Plasmalogen Blood Test Pilot Study
Rhizokids July 2018
Dear parents, we are asking for your participation in a pilot study this summer at the RhizoKids conference in Alabama to test a new finger-prick blood collection protocol for measuring plasmalogen levels. Being able to determine plasmalogen levels in RCDP children is important for determining whether a plasmalogen augmenting therapy, such as PPI-1040, is working correctly. It is also important for establishing the natural variation in plasmalogen levels over time, which could later be used as the "reference" point for evaluating treatment.
We know that conventional blood draws on children (especially those with RCDP) can be challenging, so our lab has developed a new method that uses a product called the Mitra cartridge that collects 10 microliters of blood from a finger prick. 10 microliters is a very small amount - less than a single drop. We would like to test the method this July at the Alabama Rhizokids conference, with the intention to continue to use it for the duration of the natural history and PPI-1040 intervention study.
What is involved
At the start of the conference, Drs. Tara Smith and Shawn Ritchie from Med-Life Discoveries (the company developing PPI-1040), will provide a brief update about the clinical trial plan, including this particular blood collection study, via Skype. Ms. Moussumi ?????, who is at the conference to begin creating an RCDP quality of life questionnaire, will be present to organize signing of consent forms to participate in the study. A time will be scheduled for a nurse of doctor at the conference to perform the finger prick and collect the blood sample. The sample is then allowed to dry and shipped to Med-Life Discoveries for analysis.
ELIGIBILITY and Informed Consent
All parents willing to participate must sign an informed consent form. All RCDP patients and unaffected siblings are eligible to participate. It is important to collect samples from some unaffected siblings so that we have a normal plasmalogen comparison group. There is no cost to participate in the study.
A copy of the consent form is linked above and can be reviewed prior to attending the conference. Any questions about the consent or the study can also be sent ahead of the conference using the button above.
The test kit and procedure
The product that we are using for the blood collection is a commercially available finger prick test kit, manufactured by Neoteryx. The Neoteryx Mitra Cartridge requires less blood than the cards typically used for new-born screening, which means a small-gauge lancet can be used. The product looks like a small Q-Tip with a special wick that soaks up a very small volume of blood. We have tested the product on ourselves several times and could barely feel the lancet. There are no specific pre-sampling requirements needed (i.e., subjects do not have to be fasted, and the sample can be taken any time during the day).
A video describing the procedure is shown below. Following the collection, the cartridge will be sent to Med-Life Discoveries and plasmalogens extracted and analyzed by mass spectrometry.
After the pilot
After the pilot project is complete, we anticipate continuing to collect blood samples as part of the Natural History study over the next year. Since the product is easy to use, our plan is to ship the kits to participants to be performed by parents in their home. Our goal is to collect approximately four samples over the period of 12 months.