We have received the following statement from Carlos R. Camozzi, Chief Medical Officer, Orphazyme. Arimoclomol in NP-C Clinical Programme As a follow-up to the presentation delivered at the Niemann-Pick UK (NP-UK) meeting (26-27 September 2014), it is with great anticipation and pleasure that we announce that Orphazyme is now in the final preparation stages for a Phase II/III registration-enabling clinical trial in Niemann-Pick disease type C with Arimoclomol, a small molecule inducer of heat shock proteins. As presented at the NP-UK family conference we have obtained very promising data from our preclinical studies with arimoclomol in cells from patients, as well as in the Niemann-Pick disease type C mouse model. This work has been done in collaboration with Professor Frances Platt, University of Oxford. Arimoclomol is orally available and its safety profile has been established through extensive and multiple Phase I clinical trials. In order to reach a trial design from a clinical and regulatory perspective that allows the appropriate assessment of the efficacy of arimoclomol, Orphazyme has sought the guidance of the regulatory authorities as well as clinicians and other stakeholders. The clinical programme is composed of an Observational Study, expected to start a staggered enrolment in the first quarter of 2015 (March 2015). After 6 months of follow-up, all the patients that complete the Observational Study will enter (upon consent) into the Interventional Phase II/III trial with Arimoclomol. This will be a multi-centre trial. Work is now underway to establish clinical centres in both Europe and the USA. These will be announced once the discussions are complete. Key proposed inclusion criteria for participation in the Observational Study: • NPC diagnosis genetically confirmed • Male / female aged 2-18 (+11 months) • Ability to walk independently or with assistance • Treated or not treated with Miglustat Key proposed exclusion criteria: • Severe liver insufficiency or liver transplant • Patient with established epileptic “seizures period” (>3 epileptic seizures that required medication within 6 months prior to Observational Study) • Treatment with cyclodextrin or any other Investigational Medicinal Product (IMP) within 4 weeks prior to study enrolment. To participate in the Interventional Study, individuals must have completed the Observational Study. Orphazyme will deliver regular updates on the progress of the clinical study in order to keep the NPC community informed. Carlos R. Camozzi, Chief Medical Officer, Orphazyme
Posted on: Mon, 06 Oct 2014 07:29:49 +0000
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