A Study to Evaluate the Efficacy and Safety of Alglucosidase Alfa Produced at the 4000 L Scale for Pompe Disease
NCT ID: NCT01526785
Last Updated: 2015-12-08
Study Results
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View full resultsBasic Information
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TERMINATED
PHASE4
113 participants
INTERVENTIONAL
2012-03-31
2014-12-31
Brief Summary
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Detailed Description
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Conditions
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Study Design
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NA
SINGLE_GROUP
TREATMENT
NONE
Study Groups
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Alglucosidase alfa
Alglucosidase alfa (4000 L scale) intravenous (IV) infusion administered for 52 weeks as per physician's routine practice.
Alglucosidase alfa
4000 L alglucosidase alfa administered by IV infusion at the same dose and dose regimen used for the patient's routine treatment prior to the study for 52 weeks.
Interventions
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Alglucosidase alfa
4000 L alglucosidase alfa administered by IV infusion at the same dose and dose regimen used for the patient's routine treatment prior to the study for 52 weeks.
Other Intervention Names
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Eligibility Criteria
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Inclusion Criteria
1. The participant and/or their parent/legal guardian was willing and able to provide signed informed consent.
2. The participant might be at least 1 year of age at the time of informed consent.
3. The participant had a diagnosis of Pompe disease and might have received treatment with 160 L alglucosidase alfa prior to screening.
4. The participant, if female and of childbearing potential, might have a negative pregnancy test (urine beta-human chorionic gonadotropin) at baseline. Note: all female participants of childbearing potential and sexually mature males might agree to use a medically accepted method of contraception throughout the study.
Exclusion Criteria
1. The participant had within the past 3 months received or was currently receiving any investigational product other than 160 L alglucosidase alfa or was currently participating in another clinical treatment study.
2. The participant, in the opinion of the Investigator, was clinically unstable and would not be expected to survive to completion of the 52-week treatment period.
3. The participant and/or their parent/legal guardian, in the opinion of the Investigator, was unable to adhere to the requirements of the study.
1 Year
ALL
No
Sponsors
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Genzyme, a Sanofi Company
INDUSTRY
Responsible Party
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Principal Investigators
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Medical Monitor
Role: STUDY_DIRECTOR
Genzyme, a Sanofi Company
Locations
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Birmingham, Alabama, United States
Phoenix, Arizona, United States
Little Rock, Arkansas, United States
Oakland, California, United States
Orange, California, United States
San Diego, California, United States
San Jose, California, United States
Stanford, California, United States
Aurora, Colorado, United States
Washington D.C., District of Columbia, United States
Gainesville, Florida, United States
Miami, Florida, United States
Decatur, Georgia, United States
Chicago, Illinois, United States
Indianapolis, Indiana, United States
Kansas City, Kansas, United States
Louisville, Kentucky, United States
Baton Rouge, Louisiana, United States
Shreveport, Louisiana, United States
Boston, Massachusetts, United States
Detroit, Michigan, United States
Grand Rapids, Michigan, United States
Minneapolis, Minnesota, United States
Jackson, Mississippi, United States
Columbia, Missouri, United States
Kansas City, Missouri, United States
St Louis, Missouri, United States
Omaha, Nebraska, United States
Las Vegas, Nevada, United States
New Brunswick, New Jersey, United States
Glens Falls, New York, United States
New York, New York, United States
Rochester, New York, United States
Syracuse, New York, United States
Valhalla, New York, United States
Asheville, North Carolina, United States
Durham, North Carolina, United States
Cincinnati, Ohio, United States
Cleveland, Ohio, United States
Philadelphia, Pennsylvania, United States
Pittsburgh, Pennsylvania, United States
Providence, Rhode Island, United States
Memphis, Tennessee, United States
Nashville, Tennessee, United States
Austin, Texas, United States
Fort Worth, Texas, United States
Salt Lake City, Utah, United States
Norfolk, Virginia, United States
Seattle, Washington, United States
Morgantown, West Virginia, United States
Miliwaukee, Wisconsin, United States
Countries
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References
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Duong T, Kishnani PS, An Haack K, Foster MC, Gibson JB, Wilson C, Hahn SH, Hillman R, Kronn D, Leslie ND, Pena LDM, Sparks SE, Stockton DW, Tanpaiboon P, Day JW; Pompe ADVANCE Study Consortium. Motor Responses in Pediatric Pompe Disease in the ADVANCE Participant Cohort. J Neuromuscul Dis. 2022;9(6):713-730. doi: 10.3233/JND-210784.
Byrne BJ, Colan SD, Kishnani PS, Foster MC, Sparks SE, Gibson JB, An Haack K, Stockton DW, Pena LDM, Hahn SH, Johnson J, Tanpaiboon PX, Leslie ND, Kronn D, Hillman RE, Wang RY; Pompe ADVANCE Study Consortium. Cardiac responses in paediatric Pompe disease in the ADVANCE patient cohort. Cardiol Young. 2022 Mar;32(3):364-373. doi: 10.1017/S1047951121002079. Epub 2021 Aug 23.
Kishnani PS, Gibson JB, Gambello MJ, Hillman R, Stockton DW, Kronn D, Leslie ND, Pena LDM, Tanpaiboon P, Day JW, Wang RY, Goldstein JL, An Haack K, Sparks SE, Zhao Y, Hahn SH; Pompe ADVANCE Study Consortium. Clinical characteristics and genotypes in the ADVANCE baseline data set, a comprehensive cohort of US children and adolescents with Pompe disease. Genet Med. 2019 Nov;21(11):2543-2551. doi: 10.1038/s41436-019-0527-9. Epub 2019 May 14.
Hahn SH, Kronn D, Leslie ND, Pena LDM, Tanpaiboon P, Gambello MJ, Gibson JB, Hillman R, Stockton DW, Day JW, Wang RY, An Haack K, Shafi R, Sparks S, Zhao Y, Wilson C, Kishnani PS; Pompe ADVANCE Study Consortium. Efficacy, safety profile, and immunogenicity of alglucosidase alfa produced at the 4,000-liter scale in US children and adolescents with Pompe disease: ADVANCE, a phase IV, open-label, prospective study. Genet Med. 2018 Oct;20(10):1284-1294. doi: 10.1038/gim.2018.2. Epub 2018 Mar 22.
Other Identifiers
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EFC12720
Identifier Type: OTHER
Identifier Source: secondary_id
AGLU09411
Identifier Type: -
Identifier Source: org_study_id