Date: 2017-05-17
Type of
information: update on patient enrollment
phase: 2
Announcement: update on patient enrollment
Company: Newron (Italy)
Product: sarizotan
Action
mechanism:
- 5-HT1A receptor agonist/D2 receptor antagonist. Sarizotan is a full 5-HT1a agonist and a dopamine D2-like agonist/partial agonist. This compound has been licensed from Merck KGaA.
- Sarizotan has been associated with a 70 to 85 percent reduction of apneas and hyperventilation episodes in preclinical testing with both acute and chronic dosing.
- Patients with Rett syndrome experience respiratory symptoms, such as episodes of apnea and hyperventilation, whose onset is as early as 3 years of age and that may persist for 10-15 years. These apneic episodes may occur as frequently as 10-60 times / hour during waking time. Regulatory endorsement of the study endpoints represents a growing recognition of the importance and contribution of respiratory symptoms. Sarizotan has shown consistent and statistically significant reduction in apneic episodes in genetic knock-out murine models of Rett’s syndrome that exhibit the characteristic phenotypic features of the disease. These effects were achieved with plasma levels of sarizotan that approximate those that will be achieved at the doses evaluated in the STARS study.
- Sarizotan received orphan drug designation for the treatment of Rett syndrome from both the European Commission and the FDA in 2015.
Disease: Rett syndrome
Therapeutic
area: Rare diseases - Neurodevelopmental diseases
Country: Australia, India, Italy, UK, USA
Trial
details:
- STARS (Sarizotan Treatment of Apneas in Rett Syndrome) is a randomized, double-blind, placebo-controlled study to evaluate the efficacy, safety and tolerability of sarizotan in patients with Rett syndrome with respiratory symptoms. The trial is expected to enroll 129 patients (three groups of 43), in up to 15 centers of excellence in the USA, the UK, Italy, Australia and India.
- The potentially pivotal clinical study will evaluate the efficacy, safety and tolerability of sarizotan in patients with Rett syndrome suffering from respiratory symptoms.
- The primary endpoint for the STARS study is reduction in episodes of clinically significant apneas (>10 sec) during waking time by 20 percent. During this 6-month study, patients will receive treatment with daily doses of 10 and 20 mg of sarizotan, or placebo.
- Sarizotan received Orphan Drug Designation for the treatment of Rett syndrome from both the European Commission and the FDA in 2015.
Latest
news:
- • On May 17, 2017, Newron Pharmaceuticals announced that the Sarizotan Treatment of Apneas in Rett Syndrome (STARS) study has been expanded to include Rett syndrome patients as young as six years of age. An amendment to allow the inclusion of the patients under the age of 13 was approved by the FDA after Newron submitted results from juvenile toxicology studies. Newron is also submitting the amendment to Health Authorities and Institutional Review Boards in other countries. Newron Pharmaceuticals expects to have top-line results available for release in 2018.
- Besides the STARS efficacy study, Newron, as part of its commitment to the rare disease patient community, is partnering with the global Rett community to work on the first Burden of Disease (BOD) study. The study aims to deliver data and analytics to quantify the physical, emotional and financial challenges of Rett syndrome. These learnings can help identify improved intervention programs and services designed to complement the Rett care pathway.
- • On July 21, 2016, Newron Pharmaceuticals announced the initiation of the STARS study to evaluate the efficacy, safety and tolerability of sarizotan in patients with Rett syndrome suffering from respiratory symptoms. The first US study center initiated is the Rush University Medical Center, Chicago, Peter T. Heydemann.
- • On June 16, 2016, Newron Pharmaceuticals is scheduled to present the design of the upcoming clinical trial of sarizotan addressing respiratory abnormalities in Rett syndrome (STARS) at the 2016 Rett Syndrome Symposium at the Eaglewood Resort & Spa, Itasca, Illinois, USA, from June 22-24, 2016. The presentation is part of the poster session scheduled for Thursday, June 23.
- • On May 17, 2016 , Newron Pharmaceuticals announced that its Investigational New Drug (IND) application for the evaluation of sarizotan for the treatment of patients withRett syndrome was approved by the FDA. Newron expects to initiate the potentially pivotal STARS (Sarizotan Treatment of Apneas in Rett Syndrome) in the third quarter of this year.
- • On October 28, 2015, Newron Pharmaceuticals announced the study design and details of its planned pivotal trial for sarizotan for the treatment of patients with Rett syndrome. The planned international pivotal trial with sarizotan is a double-blind, placebo-controlled efficacy study that has been designed based on extensive discussions with regulatory authorities in Europe, the U.S. and Canada as well as consultation with an international group of physicians specializing in Rett syndrome and a leading advocacy group at Rettsyndrome.com.
Is
general: Yes
