Date: 2017-02-28
Type of
information: Initiation of the trial
phase: natural history
Announcement: initiation of the trial
Company: Achillion Pharmaceuticals (USA - CT) Imperial College London (UK)
Product:
Action
mechanism:
Disease: C3 glomerulopathy
Therapeutic
area: Rare diseases - Renal diseases - Kidney diseases
Country:
Trial
details:
Latest
news:
- • On February 28, 2017, Achillion Pharmaceuticals announced that it has entered into an agreement with Imperial College London to conduct a natural history study of C3 glomerulopathy (C3G), a rare renal disorder which includes dense deposit disease (DDD) and C3 glomerulonephritis (C3GN).
- This study, funded by Achillion, is being conducted by a team of researchers led by Dr. Matthew Pickering and Dr. H. Terry Cook, both of Imperial College. The title of the study is "Natural History Study of C3 Glomerulopathy (C3G): Discovery of Histological Predictors of Outcome." The study is expected to be three years in length. It will include up to 400 participants.
- C3 glomerulopathy (C3G) describes a rare renal disease characterized by the presence of fragments of a protein called C3 in the filtering units (glomeruli) of the kidney. C3 fragment accumulation results from over-activation of part of the immune system known as the complement alternative pathway. This accumulation results in inflammation in the glomeruli (glomerulonephritis) and subsequent permanent damage (need for dialysis or transplant) in 30-50% of patients within 10 years of diagnosis. C3G affects persons of all ages, although the mean age appears to be lower in DDD patients as compared to C3GN patients. The incidence of C3G is estimated at 2-3 per 1,000,000 people.
- Achillion researchers have designed a library of nearly 2,000 small molecule inhibitors of complement factor D. Factor D occupies a critical position in the complement alternative pathway. Dysregulation of the alternative pathway can induce inflammation and tissue damage and is associated with a variety of diseases, including PNH and C3G. Achillion's lead candidate, ACH-4471, entered the clinic last year and is reported to be the first factor D inhibitor to demonstrate complement alternative pathway inhibition in humans after oral dosing.
Is
general: Yes
