Investigational Site Number 8400025

    Pittsburgh, Pennsylvania 15213
    United States

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  • Investigational Site Number 8400022

    Dallas, Texas 75246
    United States

    Site Not Available

  • Investigational Site Number 8400018

    Salt Lake City, Utah 84132
    United States

    Site Not Available

  • Investigational Site Number 8400005

    Fairfax, Virginia 22030
    United States

    Site Not Available

  • Investigational Site Number 8400024

    Morgantown, West Virginia 26506
    United States

    Site Not Available

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