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)

Part of paid clinical trials in Chicago, Illinois.

Sponsor
Sanofi
Study ID
NCT06192576
Status
Recruiting
Apply to this trial

Conditions

  • Acid Sphingomyelinase Deficiency
  • Niemann-Pick Diseases

Eligibility Criteria

Sex
ALL
Age
N/A - 2 Years
Healthy Volunteers
Not accepted

Interventions

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

Study Details

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.

Key Dates

Start date
Apr 16, 2024
Status verified
Oct 2025
Primary completion
Jan 2, 2029
Completion
Jan 15, 2029

Study Design

Enrollment
10 participants (estimated)

Arms

  • Arm: Olipudase alfa arm

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 ]

Central Contacts

  • Trial Transparency email recommended (Toll free for US & Canada)
    800-633-1610
    [email protected]

Locations (5)

FacilityCityStateZIPSite coordinators
Ann and Robert H Lurie Children's Hospital of Chicago- Site Number: 001002ChicagoIllinois60611
Joshua Baker
[email protected]
312-227-4000
Joshua Baker (PRINCIPAL_INVESTIGATOR)
Children's Hospital Medical Center- Site Number: 001003CincinnatiOhio45229-3039
Nicole K. Weaver
[email protected]
800-647-4805
Nicole K. Weaver, MD (PRINCIPAL_INVESTIGATOR)
Nationwide Children's Hospital - PIN- Site Number : 1001-1ColumbusOhio43205-2664
William Burns
[email protected]
614-722-3535
William Burns (PRINCIPAL_INVESTIGATOR)
Pulse InfoFrame US Inc.- Site Number: 001001PhiladelphiaPennsylvania19104
Femida Gwadry-Sridhar
[email protected]
519-872-1471
Femida Gwadry-Sridhar (PRINCIPAL_INVESTIGATOR)
Dell Children's Medical Center- Site Number : 1001-2AustinTexas78723
James B. Gibson
[email protected]
512-628-1810

Find similar trials in Chicago, IL

By research site
Ann and Robert H Lurie Children's Hospital of Chicago· Chicago, ILChildren's Hospital Medical Center· Cincinnati, OHNationwide Children's Hospital - PIN· Columbus, OHPulse InfoFrame US Inc.· Philadelphia, PADell Children's Medical Center· Austin, TX

Related Studies