Search clinical trials for: Familial Amyloid Polyneuropathy

Total 3501 results

AstraZeneca

Recruiting

Hereditary Transthyretin Amyloidosis Polyneuropathy in Patients With Carpal Tunnel Syndrome in Russia (LOCUS)

Carpal Tunnel Syndrome | Amyloidosis | Polyneuropathy | Hereditary Transthyretin Amyloidosis

Russian Federation
AstraZeneca

Recruiting

hATTR Polyneuropathy in Russia (PRIMER)

Hereditary Transthyretin Amyloidosis

Russian Federation
Alnylam Pharmaceuticals

Recruiting

Observational Study of Neurofilament Light Chain (NfL) as a Biomarker in Asymptomatic Carriers of the Transthyretin (TTR) Variants and Patients With Hereditary Transthyretin-mediated (hATTR) Amyloidosis With Polyneuropathy

Hereditary Amyloidosis, Transthyretin-Related | Asymptomatic Carrier State

France
RwHealth
Northern Care Alliance NHS Foundation Trust; Alnylam Pharmaceuticals

Not yet recruiting

RWE-based Treatment Patterns and Outcomes in CIDP

Hereditary Amyloidosis | Guillain-Barre Syndrome | Chronic Inflammatory Demyelinating Polyneuropathy

United Kingdom
Rennes University Hospital

Recruiting

Myocardial Effects in Patients With ATTRv With Polyneuropathy Treated With Patisiran or Vutrisiran (MyocardON-TTR)

Transthyretin Amyloidosis | Amyloidosis, Hereditary

France
Intellia Therapeutics

Recruiting

Long-Term Follow-Up (LTFU) of Subjects Dosed With NTLA-2001

Transthyretin-Related (ATTR) Familial Amyloid Cardiomyopathy | Transthyretin-Related (ATTR) Familial Amyloid Polyneuropathy | Wild-Type Transthyretin Cardiac Amyloidosis

United Kingdom, New Zealand, Sweden
Alnylam Pharmaceuticals

Recruiting

Patisiran-Lipid Nanoparticle (LNP) Pregnancy Surveillance Program

Polyneuropathy | Hereditary Transthyretin-mediated (hATTR) Amyloidosis

Netherlands, Spain, United States, Germany, France, Italy, Portugal
Alnylam Pharmaceuticals

Recruiting

ConTTRibute: A Global Observational Study of Patients With Transthyretin (TTR)-Mediated Amyloidosis (ATTR Amyloidosis) (ConTTRibute)

Transthyretin-Mediated Amyloidosis | ATTR Amyloidosis

Israel, Italy, Netherlands, Spain, United States, France, Denmark, Germany, Portugal, Brazil, Taiwan
CENTOGENE GmbH Rostock
Alnylam Pharmaceuticals

Recruiting

TRAMmoniTTR Study Genetic Screening of an At-risk Population for hATTR and Monitoring of TTR Positive Subjects (TRAMmoniTTR)

Cardiomyopathies | Polyneuropathies | Transthyretin Amyloidosis | Transthyretin-Related (ATTR) Familial Amyloid Cardiomyopathy | Transthyretin-Related (ATTR) Familial Amyloid Polyneuropathy

Germany, Austria, Switzerland
Alnylam Pharmaceuticals

Approved for marketing

Expanded Access Protocol of Patisiran for Patients With Hereditary ATTR Amyloidosis (hATTR)

Amyloid Neuropathies | Amyloid Neuropathies, Familial | TTR-mediated Amyloidosis | Amyloidosis, Hereditary | Amyloidosis, Hereditary, Transthyretin-Related | Familial Amyloid Polyneuropathies
Pfizer

Completed

Retrospective Study Collecting Neurological Follow-up of Hereditary Transthyretin Amyloidosis (ATTRv) Patients Included in B3461028 and B3461045. (TRAMA)

Polyneuropathy | Hereditary Transthyretin Amyloidosis (ATTRv)

