Safety and Efficacy of Encapsulated Allogeneic MPS-1 Therapy

• Research type

  Research Study

• Full 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)

• IRAS ID

  297897

• Contact name

  Derralynn Hughes

• Contact email

  derralynnhughes@nhs.net

• Sponsor organisation

  Sigilon Therapeutics, Inc.

• Eudract number

  2021-002025-14

• Clinicaltrials.gov Identifier

  NCT05665036

• Duration of Study in the UK

  6 years, 3 months, 31 days

• Research summary

  This is a research study of an experimental product called SIG-005 (commonly called “hIDUA producing spheres”), which is being developed for patients with attenuated MPS-1 (also called Hurler-Scheie or Scheie Syndrome). This will be the first study of SIG-005 in humans.

  SIG-005 looks like small spheres that are approximately 1.5mm in diameter or approximately the size of the tip of a ballpoint pen. The spheres are made of alginate and thousands of human cells. Alginate is a natural material that comes from seaweed and is already used in some foods and medical products. The alginate forms a protective layer around the cells and shields the cells from the immune system. The human cells have been modified to express an enzyme called human native alpha-L-iduronidase enzyme (hIDUA). The cells are designed to continuously produce hIDUA when placed into the abdominal cavity. The hIDUA is expected to move into the bloodstream, where it can reach tissues and organs and help to break down glycosaminoglycans (GAGs) that build up and cause MPS-1 symptoms.

  The study includes a Screening Period of up to 3 months, a Baseline Assessment Period of 2 weeks, SIG-005 Administration, and a Follow Up period of 5 years.
  
  The purpose of this study is to evaluate:
  • How participants tolerate SIG-005
  • Changes in IDUA activity level after SIG-005, and how long these changes last
  • Changes in GAG levels after SIG-005, and how long these changes last
  • How SIG-005 affects heart and lung function, physical and cognitive abilities,
  and liver and spleen volume
  • How SIG-005 affects overall health and quality of life

• REC name

  London - West London & GTAC Research Ethics Committee

• REC reference

  21/LO/0472

• Date of REC Opinion

  1 Sep 2021

• REC opinion

  Further Information Favourable Opinion