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)
NCT ID: NCT06192576
Last Updated: 2025-10-31
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
Get a concise snapshot of the trial, including recruitment status, study phase, enrollment targets, and key timeline milestones.
RECRUITING
10 participants
OBSERVATIONAL
2024-04-16
2029-01-15
Brief Summary
Review the sponsor-provided synopsis that highlights what the study is about and why it is being conducted.
Related Clinical Trials
Explore similar clinical trials based on study characteristics and research focus.
Safety, Tolerability, PK, and Efficacy Evaluation of Repeat Ascending Doses of Olipudase Alfa in Pediatric Patients <18 Years of Age With Acid Sphingomyelinase Deficiency
NCT02292654
A Long-Term Study of Olipudase Alfa in Patients With Acid Sphingomyelinase Deficiency
NCT02004704
A Study to Evaluate Safety and Tolerability of Olipudase Alfa in Pediatric and Adult Participants With Acid Sphingomyelinase Deficiency (ASMD) Who Completed the DFI12712 or the LTS13632 Study in France
NCT06949358
Safety Study of rhASM Enzyme Replacement Therapy in Adults With Acid Sphingomyelinase Deficiency (Niemann-Pick Disease)
NCT00410566
Tolerability and Safety Study of Recombinant Human Acid Sphingomyelinase in Acid Sphingomyelinase Deficiency Patients
NCT01722526
Detailed Description
Dive into the extended narrative that explains the scientific background, objectives, and procedures in greater depth.
Conditions
See the medical conditions and disease areas that this research is targeting or investigating.
Study Design
Understand how the trial is structured, including allocation methods, masking strategies, primary purpose, and other design elements.
COHORT
PROSPECTIVE
Study Groups
Review each arm or cohort in the study, along with the interventions and objectives associated with them.
Olipudase alfa arm
Olipudase alfa
This study will not administer any treatment, only observe the treatment as prescribed in real-world clinical practice.
Interventions
Learn about the drugs, procedures, or behavioral strategies being tested and how they are applied within this trial.
Olipudase alfa
This study will not administer any treatment, only observe the treatment as prescribed in real-world clinical practice.
Other Intervention Names
Discover alternative or legacy names that may be used to describe the listed interventions across different sources.
Eligibility Criteria
Check the participation requirements, including inclusion and exclusion rules, age limits, and whether healthy volunteers are accepted.
Inclusion Criteria
* 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 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.
2 Years
ALL
No
Sponsors
Meet the organizations funding or collaborating on the study and learn about their roles.
Pulse Infoframe Ltd.
INDUSTRY
Sanofi
INDUSTRY
Responsible Party
Identify the individual or organization who holds primary responsibility for the study information submitted to regulators.
Locations
Explore where the study is taking place and check the recruitment status at each participating site.
Ann and Robert H Lurie Children's Hospital of Chicago- Site Number: 001002
Chicago, Illinois, United States
Children's Hospital Medical Center- Site Number: 001003
Cincinnati, Ohio, United States
Nationwide Children's Hospital - PIN- Site Number : 1001-1
Columbus, Ohio, United States
Pulse InfoFrame US Inc.- Site Number: 001001
Philadelphia, Pennsylvania, United States
Dell Children's Medical Center- Site Number : 1001-2
Austin, Texas, United States
Countries
Review the countries where the study has at least one active or historical site.
Central Contacts
Reach out to these primary contacts for questions about participation or study logistics.
Trial Transparency email recommended (Toll free for US & Canada)
Role: CONTACT
Facility Contacts
Find local site contact details for specific facilities participating in the trial.
Related Links
Access external resources that provide additional context or updates about the study.
Register (healthie.net)
Other Identifiers
Review additional registry numbers or institutional identifiers associated with this trial.
U1111-1294-8169
Identifier Type: REGISTRY
Identifier Source: secondary_id
OBS18020
Identifier Type: -
Identifier Source: org_study_id
More Related Trials
Additional clinical trials that may be relevant based on similarity analysis.