Recruiting

Olipudase Alfa

Sponsor:

Sanofi

Code:

NCT06192576

Conditions

Niemann-Pick Diseases

Acid Sphingomyelinase Deficiency

Eligibility Criteria

Sex: All

Age: 0 - 2

Healthy Volunteers: Not accepted

Interventions

Olipudase alfa

Study Details

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

Study ID

NCT06192576

Start date

Apr 16, 2024

Status verified date

Oct, 2025

Completion date

Jan 15, 2029

Anticipated

Primary completion date

Jan 2, 2029

Anticipated

Eligibility Criteria

Eligibility Criteria

Sex: All

Age: 0 - 2

Healthy Volunteers: Not accepted

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.

Study Design

Enrollment

10 participants

Anticipated

Interventions and Outcome Measures

Arms

Olipudase alfa arm

Interventions

Olipudase alfa

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

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 ]
  • 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 ]

Central Contacts and Locations

Central contacts

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

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

Locations

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

Recruiting

Chicago, Illinois, United States, 60611

Contacts

Joshua Baker

312-227-4000jobaker@luriechildrens.org

Principal Investigator:

Joshua Baker

Children's Hospital Medical Center- Site Number: 001003

Recruiting

Cincinnati, Ohio, United States, 45229-3039

Contacts

Nicole K. Weaver

800-647-4805Kathryn.Weaver@cchmc.org

Principal Investigator:

Nicole K. Weaver, MD

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

Recruiting

Columbus, Ohio, United States, 43205-2664

Contacts

William Burns

614-722-3535william.burns@nationwidechildrens.org

Principal Investigator:

William Burns

Pulse InfoFrame US Inc.- Site Number: 001001

Recruiting

Philadelphia, Pennsylvania, United States, 19104

Contacts

Femida Gwadry-Sridhar

519-872-1471femida@pulseinfoframe.com

Principal Investigator:

Femida Gwadry-Sridhar

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

Recruiting

Austin, Texas, United States, 78723

Contacts

James B. Gibson

512-628-1810JBGibson@ascension.org

More Information

Sponsor

Sanofi

Last update posted

Oct 31, 2025

Last verified

Oct, 2025

Trial information was received from ClinicalTrials.gov and was last updated on 2026-09-08. This information was provided to ClinicalTrials.gov by Sanofi on 2025-10-31.