PHOENIX NEST BIOTECH

JLK-448

JLK-448, is a remote observational study of patients with Sanfilippo syndrome type C. Participants will be using the C-RARE application to film their child engaged in daily living and social activities. Caregivers will also complete questionnaires regarding their childs health and behaviors.

 

Enrollment is anticipated to begin November 2025 and will be announced through patient organizations. Caregivers of children with Sanfilippo type C will be directed to a site coordinator at UT Southwestern where they will provide documentation of their child’s diagnosis of MPS IIIC. Once screened and approved caregivers may enroll their child. This is a two- year study with filming and questionnaires conducted every 6-months for a total of five time points.  The point of the study is to help describe the disease symptoms and progression. 

 

Due to funding constraints the study is limited to 35 participants from Spanish, Portuguese, French and English speaking households. These languages are among the most common languages spoken in the MPS IIIC patient population. 

 

JLK-448 Funding Support

Sanfilippo Sud and collaborating patient groups $120,000.