NCT04541628

Safety & Efficacy of Encapsulated Allogeneic FVIII Cell Therapy in Haemophilia A

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)

• Status: Terminated
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 3 (actual)
• Sponsor: Eli Lilly and Company · Industry
• Sex: Male
• Age: 18 Years
• Healthy volunteers: Not accepted

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.

Conditions

• Hemophilia A

Interventions

Type	Name	Description
COMBINATION_PRODUCT	SIG-001	Laparoscopic administration of SIG-001 spheres, an encapsulated allogeneic cell therapy genetically modified with a non-viral vector to produce BDD-hFVIII.

Timeline

• Start date: 2020-09-28
• Primary completion: 2022-10-28
• Completion: 2023-01-09
• First posted: 2020-09-09
• Last updated: 2024-09-04
• Results posted: 2024-09-04

Locations

6 sites across 2 countries: United States, United Kingdom

Regulatory

• FDA-regulated drug study