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NCT04106544 · ClinicalTrials.gov registry record · NA
A Prospective and Retrospective Cohort Study in Patients With Chronic Forms of Acid Sphingomyelinase Deficiency (ASMD)
A NA study, sponsored by Sanofi.
- Completed
- Registry status
- NA
- Development phase
- 84
- Enrollment target
NCT04106544: Completed NA study, sponsored by Sanofi.
NCT04106544 is a NA study that has completed, run by Sanofi. The registered enrollment target is 84 participants, below the 1,404-participant average among 58,714 other NA trials with a reported enrollment target (94% lower). According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT04106544, a NA study, has completed, sponsored by Sanofi.
- COMPLETED
- Registry status
- NA
- Development phase
- 84 participants
- Enrollment target
Study Summary
Primary Objective: * To describe the clinical features and their severity at the time of diagnosis and their evolution over time in patients with confirmed chronic visceral and chronic neurovisceral forms of ASMD * To describe Clinician-Reported Outcomes (ClinROs) and Patient-Reported Outcomes (PROs) at enrollment and their evolution over time; disease severity at the time of diagnosis and its evolution over time Secondary Objectives: * To describe abnormal values in laboratory parameters and all values of specific clinical and imaging assessments at the time of diagnosis and their evolution over time * To study the use and applicability towards validation of a newly developed ASMD disease severity scoring system * To study the use and applicability towards validation of a newly developed ASMD PRO tool * To describe ASMD-related disease burden among patients with ASMD, caregivers, and healthcare resource utilization * To describe the association between patient demographics (eg, age, gender, race, Ashkenazi ancestry) and genotype with selected clinical features in patients with confirmed chronic visceral and chronic neurovisceral forms of ASMD
Interventions
- PROCEDURE Investigational Procedures
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 84 participants |
| Start Date | 2019-09-27 |
| Est. Completion | 2023-05-15 |
| Phase | NA |
What the finished NCT04106544 record still lists
NCT04106544 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 84 participants, a relatively small participant target, below the 1,404-participant average among 58,714 other NA trials with a reported enrollment target (94% lower).
The record links to 0 conditions, and to 1 intervention - of which Investigational Procedures is the first listed.
NCT04106544 does not publish any study locations in the registry export this page uses.
Frequently Asked Questions
What is clinical trial NCT04106544 about?
NCT04106544 is a clinical study titled "A Prospective and Retrospective Cohort Study in Patients With Chronic Forms of Acid Sphingomyelinase Deficiency (ASMD)". Primary Objective: * To describe the clinical features and their severity at the time of diagnosis and their evolution over time in patients with confirmed chronic visceral and chronic neurovisceral forms of ASMD * To describe Clinician-Reported Outcomes (ClinROs) and Patient-Reported Outcomes (PRO...
What is the current status of trial NCT04106544?
This trial is currently completed. It is a NA study. The enrollment target is 84 participants. The study started on 2019-09-27. Estimated completion is 2023-05-15.
What interventions are being tested in trial NCT04106544?
The interventions under investigation include: Investigational Procedures (PROCEDURE).
Who is sponsoring clinical trial NCT04106544?
This trial is sponsored by Sanofi, which has 713 total clinical trials registered on ClinicalTrials.gov.
Learn More About Clinical Trials
How this trial's enrollment target compares
Where NCT04106544's enrollment target sits among peer trials
84 983rd of 2000 higher than 1,011 of 2,000 other NA trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other NA trials. The dashed line + filled bar mark this entry. Hover or tap any bar for its full count and share, and where it sits relative to this entry.
Source ClinicalTrials.gov registry export · 2026-08-08
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