Status:
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)
Lead Sponsor:
Sanofi
Collaborating Sponsors:
Pulse Infoframe Ltd.
Conditions:
Niemann-Pick Diseases
Acid Sphingomyelinase Deficiency
Eligibility:
All Genders
Up to 2 years
Brief 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 ...
Detailed Description
This is a hybrid study design. Patients need not travel to existing study sites in order to enroll.
Eligibility Criteria
Inclusion
- 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
- 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.
Key Trial Info
Start Date :
April 16 2024
Trial Type :
OBSERVATIONAL
Allocation :
ESTIMATED
End Date :
January 15 2029
Estimated Enrollment :
10 Patients enrolled
Trial Details
Trial ID
NCT06192576
Start Date
April 16 2024
End Date
January 15 2029
Last Update
October 31 2025
Active Locations (5)
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1
Ann and Robert H Lurie Children's Hospital of Chicago- Site Number: 001002
Chicago, Illinois, United States, 60611
2
Children's Hospital Medical Center- Site Number: 001003
Cincinnati, Ohio, United States, 45229-3039
3
Nationwide Children's Hospital - PIN- Site Number : 1001-1
Columbus, Ohio, United States, 43205-2664
4
Pulse InfoFrame US Inc.- Site Number: 001001
Philadelphia, Pennsylvania, United States, 19104