Phase 3 N=24 Treatment

Patisiran in Patients With Hereditary Transthyretin-mediated Amyloidosis (hATTR Amyloidosis) Disease Progression Post-Liver Transplant

Amyloidosis, Familial · Transthyretin Amyloidosis

Bottom Line

View on ClinicalTrials.gov: NCT03862807 ↗

Enrolled (actual)

Serious AEs

21.7%

Results posted

Dec 2021

Primary outcome: Primary: Average of Month 6 and Month 12 Percentage Reduction From Baseline in Serum Transthyretin (TTR) — 91.0 percent reduction

Study Design & Population

Study type: Interventional
Phase: Phase 3
Interventions: Patisiran (Drug)
Age: Adult, Older Adult · 18+ yrs
Sex: All
Sponsor: Alnylam Pharmaceuticals
Primary completion: Oct 2020

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	—

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.

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

View full record on ClinicalTrials.gov →

Data sourced from ClinicalTrials.gov (NCT03862807). 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.