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NCT05523206 · ClinicalTrials.gov registry record
A Natural History Study of Biomarkers and Clinical Outcomes in Mucopolysaccharidosis Type IIIA (MPS IIIA; Sanfilippo Syndrome)
A clinical trial, sponsored by Sanguine Biosciences.
- Completed
- Registry status
- 15
- Enrollment target
NCT05523206 is a clinical trial that has completed, run by Sanguine Biosciences. The registered enrollment target is 15 participants.
The verdict
NCT05523206 has completed, sponsored by Sanguine Biosciences.
- COMPLETED
- Registry status
- 15 participants
- Enrollment target
Study Summary
This protocol is a decentralized, single cohort, natural history and biomarker study enrolling up to 20 participants with MPS IIIA (Sanfilippo syndrome). At least 10 participants (\~50%) must be less than four years old at the time of the Parent/LAR consent. The study will have a screening process and 7 study visits, e.g. home visits, that will consist of serum collection and completion of a remote assessment of the Vineland Adaptive Behavior Scales 3rd Edition (Vineland-3) MPS IIIA remains a devastating disease with a high unmet medical need. There is currently a limited number of available data to adequately characterize the progression of the disease. Analysis of blood biospecimens and adaptive behavior in this study will help researchers better understand the clinical progression of MPS IIIA. A better understanding of disease progression may assist in developing novel therapies for rare genetic disorders.
Interventions
- OTHER No intervention
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 15 participants |
| Start Date | 2022-09-27 |
| Est. Completion | 2025-01-28 |
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Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT05523206
The ClinicalTrials.gov registry entry for NCT05523206 describes a study currently listed as completed, categorized as an unspecified phase. The registered enrollment target is 15 participants, a figure that helps gauge the scale of data the investigators plan to collect. The listed sponsor is Sanguine Biosciences, which has 11 total studies on file at ClinicalTrials.gov.
The record links to 0 conditions, and to 1 intervention - of which No intervention is the first listed.
NCT05523206 reports 0 study locations.
Frequently Asked Questions
What is clinical trial NCT05523206 about?
NCT05523206 is a clinical study titled "A Natural History Study of Biomarkers and Clinical Outcomes in Mucopolysaccharidosis Type IIIA (MPS IIIA; Sanfilippo Syndrome)". This protocol is a decentralized, single cohort, natural history and biomarker study enrolling up to 20 participants with MPS IIIA (Sanfilippo syndrome). At least 10 participants (\~50%) must be less than four years old at the time of the Parent/LAR consent. The study will have a screening process a...
What is the current status of trial NCT05523206?
This trial is currently completed. The enrollment target is 15 participants. The study started on 2022-09-27. Estimated completion is 2025-01-28.
What interventions are being tested in trial NCT05523206?
The interventions under investigation include: No intervention (OTHER).
Who is sponsoring clinical trial NCT05523206?
This trial is sponsored by Sanguine Biosciences, which has 11 total clinical trials registered on ClinicalTrials.gov.
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