Clinical trial • Phase III • Neurology|Rare Disease
NUCRESIRAN for Hereditary transthyretin-mediated amyloidosis with polyneuropathy (hATTR-PN)
Phase III trial of NUCRESIRAN for Hereditary transthyretin-mediated amyloidosis with polyneuropathy (hATTR-PN).
Overview
- Trial Therapeutic Area
- Neurology|Rare Disease
- Trial Disease
- Hereditary transthyretin-mediated amyloidosis with polyneuropathy (hATTR-PN)
- Trial Stage
- Phase III
- Drug Modality
- Oligonucleotide
- Orphan Drug
- Yes
Key dates
- Initial CTIS Submission Date
- 20-10-2025
- First CTIS Authorization Date
- 20-02-2026
Trial design
Randomised, open-label, vutrisiran (amvuttra) 25 mg subcutaneous every 3 months (comparator arm as stated).-controlled Phase III trial in Portugal, France, Germany and others.
- Randomised
- Yes
- Open Label
- Yes
- Comparator
- Vutrisiran (Amvuttra) 25 mg subcutaneous every 3 months (comparator arm as stated).
- Target Sample Size
- 100
- Trial Duration For Participant
- 1460
Eligibility
Recruits 100 Vulnerable population selected (isVulnerablePopulationSelected = true). The trial enrols adults only (age 18 or age of legal consent to 85). Participants must be able to understand, be willing and able to comply with study requirements and provide written informed consent (see inclusion criterion 6). Subject information and informed consent forms (Main ICF) and related ICFs (pregnancy, newborn, partner consent, data protection notices) are provided in country/language-specific versions..
- Pregnancy Exclusion
- 29. Female patient who is pregnant or breastfeeding
- Vulnerable Population
- Vulnerable population selected (isVulnerablePopulationSelected = true). The trial enrols adults only (age 18 or age of legal consent to 85). Participants must be able to understand, be willing and able to comply with study requirements and provide written informed consent (see inclusion criterion 6). Subject information and informed consent forms (Main ICF) and related ICFs (pregnancy, newborn, partner consent, data protection notices) are provided in country/language-specific versions.
Inclusion criteria
- {"criterion_text":"- 1. Male or female age 18 (or age of legal consent) to 85 years, inclusive, at the time of initial informed consent.\n- 2. Have a diagnosis of hATTR amyloidosis with polyneuropathy with a documented TTR gene variant\n- 3. Have a neuropathy impairment score (NIS) of 5 to 130 (inclusive; this criterion must be met at the Baseline Visit 2)\n- 4. Have a Polyneuropathy Disability (PND) score of ≤3b (this criterion must be met at the Baseline Visit 2)\n- 5. Have a Karnofsky Performance Status (KPS) of ≥60%Informed Consent\n- 6. Patient is able to understand and is willing and able to comply with the study requirements and to provide written informed consent."}
Exclusion criteria
- {"criterion_text":"- 1. All exclusion criteria can be found in the protocol (Section 4.2). 1. Has had a liver transplant or is likely, in the opinion of the Investigator, to undergo liver transplantation during the Treatment Period of the study\n- 18. Has uncontrolled clinically significant cardiac arrhythmia or unstable angina\n- 19. Has known type 1 diabetes\n- 10. Received prior or currently receiving TTR-lowering therapy\n- 20. Has had type 2 diabetes mellitus for ≥5 years\n- 21. Has untreated hypo- or hyperthyroidism\n- 22. Has had a major surgery within the past 3 months or has a major surgery planned during the study through Month 18\n- 23. Has an active infection requiring systemic antiviral, antiparasitic, or antimicrobial therapy that will not be completed prior to dosing (Day 1)\n- 24. Has other medical conditions or comorbidities which, in the