ELX-02 in Patients with Alport Syndrome
Research type
Research Study
Full title
A Phase 2 Open Label Pilot Study to Evaluate the Safety and Efficacy of Subcutaneously Administered ELX-02 in Patients with Alport Syndrome with Col4A5 and Col4A3/4 Nonsense Mutation
IRAS ID
1005163
Contact name
Timothy Kachmar
Contact email
Sponsor organisation
Eloxx Pharmaceuticals Inc
Eudract number
2022-000604-35
ISRCTN Number
ISRCTN12014920
Research summary
The main purpose of this study is to learn whether the study drug, ELX-02, is safe for Alport Syndrome (AS) caused by nonsense mutations. Alport Syndrome caused by nonsense mutations is a genetic disorder that decreases kidney function and can also affect the ears and decrease patients' hearing. In this form of AS, the nonsense mutation prevents the synthesis of the full-length Collagen IV protein that forms the filters in the kidney and has an important role in ear function. There is currently no treatment for Alport Syndrome (AS) caused by nonsense mutations. This means that it has not been approved for treatment anywhere in the world. ELX-02 is designed to restore the full-length Collagen IV protein production, based on the same restoring protein production seen for certain antibiotics, so called Aminoglycosides. Use of aminoglycosides is associated with hearing and balance problems and worsening of the kidney function. ELX-02 was designed in a way that it is expected not to have those negative long term effects. In total, up to 8 patients will be asked to participate in the study in the UK and Australia. Patients will be in the study for approximately 26 weeks. This includes a screening period of up to 42 days, a treatment period of 60 days and a follow-up periode of 90 days.
REC name
East of England - Cambridge South Research Ethics Committee
REC reference
22/EE/0064
Date of REC Opinion
4 May 2022
REC opinion
Further Information Favourable Opinion