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)

Investigating Long-term Safety and Immune Response in Children with Acid Sphingomyelinase Deficiency

Recruiting
2 years or below
All
Phase N/A
10 participants needed
1 Location

Study Overview

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

You may be eligible for this study if you meet the following criteria:

  • Conditions: Niemann-Pick Diseases, Acid Sphingomyelinase Deficiency
  • Age: 2 years or below
  • Gender: All

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.

Updated on 17 Jun 2024. Study ID: NCT06192576

This study investigates the long-term safety and immune response to a treatment in young children with Acid Sphingomyelinase Deficiency (ASMD). ASMD is a rare genetic disorder that affects the body's ability to break down certain fats, leading to harmful buildup in various organs. The purpose of this study is to observe children under two years old who are receiving treatment for ASMD, to better understand the effects and safety of the therapy over time.

Participants in this observational study will undergo various assessments as part of their routine care, including monitoring for antibodies that may develop in response to the treatment. An observational study is a type of research study where data is collected by observing participants receiving routine care, without changing their treatment, which may focus on people using specific medications or having certain conditions to better understand how treatments work.

  • Who can participate: Children under 2 years old with ASMD type A/B or B, or any age with ASMD type A, weighing at least 2 kg, and eligible to start or have just started enzyme replacement therapy.
  • Study details: Participants will undergo assessments as part of their routine care, with additional data collection on immune response and safety without altering their treatment.
  • Study timelines: The study will last 5 years.

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