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NCT05619900 · ClinicalTrials.gov registry record
Registry of Patients Diagnosed With Lysosomal Storage Diseases
A clinical trial of Mucopolysaccharidosis II and Mucopolysaccharidosis VI, sponsored by University of California, San Francisco.
- Recruiting
- Registry status
- 250
- Enrollment target
- 1
- Study location
NCT05619900: Recruiting study of Mucopolysaccharidosis II and Mucopolysaccharidosis VI, sponsored by University of California, San Francisco.
NCT05619900 is a study of Mucopolysaccharidosis II and Mucopolysaccharidosis VI that is actively recruiting participants, run by University of California, San Francisco. The registered enrollment target is 250 participants, above the 38-participant average among 7 other Mucopolysaccharidosis II trials with a reported enrollment target (558% higher). The trial reports 1 study location across 1 state. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT05619900, a study of Mucopolysaccharidosis II and Mucopolysaccharidosis VI, is actively recruiting participants, sponsored by University of California, San Francisco.
- RECRUITING
- Registry status
- 250 participants
- Enrollment target
- 1
- Study location
Study Summary
This is an international prospective and retrospective registry of patients with Lysosomal Storage Diseases (LSDs) to understand the natural history of the disease and the outcomes of fetal therapies, with the overall goal of improving the prenatal management of patients with LSDs.
Primary Outcome
Prenatal presentation of symptoms (e.g. hydrops) appearing on fetal imaging such as ultrasound and ECHO.
Conditions Studied
Interventions
- OTHER There is no intervention
Study Locations (1)
California
- University of California San Francisco - San Francisco
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 250 participants |
| Start Date | 2022-05-31 |
| Est. Completion | 2050-05-31 |
What NCT05619900 shows while recruiting
NCT05619900 is an observational study that tracks outcomes without assigning an intervention. The registered 250 participants enrollment target is mid-sized for trials with a published cap, above the 38-participant average among 7 other Mucopolysaccharidosis II trials with a reported enrollment target (558% higher).
The record links to 8 conditions, with Mucopolysaccharidosis II appearing as the primary indexed condition, and to 1 intervention - of which There is no intervention is the first listed.
NCT05619900 reports a single indexed study location in California.
Frequently Asked Questions
What is clinical trial NCT05619900 about?
NCT05619900 is a clinical study titled "Registry of Patients Diagnosed With Lysosomal Storage Diseases". This is an international prospective and retrospective registry of patients with Lysosomal Storage Diseases (LSDs) to understand the natural history of the disease and the outcomes of fetal therapies, with the overall goal of improving the prenatal management of patients with LSDs.
What is the current status of trial NCT05619900?
This trial is currently recruiting. The enrollment target is 250 participants. The study started on 2022-05-31. Estimated completion is 2050-05-31.
What conditions does trial NCT05619900 study?
This clinical trial studies the following conditions: Mucopolysaccharidosis II, Mucopolysaccharidosis VI, Mucopolysaccharidosis I, Wolman Disease, Mucopolysaccharidosis IV A.
What interventions are being tested in trial NCT05619900?
The interventions under investigation include: There is no intervention (OTHER).
Who is sponsoring clinical trial NCT05619900?
This trial is sponsored by University of California, San Francisco, which has 1,713 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT05619900 being conducted?
This trial has 1 study location across California. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
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