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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: |
NCT01650779 |
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Date of registration:
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24/07/2012 |
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Prospective Registration:
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No |
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Primary sponsor: |
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Public title:
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A Study Evaluating Glycosphingolipid Clearance in Patients Treated With Agalsidase Alfa Who Switch to Agalsidase Beta
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Scientific title:
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Evaluation of Glycosphingolipid Clearance in Patients Treated With Agalsidase Alfa Who Switch to Agalsidase Beta (The INFORM Study) |
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Date of first enrolment:
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April 2012 |
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Target sample size:
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15 |
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Recruitment status: |
Completed |
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URL:
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https://clinicaltrials.gov/show/NCT01650779 |
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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:
- The participant and/or his parent/legal guardian is willing and able to provide signed
informed consent, and the participant, if less than (<) 18 years of age, is willing to
provide assent if deemed able to do so
- Participant is male and has been treated with agalsidase alfa at 0.2 mg/kg q2w for the
12 months prior to switching to agalsidase beta
- The participant has a confirmed diagnosis of Fabry disease by alfa-galactosidase A
(alfa-GAL) activity and/or genotyping per local standards
- The participant when switched to agalsidase beta receives the labeled dose, that is,
0.9 to 1.1 mg/kg (1 mg/kg) q2w, and must be willing to maintain the labeled dose for
the duration of the study
Exclusion Criteria:
- The participant is on dialysis or is post renal transplantation
- The participant is in end-stage cardiac failure
- The participant and/or his parent or legal guardian, in the opinion of the
investigator, is unable to adhere to the requirements of the study
- The participant has been switched from agalsidase alfa to agalsidase beta and does not
have historical blood and urine samples
Age minimum:
N/A
Age maximum:
N/A
Gender:
Male
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Health Condition(s) or Problem(s) studied
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Fabry Disease
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Intervention(s)
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Biological: Agalsidase beta
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Primary Outcome(s)
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Percent Change From Baseline in Plasma Deacylated Globotriaosylceramide (Lyso-GL-3) at Month 2, 4 and 6
[Time Frame: Baseline, Month 2, 4, 6]
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Secondary Outcome(s)
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Percent Change From Baseline in Plasma Globotriaosylceramide (GL-3) at Month 2, 4 and 6
[Time Frame: Baseline, Month 2, 4, 6]
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Percent Change From Baseline in Gastrointestinal (GI) Symptoms (Abdominal Pain, Abdominal Distention, and Bowel Irregularities) at Month 2, 4, and 6
[Time Frame: Baseline, Month 2, 4, 6]
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Percent Change From Baseline in Urine GL-3 at Month 2, 4, and 6
[Time Frame: Baseline, Month 2, 4, 6]
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Secondary ID(s)
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AGAL19412
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Source(s) of Monetary Support
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Please refer to primary and secondary sponsors
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