ClinicalTrials.gov record NCT06192576

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)

Record copied from ClinicalTrials.gov: Oct 10, 2026AI summaries generated: Sep 28, 2026

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

StatusRecruitingThe study is currently enrolling participants. Only the study team can say whether a particular person can join.
Study typeObservationalA study in which researchers do not assign participants to a treatment or other intervention. They observe or measure what happens, which may include study visits, tests, questionnaires or a review of medical records.
AgeUp to 2 years old
SexAll
LocationUnited States
AI summaryWhat this study is about, in plain languageShow ▾Hide ▴

AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.

This prospective observational study follows children with ASMD who start treatment before age 2 (people with ASMD type A can join at any age) and are treated with olipudase alfa in routine clinical care. The study does not give or assign treatments; it records safety events, laboratory results, and anti-drug antibody testing. Follow-up for each participant is at least 1 year and up to 3 years; the overall study duration is 5 years with enrollment up to 4 years. Some protocol-specified procedures (for example, assessment of anti-drug antibodies) may be collected outside usual care.

What it is studying. The study is observing long-term safety of olipudase alfa in children with ASMD who start treatment before age 2, and in people with ASMD type A of any age, including counts of adverse events (AEs), serious adverse events (SAEs), and adverse events of special interest (AESIs) such as moderate/severe or recurrent hypersensitivity and infusion-associated reactions. It is also measuring the presence and levels (titers) of anti-olipudase alfa antibodies (IgG, and IgE when relevant) and tracking abnormal laboratory test results and vital signs from baseline to up to three years.

What the researchers hope to learn. To describe the long-term safety profile of olipudase alfa in infants with ASMD; to measure immunogenicity by detecting and quantifying anti-olipudase alfa antibodies (including IgE in cases of moderate/severe or recurrent reactions); and to document abnormal laboratory results and vital signs and the number of AEs, SAEs, and AESIs from baseline to three years.

What this study is about

The study's own summary, as published on ClinicalTrials.gov. Underlined terms have a short explanation.

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.

Interventions being studied

Being studied in this trial does not mean the treatment or procedure is approved or available for this condition.

Drug
  • Olipudase alfa

Eligibility

The criteria exactly as listed on ClinicalTrials.gov. Only the study team can say whether they apply to you.

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.

Study description and what participation involves

The study's detailed description, as published on ClinicalTrials.gov.

Detailed description from the registry

This is a hybrid study design. Patients need not travel to existing study sites in order to enroll.

AI summaryWhat participation may involve, in plain languageShow ▾Hide ▴

AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.

This is an observational study that follows patients treated with olipudase alfa in routine clinical care and collects safety and immune-response information. Participants will have adverse events (AEs), serious AEs (SAEs), and adverse events of special interest (AESIs, including moderate/severe or recurrent hypersensitivity and infusion-associated reactions) recorded, along with abnormal laboratory results and vital signs. The study measures the presence and titers of serum anti-olipudase alfa antibodies (IgG ADA, and IgE ADA if moderate/severe or recurrent reactions occur). The design is hybrid; ancillary protocol‑specified procedures (for example, ADA assessment) may be performed outside standard care, the study does not assign or change treatment, and patients need not travel to existing study sites to enroll.

How the study is structured. A prospective, non-interventional cohort study that collects primary data while participants receive olipudase alfa as part of routine care. The study may include additional protocol-specified procedures (for example, antibody testing) that go beyond usual care, but it does not assign or administer treatment. The design is hybrid so patients need not travel to existing study sites to enroll. Each participant is followed for a minimum of one year and up to three years; enrollment may continue for up to four years and the overall study duration is five years.

Questions you could ask

These questions are suggestions for a conversation with the study team or your own clinician, not advice.

  • The registry lists this study under Niemann-Pick Diseases; Acid Sphingomyelinase Deficiency. Does it apply to my diagnosis?
  • This is an observational study, so the study itself gives no treatment. What would taking part involve for me, and would it change my usual care?
  • The listed sites are in United States. Is any of them reachable for me, and does the study help with travel?
  • Which of the eligibility criteria would need to be checked with tests before I could join?

