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Acid Sphingomyelinase Deficiency (ASMD) Cohort for Niemann-Pick Disease

N/A
Waitlist Available
Research Sponsored by Sanofi
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Timeline
Screening 3 weeks
Treatment Varies
Follow Up up to 2 years
Awards & highlights

Study Summary

This trial is studying how well a newly developed scoring system works to measure disease severity and patient-reported outcomes in patients with a confirmed chronic visceral and chronic neurovisceral forms of ASMD.

Eligible Conditions
  • Niemann-Pick Disease

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~up to 2 years
This trial's timeline: 3 weeks for screening, Varies for treatment, and up to 2 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
Clinician-Reported Outcomes (ClinROs) depending on participant's age, local regulation, local availability and investigator's discretion
Number of patients with at least one clinical feature and highest severity grade at the time of diagnosis and over time
Patient-Reported Outcomes (PROs) depending on participant's age, local regulation, local availability and investigator's discretion
+1 more
Secondary outcome measures
Age appropriate Z-score deviation for height and weight (children only)
Association of cerebrovascular accident with age, gender, race, Ashkenazi ancestry and genotype
Association of external bleeding episode with age, gender, race, Ashkenazi ancestry and genotype
+22 more

Trial Design

1Treatment groups
Experimental Treatment
Group I: Acid Sphingomyelinase Deficiency (ASMD) CohortExperimental Treatment1 Intervention
Patients across the full spectrum of chronic ASMD who have fulfilled the eligibility criteria and who have performed the inclusion visit
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Investigational Procedures
2019
N/A
~90

Find a Location

Who is running the clinical trial?

SanofiLead Sponsor
2,161 Previous Clinical Trials
3,511,955 Total Patients Enrolled
Clinical Sciences & OperationsStudy DirectorSanofi
857 Previous Clinical Trials
2,018,872 Total Patients Enrolled

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

Does this experiment still have space for participants?

"As per the information on clinicaltrials.gov, this medical trial has ceased to recruit participants - it was initially posted in September 2019 and its most recent edit occurred in May 2022. Despite being closed, 10 other studies are still enrolling patients as of now."

Answered by AI
Recent research and studies
clinicaltrials.govStudy
A Prospective and Retrospective Cohort Study in Patients With Chronic Forms of Acid Sphingomyelinase Deficiency (ASMD)
2019. "A Prospective and Retrospective Cohort Study in Patients With Chronic Forms of Acid Sphingomyelinase Deficiency (ASMD)". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT04106544.
~15 spots leftby Apr 2025
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