A Study to Evaluate the Efficacy and Safety of Alglucosidase Alfa Produced at the 4000 L Scale for Pompe Disease

NCT01526785 | Terminated Phase 4 Results DMC

• 2 other identifiers: AGLU09411EFC12720
• Study Type: interventional
• Target: 113
• Locations: 1 country
• Sites: 51

Brief Summary

The objective of this study was to evaluate the efficacy and safety of treatment with 4000 litre (L) alglucosidase alfa (Lumizyme®) in Pompe participants.

Conditions

Pompe Disease

Outcome Measures

Primary Outcomes (1)

• Percentage of Participants Who Were Clinically Stable or Improved at Week 52

  Clinical stability was defined as absence of death due to disease progression or new dependency on invasive ventilation and; decline in cardiac status, motor function, and pulmonary function from baseline.

  Week 52

Secondary Outcomes (6)

• Survival Rate at Week 52

  Week 52

• Invasive Ventilator-Free Survival Rate at Week 52

  Week 52

• Change From Baseline on Left Ventricular Mass Z-Score (LVM-Z) at Week 52

  Baseline, Week 52

• Change From Baseline on Gross Motor Function Measure-88 (GMFM-88) at Week 52

  Baseline, Week 52

• Change From Baseline in Forced Vital Capacity (FVC) at Week 52- At Supine Position

  Baseline, Week 52

Study Arms (1)

Alglucosidase alfa

EXPERIMENTAL

Alglucosidase alfa (4000 L scale) intravenous (IV) infusion administered for 52 weeks as per physician's routine practice.

Drug: Alglucosidase alfa

Interventions

Alglucosidase alfa DRUG

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.

Also known as: Lumizyme®

Alglucosidase alfa

Eligibility Criteria

• Age: 1 Year+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• A participant might meet all of the following criteria to be eligible for this study.
• The participant and/or their parent/legal guardian was willing and able to provide signed informed consent.
• The participant might be at least 1 year of age at the time of informed consent.
• The participant had a diagnosis of Pompe disease and might have received treatment with 160 L alglucosidase alfa prior to screening.
• 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.

You may not qualify if:

• A participant who met any of the following criteria were to be excluded from this study.
• 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.
• 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.
• The participant and/or their parent/legal guardian, in the opinion of the Investigator, was unable to adhere to the requirements of the study.

Sponsors & Collaborators

• Genzyme, a Sanofi Company: lead

Study Sites (51)

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Birmingham, Alabama, United States

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Phoenix, Arizona, United States

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Little Rock, Arkansas, United States

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Oakland, California, United States

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Orange, California, United States

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San Diego, California, United States

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San Jose, California, United States

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Stanford, California, United States

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Aurora, Colorado, United States

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Washington D.C., District of Columbia, United States

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Gainesville, Florida, United States

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Miami, Florida, United States

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Decatur, Georgia, United States

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Chicago, Illinois, United States

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Indianapolis, Indiana, United States

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Kansas City, Kansas, United States

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Louisville, Kentucky, United States

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Baton Rouge, Louisiana, United States

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Shreveport, Louisiana, United States

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Boston, Massachusetts, United States

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Detroit, Michigan, United States

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Grand Rapids, Michigan, United States

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Minneapolis, Minnesota, United States

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Jackson, Mississippi, United States

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Columbia, Missouri, United States

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Kansas City, Missouri, United States

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St Louis, Missouri, United States

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Omaha, Nebraska, United States

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Las Vegas, Nevada, United States

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New Brunswick, New Jersey, United States

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Glens Falls, New York, United States

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New York, New York, United States

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Rochester, New York, United States

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Syracuse, New York, United States

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Valhalla, New York, United States

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Asheville, North Carolina, United States

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Durham, North Carolina, United States

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Cincinnati, Ohio, United States

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Cleveland, Ohio, United States

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Philadelphia, Pennsylvania, United States

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Pittsburgh, Pennsylvania, United States

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Providence, Rhode Island, United States

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Memphis, Tennessee, United States

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Nashville, Tennessee, United States

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Austin, Texas, United States

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Fort Worth, Texas, United States

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Salt Lake City, Utah, United States

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Norfolk, Virginia, United States

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Seattle, Washington, United States

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Morgantown, West Virginia, United States

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Miliwaukee, Wisconsin, United States

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Related Publications (4)

• 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.

  PMID: 36214004 DERIVED

• 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.

  PMID: 34420548 DERIVED

• 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.

  PMID: 31086307 DERIVED

• 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.

  PMID: 29565424 DERIVED

MeSH Terms

Conditions

Glycogen Storage Disease Type II

Interventions

GAA protein, human

Condition Hierarchy (Ancestors)

Lysosomal Storage Diseases, Nervous System; Brain Diseases, Metabolic, Inborn; Brain Diseases, Metabolic; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Metabolism, Inborn Errors; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Glycogen Storage Disease; Carbohydrate Metabolism, Inborn Errors; Lysosomal Storage Diseases; Metabolic Diseases; Nutritional and Metabolic Diseases

Results Point of Contact

• Title: Trial Transparency Team
• Organization: Sanofi

Contact-US@sanofi.com

Study Officials

• Medical Monitor

  Genzyme, a Sanofi Company

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: OTHER
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 4
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: February 2, 2012
• First Posted: February 6, 2012
• Study Start: March 1, 2012
• Primary Completion: December 1, 2014
• Study Completion: December 1, 2014
• Last Updated: December 8, 2015
• Results First Posted: October 27, 2015

Record last verified: 2015-11

Locations

US (51)