Amyloidosis
Conditions
Keywords
Kiacta for AA amyloidosis
Brief summary
The primary purpose of this study is to assess the efficacy and safety of treatment with Kiacta in adult patients with AA Amyloidosis.
Interventions
DRUGKIACTA (eprodisate disodium)
Orally 1 to 3 capsules (Kiacta 400 mg) twice daily and adjusted as per the Creatine Clearance (CrCl) level increases or decreases.
DRUGPlacebo
Orally 1 to 3 capsules (placebo) twice daily and adjusted as per the Creatine Clearance (CrCl) level increases or decreases:
Sponsors
C.T. Development America, Inc.
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
PREVENTION
Masking
DOUBLE (Subject, Investigator)
Eligibility
Sex/Gender
ALL
Age
18 Years to 80 Years
Healthy volunteers
No
Inclusion criteria
* females must be of nonchildbearing potential (more than 1 yr postmenopausal)or use effective contraception for at least 2 months prior to the baseline visit and through 30 days after the last dose of study medication * confirmed diagnosis of AA amyloidosis demonstrated by positive biopsy using congo red staining and immunohistochemistry or immunoelectronmicroscopy. Mass spectroscopy will be used upon approval of the sponsor on a case to case basis. * persistent proteinuria greater than 1 g/24h at 2 distinct 24-hr urine collections * must have CrCl greater than 25 ml/min/1.73 m2 at 2 distinct 24 hr urine collections
Exclusion criteria
* evidence or suspicion of chronic kidney disease secondary to a disease other than AA amyloidosis (eg, diabetes, long-standing uncontrolled hypertension, polycystic kidney disease, recurring polynephritis, or systemic lupus erythematosus) * history of kidney transplantation * evidence or suspicion of a cause of potentially reversible acute renal failure within 3 months prior to baseline visit * presence of concomitant diseases or medication that could interfere with the interpretation of study results or compromise patient safety * presence of condition that could reduce life expectancy to less than 2 yrs * Type 1 or 2 diabetes mellitus * significant hepatic enzyme elevation * unstable angina, myocardial infarction, coronary artery bypass graft surgery, or percutaneous transluminal coronary angioplasty within 6 months prior to the baseline visit; presence of NY Heart Assoc class III or IV heart failure * presence of, or history of stroke or transient ischemic attack within 6 months prior to baseline visit * initiation of, or any changes in, angiotensin converting enzyme inhibitor, angiotensin II receptor antagonist therapy, or renin inhibitor within 3 months prior to baseline visit * initiation of, or any changes in, cytotoxic agents, anti-tumor necrosis factor agents, anti interleukin-1 or 6 agents, or colchicine therapy within 3 months prior to baseline visit * previous use of Kiacta * history of malignancy within 5 yrs prior to study entry, except for cervical carcinoma in situ, nonmelanomatous carcinoma of the skin, or ductal carcinoma in situ of the breast that has been surgically cured * use of investigational drug within 30 days prior to the first screening visit * active alcohol and/or drug abuse
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Time from baseline to a persistent decrease in Creatinine clearance (CrCL) of 40% or more, a persistent increase in Serum Creatinine(SCr) of 80% or more, or progression to end-stage renal disease(ESRD) | Up to 24 months |
Secondary
| Measure | Time frame |
|---|---|
| Progression to end-stage renal disease (ESRD) | baseline, every 3 months to end of study visit |
| estimated glomerular filtration rate (eGFR) | screening, baseline, every 3 months, 12 months , early termination, treatment completion, end of study visit |
| serum cystatin C over time | baseline, every 3 months, 12 months, early termination, treatment completion, end of study visit |
| rate of change (slope) in creatinine clearance (CrCL) over time | baseline to primary endpoint, measured every 3 months to end of study visit |
| serum amyloid A | baseline, every 3 months, 12 months, early termination, treatment completion, end of study visit |
| Time from baseline to persistent decrease in CrCL of 40% or more, a persistent increase in SCr of 80% or more, progression to ESRD, or all-cause mortality | Up to 24 months |
| urinary protein/creatinine ratio | screening, baseline, every 3 months, 12 months, early termination, treatment completion, end of study visit |
Countries
Belgium, Egypt, Estonia, Finland, France, Georgia, Germany, India, Israel, Italy, Latvia, Lithuania, Netherlands, Peru, Poland, Russia, Spain, Sweden, Tunisia, Turkey (Türkiye), Ukraine, United Kingdom, United States
Outcome results
None listed