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New study tracks safety of enzyme therapy in babies with rare genetic disease

NCT ID NCT06192576

Knowledge-focused Sponsor: Sanofi Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now This study
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jun 27, 2026 · Last updated Sep 17, 2026 · Updated 2 times

Summary

This study follows up to 10 children under 2 years old with acid sphingomyelinase deficiency (ASMD) who are already receiving olipudase alfa (Xenpozyme®) as part of their routine care. Researchers will monitor side effects and immune responses over 1 to 3 years. No new treatment is given; the study simply observes what happens in real-world medical practice.

What this could mean

Our plain-language read of the trial. This is informational only, not medical advice or a prediction.

Active substance
olipudase alfa (Xenpozyme®)
What this could lead to
If successful, this study could confirm that olipudase alfa is safe and well-tolerated in very young children with ASMD, supporting its continued use in this age group.
What could go wrong
This is a small observational study (10 participants) with no control group, so results may not apply to all patients. It only monitors safety, not effectiveness, and side effects like allergic reactions are possible.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Participants

About 10 people

The number the study aims to enrol. It can still change while the study runs.

Started

Apr 2024

Expected to finish

Jan 2029

An estimate. End dates often move.

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Who is studied

All children under the age of 2 years with a documented ASMD diagnosis as shown by ASM deficiency in a cell-based assay (peripheral leukocytes, cultured fibroblasts, or lymphocytes) and/or by genotype determination will be considered for enrollment into this ASMD study.

Ages

Up to 2 years

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

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.

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Conditions

The condition(s) this trial relates to.

acid sphingomyelinase deficiency Niemann-Pick disease Niemann-Pick disease type A Niemann-Pick disease type B

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Acid sphingomyelinase deficiency Niemann-Pick diseases

How to take part

Only the study team decides who joins. These are the ways to reach them.

  1. The study's own enquiry address

    This study publishes an address for enquiries. See it below .

  2. The places running it

    6 sites. The list below names each one and where it is.

  3. The official record

    ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.

    Open the record ↗

  4. A doctor treating you

    A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.

Contacts and locations

Study contacts

  • Contact

    Email: •••••@•••••

Locations

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

    RECRUITING

    Chicago, Illinois, 60611, United States

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

    RECRUITING

    Cincinnati, Ohio, 45229-3039, United States

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

    RECRUITING

    Austin, Texas, 78723, United States

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

    RECRUITING

    Columbus, Ohio, 43205, United States

  • Pulse InfoFrame US Inc.- Site Number: 001001

    RECRUITING

    Philadelphia, Pennsylvania, 19104, United States

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

    RECRUITING

    St. George, Utah, 84790, United States

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