For this reason, small CHMI studies usually seek to assess proof-of-concept in the first instance, with a view to validating observed efficacy results in repeat studies or progressing to a Phase IIb field study if a highly encouraging signal is seen.
The project had a strict time line for assessing proof of concept, and the field team was given a clear payback schedule in order to track the business's performance.
The LowSALT CKD study is an investigator-initiated study specifically designed to assess the proof-of-concept and efficacy of sodium restriction in patients with established CKD.
The project provided considerable basic support to assess this proof of concept.
We assessed the proof of concept of this approach by correcting ZAP-70 deficient severe combined immunodeficiency (SCID) in mice.
With a view towards economic and efficient gDNA extraction from blood, and to determine the quality of these extractions, PCR amplification of antibody variable (v -region genes from the extracted nucleic acids were assessed as a proof-of-concept for DNA-based diagnosis.
The efficacy and safety of canakinumab were initially assessed in a proof of concept study for patients with MWS carrying the NLRP3 gene mutation.
To assess this, we performed a proof-of-principle study on 14 KMT2D and two KDM6A nonsense mutations using specific compounds that mediate translational readthrough and thereby stimulate the re-expression of full-length functional proteins.
The objective of this study was to assess safety, tolerability, and explore efficacy (proof-of-concept).
As a proof-of-principle, we assessed the performance of two different endothelial sources and the impact of different perfusion programs on endothelial viability after re-endothelialization of decellularized lung scaffolds.
These models recapitulate specific molecular pathways in tumor initiation or progression and provide a biological system in which to study the disease process for assessing efficacy of new therapies and proof-of-principle for testing molecularly targeted drugs.
