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 provided considerable basic support to assess this proof of concept.
Although this proof of concept model is typical of the investigation of a new drug (8 ), similar methods have been used to assess the proof of concept of novel teaching methods for physicians (9 ), mind body techniques for stress reduction (10 ), and intersectoral collaboration between health services and fire departments to prevent elderly persons from falling (11 ).
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.
We assessed the proof of concept of this approach by correcting ZAP-70 deficient severe combined immunodeficiency (SCID) in mice.
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 address this, previous efforts have used formal proof analyses to assess the robustness of collaborative procedures to miscommunications.
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.
These changes explained the lower increase of porosity during proofing, assessed by digital camera and 2D image analysis.
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 objective of this study was to assess safety, tolerability, and explore efficacy (proof-of-concept).
