RE: Pediatric brain scaling

From: Ken Luu Date: November 16, 2015 technical Source: mail-archive.com
Hi Mark, I've recently developed a popPK model for an IT dosed drug in infants (0-2yrs) and children (2-15yrs), median age for all studies were 5 yo. The work has not been published yet as the clinical development program is ongoing. Some background: The basis of the dosing scheme for this drug was theoretically derived (prior to any modeling) based on predicted CSF volume rather than brain weight. In the publication: Matsuzawa, J., et al. (2001). "Age-related volumetric changes of brain gray and white matter in healthy infants and children." Cereb Cortex 11(4): 335-342, CSF volume changes for infants up to 2 years but does not change as they age after 2 years. For this reason, the original clinical program dosed infants based on age and children at a fixed dose. In my model (unfortunately, a lot of it I cannot share at this time), I had sparse CSF concentrations measured along with sparse and rich sampling concentrations in plasma. The model simultaneously fit CSF and Plasma profiles. In the covariate analysis, either AGE or BWT was a statistically significant covariate to Vcsf (CSF volume of distribution). The relation identified was a piece-wise ("hockey stick") relation. Neither AGE for BWT was influential on CLcsf. The final model (thanked God) supported the original dosing rationale. When I was starting this work I couldn't find anything on this topic either. I hope this helps in thinking in terms of CSF volume instead of brain weight, if it's relevant to your compound. Ken Kenneth T. Luu, Ph.D. Director | PK & Clinical Pharmacology Isis Pharmaceuticals, Inc. | 2855 Gazelle Ct Carlsbad, CA 92010 Office: 760.603.2457 | Fax: 760-603-2502 | Email: [email protected]<mailto:[email protected]>
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From: [email protected] [mailto:[email protected]] On Behalf Of Mark Sale Sent: Monday, November 16, 2015 8:56 AM To: [email protected] Subject: [NMusers] Pediatric brain scaling Dear Colleagues, I'm working on a drug that is administered intrathecally to kids and adults. I'm interested in scaling pk from adults to kids (whose brains start out relatively large, compared to the body, but reach maximum size in the teens, then stop growing). I'm a little surprised that I can't find anything about dosing CNS drugs to peds, based on brain weight (and, ideally, brain clearance). Does anyone know of anything? thanks Mark Mark Sale M.D. Vice President, Modeling and Simulation Nuventra, Inc. (tm) 2525 Meridian Parkway, Suite 280 Research Triangle Park, NC 27713 Office (919)-973-0383 [email protected]<[email protected]> http://www.nuventra.com Empower your Pipeline CONFIDENTIALITY NOTICE The information in this transmittal (including attachments, if any) may be privileged and confidential and is intended only for the recipient(s) listed above. Any review, use, disclosure, distribution or copying of this transmittal, in any form, is prohibited except by or on behalf of the intended recipient(s). If you have received this transmittal in error, please notify me immediately by reply email and destroy all copies of the transmittal.
Nov 16, 2015 Mark Sale Pediatric brain scaling
Nov 16, 2015 Ken Luu RE: Pediatric brain scaling
Nov 16, 2015 Nick Holford Re: RE: Pediatric brain scaling