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)
For patients and families
In plain language
An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.
- What is being studied
- The protocol lists: Olipudase alfa.
- Who it may be relevant to
- Registry conditions: Niemann-Pick Diseases, Acid Sphingomyelinase Deficiency. Basic parameters: up to 2 years · All.
- What needs checking
- Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
- Where it takes place
- United States
- Next step
- Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →
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Official title
A Prospective Observational Study to Assess the Long-term Safety and Immunogenicity of Olipudase Alfa Therapy During Routine Clinical Care in Pediatric Patients Less Than 2 Years of Age With Acid Sphingomyelinase Deficiency
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.
Detailed description
This is a hybrid study design. Patients need not travel to existing study sites in order to enroll.
Interventions
- Drug Olipudase alfa
This study will not administer any treatment, only observe the treatment as prescribed in real-world clinical practice.
Primary outcome measures
- 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]
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.
Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.
Healthy volunteers: No
Study design
- Observational model
- Cohort
Study locations
United States · 5 centers
- Ann and Robert H Lurie Children's Hospital of Chicago- Site Number: 001002 — Chicago
- Children's Hospital Medical Center- Site Number: 001003 — Cincinnati
- Nationwide Children's Hospital - PIN- Site Number : 1001-1 — Columbus
- Pulse InfoFrame US Inc.- Site Number: 001001 — Philadelphia
- Dell Children's Medical Center- Site Number : 1001-2 — Austin
Identifiers
NCT: NCT06192576 · OBS18020 · U1111-1294-8169