Flag of the European Union EU Clinical Trials Register Help

Clinical trials for Familial amyloid polyneuropathy

The European Union Clinical Trials Register   allows you to search for protocol and results information on:
  • interventional clinical trials that were approved in the European Union (EU)/European Economic Area (EEA) under the Clinical Trials Directive 2001/20/EC
  • clinical trials conducted outside the EU/EEA that are linked to European paediatric-medicine development

  • EU/EEA interventional clinical trials approved under or transitioned to the Clinical Trial Regulation 536/2014 are publicly accessible through the
    Clinical Trials Information System (CTIS).


    The EU Clinical Trials Register currently displays   44358   clinical trials with a EudraCT protocol, of which   7384   are clinical trials conducted with subjects less than 18 years old.   The register also displays information on   18700   older paediatric trials (in scope of Article 45 of the Paediatric Regulation (EC) No 1901/2006).

    Phase 1 trials conducted solely on adults and that are not part of an agreed paediatric investigation plan (PIP) are not publicly available (see Frequently Asked Questions ).  
     
    Examples: Cancer AND drug name. Pneumonia AND sponsor name.
    How to search [pdf]
    Search Tips: Under advanced search you can use filters for Country, Age Group, Gender, Trial Phase, Trial Status, Date Range, Rare Diseases and Orphan Designation. For these items you should use the filters and not add them to your search terms in the text field.
    Advanced Search: Search tools
     
