The Study of an Investigational Drug, Patisiran (ALN-TTR02), for the Treatment of Transthyretin (TTR)-Mediated Amyloidosis in Participants Who Have Already Been Treated With ALN-TTR02 (Patisiran)
NCT ID: NCT02510261
Last Updated: 2023-12-06
Study Results
Outcome measurements, participant flow, baseline characteristics, and adverse events have been published for this study.
View full resultsBasic Information
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COMPLETED
PHASE3
211 participants
INTERVENTIONAL
2015-07-16
2022-11-23
Brief Summary
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Detailed Description
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Conditions
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Keywords
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Study Design
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NON_RANDOMIZED
SINGLE_GROUP
TREATMENT
NONE
Study Groups
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Prior Placebo Group of Study 004
Participants who received placebo and completed parent study ALN-TTR02-004 (NCT01960348) were enrolled to receive 0.3 milligrams per kilogram (mg/kg) patisiran intravenously (IV) once every 3 weeks (Q3W) up to 65.5 months.
Patisiran
Patisiran was administered IV.
Prior Patisiran Group of Study 004
Participants who received patisiran and completed parent study ALN-TTR02-004 (NCT01960348) were enrolled to receive 0.3 mg/kg patisiran IV Q3W up to 66.9 months.
Patisiran
Patisiran was administered IV.
Prior Patisiran Group of Study 003
Participants who received patisiran and completed parent study ALN-TTR02-003 (NCT01961921) were enrolled to receive 0.3 mg/kg patisiran IV Q3W up to 61.4 months.
Patisiran
Patisiran was administered IV.
Interventions
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Patisiran
Patisiran was administered IV.
Other Intervention Names
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Eligibility Criteria
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Inclusion Criteria
* Be willing and able to comply with the protocol-required visit schedule and visit requirements and provide written informed consent
Exclusion Criteria
18 Years
85 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
La Mesa, California, United States
Clinical Trial Site
Aurora, Colorado, United States
Clinical Trial Site
Jacksonville, Florida, United States
Clinical Trial Site
Chicago, Illinois, United States
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Baltimore, Maryland, United States
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Boston, Massachusetts, United States
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Detroit, Michigan, United States
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Rochester, Minnesota, United States
Clinical Trial Site
Joplin, Missouri, United States
Clinical Trial Site
St Louis, Missouri, United States
Clinical Trial Site
Cooperstown, New York, United States
Clinical Trial Site
New York, New York, United States
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New York, New York, United States
Clinical Trial Site
Columbus, Ohio, United States
Clinical Trial Site
Buenos Aires, , Argentina
Clinical Trial Site
Westmead, , Australia
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Rio de Janeiro, , Brazil
Clinical Trial Site
São Paulo, , Brazil
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Sofia, , Bulgaria
Clinical Trial Site
Vancouver, British Columbia, Canada
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Nicosia, , Cyprus
Clinical Trial Site
Fort de France, Martinique, France
Clinical Trial Site
Saint-Pierre, Reunion Island, France
Clinical Trial Site
Créteil, , France
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Le Kremlin-Bicêtre, , France
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Lille, , France
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Marseille, , France
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Cologne, , Germany
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Heidelberg, , Germany
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Münster, , Germany
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Pavia, , Italy
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Rome, , Italy
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Sicily, , Italy
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Ehime, , Japan
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Fukuoka, , Japan
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Hiroshima, , Japan
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Kumamoto, , Japan
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Nagano, , Japan
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Okawasuji, , Japan
Clinical Trial Site
Ono, , Japan
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Ōita, , Japan
Clinical Trial Site
Kuala Lumpur, , Malaysia
Clinical Trial Site
Tlalpan, , Mexico
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Groningen, , Netherlands
Clinical Trial Site
Lisbon, , Portugal
Clinical Trial Site
Porto, , Portugal
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Seoul, , South Korea
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Barcelona, , Spain
Clinical Trial Site
Huelva, , Spain
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Madrid, , Spain
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Palma de Mallorca, , Spain
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Umeå, , Sweden
Clinical Trial Site
Taipei, , Taiwan
Clinical Trial Site
Taipei, , Taiwan
Clinical Trial Site
Istanbul, , Turkey (Türkiye)
Clinical Trial Site
London, , United Kingdom
Countries
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References
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Adams D, Wixner J, Polydefkis M, Berk JL, Conceicao IM, Dispenzieri A, Peltier A, Ueda M, Bender S, Capocelli K, Jay PY, Yureneva E, Obici L; patisiran Global OLE study group. Five-Year Results With Patisiran for Hereditary Transthyretin Amyloidosis With Polyneuropathy: A Randomized Clinical Trial With Open-Label Extension. JAMA Neurol. 2025 Mar 1;82(3):228-236. doi: 10.1001/jamaneurol.2024.4631.
Lin KP, Yang CC, Lee YC, Lee MJ, Vest J, Sweetser MT, White MT, Badri P, Hsieh ST, Chao CC. Patisiran, an RNAi therapeutic for hereditary transthyretin-mediated amyloidosis: Sub-analysis in Taiwanese patients from the APOLLO study. J Formos Med Assoc. 2024 Sep;123(9):975-984. doi: 10.1016/j.jfma.2024.03.008. Epub 2024 Mar 27.
Adams D, Polydefkis M, Gonzalez-Duarte A, Wixner J, Kristen AV, Schmidt HH, Berk JL, Losada Lopez IA, Dispenzieri A, Quan D, Conceicao IM, Slama MS, Gillmore JD, Kyriakides T, Ajroud-Driss S, Waddington-Cruz M, Mezei MM, Plante-Bordeneuve V, Attarian S, Mauricio E, Brannagan TH 3rd, Ueda M, Aldinc E, Wang JJ, White MT, Vest J, Berber E, Sweetser MT, Coelho T; patisiran Global OLE study group. Long-term safety and efficacy of patisiran for hereditary transthyretin-mediated amyloidosis with polyneuropathy: 12-month results of an open-label extension study. Lancet Neurol. 2021 Jan;20(1):49-59. doi: 10.1016/S1474-4422(20)30368-9. Epub 2020 Nov 16.
Provided Documents
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Document Type: Study Protocol
Document Type: Statistical Analysis Plan
Other Identifiers
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2014-003877-40
Identifier Type: EUDRACT_NUMBER
Identifier Source: secondary_id
ALN-TTR02-006
Identifier Type: -
Identifier Source: org_study_id