Safety and Efficacy of Encapsulated Allogeneic MPS-1 Therapy

NCT ID: NCT05665036

Last Updated: 2023-04-11

Basic Information

• Recruitment Status: WITHDRAWN
• Clinical Phase: PHASE1/PHASE2
• Study Classification: INTERVENTIONAL
• Study Start Date: 2021-11-15
• Study Completion Date: 2022-12-13

Brief 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

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

SIG-005

SIG-005 is comprised of human native alpha-L-iduronidase enzyme (hIDUA) producing spheres

Group Type: EXPERIMENTAL

SIG-005 (hIDUA Producing Spheres)

Intervention Type: COMBINATION_PRODUCT

Laparoscopic administration of SIG-005 spheres, an encapsulated allogeneic cell therapy genetically modified with a non-viral vector to produce hIDUA

Interventions

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

Intervention Type: COMBINATION_PRODUCT

Eligibility Criteria

Inclusion Criteria

• Able and willing to provide informed consent
• Male or female aged 18 or older
• Diagnosis of attenuated MPS-1 (Hurler-Scheie or Scheie)
• Alpha-L-iduronidase enzyme activity level of less than 10% of the lower limit of the normal range
• Prior treatment with ERT
• Willing to transition from ERT to SIG-005
• Female patients of childbearing potential with negative pregnancy test
• Use of highly effective method of contraception if applicable

Exclusion Criteria

• A diagnosis of severe MPS-1
• Previous haematopoietic stem cell transplantation (HSCT)
• History of elevated total (IgG) anti-IDUA antibody
• Use of assistive respiratory devices
• Unable to walk independently
• History of allergic reaction or anaphylaxis to recombinant hIDUA
• Body mass index (BMI) ≥35
• History of abdominal adhesions, medical history of Crohn's disease, inflammatory bowel disease or any disease that increases the risk of post-operative abdominal adhesions
• Significant underlying disease or comorbidities that are a contraindication for general anaesthesia or laparoscopic procedure
• Pregnant or lactating patients
• Prior administration of a gene therapy product
• Participation in another investigational medicine or device study
• Abnormal laboratory values as defined in the protocol
• Active alcoholism or drug addiction during the 12 months before the screening visit
• Active malignancy or history of malignancy in the 5 years prior to study entry
• Recent COVID-19 infection: within 60 days of recovery from infection
• Vaccination(s) within the last 60 days (including vaccines for SARS-CoV-2/COVID- 19)

• Minimum Eligible Age: 18 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Sigilon Therapeutics, Inc.

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Locations

Clinical Study Site

Porto Alegre, , Brazil

Clinical Study Site

London, , United Kingdom

Clinical Study Site

Salford, , United Kingdom

Countries

Brazil; United Kingdom

Other Identifiers

SIG-005-121

Identifier Type: -

Identifier Source: org_study_id