Phase 4
Completed N=8
A Prospective Study to Investigate Safety and Tolerability of Shorter Infusion of Fabrazyme
Fabry's Disease
Source: ClinicalTrials.gov NCT06019728 ↗
Enrolled (actual)
8
Serious AEs
0.0%
Results posted
Oct 2025
Primary outcomePrimary: Percent Reduction in Shortest Tolerated Infusion Duration From Pre-study Average of Recent 3 Infusions — 84.19 percent
◆ Published Evidence
No publication linked
No peer-reviewed publication reporting this trial's results has been linked yet. This can indicate results are unpublished — a known publication-bias signal. We re-check periodically.
Summary
This Phase 4 study will evaluate the safety and tolerability of Fabrazyme at current approved dose with increases in the infusion rate and reduced infusion volume. This study aims to generate data to provide the guidance on how infusion rate can be safely increased and minimize the burden of the life-long treatment with Fabrazyme.
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Percent Reduction in Shortest Tolerated Infusion Duration From Pre-study Average of Recent 3 Infusions |
84.19 | — |
| PRIMARY Shortest Actual Tolerated Infusion Duration |
20.00 | — |
| SECONDARY Number of Participants Who Achieved the Shortest Planned Duration of Infusion Time Without Experiencing Any Infusion Associated Reaction |
8 | — |
| SECONDARY Number of Participants Who Achieved the Shortest Planned Duration of Infusion Time Without Experiencing a Second Infusion Associated Reaction |
8 | — |
| SECONDARY Number of Participants Who Tolerated Planned Infusion Duration Shorter Than 90 Minutes Without Experiencing Any Infusion Associated Reaction |
8 | — |
| SECONDARY Number of Participants Who Tolerated Planned Infusion Duration Shorter Than 90 Minutes Without Experiencing a Second Infusion Associated Reaction |
8 | — |
| SECONDARY Number of Participants Who Tolerated Each Planned Infusion Duration Without Experiencing Any Infusion Associated Reaction |
6; 7; 8; 8; 8 | — |
| SECONDARY Number of Participants Who Tolerated Each Planned Infusion Duration Without Experiencing a Second Infusion Associated Reaction |
6; 7; 8; 8; 8 | — |
Eligibility Criteria
Inclusion Criteria
- Participants with confirmed diagnosis of FD who are ≥2 and ≤65 years of age at the time of signing the informed consent form (ICF) or assent, if applicable.
- Cohort 1: female participants with body weight ≥30 kg who have been treated with Fabrazyme for at least 3 months without IARs during the most recent 3 infusions.
- Cohort 2: non-classic male participants with body weight ≥30 kg who have been treated with Fabrazyme for at least 3 months without IARs during the most recent 3 infusions.
- Cohort 3: classic male participants with body weight ≥30 kg who have been treated with Fabrazyme for at least 3 months without IARs during the most recent 3 infusions.
- Cohort 4: participants with body weight <30 kg who have been treated with Fabrazyme for at least 3 months without IARs during the most recent 3 infusions.
Women of childbearing potential must use a highly effective method of contraception through the study.
Exclusion Criteria
- Female participants who are pregnant or breastfeeding.
- History of significant allergic disease or hypersensitivity to Fabrazyme or other medicinal products.
- Contraindication to Fabrazyme or any of the premedications or rescue medications (diphenhydramine, loratadine, cetirizine, fexofenadine, acetaminophen, montelukast, dexamethasone).
- Any other medical condition considered to make the increased infusion rate not tolerable at the Investigator's discretion.
The above information is not intended to contain all considerations relevant to a potential participation in a clinical trial.
Data sourced from ClinicalTrials.gov (NCT06019728). 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.