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NBIA NEWS & INFORMATION
Spoonbill Foundation Contract
May 2019
NBIA Disorders Association recently signed a contract for $50,000 with the Spoonbill Foundation, a nonprofit founded by Drs. Susan Hayflick and Penny Hogarth from OHSU, to help support the design, build, and testing phases of the database for the upcoming CoA-Z clinical trial in PKAN. This database, built in an application called REDCap Cloud, will be structured to meet the highest FDA standards. The database will be customized to meet the exact needs of the study, including the necessary fields to collect data and track specimens and study product, and built-in checks to monitor the data quality and accuracy. Developing this type of sophisticated database will directly promote the success of the trial and pave the way for future steps beyond the trial.
Two new grants awarded to BPAN researchers in January
April 2019
Two new BPAN grants were awarded in January from the University of Pennsylvania, with crucial input from the NBIA Disorders Association.
In both cases, the grants will enable researchers to build on their previous studies of BPAN, or Beta-Propeller Associated Neurodegeneration, which is fast becoming one of the most common forms of NBIA.
The money for the grants comes from last year’s Million Dollar Bike Ride held in May 2018 by the Orphan Disease Center at the University of Pennsylvania.
Two PKAN grants awarded this year in collaboration with sister groups
April 2019
Two new grants to study PKAN were awarded early this year by the NBIA Disorders Association in collaboration with two of our European sister organizations, AISNAF in Italy and Hoffungsbaum e. V. in Germany.
These grants mark the first time all three NBIA groups have teamed up to fund research projects.
The organizations received 12 proposals, with eight focusing on PKAN, three on BPAN and one on MPAN. All were evaluated by an International Scientific Advisory Board made up of scientists and clinicians with expertise in the field of rare, neurodegenerative diseases, including NBIA.
Three clinical trials are underway; two others are being planned
April 2019
Possible treatments for two NBIA disorders are being tested in clinical trials that are either in progress or being planned for the near future.
The best known of these is the Retrophin Inc. trial for Pantothenate Kinase-Associated Neurodegeneration, or PKAN, now underway at 20 sites in the U.S., Canada and Europe. It is the first trial of a medication that targets the underlying causes of this disorder.
Retrophin, based in San Diego, finished enrolling patients in December 2018, with approximately 82 PKAN patients between the ages of 6 and 65 years. The Fosmetpantotenate Replacement Therapy, or FORT study, is being conducted under a Special Protocol Assessment agreement. That means the U.S. Food and Drug Administration believes that the trial’s design is adequate to support the filing of a New Drug Application, assuming the results are favorable.
