Date: 2015-09-10
Type of information: Initiation of the trial
phase: 2-3
Announcement: initiation of the trial
Company: Orphazyme (Denmark)
Product: arimoclomol citrate
Action
mechanism: heat shock protein inducer. Arimoclomol is a small molecule inducer of heat shock proteins. heat shock protein inducer
Disease: Niemann-Pick disease type C
Therapeutic area: Rare diseases - Genetic diseases
Country: Denmark, France, Germany, Italy, Poland, Spain, Switzerland, UK
Trial
details: The AIDNPC programme comprises two stages: Stage 1: An observational study, which is a 6 month study used to map the individual patient’s baseline disease progression rate. The patients stay on current standard of care (including Zavesca and/or symptom management). The stage 1 will provide a baseline of each individual patient that will increase the likelihood of documenting a robust effect of arimoclomol within the following period of 12 months. Every patient completing the observational study is offered to enter Stage 2. Stage 2: The interventional study, starting immediately after completing stage 1, is a double-blind, controlled study, where patients will receive either arimoclomol or placebo in addition to their ongoing standard of care treatment for a 12-month period. Patients will be randomized 2:1, which means that two patients will receive arimoclomol for every one patient receiving placebo. Neither the patients nor the treating physicians will know during the 12 months whether they are receiving placebo or arimoclomol (i.e., ‘double-blind’).After completing the 12-month intervention study every patient will be offered arimoclomol. The primary endpoint of the study is disease progression rate, which, supported by biochemical readouts, will provide the basis for an application for market authorization at the completion of the study. Inclusion criteria: The participants in the AIDNPC trial programme must be between 2 and 18 years of age and diagnosed with Niemann-Pick disease type C. Moreover, they must be presenting with at least one neurological symptom.(NCT02435030)
Latest
news: * On September 10, 2015, Orphazyme announced update to its Niemann-Pick disease type C clinical programme — AIDNPC The control group will comprise a number of patients that will receive placebo for a period of 12 months during the conduct of the trial. However, patients participating in the trial will not be required to discontinue any registered medication that they are receiving when entering the trial. The trial design will allow two patients to receive arimoclomol for every patient receiving placebo. After 12 months, all patients will be offered to receive arimoclomol. Patients who experience abrupt and rapid deterioration during their participation in the trial will be offered a transfer to a “rescue arm” of the study where they will be offered open label arimoclomol. * On April 15, 2015, Orphazyme announced the assembly of its scientific advisory board to support the upcoming clinical trial in Niemann-Pick disease type C. This scientific advisory board comprises world-leading medical, clinical and scientific professionals in lysosomal storage diseases, including Niemann-Pick disease type C: Prof. Marc Patterson (USA, Mayo Clinic), Dr. Christine í Dali (Denmark, Rigshospitalet), Prof. Mark Walterfang (Australia, University of Melbourne) and Prof. Frances Platt (UK, University of Oxford). Orphazyme also announced that the company has planned an investigator meeting in May to effectively start patient recruitment in June. * On October 6, 2014, Orphazyme announced that the company prepares for Phase II/III registration-enabling clinical trial in Niemann-Pick disease type C with arimoclomol citrate. 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 and work is now underway to establish clinical centres in both Europe and the USA.
Orphazyme’s clinical programme AIDNPC that investigates the orally available small molecule arimoclomol in Niemann-Pick disease type C is initiating in Europe, with recruitment commencing in September. If proven effective, the programme will be moved as quickly as possible to registration to become available to all patients suffering from Niemann-Pick disease type C. After several interactions with regulatory authorities in the US and Europe it has become clear that registration of arimoclomol will require the performance of a double-blinded clinical trial with a control group.
