Actively Recruiting
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)
Led by Sanofi · Updated on 2025-10-31
10
Participants Needed
5
Research Sites
247 weeks
Total Duration
On this page
Sponsors
S
Sanofi
Lead Sponsor
P
Pulse Infoframe Ltd.
Collaborating Sponsor
AI-Summary
What this Trial Is About
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.
CONDITIONS
Official Title
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)
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Participant must have ASMD type A/B or B and be less than 2 years old at treatment start, or have ASMD type A with no age limit
- Participant must weigh at least 2 kg
- Documented diagnosis of ASMD by blood cells, fibroblasts, lymphocytes, or genetic testing
- Signed informed consent from parent or legal guardian before any study procedures
- Participant is eligible to start or has received only the first dose of olipudase alfa with available clinical, lab, and antibody data
You will not qualify if you...
- Received an investigational drug within 30 days or 5 half-lives before consent and enrollment
- Deemed unsuitable for participation by investigator due to medical, clinical, or compliance reasons
- Immediate family member of study site employees or individuals involved in the study conduct
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Trial Site Locations
Total: 5 locations
1
Ann and Robert H Lurie Children's Hospital of Chicago- Site Number: 001002
Chicago, Illinois, United States, 60611
Actively Recruiting
2
Children's Hospital Medical Center- Site Number: 001003
Cincinnati, Ohio, United States, 45229-3039
Actively Recruiting
3
Nationwide Children's Hospital - PIN- Site Number : 1001-1
Columbus, Ohio, United States, 43205-2664
Actively Recruiting
4
Pulse InfoFrame US Inc.- Site Number: 001001
Philadelphia, Pennsylvania, United States, 19104
Actively Recruiting
5
Dell Children's Medical Center- Site Number : 1001-2
Austin, Texas, United States, 78723
Actively Recruiting
Research Team
T
Trial Transparency email recommended (Toll free for US & Canada)
CONTACT
How is the study designed?
Study Type
OBSERVATIONAL
Masking
N/A
Allocation
N/A
Model
N/A
Primary Purpose
N/A
Number of Arms
1
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