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)
| NCT ID | NCT06192576 |
| Status | Recruiting |
| Phase | — |
| Sponsor | Sanofi |
| Condition | Niemann-Pick Diseases |
| Study Type | OBSERVATIONAL |
| Enrollment | 10 participants |
| Start Date | 2024-04-16 |
| Primary Completion | 2029-01-02 |
Eligibility & Interventions
Eligibility Fast-Check
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What to Expect as a Participant
This is an observational study. You will not receive an experimental treatment; researchers will collect data based on your existing condition or standard treatment.
This trial targets 10 participants in total. It began in 2024-04-16 with a primary completion date of 2029-01-02.
⚠ This information is for research awareness only. Always consult your physician before joining any clinical trial. Participation is voluntary and you may withdraw at any time.
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.
Eligibility Criteria
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.
Contact & Investigator
Trial Transparency email recommended (Toll free for US & Canada)
✉ contact-us@sanofi.com📞 800-633-1610
Frequently Asked Questions
Who can join the NCT06192576 clinical trial?
This trial is open to participants of all sexes, up to 2 Years, studying Niemann-Pick Diseases. Full inclusion and exclusion criteria are listed in the Eligibility Criteria section. Always confirm your eligibility with the research team before applying.
Is NCT06192576 currently recruiting?
Yes, NCT06192576 is actively recruiting participants. Contact the research team at contact-us@sanofi.com for enrollment information.
Where is the NCT06192576 trial being conducted?
This trial is being conducted at Chicago, United States, Cincinnati, United States, Columbus, United States, Philadelphia, United States and 1 additional location.
Who is sponsoring the NCT06192576 clinical trial?
NCT06192576 is sponsored by Sanofi. The trial plans to enroll 10 participants.