International Niemann–Pick Disease Alliance
Update #18 on the AIDNPC clinical programme
(arimoclomol in treatment of Niemann-Pick disease type C)
Interim information bulletin (following the Dec. 28th call)
001 Study: Status
The study was recently completed in May 2017. A total of 36 patients were enrolled across 12 sites in 8 countries.
The primary objective of the 001 study was to characterize the individual patient’s disease progression profile through clinical, biological and quality-of-life measures recorded prospectively, as well as the historic disease information collected from patient medical records.
The 001 Study database has been closed and data analysis is under way. The findings of this natural history study in NPC patients, which has the unique strength of being a prospective study aligned with the design of the 002 Study, will be presented at meetings in the next half year and will serve to set the expectations for findings of the 002 Study. E.g., the results will form the basis for a base line in part of the later analysis of the 002 Study.
002 Study: Status and timeline
The last patient in the 002 study (a phase 2/3 trial) enrolled in May 2017, for a total of 50 patients enrolled and randomized, of which a total of 27 patients rolled over from the 001 study. We expect to have results from this study in the autumn of 2018. As each patient has completed the blinded part of the study, he/she is being offered to enter into the open-label (arimoclomol only) extension of the study.
The following sites are participating in the 002 Study:
Great Ormond St, UK
A simplified 5–Domain NPC-CSS scoring tool
Orphazyme has concluded validation of a simplified 5–Domain NPC-CSS scoring tool, with the intention to improve tracking and scoring of NPC patients in the future. The domains in this 5–Domain NPC-CSS scoring tool is ambulation, fine motor skills, swallowing, speech and cognition. This scoring tool is being used in clinical assessment of the patients in the 002 Study.
Orphazyme present at the WORLDSymposium in San Diego
Orphazyme will present and be present at the 14th Annual WORLDSymposiumTM
in San Diego (February 5–9, 2018).
Clinical Design Overview
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 March 29th 2018 at 15h EDT.