Safety and Efficacy of Encapsulated Allogeneic MPS-1 Therapy

MPS I Clinical Trial

Official title:
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Status Withdrawn

Clinical Trial 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.

Clinical Trial Description

n/a

Study Design

Related Conditions & MeSH terms

MPS I

Clinical Trial Details

NCT number	NCT05665036
Study type	Interventional
Source	Sigilon Therapeutics, Inc.
Contact
Status	Withdrawn
Phase	Phase 1/Phase 2
Start date	November 15, 2021
Completion date	December 13, 2022

View Details

See also
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