opinion of the Investigator, could interfere with study compliance or data interpretation\n- 25. Has a history of multiple drug allergies or history of allergic reactions to any component of or excipient in the study drug\n- 26. Has a history of intolerance to SC injection(s)\n- 7. Estimated glomerular filtration rate (eGFR) ≤30 mL/min/1.73m2 (calculation will be based on the Chronic Kidney Disease Epidemiology Collaboration [CKD-EPI] equation; refer to Section 10.1)\n- 11. Is currently taking tafamidis, acoramidis, doxycycline, or tauroursodeoxycholic acid; if previously on any of these agents, must have completed a 14-day washout prior to dosing (Day 1)\n- 12. Is currently taking diflunisal; if previously on this agent, must have completed a 3-day washout prior to dosing (Day 1)\n- 2. Has known other (non-hATTR) forms of amyloidosis or clinical evidence of leptomeningeal amyloidosis\n- 3. Has a New York Heart Association (NYHA) heart failure classification >2\n- 4. Has any of the following laboratory parameter assessments at screening: a. Alanine aminotransferase (ALT) or aspartate aminotransferase (AST) >2.5 upper limit of normal (ULN) reference range b. Total bilirubin >1.5 ULN (>2 ULN in patients with Gilbert's Syndrome) c. International normalized ratio (INR) >2.0\n- 5. Platelet count <50,000/µL\n- 6. Absolute neutrophil count <1500 cells/mm³\n- 27. Has a life expectancy of <2 years based on Investigator's clinical judgement\n- 28. Is not willing to comply with the contraceptive requirements during the study period as described in Section 5.9.1\n- 29. Female patient who is pregnant or breastfeeding\n- 8. Has vitamin B12 levels below the lower limit of normal\n- 30. Unwilling or unable to limit alcohol consumption throughout the course of the study\n- 31. History of alcohol use disorder within the last 12 months before screening in the opinion of the Investigator\n- 32. History of illicit substance use within the past 5 years that in the opinion of the Investigator would interfere with compliance with study procedures or follow-up visits\n- 9. Has known human immunodeficiency virus infection; or evidence of acute or chronic hepatitis C virus or hepatitis B virus infection\n- 13. Anticipated to start an approved TTR stabilizing therapy (eg, tafamidis or acoramidis) within the first 18 months following randomization\n- 14. Received prior anti-TTR antibody treatment (ie, “TTR depleters”)\n- 15. Received an investigational agent within the last 30 days (or 5 half-lives of the investigational drug, whichever is longer), or are currently in follow-up of another clinical study. Any agent that has received health agency authorization (including for emergency use) by local or regional authorities is not considered investigational\n- 16. Has other known causes of sensorimotor or autonomic neuropathy (eg, autoimmune disease, monoclonal gammopathy) that the treating physician believes to be contributing to the neuropathy\n- 17. Had acute coronary syndrome within the past 3 months"}
Endpoints
Primary endpoints
- {"endpoint_text":"- Change from baseline in the Modified Neuropathy Impairment Score +7 (mNIS+7) compared to the external placebo group from the APOLLO study at Month 9","definition_or_measurement_approach":"Change from baseline in mNIS+7 measured at Month 9 and compared against an external placebo group derived from the APOLLO study."}
Secondary endpoints
- {"endpoint_text":"- 1. Change from baseline in Norfolk Quality of Life Diabetic Neuropathy (Norfolk QoL-DN) total score compared to the external placebo group from the APOLLO study at Month 9","definition_or_measurement_approach":"Change from baseline in Norfolk QoL-DN total score measured at Month 9 compared to external APOLLO placebo group."}
