See the DrugPatentWatch profile for galafold
What is NCT00925301 (Galafold)?
NCT00925301 is a clinical trial record in ClinicalTrials.gov that involves Galafold (also known as migalastat), a medicine used for Fabry disease in patients with specific genetic mutations.
What does Galafold (migalastat) treat?
Galafold is used for Fabry disease. The key requirement is that a patient’s mutation must be “amenable” to migalastat (the drug’s mechanism depends on the mutation type) [1].
What is the purpose of the NCT00925301 study?
ClinicalTrials.gov records like NCT00925301 typically describe trials to evaluate how well Galafold works and how safe it is in people with Fabry disease, often including outcomes such as clinical measures and biomarker changes [1].
What happens if someone asks whether any Fabry patient can use Galafold?
No. Galafold is generally for patients whose Fabry mutations are eligible (“amenable”) for the drug’s mechanism. If a person’s mutation is not amenable, Galafold is not expected to be effective for that mechanism of action [1].
Where can you check study details for NCT00925301?
The most reliable place to see the trial’s exact design (phase, status, enrollment, endpoints, and locations) is the ClinicalTrials.gov entry for NCT00925301 [1].
Sources
[1] https://clinicaltrials.gov/study/NCT00925301