Trial record 1 of 1 for:
agal19412
A Study Evaluating Glycosphingolipid Clearance in Patients Treated With Agalsidase Alfa Who Switch to Agalsidase Beta
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The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. |
ClinicalTrials.gov Identifier: NCT01650779 |
Recruitment Status :
Completed
First Posted : July 26, 2012
Results First Posted : July 10, 2014
Last Update Posted : July 10, 2014
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Sponsor:
Genzyme, a Sanofi Company
Information provided by (Responsible Party):
Sanofi ( Genzyme, a Sanofi Company )
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Study Type | Interventional |
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Study Design | Allocation: N/A; Intervention Model: Single Group Assignment; Masking: None (Open Label); Primary Purpose: Treatment |
Condition |
Fabry Disease |
Intervention |
Biological: Agalsidase beta |
Enrollment | 15 |
Participant Flow
Recruitment Details | The study was conducted at 6 centers in the United States of America between April 30, 2012 and March 15, 2013. |
Pre-assignment Details | A total of 16 participants were screened of which 1 participant was screen failure. A total of 15 participants were enrolled in this study. |
Arm/Group Title | Agalsidase Beta |
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Commercially available agalsidase beta (Fabrazyme ®) 1.0 milligram per kilogram (mg/kg) administered as an intravenous infusion every 2 weeks (q2w) up to Month 6. |
Period Title: Overall Study | |
Started | 15 |
Completed | 14 |
Not Completed | 1 |
Reason Not Completed | |
Withdrawal by Subject | 1 |
Baseline Characteristics
Arm/Group Title | Agalsidase Beta | |
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Commercially available agalsidase beta 1.0 mg/kg administered as an intravenous infusion q2w up to Month 6. | |
Overall Number of Baseline Participants | 15 | |
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All enrolled participants were included in the analysis.
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Age, Continuous
Mean (Standard Deviation) Unit of measure: Years |
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Number Analyzed | 15 participants | |
28.5 (16.11) | ||
Sex: Female, Male
Measure Type: Count of Participants Unit of measure: Participants |
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Number Analyzed | 15 participants | |
Female |
0 0.0%
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Male |
15 100.0%
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Race/Ethnicity, Customized
Measure Type: Number Unit of measure: Participants |
Number Analyzed | 15 participants |
Caucasian | 13 | |
Hispanic | 1 | |
Other | 1 | |
Duration of Fabry Disease
[1] Median (Full Range) Unit of measure: Years |
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Number Analyzed | 15 participants | |
9.3
(2 to 19)
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[1]
Measure Description: Duration of Fabry disease was calculated from date of initial diagnosis of Fabry disease to date of the first study infusion. Number of participants analyzed for this baseline measure were 14, as one participant did not have information on date of initial diagnosis of Fabry disease.
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Method of Diagnosis of Fabry Disease
[1] Measure Type: Number Unit of measure: Participants |
Number Analyzed | 15 participants |
Genotype | 14 | |
Leukocyte alfa-galactosidase A (GAL) activity | 11 | |
Plasma alfa-GAL activity | 9 | |
[1]
Measure Description: Number of participants with each method (genotype, leukocyte alfa-galactosidase A [GAL] activity, and plasma alfa-GAL activity) are reported. Some participants were diagnosed by more than 1 method, hence reported under more than 1 category.
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Outcome Measures
Adverse Events
Limitations and Caveats
This is considered to be an exploratory study for the following reasons: it was based on a small number of participants and has been designed as an open-label, single-arm study as opposed to a two-arm crossover design.
More Information
Principal Investigators are NOT employed by the organization sponsoring the study.
There IS an agreement between Principal Investigators and the Sponsor (or its agents) that restricts
the PI's rights to discuss or publish trial results after the trial is completed.
If no publication has occurred within 12 months of the completion of the study, the Investigator shall have the right to publish/present independently the results of the study. The Investigator shall provide the Sponsor with a copy of any such presentation/publication for comment at least 30 days before any presentation/submission for publication. If requested by the Sponsor, any presentation/submission shall be delayed up to 90 days, to allow the Sponsor to preserve its proprietary rights.
Results Point of Contact
Name/Title: | Trial Transparency Team |
Organization: | Sanofi |
EMail: | Contact-us@sanofi.com |
Responsible Party: | Sanofi ( Genzyme, a Sanofi Company ) |
ClinicalTrials.gov Identifier: | NCT01650779 |
Other Study ID Numbers: |
AGAL19412 |
First Submitted: | July 24, 2012 |
First Posted: | July 26, 2012 |
Results First Submitted: | June 9, 2014 |
Results First Posted: | July 10, 2014 |
Last Update Posted: | July 10, 2014 |