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)

NCT06192576 | Recruiting FDA Drug

• 2 other identifiers: OBS18020U1111-1294-8169
• Study Type: observational
• Target: 10
• Locations: 1 country
• Sites: 5

Brief 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 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.

Conditions

Niemann-Pick Diseases; Acid Sphingomyelinase Deficiency

Outcome Measures

Primary Outcomes (3)

• Number of cases in the exposed population with AEs, SAEs, and AESIs (including moderate/severe or recurrent hypersensitivity reactions and infusion-associated reactions)

  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)

  Baseline to 3 years

• Number of cases in the exposed population with abnormal laboratory test results and vital signs

  Baseline to 3 years

Study Arms (1)

Olipudase alfa arm

Drug: Olipudase alfa

Interventions

Olipudase alfa DRUG

This study will not administer any treatment, only observe the treatment as prescribed in real-world clinical practice.

Also known as: Xenpozyme®

Olipudase alfa arm

Eligibility Criteria

• Age: Up to 2 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)
• Sampling Method: Non-Probability Sample

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.

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Sanofi: lead
• Pulse Infoframe Ltd.: collaborator

Study Sites (5)

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

Chicago, Illinois, 60611, United States

RECRUITING

Children's Hospital Medical Center- Site Number: 001003

Cincinnati, Ohio, 45229-3039, United States

RECRUITING

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

Columbus, Ohio, 43205-2664, United States

RECRUITING

Pulse InfoFrame US Inc.- Site Number: 001001

Philadelphia, Pennsylvania, 19104, United States

RECRUITING

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

Austin, Texas, 78723, United States

RECRUITING

Related Links

• Register (healthie.net)

MeSH Terms

Conditions

Niemann-Pick Diseases

Interventions

olipudase alfa

Condition Hierarchy (Ancestors)

Sphingolipidoses; Lysosomal Storage Diseases, Nervous System; Brain Diseases, Metabolic, Inborn; Brain Diseases, Metabolic; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Histiocytosis, Non-Langerhans-Cell; Histiocytosis; Lymphatic Diseases; Hemic and Lymphatic Diseases; Metabolism, Inborn Errors; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Lipidoses; Lipid Metabolism, Inborn Errors; Lysosomal Storage Diseases; Metabolic Diseases; Nutritional and Metabolic Diseases; Lipid Metabolism Disorders

Central Study Contacts

Trial Transparency email recommended (Toll free for US & Canada)

CONTACT

800-633-1610; contact-us@sanofi.com

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: December 20, 2023
• First Posted: January 5, 2024
• Study Start: April 16, 2024
• Primary Completion (Estimated): January 2, 2029
• Study Completion (Estimated): January 15, 2029
• Last Updated: October 31, 2025

Record last verified: 2025-10

Data Sharing

• IPD Sharing: Will share

Locations

US (5)