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Sponsor developing new product
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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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| SIG-005 | Experimental | SIG-005 is comprised of human native alpha-L-iduronidase enzyme (hIDUA) producing spheres |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| SIG-005 (hIDUA Producing Spheres) | Combination Product | Laparoscopic administration of SIG-005 spheres, an encapsulated allogeneic cell therapy genetically modified with a non-viral vector to produce hIDUA |
| Measure | Description | Time Frame |
|---|---|---|
| Number of patients with clinically significant changes from baseline in physical examination | Safety and tolerability will be assessed by evaluating the number of patients with clinically significant changes from baseline in physical examination as assessed by an assessment of general appearance (head, eyes, ears, nose, and throat), as well as review of cardiovascular, dermatologic, gastrointestinal, genitourinary, lymphatic, musculoskeletal, neurologic and respiratory systems. Directed physical examinations at protocol-specified visits will be based on the patient's clinical status and will include general appearance, cardiovascular, gastrointestinal, neurologic, and respiratory assessments. Clinically significant changes from baseline will be captured as AEs. | Baseline up to 5 years |
| Number of patients with clinically significant changes in vital signs from baseline | Safety and tolerability will be assessed by evaluating the number of patients with clinically significant changes from baseline in vital signs, including temperature, respiratory rate, seated blood pressure, and pulse. | Baseline up to 5 years |
| Number of patients with clinically significant changes in total (IgG) IDUA antibody titres from baseline | Safety and tolerability will be assessed by evaluating the number of patients with clinically significant changes from baseline in total (IgG) antibody titres against IDUA | Baseline up to 5 years |
| Number of patients with clinically significant changes in clinical laboratory tests from baseline | Safety and tolerability will be assessed by evaluating the number of patients with clinically significant changes from baseline in clinical laboratory tests, including hematology, serum chemistry, and urinalysis. | Baseline up to 5 years |
| Number of patients with clinically significant changes in treatment-emergent adverse events (TEAE) from baseline, as assessed by CTCAE v5.0. |
| Measure | Description | Time Frame |
|---|---|---|
| To evaluate and Characterize effect of SIG-005 in levels of α-L-iduronidase (IDUA) in blood after administration of SIG-005 | To evaluate and characterize levels of IDUA and leukocyte count in blood plasma from time of baseline up to 5 years post-administration of SIG-005. | Baseline up to 5 years |
| To evaluate and Characterize effect of SIG-005 on glycosaminoglycans (GAG) levels in urine following administration of SIG-005. |
| Measure | Description | Time Frame |
|---|---|---|
| To assess the effect of SIG-005 on health assessment and health-related quality of life | The effect of SIG-005 on health and health-related quality of life will be measured as changes from baseline in responses to the 12-Item Short Form Health Survey (SF-12) and the 5-level EuroQuol EQ-5D-5L questionnaire | Baseline up to 5 years |
Inclusion Criteria:
Exclusion Criteria:
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Clinical Study Site | Porto Alegre | Brazil | ||||
| Clinical Study Site |
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| ID | Term |
|---|---|
| D008059 | Mucopolysaccharidosis I |
| ID | Term |
|---|---|
| D009083 | Mucopolysaccharidoses |
| D002239 | Carbohydrate Metabolism, Inborn Errors |
| D008661 | Metabolism, Inborn Errors |
| D030342 | Genetic Diseases, Inborn |
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Safety and tolerability will be assessed by evaluating the number of patients with clinically significant changes from baseline in TEAEs as assessed by CTCAE v5.0. |
| Baseline up to 5 years |
Total GAG, HS and DS levels in urine and any clinically significant changes will be assessed from time of baseline up to 5 years post-administration of SIG-005. |
| Baseline up to 5 years |
| To evaluate and Characterize effect of SIG-005 MRI images of liver and spleen volume | Non-contrast MRI of the liver and spleen will be performed to determine any clinically significant changes in liver or spleen volume from time of baseline up to 5 years post-administration of SIG-005. | Baseline up to 5 years |
| To evaluate and Characterize effect of SIG-005 on cardiac measurements via electrocardiogram (ECG) | Resting 12-lead ECG assessments will be performed to determine any clinically significant changes in cardiac measurements from time of baseline up to 5 years post-administration of SIG-005. | Baseline up to 5 years |
| London |
| NW3 2QG |
| United Kingdom |
| Clinical Study Site | Salford | United Kingdom |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D016464 | Lysosomal Storage Diseases |
| D017520 | Mucinoses |
| D003240 | Connective Tissue Diseases |
| D017437 | Skin and Connective Tissue Diseases |
| D008659 | Metabolic Diseases |
| D009750 | Nutritional and Metabolic Diseases |