Safety and Efficacy of Encapsulated Allogeneic MPS-1 Therapy

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.

Timeline

Start

2021-11-15

Primary completion

2022-12-13

Completion

2022-12-13

Outcome

Outcome not reported

Stopped: “Sponsor developing new product”

Drugs

Indications