Newron Pharmaceuticals S.p.A (Newron) is a biopharmaceutical company specializing in developing inventive therapeutic options. They focus primarily on conditions related to the nervous system. According to bioportfolio.com, Newron announced on June 13th 2018, that their new clinical study involving Rett syndrome treatment will finishing enrolling participants in the second half of 2018. Keep reading to learn more, or follow the original source here for further details.
Rett syndrome is a rare neurological disorder that was previously considered part of the autism spectrum. It is caused by a mutation on the X chromosome and largely affects women. Babies typically show symptoms of Rett syndrome between the ages of 12 and 18 months. Symptoms vary but often include slowed brain growth, small head, difficulties with coordination and dexterity, lack of language skills, social anxiety, and uncoordinated breathing.
Newron’s clinical study, referred to as the STARS study, is expected to post results in the first half of 2019. More than 100 patients currently qualify for the trial. Newron wants to see the number of participants reach 129 patients aged six or higher.
STARS will specifically assess patients who experience clinically significant apneas (at least 10 episodes per hour during waking activity) while living with Rett syndrome. Apneas are observed in 70% of patients with Rett syndrome, and contribute to a wide variety of other conditions which may be connected to Rett syndrome.
Once the study group has been finalized, participants will be randomly assigned treatment with sarizotan. Those not receiving sarizotan receive a palcebo instead.
The study is set to last 24 weeks and will be conducted in a double-blind format. The goal STARS aims to achieve is a 20 percent decrease in apneas during active hours of wakefulness.
Thus far, there have been no major concerns reported about sarizotan. Some patients have, however, stopped treatment due to adverse affects or because the treatment did not appear to work for them. Safety will continue to be monitored by an independent board.