Actively Recruiting
Natural History Study of Participants With Sanfilippo Syndrome Type IIIC
Led by Phoenix Nest · Updated on 2026-05-12
30
Participants Needed
2
Research Sites
211 weeks
Total Duration
On this page
Sponsors
P
Phoenix Nest
Lead Sponsor
V
Vaincres Les Maladies Lysosomales
Collaborating Sponsor
AI-Summary
What this Trial Is About
This study is planned to document, through retrospective and prospective data collection, syndrome progression in children and young adults with MPS IIIC.
CONDITIONS
Official Title
Natural History Study of Participants With Sanfilippo Syndrome Type IIIC
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Confirmed diagnosis of Sanfilippo syndrome type C by enzyme deficiency, genetic testing, and urine analysis
- Signs or symptoms consistent with Sanfilippo syndrome type C or eligibility determined by sponsor and investigator
- Written informed consent from parent or guardian and assent from patient if required
- Parent or guardian willing to attend all study visits with the patient
- Ability to comply with study requirements as judged by the investigator
- Negative urine pregnancy test at screening for nonsterile females of childbearing potential
- Able to eat food or liquids by mouth and able to walk with or without assistance
- Vineland Adaptive Behavior Scales age equivalent of at least 1 year
You will not qualify if you...
- Received investigational drug within 30 days prior to baseline visit
- Medical condition or circumstance that may affect compliance, safety, or data interpretation as judged by investigator
- Significant non-MPS IIIC-related central nervous system impairment or behavioral disturbances that could affect study results
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Trial Site Locations
Total: 2 locations
1
The University of Texas Southwestern Medical Center
Dallas, Texas, United States, 75390
Actively Recruiting
2
Hospices Civils De Lyon
Bron, France, 690007539
Actively Recruiting
Research Team
N
Nathalie Reynes
CONTACT
N
Nathalie Guffon, MD
CONTACT
How is the study designed?
Study Type
OBSERVATIONAL
Masking
N/A
Allocation
N/A
Model
N/A
Primary Purpose
N/A
Number of Arms
3
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