A clinical trial is a study that researchers conduct in order to determine whether a new treatment is safe and effective in patients. These tightly-monitored and highly-controlled trials are required by agencies such as the U.S. Food and Drug Administration (FDA), European Medicines Agency (EMA) and the Australian Therapeutic Goods Administration (TGA) for the approval of any new drug.
ArQule has designed the MOSAIC clinical trial to determine whether its drug candidate miransertib can effectively treat individuals with Proteus syndrome and PIK3CA-related overgrowth spectrum syndromes. Based on the mechanism of action of miransertib, you or your loved one must have a mutation in either the AKT1 or PIK3CA gene to be eligible to participate in the MOSAIC trial. For additional eligibility criteria, please visit our clinicaltrials.gov page (NCT03094832).
The MOSAIC trial will begin enrollment soon. If you wish to be notified once enrollment begins, please sign up to receive alerts here:
Proteus syndrome is a rare genetic overgrowth disorder caused by a mutation in the AKT1 gene, which is involved in controlling cell growth. A mutation in this gene can lead to the overgrowth of organs and tissues throughout the body such as bones and skin. The overgrowth associated with PS often starts in infancy and accelerates during early adolescence.
PIK3CA-related overgrowth spectrum is a group of rare genetic overgrowth disorders caused by mutations to the PIK3CA gene, which is involved in the same pathway as AKT1 and controls cell growth. Like in Proteus syndrome, these mutations lead to overgrowth of tissues throughout the body. PROS is typically congenital or emerges in early childhood.
Some of the syndromes/diseases with the PIK3CA mutation are:
Miransertib is a small molecule drug candidate that inhibits the PI3K/AKT pathway, a pathway in our cells that helps to control cell growth. Miransertib is administered by mouth once a day. ArQule believes that by inhibiting the PI3K/AKT pathway, miransertib can reduce cell overgrowth and therefore become a transformative treatment option for those individuals with overgrowth disorders that are caused by mutations in the PI3K/AKT pathway.
Miransertib targets the pathway associated with both PS and PROS
If you or a loved one has been diagnosed with Proteus syndrome or one of a number of distinct overgrowth syndromes characterized by a mutation in the PIK3CA gene, we encourage you to reach out to the below resources.
Or, contact firstname.lastname@example.org for more information about the trial.