A Real-world Long-term Safety and Immunogenicity Study of Olipudase Alfa Therapy in Pediatric Patients Less Than 2 Years of Age With Acid Sphingomyelinase Deficiency (ASMD)
Olipudase alfa: This study will not administer any treatment, only observe the treatment as prescribed in real-world clinical practice.
Study summary
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.
Eligibility
Sex
ALL
Min age
—
Max age
2 Years
Healthy volunteers
No
Inclusion Criteria:
* The participant must have ASMD type A/B or B and must be \<2 years of age at the time of treatment initiation, OR ASMD type A (without age restriction).
* The participant must weigh ≥ 2 kg \[The United States Prescribing Information (USPI)\] for olipudase alfa specifies this minimum weight for infants receiving olipudase alfa).
* The participant must have documented ASMD, as determined in peripheral leukocytes, cultured fibroblasts, or lymphocytes and/or by genotype determination.
* Signed informed consent must be provided by the participant's parent(s)/legal guardian(s), including compliance with the requirements and restrictions listed in the informed consent form (ICF) and in this protocol. The signed ICF must be provided before any protocol-related procedures are performed.
* The participant is eligible to start olipudase alfa enzyme replacement therapy or has received the first dose (and no more) of olipudase alfa, and has retrievable clinical, laboratory, and ADA data.
Exclusion Criteria:
* The participant has received an investigational drug within 30 days or 5 drug half-lives before signature of the ICF and study enrollment.
* The participant is not suitable for participation for reasons determined by the Investigator, including medical or clinical conditions, or potential risk of noncompliance with study procedures.
* The participant is an immediate family member of employees of the study site or other individuals directly involved in study conduct, in conjunction with Section 1.61 of ICH-GCP Ordinance E6.
The above information is not intended to contain all considerations relevant to a patient's potential participation in a clinical trial.
Primary outcome measure(s)
Number of cases in the exposed population with AEs, SAEs, and AESIs (including moderate/severe or recurrent hypersensitivity reactions and infusion-associated reactions) — 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) — Baseline to 3 years
Number of cases in the exposed population with abnormal laboratory test results and vital signs — Baseline to 3 years
Trial sites (6)
Facility
City
Region
Status
Ann and Robert H Lurie Children's Hospital of Chicago- Site Number: 001002
Chicago
Illinois
Recruiting
Children's Hospital Medical Center- Site Number: 001003
Cincinnati
Ohio
Recruiting
Nationwide Children's Hospital- Site Number : 1001-1
Columbus
Ohio
Recruiting
Pulse InfoFrame US Inc.- Site Number: 001001
Philadelphia
Pennsylvania
Recruiting
Dell Children's Medical Center- Site Number : 1001-2
Austin
Texas
Recruiting
St. George Regional Hospital, Intermountain Health - Site Number: 1001-3
This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.
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