- {"endpoint_text":"- 2. Percent reduction in serum TTR levels in the nucresiran group compared to the in-study vutrisiran group through Month 9","definition_or_measurement_approach":"Percent reduction in serum transthyretin (TTR) concentration in nucresiran arm versus in-study vutrisiran arm through Month 9; measured via serum assays."}
- {"endpoint_text":"- 3. Change from baseline in modified body mass index (mBMI) compared to the external placebo group from the APOLLO study at Month 9","definition_or_measurement_approach":"Change from baseline in mBMI at Month 9 compared to external APOLLO placebo group."}
- {"endpoint_text":"- 4. Change from baseline in the following parameters compared to the external placebo group from the APOLLO study at Month 18: o mNIS+7; o Norfolk QoL-DN total score; o mBMI; o Rasch-built Overall Disability Scale R-ODS; o Timed 10-meter walk test (10-MWT)","definition_or_measurement_approach":"Change from baseline at Month 18 for listed clinical and functional measures (mNIS+7, Norfolk QoL-DN, mBMI, R-ODS, 10-MWT) compared to external APOLLO placebo group; each measured using the respective validated instrument or timed test."}
- {"endpoint_text":"- 5. Percent reduction in serum TTR levels in the nucresiran group compared to the in-study vutrisiran group through Month 18","definition_or_measurement_approach":"Percent reduction in serum TTR in nucresiran arm versus in-study vutrisiran arm through Month 18."}
- {"endpoint_text":"- 6. Percent reduction in serum TTR levels in the nucresiran group compared to the in-study vutrisiran group at Week 6","definition_or_measurement_approach":"Percent reduction in serum TTR in nucresiran arm versus in-study vutrisiran arm measured at Week 6."}
Recruitment
- Digital Remote Recruitment
- True, recruitment and participant engagement via Proofpilot patient and site engagement portal, Neonstone virtual platform for patient/site engagement and recruitment tracking, online trial posting/social imagery and registration postcards referenced in recruitment materials.
- Planned Sample Size
- 100
- Recruitment Window Months
- 53
- Consent Approach
- Participants must provide written informed consent (inclusion criterion requires ability to provide written informed consent). Main informed consent forms and related ICFs (pregnancy, newborn, partner consent, data protection notices, optional future research consents) are provided in country- and language-specific versions (examples in Portuguese, French, Greek, German, English, Italian, Spanish, Swedish). Consent is obtained from the participant (adults only; age 18 or age of legal consent).
Methods
- Country-specific recruitment materials: Patient Information Brochures, Visual Talking Points, HCP Fact Cards, GP Letters, Study Visit Guides and Trial Posting Information (K1/K2 recruitment-arrangements documents) distributed in multiple country/language versions.
- Digital participant engagement platforms: Patient and Site Engagement Portal (Proofpilot Inc.) and a virtual platform for patient and site engagement and recruitment tracking (Neonstone Limited).
- Site-based recruitment via participating hospitals and neurology clinics (trial sites listed per country).
- Printed recruitment items and outreach: Registration postcards, Post-It pads, Retention/Visit reminders and Trial social imagery for trial posting.
Geography
- Total Number Of Sites
- 25
- Total Number Of Participants
- 86
Portugal
- Earliest CTIS Part Ii Submission Date
- 12-11-2025
- Latest Decision Or Authorization Date
- 25-02-2026
- Processing Time Days
- 105
- Number Of Sites