More on Niemann-Pick disease type C

Clinical trials15 trials listed for Niemann-Pick disease type CEvery trial the registry lists for Niemann-Pick disease type C, with recruitment status, phase and sites, updated from ClinicalTrials.gov.See all trials →Research updatesWhat changed in Niemann-Pick disease type C researchLatest monthly update: September 2026. Includes: Gene change range. New research summarized each month in plain language, with links to every source paper.Read the latest update →

Important dates

Study start
Apr 16, 2024
Primary completion
Jan 2, 2029 (estimated)
Study completion
Jan 15, 2029 (estimated)
Target enrollment
10 participants (estimated)
Last updated on registry
Sep 16, 2026
Data verified
Oct 10, 2026

Sponsor and research team

The lead sponsor funds and oversees the trial. A pharmaceutical or biotech company sponsor typically means the study is testing that company's drug or device; a hospital, university, or government agency usually indicates investigator-initiated or publicly funded research.

Lead sponsor
Sanofi
Collaborators
  • Pulse Infoframe Ltd.

Central study contact

If you're interested in participating, the central contact can answer general questions about the study — including eligibility requirements and how to find a recruiting site near you. Actual eligibility is determined by the site's medical team.

Participating sites (6)

  • Ann and Robert H Lurie Children's Hospital of Chicago- Site Number: 001002Recruiting

    Chicago, Illinois, 60611 · United States

  • Children's Hospital Medical Center- Site Number: 001003Recruiting

    Cincinnati, Ohio, 45229-3039 · United States

  • Nationwide Children's Hospital- Site Number : 1001-1Recruiting

    Columbus, Ohio, 43205 · United States

  • Pulse InfoFrame US Inc.- Site Number: 001001Recruiting

    Philadelphia, Pennsylvania, 19104 · United States

  • Dell Children's Medical Center- Site Number : 1001-2Recruiting

    Austin, Texas, 78723 · United States

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

    St. George, Utah, 84790 · United States

Terms used on this page · explanations by MedicalUpdates.ai

cohort
A group of people who share a characteristic, such as a diagnosis, and are studied together. 'Prospective' means they are followed forward from enrolment; 'retrospective' means existing records are reviewed.
open label
No masking is used, so the assigned intervention is not deliberately hidden from participants or study staff.
standard of care
Treatment generally considered appropriate for a condition in a particular clinical setting. It can vary by patient, place and over time. In many studies the new treatment is added to, or compared with, standard of care.
follow-up
The period after treatment or enrolment during which participants are observed or contacted, for example to record side effects or outcomes.
enrollment
The number of participants a study plans to include, or has included. 'Estimated' means the planned number; 'actual' means the number who joined.
informed consent
The process of explaining a study, including its risks and what participation involves, so that a person can decide freely whether to take part. Consent is usually documented in writing, and a participant can choose to leave the study later.
legal guardian
A person who is legally allowed to make decisions, including consent to research, on behalf of someone who cannot consent for themselves.
investigational drug
A treatment that is being tested and has not been approved for this use. It may be approved for a different condition. Being studied does not mean it works or is available outside the study.
half-lives
The time it takes for the amount of a drug in the body to fall by half. 'Five half-lives' is often used as the point at which a previous drug has essentially left the body.
clinical trial
A study in which researchers assign participants to one or more interventions, such as a drug, procedure, device, behavioural programme or other approach, to measure their effects.
Inclusion Criteria
The conditions a person must meet to be considered for the study, for example a confirmed diagnosis, an age range or a test result.
Exclusion Criteria
The conditions that rule a person out even if they meet the inclusion criteria, for example other illnesses, certain medicines or pregnancy.

The study record on this page is reproduced from ClinicalTrials.gov, a database of the U.S. National Library of Medicine, where it was submitted by the study sponsor. MedicalUpdates.ai does not own this content, is not affiliated with or endorsed by ClinicalTrials.gov, NIH, or the U.S. Government, and does not verify the record. Plain-language notes, the glossary and the questions above are added by this site; text in the boxes marked "AI summary" was written by an AI model and may contain errors. Registry records change over time. Check the original record for the current version and discuss eligibility or participation with a qualified healthcare professional.

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