Long-term safety and immune response of olipudase alfa in young children with acid sphingomyelinase deficiency
A Prospective Observational Study to Assess the Long-term Safety and Immunogenicity of Olipudase Alfa Therapy During Routine Clinical Care in Pediatric Patients Less Than 2 Years of Age With Acid Sphingomyelinase Deficiency
This study is testing the long-term safety and immune response of a treatment called olipudase alfa in young children under 2 years old who have acid sphingomyelinase deficiency.
Quick facts
| Study type | Observational |
|---|---|
| Enrollment | 10 (estimated) |
| Ages | N/A to 2 Years |
| Sex | All |
| Sponsor | Sanofi Industry-sponsored |
| Locations | 5 sites (Chicago, Illinois and 4 other locations) |
| Trial ID | NCT06192576 on ClinicalTrials.gov |
What this trial studies
This observational study aims to assess the long-term safety and immunogenicity of olipudase alfa therapy in pediatric patients under 2 years of age diagnosed with acid sphingomyelinase deficiency (ASMD). It is a multicenter, open-label study that will collect primary data over a total duration of 5 years, with a follow-up period ranging from 1 to 3 years. The study allows for enrollment without requiring participants to travel to existing study sites, facilitating broader access to the study. The methodology includes ancillary procedures to evaluate anti-drug antibodies (ADA) while remaining non-interventional regarding treatment decisions.
Who should consider this trial
Good fit: Ideal candidates for this study are pediatric patients under 2 years of age with a confirmed diagnosis of ASMD type A/B or B.
Not a fit: Patients who are older than 2 years or do not have a documented diagnosis of ASMD will not benefit from this study.
Why it matters
Potential benefit: If successful, this study could provide valuable insights into the safety and immune response of olipudase alfa, potentially improving treatment strategies for young children with ASMD.
How similar studies have performed: While this study employs a novel observational approach, similar studies on enzyme replacement therapies have shown promising results in other conditions.
Eligibility criteria
Show full inclusion / exclusion criteria
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.
Where this trial is running
Chicago, Illinois and 4 other locations
- Ann and Robert H Lurie Children's Hospital of Chicago- Site Number: 001002 — Chicago, Illinois, United States (Recruiting)
- Children's Hospital Medical Center- Site Number: 001003 — Cincinnati, Ohio, United States (Recruiting)
- Nationwide Children's Hospital - PIN- Site Number : 1001-1 — Columbus, Ohio, United States (Recruiting)
- Pulse InfoFrame US Inc.- Site Number: 001001 — Philadelphia, Pennsylvania, United States (Recruiting)
- Dell Children's Medical Center- Site Number : 1001-2 — Austin, Texas, United States (Recruiting)
Study contacts
- Study coordinator: Trial Transparency email recommended (Toll free for US & Canada)
- Email: contact-us@sanofi.com
- Phone: 800-633-1610
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.