As a collaborator on this study, Cure Sanfilippo Foundation is thrilled to announce the MPSIII (Sanfilippo) Anakinra clinical trial is now listed as “recruiting” on ClinicalTrials.gov.
This is an option for any Type of Sanfilippo (A, B, C, D).
Read the full listing at https://clinicaltrials.gov/ct2/show/NCT04018755.
Study Description
Inflammation has been connected with disease pathogenesis in the MPS disorders. Therapies aimed at decreasing inflammation are currently being studied in many MPS disorders and benefits in both brain and other parts of the body have been reported.
Decreasing interleukin-1 (IL-1) in an animal model of MPS III showed benefits in brain disease and behavior. Thus, we think that Anakinra (Kineret), which decreases IL-1 levels in the body, will improve behavioral and other problems in children with MPS III.
The expected potential direct benefits include, but are not limited to, improved behavior, sleep, stooling, communication, mood, and gait; as well as decreased seizure frequency, disordered movement, and fatigue.
However, there is no guarantee that participants will get any benefit from being in this study.
Study Contact Information
If interested, please contact the Clinical Trial site, Lundquist Institute (formerly LA Biomed). All contact details can be found at link below.
If you have any questions for the study collaborator, Cure Sanfilippo Foundation, e-mail Contact@CureSanfilippoFoundation.org./.