015 - extension study of CL-503012 in patients with AA Amyloidosis
Research type
Research Study
Full title
International Open-Label Extension of the Phase 3 Study CL-503012 with KIACTA™ in Patients with AA Amyloidosis
IRAS ID
143132
Contact name
Helen Lachmann
Contact email
Sponsor organisation
A.T. Development Switzerland SARL
Eudract number
2013-004150-16
ISRCTN Number
n/a
Research summary
Amyloidosis is the general term for a number of diseases where the build-up of abnormal proteins (amyloid) leads to dysfunction or failure of the involved organs. AA amyloidosis results from the build-up of amyloid A protein. It is a complication of chronic inflammatory conditions such as arthritis, chronic infections and familial Mediterranean fever.
Rheumatoid arthritis is the major cause of AA amyloidosis in Western Europe.AA amyloidosis is a rare life-threatening disease which mainly affects the kidney. The most common feature is progressive kidney dysfunction leading to end-stage kidney failure (dialysis) and death. Average survival time from diagnosis varies from 10-12 years.
There is no specific treatment for AA amyloidosis. Current treatment is based on controlling underlying inflammatory disease to slow progression of AA amyloidosis. Kiacta specifically inhibits the build-up of amyloid A protein. Kiacta has been administered in 3 previous clinical trials and was found to be safe and well tolerated.
This is an open-label extension study to the Phase 3 study CL-503012. The purpose of this study is to provide access to Kiacta for patients who have completed the CL-503012 study. Participants will receive Kiacta as 1 to 3 capsules twice daily. The daily dose received depends on kidney function.
Participants will receive study drug for a maximum of 12 months, unless Kiacta becomes commercially available. During this time, participants will attend the clinic every 6 months. Procedures include: vital signs, urine and blood samples. Telephone calls to assess AEs/SAEs, and effectiveness will be performed at 1, 3 and 9 months. Efficacy will be assessed by looking at patient survival status and progression to end-stage renal disease.
This study is sponsored by A.T. Development Switzerland SARL. Participants completing the CL-503012 study and fulfilling selection criteria may participate in this study, with approximately 10 patients expected from the UK.
REC name
London - Surrey Borders Research Ethics Committee
REC reference
14/LO/0354
Date of REC Opinion
14 Apr 2014
REC opinion
Further Information Favourable Opinion