    19 result(s) found for: Familial amyloid polyneuropathy. Displaying page 1 of 1.
    EudraCT Number: 2014-001586-27 Sponsor Protocol Number: SOMCT01-C Start Date*: 2014-07-25
    Sponsor Name:SOM Innovation Biotech SL (SOM Biotech)
    Full Title: Study of SOM0226 in familial amyloid polyneuropathy (FAP) patients and asymptomatic carriers to evaluate protein stabilization activity
    Medical condition: Patients with familial amyloid polyneuropathy (FAP), asymptomatic carriers and healthy volunteers
    Disease: Version SOC Term Classification Code Term Level
    17.0 100000004850 10057949 Familial amyloid polyneuropathy LLT
    Population Age: Adults Gender: Male, Female
    Trial protocol: ES (Prematurely Ended)
    Trial results: View results
    EudraCT Number: 2015-002603-29 Sponsor Protocol Number: ALN-TTRSC-005 Start Date*: 2015-12-01
    Sponsor Name:Alnylam Pharmaceuticals, Inc
    Full Title: An Open-Label Study To Evaluate The Efficacy And Safety Of Revusiran In Patients With Transthyretin-Mediated Familial Amyloidotic Polyneuropathy With Disease Progression Post Orthotopic Liver Trans...
    Medical condition: Transthyretin (TTR)-mediated Familial Amyloidotic Polyneuropathy (FAP)
    Disease: Version SOC Term Classification Code Term Level
    18.0 10010331 - Congenital, familial and genetic disorders 10019889 Hereditary neuropathic amyloidosis PT
    Population Age: Adults Gender: Male, Female
    Trial protocol: SE (Completed) PT (Completed) ES (Completed) GB (Completed)
    Trial results: View results
    EudraCT Number: 2012-001831-30 Sponsor Protocol Number: ISIS420915-CS2 Start Date*: 2013-04-22
    Sponsor Name:Ionis Pharmaceuticals, Inc.
    Full Title: A Phase 2/3 Randomized, Double-Blind, Placebo-Controlled Study to Assess the Efficacy and Safety of ISIS 420915 in Patients with Familial Amyloid Polyneuropathy
    Medical condition: Familial Amyloid Polyneuropathy
    Disease: Version SOC Term Classification Code Term Level
    19.0 100000004850 10057949 Familial amyloid polyneuropathy LLT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: GB (Completed) PT (Completed) DE (Completed) IT (Completed) ES (Completed) BG (Completed)
    Trial results: View results
    EudraCT Number: 2017-005115-13 Sponsor Protocol Number: ISIS420915-CS4 Start Date*: 2018-04-25
    Sponsor Name:Ionis Pharmaceuticals, Inc.
    Full Title: A Non-Interventional Study to Monitor the Survival Status of Patients that Discontinued from ISIS 420915-CS2 or ISIS 420915-CS3
    Medical condition: Familial Amyloid Polyneuropathy
    Disease: Version SOC Term Classification Code Term Level
    20.0 100000004850 10057949 Familial amyloid polyneuropathy LLT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: ES (Ongoing) FR (Ongoing) GB (Completed) PT (Prematurely Ended)
    Trial results: (No results available)
    EudraCT Number: 2013-004561-13 Sponsor Protocol Number: ISIS420915-CS3 Start Date*: 2014-07-04
    Sponsor Name:Ionis Pharmaceuticals, Inc
    Full Title: An Open-Label Extension Study to Assess the Long-Term Safety and Efficacy of ISIS 420915 in Patients with Familial Amyloid Polyneuropathy (FAP)
    Medical condition: Familial Amyloid Polyneuropathy
    Disease: Version SOC Term Classification Code Term Level
    20.0 100000004850 10057949 Familial amyloid polyneuropathy LLT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: PT (Completed) GB (GB - no longer in EU/EEA) DE (Completed) FR (Completed) ES (Ongoing)
    Trial results: View results
    EudraCT Number: 2014-003877-40 Sponsor Protocol Number: ALN-TTR02-006 Start Date*: 2015-05-11
    Sponsor Name:Alnylam Pharmaceuticals, Inc.
    Full Title: A Multicenter, Open-Label, Extension Study to Evaluate the Long-term Safety and Efficacy of Patisiran in Patients with Familial Amyloidotic Polyneuropathy Who Have Completed a Prior Clinical Study ...
    Medical condition: Transthyretin mediated amyloidosis (ATTR)
    Disease: Version SOC Term Classification Code Term Level
    17.1 10007541 - Cardiac disorders 10007509 Cardiac amyloidosis PT
    17.1 10010331 - Congenital, familial and genetic disorders 10019889 Hereditary neuropathic amyloidosis PT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: ES (Ongoing) SE (Completed) PT (Completed) DE (Completed) NL (Completed) GB (GB - no longer in EU/EEA) CY (Completed) IT (Completed)
    Trial results: View results
    EudraCT Number: 2006-001066-16 Sponsor Protocol Number: H23750 Start Date*: 2007-01-04
    Sponsor Name:John L. Berk, M.D.
    Full Title: The effect of diflunisal (IND 68092) on familial amyloidosis
    Medical condition: Familial Amyloid Polyneuropathy (FAP)
    Disease:
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: SE (Completed) IT (Ongoing) GB (GB - no longer in EU/EEA)
    Trial results: (No results available)
    EudraCT Number: 2018-004670-10 Sponsor Protocol Number: AG10-333 Start Date*: 2020-07-07
