Acid Sphingomyelinase Deficiency, Niemann-Pick Diseases
Conditions
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.
Detailed description
This is a hybrid study design. Patients need not travel to existing study sites in order to enroll.
Interventions
DRUGOlipudase alfa
This study will not administer any treatment, only observe the treatment as prescribed in real-world clinical practice.
Sponsors
Sanofi
Pulse Infoframe Ltd.
Study design
Observational model
COHORT
Time perspective
PROSPECTIVE
Eligibility
Sex/Gender
ALL
Age
No minimum to 2 Years
Healthy volunteers
No
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. The above information is not intended to contain all considerations relevant to a patient's potential participation in a clinical trial.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Number of cases in the exposed population with AEs, SAEs, and AESIs (including moderate/severe or recurrent hypersensitivity reactions and infusion-associated reactions) | 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) | Baseline to 3 years |
| Number of cases in the exposed population with abnormal laboratory test results and vital signs | Baseline to 3 years |
Countries
United States
Contacts
CONTACTTrial Transparency email recommended (Toll free for US & Canada)
Outcome results
None listed