Niemann-Pick DiseasesNCT06192576
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)
An observational study of Niemann-Pick Diseases and Acid Sphingomyelinase Deficiency, run by Sanofi at 6 sites in United States.
Trial 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 outside the standard of care for acid sphingomyelinase deficiency (ASMD), but the study methodology remains… Read more about this study
- Who is it for?
- Ages Up to 2 years
- Conditions the study is for
- Niemann-Pick Diseases, Acid Sphingomyelinase Deficiency
- What is being tested?
- Olipudase alfa
- How many people
- 10 participants
- Where
- 6 sitesUnited States
- Study dates
- 16 Apr 2024to 15 Jan 2029
- Phase
- Observational
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Up to 2 years
- All sexes
- No healthy volunteers
- Child
Study population: 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.
Who may be able to join
- 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.
Who cannot join
- 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.
Trial locations and contacts
6 sites in 1 country. Contact the study team or a site near you to ask about taking part.
Trial Transparency email recommended (Toll free for US & Canada) contact
6 sites in 1 country
United States6 sites, 6 recruiting
- Recruiting
Dell Children's Medical Center- Site Number : 1001-2
Austin, Texas, 78723
James B. Gibson, 512-628-1810, JBGibson@ascension.org
- Recruiting
Ann and Robert H Lurie Children's Hospital of Chicago- Site Number: 001002
Chicago, Illinois, 60611
Joshua Baker, 312-227-4000, jobaker@luriechildrens.org
Joshua Baker
- Recruiting
Children's Hospital Medical Center- Site Number: 001003
Cincinnati, Ohio, 45229-3039
Nicole K. Weaver, 800-647-4805, Kathryn.Weaver@cchmc.org
Nicole K. Weaver, MD
- Recruiting
Nationwide Children's Hospital- Site Number : 1001-1
Columbus, Ohio, 43205
William Burns, 614-722-3535, william.burns@nationwidechildrens.org
William Burns
- Recruiting
Pulse InfoFrame US Inc.- Site Number: 001001
Philadelphia, Pennsylvania, 19104
Femida Gwadry-Sridhar, 519-872-1471, femida@pulseinfoframe.com
Femida Gwadry-Sridhar
- Recruiting
St. George Regional Hospital, Intermountain Health - Site Number: 1001-3
St. George, Utah, 84790
About this study
Official title: 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
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.
Read the detailed description
This is a hybrid study design. Patients need not travel to existing study sites in order to enroll.
- Being tested
- Olipudase alfa
- Medical subject headings
- Niemann-Pick Diseases
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Olipudase alfaDrug
Also called Xenpozyme®
This study will not administer any treatment, only observe the treatment as prescribed in real-world clinical practice.
| Study group | Role | What they receive |
|---|---|---|
| Olipudase alfa arm | Not described Receives: Drug: Olipudase alfa |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of cases in the exposed population with AEs, SAEs, and AESIs (including moderate/severe or recurrent hypersensitivity reactions and infusion-associated reactions)
Measured: Baseline to 3 years
Presence and titers of serum anti-olipudase alfa IgG anti-drug antibodies (ADA) (and IgE ADA in cases of moderate/severe or recurrent reactions)
Measured: Baseline to 3 years
Number of cases in the exposed population with abnormal laboratory test results and vital signs
Measured: Baseline to 3 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted5 Jan 2024
- Study start16 Apr 2024
- Primary completion2 Jan 2029 (estimated)
- Study completion15 Jan 2029 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Niemann-Pick DiseasesStudies of the same condition, open and under way.
- More trials run by SanofiThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 10 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Sanofi
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Pulse Infoframe Ltd.
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06192576
- Sponsor's protocol ID
- OBS18020
- Other IDs
- U1111-1294-8169
- Status last verified
- Sep 2026
- First submitted
- 20 Dec 2023
- Last updated
- 16 Sep 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Yes: Qualified researchers may request access to patient level data and related study documents including the clinical study report, study protocol with any amendments, blank case report form, statistical analysis plan, and dataset specifications. Patient level data will be anonymized and study documents will be redacted to protect the privacy of trial participants. Further details on Sanofi's data sharing criteria, eligible studies, and process for requesting access can be found at: https://vivli.org
Study at a glance
From the registry record, last updated by the sponsor on 16 Sep 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- 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
- ClinicalTrials.gov ID
- NCT06192576
- Acronym / sponsor ID
- OBS18020
- Study type
- Observational
- Recruitment status
- Recruiting
- Enrolment
- 10 participants (estimated)
- Lead sponsor
- SanofiIndustry (pharma, biotech, devices)
- Collaborators
- Pulse Infoframe Ltd.
- Study start
- 16 Apr 2024 (actual)
- Primary completion
- 2 Jan 2029 (estimated)
- Study completion
- 15 Jan 2029 (estimated)
- First posted
- 5 Jan 2024 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Detailed description
This is a hybrid study design. Patients need not travel to existing study sites in order to enroll.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Olipudase alfa arm | – | Drug: Olipudase alfa |
Who may qualify
- Ages
- Up to 2 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
- Study population
- 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.
Key inclusion criteria (5 listed)
- 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.
Key exclusion criteria (4 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Olipudase alfaAlso: Xenpozyme®This study will not administer any treatment, only observe the treatment as prescribed in real-world clinical practice. | Drug | Olipudase alfa arm |
Primary outcome
Number of cases in the exposed population with AEs, SAEs, and AESIs (including moderate/severe or recurrent hypersensitivity reactions and infusion-associated reactions)
Time frame: Baseline to 3 years
Presence and titers of serum anti-olipudase alfa IgG anti-drug antibodies (ADA) (and IgE ADA in cases of moderate/severe or recurrent reactions)
Time frame: Baseline to 3 years
Number of cases in the exposed population with abnormal laboratory test results and vital signs
Time frame: Baseline to 3 years
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 16 Sep 2026.
- Register (healthie.net)Link provided by the sponsor.
- Individual participant dataSharing planned: Yes. Qualified researchers may request access to patient level data and related study documents including the clinical study report, study protocol with any amendments, blank case report form, statistical analysis plan, and dataset specifications. Patient level data will be anonymized and study documents will be redacted to protect the privacy of trial participants. Further details on Sanofi's data sharing criteria, eligible studies, and process for requesting access can be found at: https://vivli.org
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Trial Transparency email recommended (Toll free for US & Canada) contact
6 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Niemann-Pick Diseases and Acid Sphingomyelinase Deficiency, run by Sanofi at 6 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 5 inclusion and 4 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 16 Sep 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 6 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Trial Transparency email recommended (Toll free for US & Canada) (800-633-1610 ext. option 6, contact-us@sanofi.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Number of cases in the exposed population with AEs, SAEs, and AESIs (including moderate/severe or recurrent hypersensitivity reactions and infusion-associated reactions) (time frame: Baseline to 3 years). The study lists 3 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 16 Sep 2026.