    Sponsor Name:Eidos Therapeutics, Inc.
    Full Title: A Phase 3, Randomized, Double-Blind, Placebo-Controlled Study of the Efficacy and Safety of AG10 in Subjects with Symptomatic Transthyretin Amyloid Polyneuropathy (ATTRibute-PN Trial)
    Medical condition: Symptomatic Transthyretin Amyloid Polineuropathy (ATTR-PN)
    Disease: Version SOC Term Classification Code Term Level
    20.0 100000004850 10057949 Familial amyloid polyneuropathy LLT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: HU (Prematurely Ended) DE (Prematurely Ended) PT (Prematurely Ended) FR (Completed) BG (Prematurely Ended) NL (Prematurely Ended) IT (Prematurely Ended)
    Trial results: (No results available)
    EudraCT Number: 2009-011535-12 Sponsor Protocol Number: Fx1A-303 Start Date*: 2009-08-26
    Sponsor Name:Pfizer Inc.
    Full Title: Open-Label Safety and Efficacy Evaluation of Fx-1006A in Patients with Transthyretin Amyloidosis
    Medical condition: Transthyretin Amyloidosis (ATTR)
    Disease: Version SOC Term Classification Code Term Level
    14.1 10010331 - Congenital, familial and genetic disorders 10057949 Familial amyloid polyneuropathy LLT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: DE (Completed) SE (Completed) FR (Completed) PT (Completed) IT (Completed)
    Trial results: View results
    EudraCT Number: 2019-001698-10 Sponsor Protocol Number: ION-682884-CS3 Start Date*: 2020-03-06
    Sponsor Name:Ionis Pharmaceuticals, Inc.
    Full Title: A Phase 3 Global, Open-Label, Randomized Study to Evaluate the Efficacy and Safety of ION-682884 in Patients with Hereditary Transthyretin-Mediated Amyloid Polyneuropathy
    Medical condition: Hereditary Transthyretin-Mediated Amyloid Polyneuropathy
    Disease: Version SOC Term Classification Code Term Level
    20.0 100000004850 10057949 Familial amyloid polyneuropathy LLT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: GB (GB - no longer in EU/EEA) PT (Completed) DE (Completed) ES (Ongoing) IT (Completed) SE (Completed) GR (Prematurely Ended) FR (Completed) CY (Completed) NL (Prematurely Ended)
    Trial results: View results
    EudraCT Number: 2013-002987-17 Sponsor Protocol Number: ALN-TTR02-004 Start Date*: 2013-11-25
    Sponsor Name:Alnylam Pharmaceuticals, Inc
    Full Title: 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 Polyneu...
    Medical condition: Transthyretin mediated amyloidosis (ATTR)
    Disease: Version SOC Term Classification Code Term Level
    19.0 10007541 - Cardiac disorders 10007509 Cardiac amyloidosis PT
    19.0 10010331 - Congenital, familial and genetic disorders 10019889 Hereditary neuropathic amyloidosis PT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: SE (Completed) ES (Completed) PT (Completed) IT (Completed) DE (Completed) NL (Completed) GB (Completed)
    Trial results: View results
    EudraCT Number: 2008-001262-87 Sponsor Protocol Number: Fx-006 Start Date*: 2008-07-04
    Sponsor Name:FoldRx Pharmaceuticals, Inc.
    Full Title: An Open-Label Extension of Study Fx-005 Evaluating Long-Term Safety and Clinical Outcomes of Fx-1006A in Patients with Transthyretin Amyloid Polyneuropathy
    Medical condition: Transthyretin Amyloid Polyneuropathy (ATTR-PN)
    Disease: Version SOC Term Classification Code Term Level
    9.1 10057949 Familial amyloid polyneuropathy LLT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: PT (Completed) SE (Completed) FR (Completed) DE (Completed)
    Trial results: View results
    EudraCT Number: 2018-002098-23 Sponsor Protocol Number: ALN-TTRSC02-002 Start Date*: 2019-05-09
    Sponsor Name:Alnylam Pharmaceuticals, Inc.
    Full Title: HELIOS-A: A Phase 3 Global, Randomized, Open-label Study to Evaluate the Efficacy and Safety of ALN-TTRSC02 in Patients with Hereditary Transthyretin Amyloidosis (hATTR Amyloidosis)
    Medical condition: Hereditary transthyretin-mediated amyloidosis (hATTR amyloidosis)
    Disease: Version SOC Term Classification Code Term Level
    20.0 10007541 - Cardiac disorders 10007509 Cardiac amyloidosis PT
    20.0 10010331 - Congenital, familial and genetic disorders 10019889 Hereditary neuropathic amyloidosis PT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: DE (Completed) PT (Trial now transitioned) GB (GB - no longer in EU/EEA) BG (Trial now transitioned) ES (Ongoing) BE (Trial now transitioned) GR (Completed) NL (Trial now transitioned) CY (Trial now transitioned) IT (Trial now transitioned)
    Trial results: (No results available)
    EudraCT Number: 2021-001427-40 Sponsor Protocol Number: ION-682884-CS13 Start Date*: 2022-06-07
    Sponsor Name:Ionis Pharmaceuticals, Inc.
    Full Title: An Open-Label, Extension Study to Assess the Long-Term Safety and Efficacy of ION-682884 in Patients with Hereditary Transthyretin-Mediated Amyloid Polyneuropathy
    Medical condition: Hereditary Transthyretin-Mediated Amyloid Polyneuropathy
    Disease: Version SOC Term Classification Code Term Level
