What clinical trials has Fabrazyme (agalsidase beta) been studied in?
Fabrazyme’s main clinical-trial evidence comes from studies of intravenous agalsidase beta in patients with Fabry disease, with endpoints that typically include changes in disease biomarkers (especially globotriaosylceramide/GL-3), organ-related measures, and clinical outcomes over time. The available trial literature and registries are the basis for its clinical use in Fabry disease.
Which outcomes did Fabrazyme trials measure (GL-3, kidney, heart, neuropathy)?
Across Fabrazyme studies, researchers commonly tracked:
- Biochemical effects, including reductions in GL-3-related measures after dosing.
- Organ involvement progression, particularly kidney function and pain/neuropathy outcomes.
- Cardiac findings and other systemic manifestations associated with Fabry disease.
How long were Fabrazyme trials and what were the typical follow-up periods?
Because Fabrazyme has been used for years, clinical trial programs include both shorter studies to demonstrate biochemical response and longer follow-up to observe sustained effects and progression in Fabry disease manifestations. Follow-up length varies by specific study design (for example, early efficacy/biomarker studies versus longer-term studies focused on durability and progression).
Are there different Fabrazyme trial programs for men vs women or for “classic” vs “late-onset” Fabry?
Fabry disease affects people differently depending on genotype and symptom timing, and women can have disease involvement even if classic presentations are more common in men. Clinical programs for Fabrazyme include analyses across relevant patient subgroups, reflecting the broader Fabry population rather than only one narrow phenotype.
What safety outcomes were reported in Fabrazyme clinical trials?
Key safety monitoring in Fabrazyme trials typically includes infusion-associated reactions and hypersensitivity, plus longer-term tolerability. Because Fabrazyme is a biologic enzyme replacement therapy, trials also monitor immune responses (including the formation of antibodies) and whether immune-related events affect safety or effectiveness.
Were Fabrazyme trials compared against placebo or other enzyme replacement therapies?
Some Fabrazyme studies are placebo-controlled or use designs that compare agalsidase beta against another regimen, while others use open-label or single-arm approaches paired with historical comparisons, depending on feasibility and ethical considerations in rare disease populations.
What should you look for if you’re trying to find the exact NCT trial records?
If your goal is to read the exact protocol, inclusion criteria, endpoints, and results, the most direct route is to search ClinicalTrials.gov by:
- “Fabrazyme” or “agalsidase beta”
- “Fabry disease”
- filter by study status (completed/terminated), and by recruitment dates
This is usually how patients, clinicians, or researchers locate the specific trial identifiers and publications for each study.
Which trial results are considered the key evidence for approvals?
Regulatory approvals for enzyme replacement therapies like Fabrazyme are generally supported by clinical development programs that show consistent biochemical response and clinically meaningful effects on organ systems relevant to Fabry disease, with safety data establishing tolerability and management of immune/infusion-related events.
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Sources:
I don’t have the specific Fabrazyme trial identifiers/publications in the information provided here, so I can’t reliably list exact trial names, NCT numbers, or primary endpoints without risking inaccuracies. If you share what you mean by “clinical trials” (e.g., “show me key Fabrazyme NCT studies,” “paste the trials for kidney outcomes,” or “which trials support FDA approval”), I can produce a precise, citation-backed list.