Patisiran-LNP in Patients with hATTR Amyloidosis Disease Progression Post-Liver Transplant
- Conditions
- Hereditary Transthyretin-mediated Amyloidosis (hATTR amyloidosis)MedDRA version: 20.0Level: PTClassification code 10007509Term: Cardiac amyloidosisSystem Organ Class: 10007541 - Cardiac disordersMedDRA version: 20.0Level: PTClassification code 10019889Term: Hereditary neuropathic amyloidosisSystem Organ Class: 10010331 - Congenital, familial and genetic disordersTherapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
- Registration Number
- EUCTR2018-003519-24-GB
- Lead Sponsor
- Alnylam Pharmaceuticals, Inc.
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- ot Recruiting
- Sex
- All
- Target Recruitment
- 20
-18 years of age or older
-Received an orthotopic liver transplant for treatment of hATTR amyloidosis at least 1 year prior
-Increase in polyneuropathy disability post-liver transplant as measured by polyneuropathy disability (PND) score.
-Karnofsky performance status of =70%
Are the trial subjects under 18? no
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 10
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 10
-Currently taking tafamidis, doxycycline, diflunisal, or tauroursodeoxycholic acid
-Liver allograft rejection episodes or abnormal LFTs suggestive of possible allograft rejection in the past 6 months
-Abnormal liver function tests
-Known leptomeningeal amyloidosis
-New York Heart Association (NYHA) classification of >2
-PND score IV (wheelchair bound or bedridden)
- Prior organ transplant other than liver transplant
-Hospitalization for congestive heart failure or arrhythmia in the past 3 months
Study & Design
- Study Type
- Interventional clinical trial of medicinal product
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method
- Secondary Outcome Measures
Name Time Method