Outcome
Type |
Measure |
Description |
Time frame |
Safety issue |
Other |
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 |
|
Primary |
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 |
|
Primary |
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 |
|
Primary |
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 |
|
Primary |
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 |
|
Primary |
Number of patients with clinically significant changes in treatment-emergent adverse events (TEAE) from baseline, as assessed by CTCAE v5.0. |
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 |
|
Secondary |
To evaluate and Characterize effect of SIG-005 in levels of a-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 |
|
Secondary |
To evaluate and Characterize effect of SIG-005 on glycosaminoglycans (GAG) levels in urine following administration of SIG-005. |
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 |
|
Secondary |
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 |
|
Secondary |
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 |
|