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NCT05648851 · ClinicalTrials.gov registry record

A Natural History Study of Sanfilippo Syndrome Type D

A clinical trial, sponsored by Phoenix Nest.

Completed
Registry status
10
Enrollment target

NCT05648851: Completed study, sponsored by Phoenix Nest.

NCT05648851 is a clinical trial that has completed, run by Phoenix Nest. The registered enrollment target is 10 participants. According to ClinicalTrials.gov, the official US trial registry.

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The verdict

NCT05648851 has completed, sponsored by Phoenix Nest.

COMPLETED
Registry status
10 participants
Enrollment target

Study Summary

Sanfilippo syndrome type D is a ultra rare syndrome with limited available natural history data. This study is planned to document, through retrospective and prospective data collection, syndrome progression in children and young adults with Sanfilippo syndrome type D. The results from this study may inform future clinical studies in targeted therapies for patients with Sanfilippo syndrome type D and may serve as an external control since there are very few patients with Sanfilippo syndrome type D.

Primary Outcome

Assessed using the Leiter-3, evaluation of nonverbal cognitive, neuropsychological abilities. The Leiter-3 emphasizes fluid intelligence a measure of the individuals innate cognitive abilities. Children's Color Trail Test (CCTT) assesses sustained attention, sequencing, and other executive functions while reducing reliance on language.

Trial Details

FieldValue
Enrollment Target 10 participants
Start Date 2023-01-22
Est. Completion 2024-11-01
Phoenix Nest

2 total trials

What the finished NCT05648851 record still lists

NCT05648851 is an observational study that tracks outcomes without assigning an intervention. The registry caps enrollment at 10 participants, a relatively small participant target.

The record links to 0 conditions, and to 0 interventions.

NCT05648851 does not publish any study locations in the registry export this page uses.

Frequently Asked Questions

What is clinical trial NCT05648851 about?

NCT05648851 is a clinical study titled "A Natural History Study of Sanfilippo Syndrome Type D". Sanfilippo syndrome type D is a ultra rare syndrome with limited available natural history data. This study is planned to document, through retrospective and prospective data collection, syndrome progression in children and young adults with Sanfilippo syndrome type D. The results from this study ma...

What is the current status of trial NCT05648851?

This trial is currently completed. The enrollment target is 10 participants. The study started on 2023-01-22. Estimated completion is 2024-11-01.

Who is sponsoring clinical trial NCT05648851?

This trial is sponsored by Phoenix Nest, which has 2 total clinical trials registered on ClinicalTrials.gov.

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Source: ClinicalTrials.gov NCT05648851, the US trial registry maintained by the National Library of Medicine. NCT05648851 (small enrollment · none site footprint · completed) retrieved and formatted by PlainTrial, see methodology.