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NCT05665036
Safety and Efficacy of Encapsulated Allogeneic MPS-1 Therapy
Conditions: MPS I, MPS1-S, MPS1-HS
Sex: All
Ages: 18 Years – N/A
Phase: PHASE1, PHASE2
Sponsor: Sigilon Therapeutics, Inc.
Location: Brazil
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.
Eligibility Criteria
Inclusion Criteria:Able and willing to provide informed consentMale or female aged 18 or olderDiagnosis 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 rangePrior treatment with ERTWilling to transition from ERT to SIG-005Female patients of childbearing potential with negative pregnancy testUse of highly effective method of contraception if applicableExclusion Criteria:A diagnosis of severe MPS-1Previous haematopoietic stem cell transplantation (HSCT)History of elevated total (IgG) anti-IDUA antibodyUse of assistive respiratory devicesUnable to walk independentlyHistory of allergic reaction or anaphylaxis to recombinant hIDUABody mass index (BMI) ≥35History of abdominal adhesions, medical history of Crohn's disease, inflammatory bowel disease or any disease that increases the risk of post-operative abdominal adhesionsSignificant underlying disease or comorbidities that are a contraindication for general anaesthesia or laparoscopic procedurePregnant or lactating patientsPrior administration of a gene therapy productParticipation in another investigational medicine or device studyAbnormal laboratory values as defined in the protocolActive alcoholism or drug addiction during the 12 months before the screening visitActive malignancy or history of malignancy in the 5 years prior to study entryRecent COVID-19 infection: within 60 days of recovery from infectionVaccination(s) within the last 60 days (including vaccines for SARS-CoV-2/COVID- 19)
Source: ClinicalTrials.gov (NCT05665036). StuddyBuddy aggregates publicly available trial information.
