Patisiran in Patients With Hereditary Transthyretin-mediated Amyloidosis (hATTR Amyloidosis) Disease Progression Post-Liver Transplant
NCT ID: NCT03862807
Last Updated: 2024-04-22
Study Results
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View full resultsBasic Information
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COMPLETED
PHASE3
24 participants
INTERVENTIONAL
2019-03-27
2020-10-20
Brief Summary
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Detailed Description
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Conditions
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Study Design
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NA
SINGLE_GROUP
TREATMENT
NONE
Study Groups
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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.
Patisiran
Patisiran was administered via IV infusion.
Interventions
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Patisiran
Patisiran was administered via IV infusion.
Other Intervention Names
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Eligibility Criteria
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Inclusion Criteria
* 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 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
18 Years
ALL
No
Sponsors
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Alnylam Pharmaceuticals
INDUSTRY
Responsible Party
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Principal Investigators
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Medical Director
Role: STUDY_DIRECTOR
Alnylam Pharmaceuticals
Locations
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Clinical Trial Site
Créteil, , France
Clinical Trial Site
Le Kremlin-Bicêtre, , France
Clinical Trial Site
Münster, , Germany
Clinical Trial Site
Messina, , Italy
Clinical Trial Site
Porto, , Portugal
Clinical Trial Site
Barcelona, , Spain
Clinical Trial Site
Huelva, , Spain
Clinical Trial Site
Umeå, , Sweden
Clinical Trial Site
London, , United Kingdom
Countries
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References
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Badri P, Habtemariam B, Melch M, Clausen VA, Arum S, Li X, Jay PY, Vest J, Robbie GJ. Pharmacokinetics and Pharmacodynamics of Patisiran in Patients with hATTR Amyloidosis and with Polyneuropathy After Liver Transplantation. Clin Pharmacokinet. 2023 Oct;62(10):1509-1522. doi: 10.1007/s40262-023-01292-w. Epub 2023 Aug 28.
Schmidt HH, Wixner J, Plante-Bordeneuve V, Munoz-Beamud F, Llado L, Gillmore JD, Mazzeo A, Li X, Arum S, Jay PY, Adams D; Patisiran Post-LT Study Group. Patisiran treatment in patients with hereditary transthyretin-mediated amyloidosis with polyneuropathy after liver transplantation. Am J Transplant. 2022 Jun;22(6):1646-1657. doi: 10.1111/ajt.17009. Epub 2022 Mar 26.
Seibert K, Wlodarski R, Sarswat N, Appelbaum D, Issa NP, Soliven B, Rezania K. Progressive Multiple Mononeuropathy in a Patient With Familial Transthyretin Amyloidosis After Liver Transplantation. J Clin Neuromuscul Dis. 2022 Mar 1;23(3):143-147. doi: 10.1097/CND.0000000000000368.
Provided Documents
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Document Type: Study Protocol
Document Type: Statistical Analysis Plan
Other Identifiers
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2018-003519-24
Identifier Type: EUDRACT_NUMBER
Identifier Source: secondary_id
ALN-TTR02-008
Identifier Type: -
Identifier Source: org_study_id
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