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Olipudase alfa arm for Niemann-Pick Disease

N/A
Recruiting
Research Sponsored by Sanofi
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 weeks
Treatment ~ Varies
Follow Up ~baseline to 3 years
This trial's timeline: 3 weeks for screening, Varies for treatment, and baseline to 3 years for reporting.

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 • NCT02292654
50%
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

Find a Location

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

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.
Recent research and studies
clinicaltrials.govStudy
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)
2024. "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)". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT06192576.
All > Chicago > Cincinnati
~7 spots leftby Jan 2029
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