- 2
- Number Of Participants
- 8
Sites
- Site Name
- Unidade Local de Saúde de Santa Maria - Hopital de Santa Maria
- Principal Investigator Name
- Isabel Conceição
- Principal Investigator Email
- imsconceicao@gmail.com
- Contact Person Name
- Isabel Conceição
- Contact Person Email
- imsconceicao@gmail.com
- Site Name
- Unidade Local de Saúde de Santo António - Hospital de Santo António
- Principal Investigator Name
- Teresa Coelho
- Principal Investigator Email
- responsavel.paramiloidose@chporto.min-saude.pt
- Contact Person Name
- Teresa Coelho
- Contact Person Email
- responsavel.paramiloidose@chporto.min-saude.pt
France
- Earliest CTIS Part Ii Submission Date
- 10-02-2026
- Latest Decision Or Authorization Date
- 25-02-2026
- Processing Time Days
- 15
- Number Of Sites
- 3
- Number Of Participants
- 9
Sites
- Site Name
- Assistance Publique Hopitaux De Paris
- Department Name
- Département de Neurologie
- Principal Investigator Name
- Violaine PLANTE-BORDENEUVE
- Principal Investigator Email
- violaine.plante@aphp.fr
- Contact Person Name
- Violaine PLANTE-BORDENEUVE
- Contact Person Email
- violaine.plante@aphp.fr
- Site Name
- Centre Hospitalier Regional De Marseille
- Department Name
- Centre de référence des Maladies neuromusculaires et de la SLA
- Principal Investigator Name
- Shahram ATTARIAN
- Principal Investigator Email
- shahram.attarian@ap-hm.fr
- Contact Person Name
- Shahram ATTARIAN
- Contact Person Email
- shahram.attarian@ap-hm.fr
- Site Name
- Bicetre Hospital
- Department Name
- CRMR Neurologie Adultes
- Principal Investigator Name
- Andoni ECHANIZ-LAGUNA
- Principal Investigator Email
- andoni.echaniz-laguna@aphp.fr
- Contact Person Name
- Andoni ECHANIZ-LAGUNA
- Contact Person Email
- andoni.echaniz-laguna@aphp.fr
Germany
- Earliest CTIS Part Ii Submission Date
- 11-12-2025
- Latest Decision Or Authorization Date
- 24-02-2026
- Processing Time Days
- 75
- Number Of Sites
- 3
- Number Of Participants
- 8
Sites
- Site Name
- Universitaetsklinikum Heidelberg AöR
- Department Name
- Department of Neurology
- Principal Investigator Name
- Markus Weiler
- Principal Investigator Email
- markus.weiler@med.uni-heidelberg.de
- Contact Person Name
- Markus Weiler
- Contact Person Email
- markus.weiler@med.uni-heidelberg.de
- Site Name
- Charite Universitaetsmedizin Berlin KöR
- Department Name
- Klinik für Kardiologie Angologie und Intensivmedizin
- Principal Investigator Name
- Katrin Hahn
- Principal Investigator Email
- katrin.hahn@charite.de
- Contact Person Name
- Katrin Hahn
- Contact Person Email
- katrin.hahn@charite.de
- Site Name
- Universitaet Muenster
- Department Name
- Medizinische Klinik B (Gastroenterologie, Hepatologie, Endokrinologie, Klinische Infektiologie)
- Principal Investigator Name
- Michael Praktiknjo
- Principal Investigator Email
- Michael.Praktiknjo@ukmuenster.de
- Contact Person Name
- Michael Praktiknjo
- Contact Person Email
- Michael.Praktiknjo@ukmuenster.de
Greece
- Earliest CTIS Part Ii Submission Date
- 12-11-2025
- Latest Decision Or Authorization Date
- 24-02-2026
- Processing Time Days
- 104
- Number Of Sites
- 2
- Number Of Participants
- 5
Sites
- Site Name
- General Hospital Of Athens Alexandra
- Department Name
- Plasma Cell Dyscrasias Unit, Department of Clinical Therapeutics
- Principal Investigator Name
- Efstathios Kastritis
- Principal Investigator Email
- ekastritis@gmail.com
- Contact Person Name
- Efstathios Kastritis
- Contact Person Email
- ekastritis@gmail.com
- Site Name
- University General Hospital Of Heraklion
- Department Name
- Neurology Department
- Principal Investigator Name
- Ioannis Zaganas
- Principal Investigator Email
- johnzag@yahoo.com
