NCT05665036

Safety and Efficacy of Encapsulated Allogeneic MPS-1 Therapy

A Phase 1/2 Open-Label, Sequential Dose-Escalation, Safety, Tolerability and Efficacy Study of SIG-005 in Adult Patients With Mucopolysaccharidoses 1 (MPS-1)

• Status: Withdrawn
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 0 (actual)
• Sponsor: Sigilon Therapeutics, Inc. · Industry
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

Summary

SIG-005-121 is a FIH, Phase 1/2, multi-centre, open-label, sequential dose-escalating study to assess the safety, tolerability, and preliminary efficacy of SIG-005 in adults with MPS-1. The study will evaluate up to three ascending dose levels of SIG-005 in male and female patients with attenuated MPS-1 (Scheie or Hurler-Scheie), 18 years of age or greater, who received Enzyme Replacement Therapy (ERT) for a minimum of 12 months prior to the study entry. Each cohort will include 3 patients.

Conditions

• MPS I
• MPS1-S
• MPS1-HS

Interventions

Type	Name	Description
COMBINATION_PRODUCT	SIG-005 (hIDUA Producing Spheres)	Laparoscopic administration of SIG-005 spheres, an encapsulated allogeneic cell therapy genetically modified with a non-viral vector to produce hIDUA

Timeline

• Start date: 2021-11-15
• Primary completion: 2022-12-13
• Completion: 2022-12-13
• First posted: 2022-12-27
• Last updated: 2023-04-11

Locations

3 sites across 2 countries: Brazil, United Kingdom

Regulatory

• FDA-regulated drug study