2nd Quarter 2012 Research Report Card

To keep you better informed, the PKD Foundation will publish quarterly PKD Research and Development Report Cards, featuring updates on all PKD Foundation research and development initiatives, including drug development/repurposing, targeted and core grants, scientific meetings and more.

Drug Development/Repurposing

During this fiscal year, five partnerships with Biotech and Pharma have been established to evaluate distinctly different mechanisms/pathways believed to be important in disease expression and progression of PKD. All of the compounds to be studied are at the candidate selection or beyond stage of development.



No new information.


Scientific Meeting Support

No new information.


PKD Outcomes Consortium Project (PKDOC)

Data Mapping Progress:
The Colorado, Emory, Mayo and CRISPI datasets have been validated and loaded into the C-Path Online Data Repository (CODR). The CRISP2 dataset, which extends the longitudinal clinical information for patients from CRISP1, is expected to be loaded into CODR by June 29. This is a major milestone in the PKDOC Project The data comes from a total number of 2547 patients; 1065 of those have two or more images (both US, CT or MRI) at least six months apart. Inclusion of the CRISP2 dataset is invaluable for the analysis phase of the project and significantly expands the imaging information to support TKV as a biomarker in clinical trials.

Data Analysis/Model Evaluation:
A draft analysis plan, 'Disease Progression Model for Total Kidney Volume' (TKV) is being developed. Emphasis is placed on the use of available data to identify the primary clinical outcomes, such as hypertension, hematuria, aneurysms, that will be modeled and analyzed relative to TKV expansion.

Biomarker Qualification Process:
The Qualification process began with the submission of a Letter of Intent to the FDA in January, 2012, and its acceptance by the FDA in February, 2012. Preparation of the submission package is just beginning. A face-to-face meeting with the FDA prior to the final package submission is tentatively scheduled for October.

Discarded Tissue:
We have had eight contacts this quarter.

Clinical Trial Awareness Program:
The development and implementation of the Accelerating Clinical Trials (ACT) Alerts was achieved this fiscal year. The first project involved a 2 phase ACT Alert partnership with Pfizer reaching up to 24 chapter patient populations. A second project has involved a recent and successful kickoff of our partnership with the Clinical Research Organization (CRO) Accelovance. This will include a nationwide outreach program.


HALT Study Update

There is a possibility that the HALT Clinical Trial will be extended by the NIH through July 2014. This clinical trial is evaluating whether or not the maintenance of normal blood pressure through the use of ACE (angiotensin converting enzyme) inhibitors alone or used with ARBs (angiotensin receptor blockers) can slow the progression of renal cystic disease. The study is proceeding well. It is important to note that this is the first interventional trial in ADPKD to use kidney volume as the primary endpoint. The HALT trial includes the largest collection of MRI images of the heart in ADPKD and extensive biomaterials, such as blood and urine samples, which will provide materials for future analyses related to clinical outcomes in ADPKD.

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©2016, PKD Foundation ·The PKD Foundation is a 501 (c)(3), 509 (a)(1) public charity.

©2016, PKD Foundation ·The PKD Foundation is a 501 (c)(3), 509 (a)(1) public charity.