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A Study to Evaluate the Efficacy and Safety of Alglucosidase Alfa Produced at the 4000 L Scale for Pompe Disease | Clinical Trials | Clareo Health

TERMINATEDPHASE4

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

A Phase 4 Open Label, Prospective Study in Patients With Pompe Disease to Evaluate The Efficacy and Safety of Alglucosidase Alfa Produced at the 4000L Scale

NCT01526785•Genzyme, a Sanofi Company

View on ClinicalTrials.gov

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.

Eligibility

Age

1 - No limit years

Sex

ALL

Healthy Volunteers

Inclusion Criteria

•A participant might meet all of the following criteria to be eligible for this study.
•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

•A participant who met any of the following criteria were to be excluded from this study.
•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.

Locations (51)

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

Key Dates

Start Date

March 1, 2012

Primary Completion Date

December 1, 2014

Completion Date

December 1, 2014

Last Updated

December 8, 2015

Enrollment

113

ACTUAL participants

Conditions

Pompe Disease

Interventions

Alglucosidase alfa

DRUG

A Global Prospective Observational Registry of Patients With Pompe Disease

NCT06121011

Pompe Disease

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RECRUITING

Determination of CRIM Status and Longitudinal Follow-up of Individuals With Pompe Disease

NCT01665326

Pompe Disease

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ACTIVE_NOT_RECRUITINGEARLY_PHASE1

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Data Source & Attribution

This clinical trial information is sourced from ClinicalTrials.gov, a service of the U.S. National Institutes of Health.

ClinicalTrials.gov last update: December 8, 2015Data synced to Clareo: March 29, 2026

Modifications: This data has been reformatted for display purposes. Eligibility criteria have been parsed into inclusion/exclusion sections. Location data has been geocoded to enable distance-based search. For the authoritative and most current information, please visit ClinicalTrials.gov.

Neither the United States Government nor Clareo Health make any warranties regarding the data. Check ClinicalTrials.gov frequently for updates.

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

Inclusion Criteria

Exclusion Criteria

A Global Prospective Observational Registry of Patients With Pompe Disease

Determination of CRIM Status and Longitudinal Follow-up of Individuals With Pompe Disease

Study to Assess the Safety, Tolerability, PK and PD of ABX1100

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