Patisiran in Patients With Hereditary Transthyretin-mediated Amyloidosis (hATTR Amyloidosis) Disease Progression Post-Liver Transplant
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ClinicalTrials.gov Identifier: NCT03862807 |
Recruitment Status :
Completed
First Posted : March 5, 2019
Results First Posted : December 21, 2021
Last Update Posted : December 21, 2021
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Condition or disease | Intervention/treatment | Phase |
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Amyloidosis, Familial Transthyretin Amyloidosis | Drug: Patisiran | Phase 3 |
Study Type : | Interventional (Clinical Trial) |
Actual Enrollment : | 24 participants |
Allocation: | N/A |
Intervention Model: | Single Group Assignment |
Masking: | None (Open Label) |
Primary Purpose: | Treatment |
Official Title: | 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 |
Actual Study Start Date : | March 27, 2019 |
Actual Primary Completion Date : | October 6, 2020 |
Actual Study Completion Date : | October 20, 2020 |

Arm | Intervention/treatment |
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Experimental: Patisiran
Participants received patisiran 0.3 milligrams/kilogram (mg/kg) via intravenous (IV) infusion once every 3 weeks (q3w) for 12 months. Dosing was based on actual body weight. For participants weighing 100 kg or more, patisiran was administered at a total dose of 30 mg IV q3w.
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Drug: Patisiran
Patisiran was administered via IV infusion.
Other Names:
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- Average of Month 6 and Month 12 Percentage Reduction From Baseline in Serum Transthyretin (TTR) [ Time Frame: Baseline, Months 6 and 12 ]Serum TTR was assessed using enzyme linked immunosorbent assay (ELISA). The average of the percentage reduction in serum TTR observed at Month 6 and at Month 12 is first calculated for each patient and then the median (95% CI) of these averaged values is summarized for the Safety Analysis Set.
- Change From Baseline in the Neuropathy Impairment Score (NIS) at Month 12 [ Time Frame: Baseline, Month 12 ]The NIS is a composite neurologic impairment score that assesses motor weakness (NIS-W), sensation (NIS-S) and reflexes (NIS-R) by physical exam. The minimum and maximum values are 0 and 244, respectively. A higher score indicates a worse outcome.
- Change From Baseline in Norfolk Quality of Life-Diabetic Neuropathy (Norfolk QoL-DN) Score at Month 12 [ Time Frame: Baseline, Month 12 ]The Norfolk QoL-DN questionnaire is a standardized 35-item patient-reported outcomes measure that is sensitive to the different features of diabetic neuropathy - small fiber, large fiber, and autonomic nerve function. The minimum and maximum values are -4 and 136, respectively. A higher score indicates a worse outcome.
- Change From Baseline in the Rasch-Built Overall Disability Scale (R-ODS) at Month 12 [ Time Frame: Baseline, Month 12 ]The R-ODS is comprised of a 24-item linearly weighted scale that specifically captures activity and social participation limitations. The minimum and maximum values are 0 and 48, respectively. A higher score indicates a better outcome.
- Change From Baseline in the Composite Autonomic Symptom Score (COMPASS-31) at Month 12 [ Time Frame: Baseline, Month 12 ]The COMPASS-31 questionnaire is a measure of autonomic neuropathy symptoms. The questions evaluate 6 autonomic domains (orthostatic intolerance, vasomotor, secretomotor, gastrointestinal, bladder, and pupillomotor). The minimum and maximum values are 0 and 100, respectively. A higher score indicates a worse outcome.
- Change From Baseline in the Modified Body Mass Index (mBMI) at Month 12 [ Time Frame: Baseline, Month 12 ]Nutritional status of participants was evaluated using the mBMI, calculated as BMI (kg/m^2) multiplied by albumin (g/L). An increase from baseline in mBMI suggests improvement, and a decrease from baseline suggests worsening.
- Percentage of Participants With Adverse Events [ Time Frame: From baseline to end of study at Month 13 ]An AE is any untoward medical occurrence in a participant or clinical investigational patient administered a pharmaceutical product and which does not necessarily have a causal relationship with this treatment.

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.
Ages Eligible for Study: | 18 Years and older (Adult, Older Adult) |
Sexes Eligible for Study: | All |
Accepts Healthy Volunteers: | No |
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

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03862807
France | |
Clinical Trial Site | |
Créteil, France | |
Clinical Trial Site | |
Le Kremlin-Bicêtre, France | |
Germany | |
Clinical Trial Site | |
Münster, Germany | |
Italy | |
Clinical Trial Site | |
Messina, Italy | |
Portugal | |
Clinical Trial Site | |
Porto, Portugal | |
Spain | |
Clinical Trial Site | |
Barcelona, Spain | |
Clinical Trial Site | |
Huelva, Spain | |
Sweden | |
Clinical Trial Site | |
Umeå, Sweden | |
United Kingdom | |
Clinical Trial Site | |
London, United Kingdom |
Study Director: | Medical Director | Alnylam Pharmaceuticals |
Documents provided by Alnylam Pharmaceuticals:
Responsible Party: | Alnylam Pharmaceuticals |
ClinicalTrials.gov Identifier: | NCT03862807 |
Other Study ID Numbers: |
ALN-TTR02-008 2018-003519-24 ( EudraCT Number ) |
First Posted: | March 5, 2019 Key Record Dates |
Results First Posted: | December 21, 2021 |
Last Update Posted: | December 21, 2021 |
Last Verified: | November 2021 |
Individual Participant Data (IPD) Sharing Statement: | |
Plan to Share IPD: | No |
Studies a U.S. FDA-regulated Drug Product: | Yes |
Studies a U.S. FDA-regulated Device Product: | No |
Product Manufactured in and Exported from the U.S.: | Yes |
Amyloidosis, Familial Amyloidosis Disease Progression Proteostasis Deficiencies Metabolic Diseases |
Disease Attributes Pathologic Processes Metabolism, Inborn Errors Genetic Diseases, Inborn |