International Niemann–Pick Disease Alliance
The AIDNPC clinical trial programme consists of two studies:
All patients have now started treatment with study medicine. At the latest end-of-the-month AIDNPC telephone conference hosted by the sponsor, Orphazyme ApS could announce that it had met its recruitment target of 46 patients. The following sites are participating in the ‘-002’ Study:
Great Ormond St, UK
Today, Orphazyme is pleased to announce that all 49 patients that have entered into the ‘-002’ study have started treatment with study medication. This means that the last patient’s last visit in the blinded part of the study will take place May 30th 2018, after which database lock and data analyse can commence. As each patient has his/her last visit in the blinded part of the study, he/she will enter into an open-label extension of the study where all patients will receive only the active substance, arimoclomol, as treatment (i.e., no one will receive placebo during the open-label extension of the study).
Orphazyme to present at Spanish NPC Association on June 24th
Orphazyme will be participating at the Scientific-Family Conference of the NPC Association of Fuenlabrada to be held on June 24th in Fuenlabrada (outside Madrid).
The graphic below illustrates the design of the ‘-002’ Study, including number and timing of patients visits to the sites. An escape route is provided for patients that experience an unacceptable rate of progression of the disease.
We encourage the sharing of above information with the patient community.
The next AIDNPC call is schedule for Thursday June 29th 2017 at 15h EDT.
Visit the AIDNPC Clinical Programme website: www.AIDNPC.com