Status:
COMPLETED
Patisiran in Patients With Hereditary Transthyretin-mediated Amyloidosis (hATTR Amyloidosis) Disease Progression Post-Liver Transplant
Lead Sponsor:
Alnylam Pharmaceuticals
Conditions:
Amyloidosis, Familial
Transthyretin Amyloidosis
Eligibility:
All Genders
18+ years
Phase:
PHASE3
Brief Summary
The purpose of this study is to evaluate the efficacy, safety and pharmacokinetics of patisiran in participants with hereditary transthyretin-mediated amyloidosis (hATTR amyloidosis) with disease prog...
Eligibility Criteria
Inclusion
- Received liver transplant for treatment of hATTR amyloidosis ≥12 months before study start
- Has increase in polyneuropathy disability (PND) score after liver transplant
- Has received stable immunosuppressive regimen with ≤10 mg/day of prednisone for at least 3 months before study start
- Has Karnofsky Performance Status (KPS) of ≥70%
- Has vitamin A level greater than or equal to lower limit of normal
Exclusion
- Has previously received inotersen or patisiran
- Has clinically significant liver function test abnormalities
- Has known portal hypertension with ascites
- Has estimated glomerular filtration rate (eGFR) ≤30 mL/min/1.73 m\^2
- Has known leptomeningeal amyloidosis
- Has infection with hepatitis B, hepatitis C or human immunodeficiency virus (HIV)
- Has New York Heart Association heart failure classification of \>2
- Is wheelchair bound or bedridden
- Has received organ transplants other than liver transplant
- Will be using another tetramer stabilizer during the study
Key Trial Info
Start Date :
March 27 2019
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
October 20 2020
Estimated Enrollment :
24 Patients enrolled
Trial Details
Trial ID
NCT03862807
Start Date
March 27 2019
End Date
October 20 2020
Last Update
April 22 2024
Active Locations (9)
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1
Clinical Trial Site
Créteil, France
2
Clinical Trial Site
Le Kremlin-Bicêtre, France
3
Clinical Trial Site
Münster, Germany
4
Clinical Trial Site
Messina, Italy