US, multicenter, cohort, open label observational study with primary data collection. Ancillary protocol-specified procedures to address the study objectives (eg, assessment of ADA) may be considered outside the standard of care for acid sphingomyelinase deficiency (ASMD), but the study methodology remains non-interventional, as the additional collection of data from participants will not dictate treatment. The total overall study duration will be 5 years. The follow-up period will be a minimum of 1 year to a maximum of 3 years. The enrollment period will be up to 4 years, to allow a minimum of 1 year of follow-up for the last participant enrolled.
This is a hybrid study design. Patients need not travel to existing study sites in order to enroll.
Study Type
OBSERVATIONAL
Enrollment
10
This study will not administer any treatment, only observe the treatment as prescribed in real-world clinical practice.
Ann and Robert H Lurie Children's Hospital of Chicago- Site Number: 001002
Chicago, Illinois, United States
RECRUITINGChildren's Hospital Medical Center- Site Number: 001003
Cincinnati, Ohio, United States
RECRUITINGNationwide Children's Hospital - PIN- Site Number : 1001-1
Columbus, Ohio, United States
RECRUITINGPulse InfoFrame US Inc.- Site Number: 001001
Philadelphia, Pennsylvania, United States
RECRUITINGDell Children's Medical Center- Site Number : 1001-2
Austin, Texas, United States
RECRUITINGNumber of cases in the exposed population with AEs, SAEs, and AESIs (including moderate/severe or recurrent hypersensitivity reactions and infusion-associated reactions)
Time frame: Baseline to 3 years
Presence and titers of serum anti-olipudase alfa IgG anti-drug antibodies (ADA) (and IgE ADA in cases of moderate/severe or recurrent reactions)
Time frame: Baseline to 3 years
Number of cases in the exposed population with abnormal laboratory test results and vital signs
Time frame: Baseline to 3 years
Trial Transparency email recommended (Toll free for US & Canada)
CONTACT
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