Study Summary
SIG-001-121 is a first-in-human (FIH), phase 1/2, multi-centre, open-label, dose escalation study to assess the safety, tolerability, and preliminary efficacy of SIG-001 in adults with severe or moderately severe haemophilia A without inhibitors. Up to three dose cohorts (3 patients each) are planned. Cohort expansions (up to 3 additional patients) may be triggered to collect additional information about safety and efficacy.
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SIG-001COMBINATION_PRODUCT
Laparoscopic administration of SIG-001 spheres, an encapsulated allogeneic cell therapy genetically modified with a non-viral vector to produce BDD-hFVIII.
Study Locations
| Facility | City | State | Country |
|---|---|---|---|
| Clinical Study Site | Indianapolis | Indiana | United States |
| Clinical Study Site | Boston | Massachusetts | United States |
| Clinical Study Site | Seattle | Washington | United States |
| Clinical Study Site | London | United Kingdom | |
| Clinical Study Site | Manchester | United Kingdom | |
| Clinical Study Site | Southampton | United Kingdom |