HERA
Research type
Research Study
Full title
A phase 2, ranodmised, double-blind, placebo controlled study to evaluate the safety, efficacy, pharmacodynamics, and pharmacokinetics of SAR339375 for subutaneous injection administered every week in patients with Alport Syndrome
IRAS ID
269460
Contact name
Claire Jones
Contact email
Sponsor organisation
Sanofi
Eudract number
2019-004394-10
Clinicaltrials.gov Identifier
Duration of Study in the UK
3 years, 1 months, 6 days
Research summary
This is a randomised, double blind placebo controlled multicentre, phase 2 study conducted in approximately 45 patients with Alport Syndrome to assess the safety, tolerability and efficacy of SAR339375. Alport Syndrome is an inherited disease of collagen tissue in the kidney which over time leads to renal function loss resulting in dialysis or renal transplantation. Other organs can also be involved such as the ears and eyes with eye defects and hearing loss as common symptoms.
SAR339375 has been studied in two studies with healthy volunteers, a natural history study (designed to collect additional information about Alport syndrome) and two ongoing studies in patients with Alport syndrome (the Phase 1b PDY16327 and the Phase 2 ACT16248 studies).
MicroRNAs are small genetic building blocks that are important in turning genes on and off. They have been found to be overexpressed in patients and animal models with Alport Syndrome. SAR339375 works by stopping the function of MicroRNA in the kidney cells. So by stopping the MicroRNA functioning properly, SAR339375 could possibly help slow the decline in kidney function in subjects with Alport syndrome.
This study will be conducted globally and in three centres in the UK.
REC name
London - Hampstead Research Ethics Committee
REC reference
19/LO/1753
Date of REC Opinion
4 Dec 2019
REC opinion
Further Information Favourable Opinion