Ethics approval for plasmalogen sampling in children unaffected by RCDP

The Roy Romanow Provincial Laboratory (RRPL) in Regina Saskatchewan.

MLD, in collaboration with the Roy Romanow Provincial Laboratory (RRPL) in Saskatchewan, has recently obtained ethics approval to begin a prospective study to analyze plasmalogen levels among children. Our quantitative plasmalogen assay has nearly completed formal FDA-level validation, and is superior to other currently available approaches, including the conventional DMA methods. MLD’s assay is being used to quantify plasmalogen species among RCDP patients as part of the RCDP Natural History Study and subsequent PPI-1040 clinical trial.

The purpose of the current study in Saskatchewan is to create a robust pediatric population distribution of plasmalogen levels. In addition to the IND safety pharmacology and adult human Phase I trial, this study will allow us to more accurately compare levels between RCDP patients and non-affected children, as well as aid in determining optimal PPI-1040 dosing and plasmalogen pharmacokinetic responses in patients with RCDP upon dosing.