Long-term safety and immune response of olipudase alfa in young children with acid sphingomyelinase deficiency

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

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.

Why it matters

Who can join: the full rules

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.

Where it's running

• 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- 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
• St. George Regional Hospital, Intermountain Health - Site Number: 1001-3
  St. George, Utah, 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

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How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.