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Main
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Note: This record shows only 22 elements of the WHO Trial Registration Data Set. To view changes that have been made to the source record, or for additional information about this trial, click on the URL below to go to the source record in the primary register. |
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Register:
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ClinicalTrials.gov |
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Last refreshed on:
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19 October 2017 |
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Main ID: |
NCT01526785 |
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Date of registration:
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02/02/2012 |
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Prospective Registration:
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Yes |
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Primary sponsor: |
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Public title:
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A Study to Evaluate the Efficacy and Safety of Alglucosidase Alfa Produced at the 4000 L Scale for Pompe Disease
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Scientific title:
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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 |
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Date of first enrolment:
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March 2012 |
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Target sample size:
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113 |
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Recruitment status: |
Terminated |
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URL:
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https://clinicaltrials.gov/show/NCT01526785 |
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Study type:
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Interventional |
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Study design:
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Phase:
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Phase 4
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Countries of recruitment
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United States
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Contacts
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Name:
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Medical Monitor |
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Address:
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Telephone:
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Email:
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Affiliation:
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Genzyme, a Sanofi Company |
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Key inclusion & exclusion criteria
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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.
Age minimum:
1 Year
Age maximum:
N/A
Gender:
All
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Health Condition(s) or Problem(s) studied
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Pompe Disease
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Intervention(s)
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Drug: Alglucosidase alfa
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Primary Outcome(s)
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Percentage of Participants Who Were Clinically Stable or Improved at Week 52
[Time Frame: Week 52]
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Secondary Outcome(s)
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Survival Rate at Week 52
[Time Frame: Week 52]
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Change From Baseline on Left Ventricular Mass Z-Score (LVM-Z) at Week 52
[Time Frame: Baseline, Week 52]
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Change From Baseline in Forced Vital Capacity (FVC) at Week 52- At Sitting Position
[Time Frame: Baseline, Week 52]
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Change From Baseline in Forced Vital Capacity (FVC) at Week 52- At Supine Position
[Time Frame: Baseline, Week 52]
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Change From Baseline on Gross Motor Function Measure-88 (GMFM-88) at Week 52
[Time Frame: Baseline, Week 52]
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Invasive Ventilator-Free Survival Rate at Week 52
[Time Frame: Week 52]
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Secondary ID(s)
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EFC12720
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AGLU09411
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Source(s) of Monetary Support
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Please refer to primary and secondary sponsors
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