Can-Fite Submits First Piclidenoson Phase 2 Protocol for Lowe Syndrome

Can-Fite BioPharma submitted a Phase 2 protocol to Bambino Gesù Children’s Hospital in Rome for the first clinical evaluation of Piclidenoson in patients with Lowe syndrome. The open-label, single-center study will give oral Piclidenoson twice daily for six months to five adults with genetically confirmed disease. Its primary endpoint measures improvement in renal uptake of 99mTc-DMSA, with secondary measures covering tubular-function biomarkers, Fanconi syndrome parameters and safety. Can-Fite said preclinical studies found restoration of OCRL-dependent cellular function; Lowe syndrome currently has no approved disease-modifying therapy.