The natural history study of Sanfilippo syndrome Type C (study ID #JLK-447) sponsored by Phoenix Nest, began recruiting this week at one of its two locations! The first site is at the Hospices Civils De Lyon in Lyon, France and will be followed next year by the opening of a second site at UT Southwestern Medical Center in Texas, USA.
This is the first prospective, observational (“natural history”) study dedicated to individuals with Sanfilippo type C. Over the two-year study, the progression of Sanfilippo Type C disease will be documented in children and young adults through retrospective review of medical records and an ongoing collection of clinical data through study site visits and at-home recording devices. Learnings from this study will be critical to further drug development and future clinical treatment trials.
For further information about this study, including contact information for the study sites, visit the study listing on ClinicalTrials.gov.
Summary information about this and other clinical trials and studies for all types of Sanfilippo is available on the Foundation’s website.
Read the update from Phoenix Nest, which contains additional specifics about the Natural History Study and the ongoing work to develop a gene therapy treatment for those with Sanfilippo type C.
“Cure Sanfilippo Foundation continues to proudly support this ongoing work, thanks to its generous donors,” said Dr. Cara O’Neill, Co-Founder & Chief Science Officer of Cure Sanfilippo Foundation. “In partnership with Phoenix Nest, we are hopeful and focused on the goal of bringing forward the first enzyme-restorative therapy for individuals with Sanfilippo type C.”
We applaud the scientists who are working hard to complete preclinical activities and are encouraged by the positive data coming out of the ongoing study.
Jill Wood, CEO Phoenix Nest, shared in an email with the Foundation that, “JLK-247, [the Phoenix Nest-sponsored] gene therapy program is progressing well. New data shows an improvement in cognitive function and survival in our MPS IIIC mouse model. Toxicology studies in rats will begin in the winter of 2024. The data from both studies will support the filing of an Investigational New Drug (IND) [application for clinical trial allowance].”