To evaluate the safety and efficacy of a novel microbial lipase (NM-BL) in a liquid formulation for the treatment of exocrine pancreatic insufficiency (EPI) in patients with cystic fibrosis (CF) in a phase IIa proof-of-concept study.
The goal of the study is to establish safety, tolerability, and CNS penetration in patients with AD, in preparation for a phase IIa proof-of-concept clinical trial beginning in 2014.
A Phase IIa proof-of-principle study showed that miravirsen monotherapy can achieve a HCV RNA decline in serum of up to 2.7 log10 IU/ml (Janssen et al, 2013).
Phase I trials, typically conducted in healthy volunteers to assess safety, are followed by phase IIa trials of "proof of concept" and phase IIb trials for "dose finding" in participants with the condition of interest.
This study was designed as an exploratory, signal-detection, Phase IIa study to provide proof-of-concept for this novel compound prior to embarking on a larger scale Phase IIb program.
A previously published proof of principle phase IIa trial with 113 patients from Kabul showed that bipolar high-frequency (HF) electro-cauterization (EC) of cutaneous leishmaniasis (CL) ulcers and subsequent moist wound treatment (MWT) closed 85% of all Leishmania (L).
A phase Ib study of saracatinib (AZD0530), a Src family kinase inhibitor with high potency for Fyn and Src kinase, is currently underway to treat patients with AD (NCT01864655), with a planned proof-of-concept phase IIa trial in 2014.
Demonstration of proof-of-concept in a Phase IIa CHMI study that an effective BSV can significantly impact PMR in vaccinated volunteers.
Tissot, A. C. et al. Effect of immunisation against angiotensin II with CYT006-AngQb on ambulatory blood pressure: a double-blind, randomised, placebo-controlled phase IIa study.
Pockros, P. J. et al. Oral resiquimod in chronic HCV infection: safety and efficacy in 2 placebo-controlled, double-blind phase IIa studies.
