Patisiran-LNP in Patients with hATTR Amyloidosis Disease Progression Post-Liver Transplant

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

Status

Active, not recruiting

Phases

Phase 3

Study type

Interventional

Source

EU CTR

Registry ID

EUCTR2018-003519-24-GB

Enrollment

Registered

2018-11-22

Start date

2019-03-12

Completion date

Unknown

Last updated

2021-01-05

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Hereditary Transthyretin-mediated Amyloidosis (hATTR amyloidosis) MedDRA version: 20.0 Level: PT Classification code 10007509 Term: Cardiac amyloidosis System Organ Class: 10007541 - Cardiac disorders MedDRA version: 20.0 Level: PT Classification code 10019889 Term: Hereditary neuropathic amyloidosis System Organ Class: 10010331 - Congenital, familial and genetic disorders

Interventions

Trade Name: Onpattro Product Name: patisiran-LNP Product Code: ALN-TTR02 Pharmaceutical Form: Concentrate for solution for infusion INN or Proposed INN: PATISIRAN Current Sponsor code: ALN-TTR02 Conce

Eligibility

Sex/Gender

All

Inclusion 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

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

Design outcomes

Primary

Measure	Time frame
Main Objective: To evaluate the TTR reduction of patisiran-LNP in hATTR amyloidosis patients with disease progression after OLT ;Secondary Objective: - To evaluate the effect of patisiran-LNP on neuropathy - To evaluate the effect of patisiran-LNP on patient reported outcomes including QoL, activities of daily living and autonomic neuropathy symptoms - To characterize the effect on nutritional status ;Primary end point(s): Percent change from baseline in serum TTR levels;Timepoint(s) of evaluation of this end point: Month 6, Month 12	—

Measure

Time frame

Main Objective: To evaluate the TTR reduction of patisiran-LNP in hATTR amyloidosis patients with disease progression after OLT ;Secondary Objective: - To evaluate the effect of patisiran-LNP on neuropathy - To evaluate the effect of patisiran-LNP on patient reported outcomes including QoL, activities of daily living and autonomic neuropathy symptoms - To characterize the effect on nutritional status ;Primary end point(s): Percent change from baseline in serum TTR levels;Timepoint(s) of evaluation of this end point: Month 6, Month 12

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Secondary

Measure	Time frame
Secondary end point(s): -Change from baseline in Neuropathy Impairment Score (NIS) -Change from baseline in quality of life and disability as assessed by Norfolk Quality of Life (QoL-DN), Rasch-built Overall Disability Scale (R-ODS), and Autonomic Symptoms Questionnaire (COMPASS 31) -Change from baseline in modified Body Mass Index (mBMI);Timepoint(s) of evaluation of this end point: 12 months (this is applicable to all 3 of the secondary end points)	—

Measure

Time frame

Secondary end point(s): -Change from baseline in Neuropathy Impairment Score (NIS) -Change from baseline in quality of life and disability as assessed by Norfolk Quality of Life (QoL-DN), Rasch-built Overall Disability Scale (R-ODS), and Autonomic Symptoms Questionnaire (COMPASS 31) -Change from baseline in modified Body Mass Index (mBMI);Timepoint(s) of evaluation of this end point: 12 months (this is applicable to all 3 of the secondary end points)

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Countries

France, Germany, Italy, Netherlands, Portugal, Spain, Sweden, United Kingdom

Contacts

Public ContactClinical Trial Information Line

Alnylam Pharmaceuticals, Inc

clinicaltrials@alnylam.com0018772569526

Outcome results

None listed

Source: EU CTR (via WHO ICTRP) · Data processed: Feb 4, 2026