Excited to see JCR Pharmaceuticals’ intravenous JR-446 enzyme replacement therapy (ERT) for Sanfilippo Syndrome Type B is now listed on ClinicalTrials.gov for a trial in the U.S., Germany, and United Kingdom. This is in addition to the trial JCR has been running in Japan since 2024.
While the U.S./German/U.K. trial is not yet recruiting, families can review the listing to learn about the trial sites in preparation for enrollment opening. There are three locations listed in the U.S.:
- Children’s Hospital Oakland Research Institution, Oakland, CA, U.S.
Contact: Irene Chang
Phone: 415-476-2757
Email: Irene.chang@ucsf.edu - UPMC Children’s Hospital of Pittsburgh, Pittsburg, PA, U.S.
Contact: Deepa Rajan
Phone: 412-692-6787
Email: rajands@upmc.edu - Lysosomal and Rare Disorders Research and Treatment Center, Faifax, VA, U.S.
Contact: Ozlem Goker-Alpan
Phone: 703-261-6220 Email: ogoker-alpan@ldrtc.org
There are also trial sites listed in Europe:
University Medical Center Hamburg-Eppendorf, Hamburg, Germany
Contact: Nicole Maria Muschol
Phone: +49 (0) 40 7410 53714
Email: muschol@uke.deGreat Ormond Street Hospital for Children, London, United Kingdom
Contact: James Davison
Phone: 0207 405 9200, ext 0074
Email: james.davison@gosh.nhs.uk
In addition to the sites, the listing includes this trial’s inclusion and exclusion criteria.
Read the JR-446 listing on ClinicalTrials.gov at https://clinicaltrials.gov/study/NCT07640984.