- Contact Person Name
- Ioannis Zaganas
- Contact Person Email
- johnzag@yahoo.com
Ireland
- Earliest CTIS Part Ii Submission Date
- 08-01-2026
- Latest Decision Or Authorization Date
- 27-02-2026
- Processing Time Days
- 50
- Number Of Sites
- 1
- Number Of Participants
- 4
Sites
- Site Name
- Tallaght University Hospital
- Department Name
- Neurology
- Principal Investigator Name
- Sinead Murphy
- Principal Investigator Email
- Sinead.murphy@tuh.ie
- Contact Person Name
- Sinead Murphy
- Contact Person Email
- Sinead.murphy@tuh.ie
Italy
- Earliest CTIS Part Ii Submission Date
- 29-01-2026
- Latest Decision Or Authorization Date
- 25-02-2026
- Processing Time Days
- 27
- Number Of Sites
- 6
- Number Of Participants
- 25
Sites
- Site Name
- Fondazione Policlinico Universitario Agostino Gemelli IRCCS
- Department Name
- UOC Neurologia
- Principal Investigator Name
- Marco Luigetti
- Principal Investigator Email
- marco.luigetti@policlinicogemelli.it
- Contact Person Name
- Marco Luigetti
- Contact Person Email
- marco.luigetti@policlinicogemelli.it
- Site Name
- Azienda Ospedaliero Universitaria Careggi
- Department Name
- UOC Neurologia D'Urgenza, Padiglione 25 CTO
- Principal Investigator Name
- Alessandro Barilaro
- Principal Investigator Email
- alessandro.barilaro@unifi.it
- Contact Person Name
- Alessandro Barilaro
- Contact Person Email
- alessandro.barilaro@unifi.it
- Site Name
- Azienda Ospedaliera di Padova
- Department Name
- UOC Clinica Neurologica, ground floor Palazzina di Neuroscienze B1
- Principal Investigator Name
- Chiara Briani
- Principal Investigator Email
- chiara.briani@unipd.it
- Contact Person Name
- Chiara Briani
- Contact Person Email
- chiara.briani@unipd.it
- Site Name
- Azienda Ospedaliera Universitaria Gaetano Martino Messina
- Department Name
- Department of Clinical and Experimental Medicine, University of Messina
- Principal Investigator Name
- Massimo Russo
- Principal Investigator Email
- russom@unime.it
- Contact Person Name
- Massimo Russo
- Contact Person Email
- russom@unime.it
- Site Name
- Fondazione IRCCS Policlinico San Matteo
- Department Name
- U.O.C. Medicina Gen 2- Centro Amiloidosi Sistemiche e Malattie ad Alta Complessità
- Principal Investigator Name
- Laura Piera Obici
- Principal Investigator Email
- l.obici@smatteo.pv.it
- Contact Person Name
- Laura Piera Obici
- Contact Person Email
- l.obici@smatteo.pv.it
- Site Name
- IRCCS Foundation Istituto Neurologico Carlo Besta
- Department Name
- Clinical Neurology, Head of Rare Neurological Diseases Unit
- Principal Investigator Name
- Davide Pareyson
- Principal Investigator Email
- davide.pareyson@istituto-besta.it
- Contact Person Name
- Davide Pareyson
- Contact Person Email
- davide.pareyson@istituto-besta.it
Spain
- Earliest CTIS Part Ii Submission Date
- 22-01-2026
- Latest Decision Or Authorization Date
- 27-02-2026
- Processing Time Days
- 36
- Number Of Sites
- 5
- Number Of Participants
- 15
Sites
- Site Name
- Bellvitge University Hospital
- Department Name
- Neurology
- Principal Investigator Name
- Carlos Casasnovas Pons
- Principal Investigator Email
- clinicalresearchwindow@bellvitgehospital.cat
- Contact Person Name
- Carlos Casasnovas Pons
- Contact Person Email
- clinicalresearchwindow@bellvitgehospital.cat
- Site Name
- Hospital Clinico San Carlos
- Department Name
- Neurology
- Principal Investigator Name
- Lucía Galán Dávila
- Principal Investigator Email
- lgalan@salud.madrid.org
- Contact Person Name
- Lucía Galán Dávila
- Contact Person Email
- lgalan@salud.madrid.org
- Site Name
- Hospital Universitario Juan Ramon Jimenez
- Department Name
- Internal Medicine
- Principal Investigator Name
