Home›Trial›NCT05768386
NCT05768386ENROLLING BY INVITATION

A Long-Term Follow-Up Study in Severe Hemophilia A Subjects Who Received BMN 270 in a Prior BioMarin Clinical Trial (270-401)

Study Summary

The BMN 270 clinical development program consists of multiple interventional studies designed to assess the safety and efficacy of a single infusion of BMN 270 for at least 5 years post-infusion. This long-term follow-up study is needed to help further understand the long-term safety of BMN 270 beyond 5 years and to assess the durability of efficacy.

Conditions
Hemophilia A
Interventions / Drugs
View on ClinicalTrials.gov ↗
Sponsor
BMRN
BioMarin Pharmaceutical
Rare Disease
D49/100
Study Details
Enrollment172 participants
Primary Completion—
Lead SponsorBioMarin Pharmaceutical
Data Retrieved2026-04-11 02:55:23.531545+00:00
What This Trial Means for Biotech Investors
Trial Phase Context
Status & Expected Data

Current status: ENROLLING BY INVITATION.

Enrolled: 172 participants
Sponsor Catalyst Profile

BioMarin Pharmaceutical (BMRN) is the sponsoring company for this trial. BiotechSign currently grades this company D (49/100) based on composite catalyst signals across its full pipeline. This trial is one data point in that overall catalyst picture.

BMRN Full Pipeline →All Companies →
Not financial advice. BiotechSign data is sourced from ClinicalTrials.gov, SEC EDGAR, and other public databases for research and informational purposes only. It does not constitute investment recommendations. Always do your own due diligence before making investment decisions.