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Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial Must have
Be younger than 18 years old
Timeline
Screening 3 weeks
Treatment Varies
Follow Up baseline to 3 years
Awards & highlights
Study 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.
Eligible Conditions
- Niemann-Pick Disease
Timeline
Screening ~ 3 weeks3 visits
Treatment ~ Varies
Follow Up ~ baseline to 3 years
Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~baseline to 3 years
Treatment Details
Study Objectives
Outcome measures can provide a clearer picture of what you can expect from a treatment.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)
Number of cases in the exposed population with abnormal laboratory test results and vital signs
Presence and titers of serum anti-olipudase alfa IgG anti-drug antibodies (ADA) (and IgE ADA in cases of moderate/severe or recurrent reactions)
Side effects data
From 2019 Phase 1 & 2 trial • 20 Patients • NCT0229265450%
Cough
50%
Vomiting
50%
Nasopharyngitis
50%
Headache
50%
Diarrhoea
25%
Pyrexia
100%
80%
60%
40%
20%
0%
Study treatment Arm
Olipudase Alfa: Adolescent Cohort
Olipudase Alfa: Child Cohort
Olipudase Alfa: Infant/Early Child Cohort
Total Participants
Trial Design
1Treatment groups
Experimental Treatment
Group I: Olipudase alfa armExperimental Treatment1 Intervention
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Olipudase alfa
2015
Completed Phase 2
~20
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Who is running the clinical trial?
Pulse Infoframe Ltd.UNKNOWN
SanofiLead Sponsor
2,164 Previous Clinical Trials
3,514,970 Total Patients Enrolled
Frequently Asked Questions
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