Phase 4
Completed N=15
A Study Evaluating Glycosphingolipid Clearance in Patients Treated With Agalsidase Alfa Who Switch to Agalsidase Beta
Source: ClinicalTrials.gov NCT01650779 ↗Enrolled (actual)
15
Serious AEs
0.0%
Results posted
Jul 2014
Primary outcomePrimary: Percent Change From Baseline in Plasma Deacylated Globotriaosylceramide (Lyso-GL-3) at Month 2, 4 and 6 — -31.71; -39.04; -39.54 percent change — p=0.0002
Summary
This is an exploratory study to evaluate changes in glycosphingolipid levels and other (exploratory) Fabry disease parameters in male Fabry disease participants who were previously treated with agalsidase alfa (Replagal®) 0.2 milligram per kilogram (mg/kg) every two weeks (q2w) and who are being switched to agalsidase beta (Fabrazyme®) 1.0 mg/kg q2w.
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Percent Change From Baseline in Plasma Deacylated Globotriaosylceramide (Lyso-GL-3) at Month 2, 4 and 6 |
-31.71; -39.04; -39.54 | 0.0002 sig |
| SECONDARY Percent Change From Baseline in Plasma Globotriaosylceramide (GL-3) at Month 2, 4 and 6 |
-10.33; -12.80; -17.89 | 0.1178 |
| SECONDARY Percent Change From Baseline in Urine GL-3 at Month 2, 4, and 6 |
-44.71; -41.49; -33.75 | 0.5811 |
| SECONDARY Percent Change From Baseline in Gastrointestinal (GI) Symptoms (Abdominal Pain, Abdominal Distention, and Bowel Irregularities) at Month 2, 4, and 6 |
— | — |
Eligibility Criteria
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
Data sourced from ClinicalTrials.gov (NCT01650779). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication. Informational only — not medical advice.