Phase 3
Completed N=24
Patisiran in Patients With Hereditary Transthyretin-mediated Amyloidosis (hATTR Amyloidosis) Disease Progression Post-Liver Transplant
Amyloidosis, Familial · Transthyretin Amyloidosis
Source: ClinicalTrials.gov NCT03862807 ↗
Enrolled (actual)
24
Serious AEs
21.7%
Results posted
Dec 2021
Primary outcomePrimary: Average of Month 6 and Month 12 Percentage Reduction From Baseline in Serum Transthyretin (TTR) — 91.0 percent reduction
◆ Published Evidence
Established
55citations · ~14 / year
Patisiran treatment in patients with hereditary transthyretin-mediated amyloidosis with polyneuropathy after liver transplantation.
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 progression after liver transplant.
Linked Publications (3)
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Patisiran treatment in patients with hereditary transthyretin-mediated amyloidosis with polyneuropathy after liver transplantation.
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Pharmacokinetics and Pharmacodynamics of Patisiran in Patients with hATTR Amyloidosis and with Polyneuropathy After Liver Transplantation.
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Progressive Multiple Mononeuropathy in a Patient With Familial Transthyretin Amyloidosis After Liver Transplantation.
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Average of Month 6 and Month 12 Percentage Reduction From Baseline in Serum Transthyretin (TTR) |
91.0 | — |
| SECONDARY Change From Baseline in the Neuropathy Impairment Score (NIS) at Month 12 |
-3.7 | — |
| SECONDARY Change From Baseline in Norfolk Quality of Life-Diabetic Neuropathy (Norfolk QoL-DN) Score at Month 12 |
-6.5 | — |
| SECONDARY Change From Baseline in the Rasch-Built Overall Disability Scale (R-ODS) at Month 12 |
-0.1 | — |
| SECONDARY Change From Baseline in the Composite Autonomic Symptom Score (COMPASS-31) at Month 12 |
-5.0 | — |
| SECONDARY Change From Baseline in the Modified Body Mass Index (mBMI) at Month 12 |
4.4 | — |
| SECONDARY Percentage of Participants With Adverse Events |
100 | — |
Eligibility Criteria
Inclusion Criteria
- 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 Criteria
- 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
Data sourced from ClinicalTrials.gov (NCT03862807) and the linked publication. Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication. Informational only — not medical advice.