MEdPACTO

A phase 1/2 clinical trial for adolescents and adults with recurrent, refractory, or progressive osteosarcoma

On July 11th, MedPacto, a genome-based drug discovery and clinical-stage biotechnology company, announced the submission of an investigational new drug (IND) application to the U.S. Food and Drug Administration (FDA) for its Vactosertib as a single agent in patients with recurrent, refractory or progressive osteosarcoma.

Case Western Reserve University School of Medicine, UH Rainbow Babies and Children's Hospital is designated as one of the clinical trial institutions. It is an upcoming global and multicenter clinical trial, a phase 1/2 study to assess the safety, tolerability, pharmacokinetics, and antitumor activity of Vactosertib as a single agent in a total of 48 patients including adolescents (ages 14-18) and adults with recurrent, refractory or progressive osteosarcoma.

Osteosarcoma, a malignant osseous neoplasm that usually develops in connective tissue, such as bone, cartilage, or muscle, is one of the most commonly reported rare cancers that accounts for 5.6% of sarcoma cancer. It can develop at any age, but most cases occur during childhood, early adolescence, and young adulthood, and among the reported cases, 25~50% experience lung metastasis even during chemotherapy.

Osteosarcoma treatment typically involves surgery, chemotherapy, and radiation therapy. Surgeries in parallel with chemotherapy makes up roughly 45.5% of all total treatments while drug treatment makes up only 15.9%, hence there is a growing need for new treatment options.

“This upcoming clinical trial is to assess the safety, tolerability, pharmacokinetics, and antitumor activity of Vactosertib as a single agent in adolescents and adults with recurrent, refractory or progressive osteosarcoma.”, “We may expand into safety/efficacy into a larger population upon results from an interim analysis,” stated a MedPacto representative.