APOLLO: The Study of an Investigational Drug, Patisiran (ALN-TTR02), for the Treatment of Transthyretin (TTR)-Mediated Amyloidosis
APOLLO: A Phase 3 Multicenter, Multinational, Randomized, Double-blind, Placebo-controlled Study to Evaluate the Efficacy and Safety of Patisiran (ALN-TTR02) in Transthyretin (TTR)-Mediated Polyneuropathy (Familial Amyloidotic Polyneuropathy-FAP)
2 other identifiers
interventional
225
21 countries
52
Brief Summary
The purpose of this study is to evaluate the safety and efficacy of patisiran (ALN-TTR02) in patients with transthyretin (TTR) mediated amyloidosis. An open-label, single-arm, long-term follow-up extension study NCT02510261 (ALN-TTR02-006) was initiated to provide participants who completed this study with continued patisiran-LNP (lipid nanoparticle) treatment.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P25-P50 for phase_3
Started Nov 2013
Typical duration for phase_3
52 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
Click on a node to explore related trials.
Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
October 9, 2013
CompletedFirst Posted
Study publicly available on registry
October 10, 2013
CompletedStudy Start
First participant enrolled
November 1, 2013
CompletedPrimary Completion
Last participant's last visit for primary outcome
August 1, 2017
CompletedStudy Completion
Last participant's last visit for all outcomes
August 1, 2017
CompletedResults Posted
Study results publicly available
September 6, 2018
CompletedApril 22, 2024
November 1, 2018
3.8 years
October 9, 2013
August 7, 2018
April 17, 2024
Conditions
Keywords
Outcome Measures
Primary Outcomes (1)
Modified Neuropathy Impairment Score +7 (mNIS+7)
The difference between the patisiran (ALN-TTR02) and placebo groups in the change from baseline in mNIS+7 at 18 months. The mNIS+7 is a composite score that quantitates motor, sensory, and autonomic neurologic impairment due to injury of large and small nerves. The minimum and maximum values are 0 and 304, respectively. A higher score indicates a worse outcome.
18mo
Secondary Outcomes (6)
Norfolk Quality of Life-Diabetic Neuropathy (Norfolk QoL-DN) Questionnaire
18mo
Neurological Impairment Score-Weakness (NIS-W) Score
18mo
Rasch-built Overall Disability Scale (R-ODS) Score
18mo
Timed 10-meter Walk Test (10-MWT, Gait Speed)
18mo
Modified Body Mass Index (mBMI)
18mo
- +1 more secondary outcomes
Study Arms (2)
patisiran (ALN-TTR02)
ACTIVE COMPARATORSterile Normal Saline (0.9% NaCl)
PLACEBO COMPARATORInterventions
administered by intravenous (IV) infusion
Eligibility Criteria
You may qualify if:
- Male or female of 18 to 85 years of age (inclusive);
- Have a diagnosis of FAP
- Neuropathy Impairment Score requirement of 5-130
- Meet Karnofsky performance status requirements
- Have adequate complete blood counts and liver function tests
- Have adequate cardiac function
- Have negative serology for hepatitis B virus (HBV) and hepatitis C virus (HCV)
You may not qualify if:
- Had a prior liver transplant or is planned to undergo liver transplant during the study period;
- Has untreated hypo- or hyperthyroidism;
- Has known human immunodeficiency virus (HIV) infection;
- Had a malignancy within 2 years, except for basal or squamous cell carcinoma of the skin or carcinoma in situ of the cervix that has been successfully treated;
- Recently received an investigational agent or device
- Is currently taking diflunisal, tafamidis, doxycycline, or tauroursodeoxycholic acid
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (52)
Clinical Trial Site
La Mesa, California, United States
Clinical Trial Site
Orange, California, United States
Clinical Trial Site
Denver, Colorado, 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
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St Louis, Missouri, United States
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New York, New York, 10029, United States
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New York, New York, 10032, United States
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Durham, North Carolina, United States
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Portland, Oregon, United States
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Buenos Aires, Argentina
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Westmead, Australia
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Ribeirão Preto, Brazil
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Rio de Janeiro, Brazil
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São Paulo, Brazil
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Sofia, Bulgaria
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Vancouver, British Columbia, Canada
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Nicosia, Cyprus
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Bourdeaux, France
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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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Heidelberg, Germany
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Münster, Germany
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Regensburg, Germany
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Pavia, Italy
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Rome, Italy
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Sicily, Italy
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Matsumoto, Nagano, Japan
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Aichi, Japan
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Kumamoto, Japan
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Kuala Lumpur, Malaysia
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Mexico City, Mexico
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Groningen, Netherlands
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Lisbon, Portugal
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Porto, Portugal
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Seoul, 135-710, South Korea
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Seoul, 143-729, South Korea
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Barcelona, Spain
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Huelva, Spain
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Madrid, Spain
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Palma de Mallorca, Spain
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Umeå, Sweden
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Taipai, 11217, Taiwan
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Taipei, 10002, Taiwan
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Istanbul, Turkey (Türkiye)
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London, NW32PF, United Kingdom
Clinical Trial Site
London, SW17 0RE, United Kingdom
Related Publications (7)
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.