    20.0 100000004850 10057949 Familial amyloid polyneuropathy LLT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: PT (Trial now transitioned) ES (Ongoing) SE (Trial now transitioned) IT (Trial now transitioned) FR (Trial now transitioned) CY (Trial now transitioned)
    Trial results: (No results available)
    EudraCT Number: 2013-001644-65 Sponsor Protocol Number: ALN-TTR02-003 Start Date*: 2013-07-19
    Sponsor Name:Alnylam Pharmaceuticals, Inc.
    Full Title: A Phase 2, Multicenter, Open-Label, Extension Study to Evaluate the Long-Term Safety, Clinical Activity, and Pharmacokinetics of ALN-TTR02 in Patients With Familial Amyloidotic Polyneuropathy Who ...
    Medical condition: Transthyretin mediated amyloidosis (ATTR)
    Disease: Version SOC Term Classification Code Term Level
    14.1 10007541 - Cardiac disorders 10007509 Cardiac amyloidosis PT
    14.1 10010331 - Congenital, familial and genetic disorders 10019889 Hereditary neuropathic amyloidosis PT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: PT (Completed) SE (Completed) ES (Completed)
    Trial results: View results
    EudraCT Number: 2012-000467-24 Sponsor Protocol Number: ALN-TTR02-002 Start Date*: 2012-05-08
    Sponsor Name:Alnylam Pharmaceuticals, Inc.
    Full Title: A Phase 2, Open-Label, Multi-Dose, Dose Escalation Trial to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Intravenous Infusions of ALN-TTR02 in Patients with TTR Amyl...
    Medical condition: Transthyretin mediated amyloidosis (ATTR)
    Disease: Version SOC Term Classification Code Term Level
    15.1 10007541 - Cardiac disorders 10007509 Cardiac amyloidosis PT
    15.1 10010331 - Congenital, familial and genetic disorders 10019889 Hereditary neuropathic amyloidosis PT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: SE (Completed) PT (Completed) DE (Completed) ES (Completed)
    Trial results: View results
    EudraCT Number: 2006-002792-41 Sponsor Protocol Number: Fx-005 Start Date*: 2006-10-30
    Sponsor Name:FoldRx Pharmaceuticals Limited
    Full Title: Safety and Efficacy of Orally Administered Fx-1006A in Patients with Familial Amyloid Polyneuropathy (FAP): a Phase II/III, Randomised, Double-Blind, Placebo-Controlled Study
    Medical condition: Familial Amyloid Polyneuropathy (FAP).
    Disease: Version SOC Term Classification Code Term Level
    9.1 10057949 Familial amyloid polyneuropathy LLT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: SE (Completed) PT (Completed) GB (Completed) ES (Completed) DE (Completed)
    Trial results: View results
    EudraCT Number: 2018-003519-24 Sponsor Protocol Number: ALN-TTR02-008 Start Date*: 2019-03-12
    Sponsor Name:Alnylam Pharmaceuticals, Inc.
    Full 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 Progressi...
    Medical condition: Hereditary Transthyretin-mediated Amyloidosis (hATTR amyloidosis)
    Disease: Version SOC Term Classification Code Term Level
    20.0 10007541 - Cardiac disorders 10007509 Cardiac amyloidosis PT
    20.0 10010331 - Congenital, familial and genetic disorders 10019889 Hereditary neuropathic amyloidosis PT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: GB (Completed) FR (Completed) PT (Completed) DE (Completed) ES (Completed) IT (Completed)
    Trial results: View results
    EudraCT Number: 2012-000633-39 Sponsor Protocol Number: FEC-TH Start Date*: 2012-05-21
    Sponsor Name:Fundación para la Formación e Investigación Sanitaria
    Full Title: Extracorporeal photopheresis in liver transplantation. Clinical trial phase I/II safety and efficacy in patients with progressive withdrawal of immunosuppression.
    Medical condition: Liver transplantation more than 2 years of evolution to complications of immunosuppression.
    Disease:
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: ES (Ongoing)
    Trial results: (No results available)
    Download Options:
    Number of Trials to download:
    Download Content:
    Download Format:
    Note, where multi-state trials are shown in search results, selecting "Full Trial details" will download full information for each of the member states/countries involved in the trial.
    Query did not match any studies.
    For support, Contact us.
    The status and protocol content of GB trials is no longer updated since 1 January 2021. For the UK, as of 31 January 2021, EU Law applies only to the territory of Northern Ireland (NI) to the extent foreseen in the Protocol on Ireland/NI. Legal notice
    As of 31 January 2023, all EU/EEA initial clinical trial applications must be submitted through CTIS . Updated EudraCT trials information and information on PIP/Art 46 trials conducted exclusively in third countries continues to be submitted through EudraCT and published on this website.

    European Medicines Agency © 1995-Sat Aug 30 06:18:22 CEST 2025 | Domenico Scarlattilaan 6, 1083 HS Amsterdam, The Netherlands
    EMA HMA