A Phase 2/3 trial evaluating the treatment candidate Anavex-2-73 (blarcamesine) in children and adolescents with Rett syndrome has started dosing participants, according to Anavex Life Sciences, the therapy’s developer.
The trial, named EXCELLENCE (NCT04304482), is testing the safety and efficacy of the small molecule therapy as a treatment for the neurodevelopmental disorder. At least 69 participants, ages 5-18, will be randomly assigned to receive either Anavex-2-73 or a placebo every day for 12 weeks. All participants may then continue to be treated with the oral liquid therapy during a voluntary extension period.
The study is still recruiting participants in Australia, with further clinical sites across the world expected to open in the future. More information on the trial, its contacts, and how to enroll is available at www.rettsyndrometrial.com and at the trial’s page here.
“We are delighted to expand the ANAVEX®2-73 (blarcamesine) treatment program for Rett syndrome to include pediatric patients,” Christopher U. Missling, PhD, president and CEO of Anavex, said in a press release.
Anavex-2-73 is a man-made (synthetic) small molecule that works by activating the sigma-1 receptor, a protein involved in ensuring that other proteins have the correct 3D structure. The therapy is intended to decrease the amount of proteins with a wrong shape, which could in turn result in lower cellular stress and the improved functioning of cells’ mitochondria — their “powerhouses.”
In a mouse model of Rett syndrome, Anavex-2-73 significantly improved motor and reflex responses to stimuli, compared with a placebo. In addition, mice given the potential therapy also showed better balance and gait (walking) patterns, as well as eased hind limb clasping. Hind limb clasping resembles the characteristic hand stereotypes — or purposeless, repeated movements — seen in people with Rett.
Anavex is currently exploring the safety, tolerability, and efficacy of its experimental therapy in two other trials, both of which are still recruiting participants ages 18-45. A Phase 2 study (NCT03758924) is underway in the U.S.; contact information is here. Meanwhile, the AVATAR trial (NCT03941444) is ongoing in Australia and the U.K. Enrollment information is here.
The U.S. Food and Drug Administration has granted Anavex-2-73 orphan drug, fast track, and rare pediatric disease designations for the treatment of Rett syndrome. Each designation works to facilitate the development of medications to treat serious conditions and fill an unmet medical need.
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