Clinical trialCLINICALTRIALSApr 14
Researchers tested a new gene therapy called ST-920 for Fabry disease in 36 patients. This treatment uses a modified virus to deliver instructions that help the body make an enzyme called alpha-galactosidase A, which people with Fabry disease don't produce enough of. The trial is now complete and tested whether different doses were safe and well-tolerated.
WHY IT MATTERSThis completed Phase 1/2 trial is the first human test of ST-920, meaning results could help determine if gene therapy can provide long-term relief for Fabry disease patients who currently require lifelong enzyme replacement infusions.
Clinical trialCLINICALTRIALSMar 26
Researchers are testing a drug called cabozantinib-s-malate to treat children and young adults with rare cancers, including sarcomas (muscle cancers), Wilms tumor (kidney cancer), and other solid tumors that have come back after treatment or didn't respond to initial therapy. The drug works by blocking proteins that help tumors grow and form new blood vessels. This phase II trial has enrolled 109 patients and is no longer recruiting new participants.
WHY IT MATTERSIf your child has a recurrent or treatment-resistant sarcoma, Wilms tumor, or other rare pediatric solid tumor, this completed trial data may help inform whether cabozantinib could be an option to discuss with their oncologist.
Clinical trialCLINICALTRIALSMar 26
Researchers tested a cancer drug called osimertinib (AZD9291) in 19 patients with advanced cancers that have specific genetic changes in a gene called EGFR. The drug works by blocking a mutated protein that helps cancer cells grow. This trial is now complete and results have been posted.
WHY IT MATTERSThis trial provides evidence that osimertinib may work against cancers with EGFR mutations across different cancer types, potentially offering a treatment option for patients whose tumors have this specific genetic change.