PMID: 38548524DERIVEDQuan D, Obici L, Berk JL, Ando Y, Aldinc E, White MT, Adams D. Impact of baseline polyneuropathy severity on patisiran treatment outcomes in the APOLLO trial. Amyloid. 2023 Mar;30(1):49-58. doi: 10.1080/13506129.2022.2118043. Epub 2022 Sep 18.
PMID: 36120830DERIVEDZhang X, Goel V, Attarwala H, Sweetser MT, Clausen VA, Robbie GJ. Patisiran Pharmacokinetics, Pharmacodynamics, and Exposure-Response Analyses in the Phase 3 APOLLO Trial in Patients With Hereditary Transthyretin-Mediated (hATTR) Amyloidosis. J Clin Pharmacol. 2020 Jan;60(1):37-49. doi: 10.1002/jcph.1480. Epub 2019 Jul 19.
PMID: 31322739DERIVEDMinamisawa M, Claggett B, Adams D, Kristen AV, Merlini G, Slama MS, Dispenzieri A, Shah AM, Falk RH, Karsten V, Sweetser MT, Chen J, Riese R, Vest J, Solomon SD. Association of Patisiran, an RNA Interference Therapeutic, With Regional Left Ventricular Myocardial Strain in Hereditary Transthyretin Amyloidosis: The APOLLO Study. JAMA Cardiol. 2019 May 1;4(5):466-472. doi: 10.1001/jamacardio.2019.0849.
PMID: 30878017DERIVEDSolomon SD, Adams D, Kristen A, Grogan M, Gonzalez-Duarte A, Maurer MS, Merlini G, Damy T, Slama MS, Brannagan TH 3rd, Dispenzieri A, Berk JL, Shah AM, Garg P, Vaishnaw A, Karsten V, Chen J, Gollob J, Vest J, Suhr O. Effects of Patisiran, an RNA Interference Therapeutic, on Cardiac Parameters in Patients With Hereditary Transthyretin-Mediated Amyloidosis. Circulation. 2019 Jan 22;139(4):431-443. doi: 10.1161/CIRCULATIONAHA.118.035831.
PMID: 30586695DERIVEDAdams D, Gonzalez-Duarte A, O'Riordan WD, Yang CC, Ueda M, Kristen AV, Tournev I, Schmidt HH, Coelho T, Berk JL, Lin KP, Vita G, Attarian S, Plante-Bordeneuve V, Mezei MM, Campistol JM, Buades J, Brannagan TH 3rd, Kim BJ, Oh J, Parman Y, Sekijima Y, Hawkins PN, Solomon SD, Polydefkis M, Dyck PJ, Gandhi PJ, Goyal S, Chen J, Strahs AL, Nochur SV, Sweetser MT, Garg PP, Vaishnaw AK, Gollob JA, Suhr OB. Patisiran, an RNAi Therapeutic, for Hereditary Transthyretin Amyloidosis. N Engl J Med. 2018 Jul 5;379(1):11-21. doi: 10.1056/NEJMoa1716153.
PMID: 29972753DERIVEDAdams D, Suhr OB, Dyck PJ, Litchy WJ, Leahy RG, Chen J, Gollob J, Coelho T. Trial design and rationale for APOLLO, a Phase 3, placebo-controlled study of patisiran in patients with hereditary ATTR amyloidosis with polyneuropathy. BMC Neurol. 2017 Sep 11;17(1):181. doi: 10.1186/s12883-017-0948-5.
PMID: 28893208DERIVED
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Results Point of Contact
- Title
- Chief Medical Officer
- Organization
- Alnylam Pharmaceuticals, Inc.
Study Officials
- STUDY DIRECTOR
Jared Gollob
Alnylam Pharmaceuticals
Publication Agreements
- PI is Sponsor Employee
- No
- Restriction Type
- LTE60
- Restrictive Agreement
- Yes
Study Design
- Study Type
- interventional
- Phase
- phase 3
- Allocation
- RANDOMIZED
- Masking
- DOUBLE
- Who Masked
- PARTICIPANT, INVESTIGATOR
- Purpose
- TREATMENT
- Intervention Model
- PARALLEL
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
- Expanded Access
- Yes
Study Record Dates
First Submitted
October 9, 2013
First Posted
October 10, 2013
Study Start
November 1, 2013
Primary Completion
August 1, 2017
Study Completion
August 1, 2017
Last Updated
April 22, 2024
Results First Posted
September 6, 2018
Record last verified: 2018-11
Data Sharing
- IPD Sharing
- Will share
Access to Anonymized individual participant data that support these results is made available 12 months after study completion and not less than 12 months after the product and indication have been approved in the US and/or the EU. Data will be provided contingent upon the approval of a research proposal and the execution of a data sharing agreement. Requests for access to data can be submitted via the website www.vivli.org.