NCT04541628

A Phase 1/2 Open-Label, Dose-Escalation, Safety, Tolerability, and Efficacy Study of SIG-001 in Adult Patients With Severe or Moderately-Severe Haemophilia A Without Inhibitors (SIG-001-121)

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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Interventions

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

FacilityCityStateCountry
Clinical Study SiteIndianapolisIndianaUnited States
Clinical Study SiteBostonMassachusettsUnited States
Clinical Study SiteSeattleWashingtonUnited States
Clinical Study SiteLondonUnited Kingdom
Clinical Study SiteManchesterUnited Kingdom
Clinical Study SiteSouthamptonUnited Kingdom

Official Trial Information

View on ClinicalTrials.gov

Data sourced from ClinicalTrials.gov. Last updated: April 14, 2026