- Francisco Muñoz Beamud
- Principal Investigator Email
- eecc.amiloidosis.hujrj@fabis.org
- Contact Person Name
- Francisco Muñoz Beamud
- Contact Person Email
- eecc.amiloidosis.hujrj@fabis.org
- Site Name
- Hospital Universitari Vall D Hebron
- Department Name
- Internal Medicine
- Principal Investigator Name
- Fernando Martínez Valle
- Principal Investigator Email
- Fernando.martinez@vallhebron.cat
- Contact Person Name
- Fernando Martínez Valle
- Contact Person Email
- Fernando.martinez@vallhebron.cat
- Site Name
- Hospital Son Llatzer
- Department Name
- Internal Medicine
- Principal Investigator Name
- Inés Asunción Losada López
- Principal Investigator Email
- ialosada@hsll.es
- Contact Person Name
- Inés Asunción Losada López
- Contact Person Email
- ialosada@hsll.es
Sweden
- Earliest CTIS Part Ii Submission Date
- 12-11-2025
- Latest Decision Or Authorization Date
- 20-02-2026
- Processing Time Days
- 100
- Number Of Sites
- 2
- Number Of Participants
- 7
Sites
- Site Name
- Region Stockholm – SLSO
- Department Name
- Studieenheten Akademiskt specialistcentrum, Dalagatan 9, 11361 Stockholm
- Principal Investigator Name
- Kristin Samuelsson
- Principal Investigator Email
- kristin.samuelsson@regionstockholm.se
- Contact Person Name
- Kristin Samuelsson
- Contact Person Email
- kristin.samuelsson@regionstockholm.se
- Site Name
- Region Vaesterbotten
- Department Name
- Norrlands Universitetssjukhus,Medicin Centrum E41,Amyloidos Centrum, Försörjningsvägen 1C 90185 Umea
- Principal Investigator Name
- Jonas Wixner
- Principal Investigator Email
- jonas.wixner@umu.se
- Contact Person Name
- Jonas Wixner
- Contact Person Email
- jonas.wixner@umu.se
Cyprus
- Earliest CTIS Part Ii Submission Date
- 20-02-2026
- Latest Decision Or Authorization Date
- 06-03-2026
- Processing Time Days
- 14
- Number Of Sites
- 1
- Number Of Participants
- 5
Sites
- Site Name
- The Cyprus Foundation For Muscular Dystrophy Research
- Department Name
- Neuroepidemiology Department and EMG (Electromyography) Laboratory
- Principal Investigator Name
- Eleni Zamba Papanicolaou
- Principal Investigator Email
- ezamba@cing.ac.cy
- Contact Person Name
- Eleni Zamba Papanicolaou
- Contact Person Email
- ezamba@cing.ac.cy
Sponsor
Primary sponsor
- Full Name
- Alnylam Pharmaceuticals Inc.
- Organisation Type
- Pharmaceutical company
- Country Of Registered Address
- United States
Contract research organisations
- Name
- Syneos Health Inc.
- Responsibilities
- Operational / clinical trial services (sponsorDuties code: 8)
- Name
- PPD Global Ltd.
- Responsibilities
- Project management duties or monitoring/regulatory
- Name
- PPD Development LP
- Responsibilities
- Multiple operational roles (codes: 1,10,11,12,13,2,5,6,7,8,9) as listed in sponsorDuties
- Name
- Pharmaceutical Product Development LLC
- Responsibilities
- Laboratory Logistics and other operational support
- Name
- Advanced Clinical LLC
- Responsibilities
- Operational support (sponsorDuties code: 6)
- Name
- Suvoda LLC
- Responsibilities
- Operational services (sponsorDuties code: 3)
Third parties
- {"country":"Germany","full_name":"Fisher Clinical Services GmbH","duties_or_roles":"sponsorDuties codes: 14","organisation_type":"Pharmaceutical company"}
- {"country":"United States","full_name":"Syneos Health Inc.","duties_or_roles":"sponsorDuties codes: 8","organisation_type":"Pharmaceutical company"}
- {"country":"Germany","full_name":"Centogene GmbH","duties_or_roles":"sponsorDuties codes: 4","organisation_type":"Laboratory/Research/Testing facility"}
- {"country":"United Kingdom","full_name":"Neonstone Limited","duties_or_roles":"Virtual platform for patient and site engagament, recruitment tracking","organisation_type":"Non-Pharmaceutical company"}
