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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 is a NA study that has completed, run by Sanofi. The registered enrollment target is 84 participants.
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 |
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Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT04106544
The ClinicalTrials.gov registry entry for NCT04106544 describes a study currently listed as completed, categorized as NA. The registered enrollment target is 84 participants, a figure that helps gauge the scale of data the investigators plan to collect. The listed sponsor is Sanofi, which has 713 total studies on file at ClinicalTrials.gov.
The record links to 0 conditions, and to 1 intervention - of which Investigational Procedures is the first listed.
NCT04106544 reports 0 study locations.
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.
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