Ascelia Pharma AB (publ) (STO: ACE), an oncology-dedicated orphan drug development company, on Monday reported that there are a number of sites now open for enrolment of patients in both the US and Europe in the global pivotal phase III clinical study SPARKLE with Mangoral. Enrolment of the first patient is expected shortly.

SPARKLE is a global multicentre and registration enabling study of Mangoral in up to 200 patients with severely reduced renal function and with known or suspected liver metastases. Ascelia Pharma expects top line results to be obtained by the end of 2020 or beginning of 2021.

According to the company, following national approvals in the US by the FDA, in Germany by BfArM and in Sweden by Läkemedelsverket, five sites were opened during December 2019 and January 2020 and are now ready to recruit patients. The current sites open for recruitment are Yale University School of Medicine in Connecticut, University of Wisconsin and Southwest Medical Imaging in Arizona in the US, as well as Karolinska University Hospital in Sweden and Universitätsmedizin Göttingen in Germany. Additional sites will be opened for recruitment shortly.

Mangoral is the first oral imaging drug being developed for liver MRI. In addition, Mangoral is covered by an Orphan Drug Designation in the US, targeting the large unmet need in patients, who cannot tolerate currently available contrast agents on the market due to impaired kidney function.

Ascelia Pharma added that SPARKLE aims to demonstrate the efficacy and safety of Mangoral compared to unenhanced liver MRI, with each patient being his/her own control subject. Primary efficacy, in terms of lesion visualisation compared to unenhanced MRI, will be evaluated by three independent blinded readers.

Also, liver MRI will be performed before and within few hours after oral Mangoral administration and basic safety parameters will be evaluated for up to five days after administration of Mangoral.