- {"country":"United Kingdom","full_name":"Illingworth Research Group Limited","duties_or_roles":"Home Nursing","organisation_type":"Hospital/Clinic/Other health care facility"}
- {"country":"United States","full_name":"The Brigham And Women’s Hospital Inc.","duties_or_roles":"Central Medical Reading of subjects' imaging (ECHO imaging reading)","organisation_type":"Hospital/Clinic/Other health care facility"}
- {"country":"United States","full_name":"Proofpilot Inc.","duties_or_roles":"Patient and Site Engagement Portal","organisation_type":"Pharmaceutical company"}
- {"country":"Greece","full_name":"PPD Global Ltd.","duties_or_roles":"Project management duties or monitoring/regulatory","organisation_type":"Pharmaceutical company"}
- {"country":"Canada","full_name":"Charles River Laboratories Montreal ULC","duties_or_roles":"sponsorDuties codes: 4","organisation_type":"Pharmaceutical company"}
- {"country":"United States","full_name":"Mayo Clinic Hospital Rochester","duties_or_roles":"Central Medical Reading of subjects' NIS, mNIS+7 assessment","organisation_type":"Hospital/Clinic/Other health care facility"}
- {"country":"United States","full_name":"Advanced Clinical LLC","duties_or_roles":"sponsorDuties codes: 6","organisation_type":"Pharmaceutical company"}
- {"country":"United States","full_name":"Aliri USA Inc.","duties_or_roles":"sponsorDuties codes: 4","organisation_type":"Laboratory/Research/Testing facility"}
- {"country":"United States","full_name":"PPD Development LP","duties_or_roles":"sponsorDuties codes: 1,10,11,12,13,2,5,6,7,8,9 (multiple operational roles listed)","organisation_type":"Pharmaceutical company"}
- {"country":"United States","full_name":"Pharmaceutical Product Development LLC","duties_or_roles":"Laboratory Logistics; sponsorDuties codes: 15 and 4","organisation_type":"Pharmaceutical company"}
- {"country":"United States","full_name":"Suvoda LLC","duties_or_roles":"sponsorDuties codes: 3","organisation_type":"Non-Pharmaceutical company"}
- {"country":"United States","full_name":"Greenphire LLC","duties_or_roles":"Patient travel and reimbursement","organisation_type":"Non-Pharmaceutical company"}
- {"country":"United States","full_name":"EPL Pathology Archives LLC","duties_or_roles":"sponsorDuties codes: 4","organisation_type":"Laboratory/Research/Testing facility"}
- {"country":"United States","full_name":"Clario Medical Imaging Inc.","duties_or_roles":"Central Medical Reading of subjects' imaging (ECG imaging reading)","organisation_type":"Pharmaceutical company"}
- {"country":"United States","full_name":"Aliri USA Inc.","duties_or_roles":"sponsorDuties codes: 4","organisation_type":"Laboratory/Research/Testing facility"}
Investigational products
- Investigational Product Name
- ALN-TTRSC04
- Active Substance
- NUCRESIRAN
- Modality
- Oligonucleotide
- Routes Of Administration
- Solution for injection in pre-filled syringe (subcutaneous injection per protocol)
- Route
- Subcutaneous
- Authorisation Status
- Investigational (no marketing authorisation indicated in product dictionary)
- Starting Dose
- 300 mg
- Dose Levels
- 300 mg
- Frequency
- Every 6 months
- Investigational Product Name
- Amvuttra 25 mg solution for injection in pre-filled syringe
- Active Substance
- VUTRISIRAN
- Modality
- Oligonucleotide
- Routes Of Administration
- Subcutaneous
- Route
- Subcutaneous
- Authorisation Status
- Authorised in EU (marketing authorisation number EU/1/22/1681/001 listed)
- Orphan Designation
- Yes
- Starting Dose
- 25 mg
- Dose Levels
- 25 mg
- Frequency
- Every 3 months
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