PHOENIX NEST 

Natural History Study of MPS IIIC

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JLK-447 

JLK-447 is a multi-center, retrospective and prospective observational study of patients with Sanfilippo syndrome type C.  The study will include 3 clinic visits (baseline, 12, and 24-months) where a myriad of clinical, behavioral, and cognitive assessments will be performed.

 

Additionally, on and between clinical visits prospective real-world data will be collected 6 times at home via C-RARE a video recording application.

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For study details and contact information please refer to NCT# 05825131.

The French site is now closed for enrollment, the US site enrolling now until October 1st 2026

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Funding Support

JLK-447 has received funding from:

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Vaincre les Maladies Lysosomales - Rare mais pas seul: Identification of Potential Biomarkers and Clinical Tools for use in Mucopolysaccharidoses IIIC Patients $541,000

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NIH/NINDS 1R44NS137892-01: 07/01/24-07/01/27, Identification of Potential Biomarkers and Clinical Tools for use in Mucopolysaccharidoses IIIC Patients $2,994,701