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NCT05825131 · ClinicalTrials.gov registry record
Natural History Study of Participants With Sanfilippo Syndrome Type IIIC
A clinical trial of Sanfilippo Syndrome Type C, sponsored by Phoenix Nest.
- Recruiting
- Registry status
- 30
- Enrollment target
- 2
- Study locations
NCT05825131: Recruiting study of Sanfilippo Syndrome Type C, sponsored by Phoenix Nest.
NCT05825131 is a study of Sanfilippo Syndrome Type C that is actively recruiting participants, run by Phoenix Nest. The registered enrollment target is 30 participants. The trial reports 2 study locations across 2 states. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT05825131, a study of Sanfilippo Syndrome Type C, is actively recruiting participants, sponsored by Phoenix Nest.
- RECRUITING
- Registry status
- 30 participants
- Enrollment target
- 2
- Study locations
Study Summary
This study is planned to document, through retrospective and prospective data collection, syndrome progression in children and young adults with MPS IIIC.
Primary Outcome
The Bayley Scales of Infant and Toddler Development, 4th edition (Bayley-4) is a standardized developmental assessment that provides raw scores for 5 subtests (cognitive, expressive communication, receptive communication, fine motor, gross motor) and standard score norms converted to percentiles for 3 scales (cognition, communication, motor). The Bayley-4 raw scores range from 0-162 for the cognitive subtest, 0-84 for the receptive communication subtest, 0-74 for the expressive communication sub
Conditions Studied
Study Locations (2)
Texas
- The University of Texas Southwestern Medical Center - Dallas
Other
- Hospices Civils De Lyon - Bron
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 30 participants |
| Start Date | 2024-11-10 |
| Est. Completion | 2027-11-01 |
What NCT05825131 shows while recruiting
NCT05825131 is an observational study that tracks outcomes without assigning an intervention. The registry caps enrollment at 30 participants, a relatively small participant target.
The record links to 1 condition, with Sanfilippo Syndrome Type C appearing as the primary indexed condition, and to 0 interventions.
NCT05825131 reports a single indexed study location in Texas, Other.
Frequently Asked Questions
What is clinical trial NCT05825131 about?
NCT05825131 is a clinical study titled "Natural History Study of Participants With Sanfilippo Syndrome Type IIIC". This study is planned to document, through retrospective and prospective data collection, syndrome progression in children and young adults with MPS IIIC.
What is the current status of trial NCT05825131?
This trial is currently recruiting. The enrollment target is 30 participants. The study started on 2024-11-10. Estimated completion is 2027-11-01.
What conditions does trial NCT05825131 study?
This clinical trial studies the following conditions: Sanfilippo Syndrome Type C.
Who is sponsoring clinical trial NCT05825131?
This trial is sponsored by Phoenix Nest, which has 2 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT05825131 being conducted?
This trial has 2 study locations across Texas. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
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