Date: 2014-07-07
Type of information: Filing of an IND Clinical trial autorisation
phase: 1
Announcement: submission of an IND
Company: OPKO Health (USA - FL)
Product: Rayaldee™ ( calcifediol - vitamin D prohormone)
Action
mechanism: Rayaldee™ is a first-in-class oral vitamin D prohormone treatment being developed for secondary hyperparathyroidism in stage 3 and 4 CKD patients with vitamin D insufficiency. It has a proprietary modified-release formulation designed to gradually and reliably raise serum total 25-hydroxyvitamin D (prohormone) concentrations to targeted levels (at least 30 ng/mL) while avoiding upregulation of CYP24, a cytochrome P-450 enzyme that reduces the PTH lowering potency of current vitamin D supplements. Activation of calcifediol, the active ingredient in Rayaldee™, by the kidney is tightly regulated, preventing excessive elevation of serum calcium and related side effects which encumber current vitamin D hormone therapies and promote vascular and renal calcification. Once approved, Rayaldee™ is expected to address an approximate 4 million CKD stage 3 and 4 patients in the U.S. and many more, elsewhere, with SHPT and vitamin D insufficiency.
Disease: adjunctive therapy for the prevention of skeletal-related events (SREs) in patients with bone metastases undergoing anti-resorptive therapy
Therapeutic area: Cancer - Oncology
Country: USA
Trial
details: The initial investigation is a phase 1 dose titration study designed to evaluate the safety and tolerability of Rayaldee™ in patients with breast or prostate cancer that has metastasized to bone who are receiving treatment with zoledronic acid (a bisphosphonate) or denosumab (a RANKL inhibitor). Approximately 24 subjects (12 with each cancer type) will receive Rayaldee™ at a starting daily dose of 30 µg (1 capsule) for 4 weeks. The dose will escalate in 30 µg increments at 4-week intervals until predetermined biochemical endpoints are reached, at which time the subjects will enter a 12-week maintenance phase. Safety parameters and various markers of bone metabolism, immune function and tumor burden will be monitored at regular intervals.
Latest
news: * On August 7, 2014, OPKO Health announced the submission of an Investigational New Drug (IND) Application to the FDA under which Rayaldee™ will begin clinical evaluation as an adjunctive therapy for the prevention of skeletal-related events (SREs) in patients with bone metastases undergoing anti-resorptive therapy. This study is expected to commence later this year and to take approximately 18 months to complete. Following evaluation of this initial study, OPKO plans to conduct subsequent studies investigating the effects of Rayaldee™ on SREs and other parameters deemed to indicate potential clinical benefits in the targeted population.
