EUCTR2018-003519-24-FR
Active, not recruiting
Phase 1
An Open-label Study to Evaluate Safety, Efficacy and Pharmacokinetics (PK) of Patisiran-LNP in Patients with Hereditary Transthyretin-mediated Amyloidosis (hATTR amyloidosis) with Disease Progression Post-Orthotopic Liver Transplant
ConditionsHereditary 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]
DrugsOnpattro
Overview
- Phase
- Phase 1
- Intervention
- Not specified
- Conditions
- Hereditary Transthyretin-mediated Amyloidosis (hATTR amyloidosis)
- Sponsor
- Alnylam Pharmaceuticals, Inc.
- Enrollment
- 20
- Status
- Active, not recruiting
- Last Updated
- 5 years ago
Overview
Brief Summary
No summary available.
Investigators
Eligibility Criteria
Inclusion Criteria
- •\-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
Exclusion Criteria
- •\-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
Outcomes
Primary Outcomes
Not specified
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