Spain
Alnylam Pharmaceuticals

Completed

APOLLO: The Study of an Investigational Drug, Patisiran (ALN-TTR02), for the Treatment of Transthyretin (TTR)-Mediated Amyloidosis

Amyloid Neuropathies | Amyloid Neuropathies, Familial | TTR-mediated Amyloidosis | Amyloidosis, Hereditary | Amyloidosis, Hereditary, Transthyretin-Related | Familial Amyloid Polyneuropathies

United States, Canada, Argentina, Australia, Bulgaria, Cyprus, Italy, Japan, Malaysia, Mexico, Netherlands, Portugal, Spain, Sweden, Germany, France, Brazil, Turkey, Korea, Republic of, Taiwan, United Kingdom
University of Pennsylvania
AstraZeneca; Ionis Pharmaceuticals, Inc.

Active, not recruiting

Early Detection of Neuropathy in ATTRv (EDONA)

Hereditary Amyloidosis, Transthyretin-Related

United States
Alnylam Pharmaceuticals

Completed

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)

Amyloidosis

United States, Canada, Argentina, Australia, Bulgaria, Cyprus, Italy, Japan, Malaysia, Netherlands, Portugal, Spain, Sweden, Taiwan, United Kingdom, Germany, France, Korea, Republic of, Brazil, Mexico, Turkey
Ionis Pharmaceuticals, Inc.

Completed

Extension Study Assessing Long Term Safety and Efficacy of IONIS-TTR Rx in Familial Amyloid Polyneuropathy (FAP)

Amyloidosis | Familial Amyloid Polyneuropathy | FAP | TTR | Transthyretin

United States, Spain, Argentina, Brazil, France, Germany, Italy, Portugal, United Kingdom
Intellia Therapeutics

Active, not recruiting

Study to Evaluate Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of NTLA-2001 in Patients With Hereditary Transthyretin Amyloidosis With Polyneuropathy (ATTRv-PN) and Patients With Transthyretin Amyloidosis-Related Cardiomyopathy (ATTR-CM)

Transthyretin-Related (ATTR) Familial Amyloid Cardiomyopathy | Transthyretin-Related (ATTR) Familial Amyloid Polyneuropathy | Wild-Type Transthyretin Cardiac Amyloidosis

France, United Kingdom, New Zealand, Sweden
Pfizer

Completed

This Study Will Examine the Clinical Effectiveness of Tafamidis in Patients With Mixed Phenotype Hereditary Transthyretin Amyloidosis

Hereditary Transthyretin Amyloidosis (ATTRv) Cardiomyopathy (CM), Mixed Phenotype

United States
Pfizer

Completed

Burden of Disease Study In Patients With Transthyretin Familial Amyloidosis Polyneuropathy (TTR-FAP) orTransthyretin Cardiomyopathy (TTR-CM) And Caregivers

Transthyretin Familial Amyloidosis Polyneuropathy (TTR-FAP) | Transthyretin Cardiomyopathy (TTR-CM) | Familial Amyloid Cardiomyopathy | Senile Systemic Amyloidosis (SSA)
CENTOGENE GmbH Rostock

Withdrawn

Biomarker for Transthyretin-Related Familial Amyloidotic Polyneuropathy (BioTRAP) (BioTRAP)

Transthyretin Amyloidosis | Transthyretin Amyloid Cardiopathy | Transthyretin-Related (ATTR) Familial Amyloid Polyneuropathy

India, Germany
CENTOGENE GmbH Rostock

Completed

Screening for the Transthyretin-Related Familial Amyloidotic Polyneuropathy (TTR FAP) (TRAP2-1)

Neuropathic Pain | Orthostatic Hypotension | Cardiac Failure | Gastrointestinal Disorders | Polyneuropathy, Amyloid

Spain, Austria, Hungary, North Macedonia, Poland, Serbia
Alnylam Pharmaceuticals

Completed

A Multicenter Observational Study to Evaluate the Effectiveness of Patisiran in Patients With Polyneuropathy of ATTRv Amyloidosis With a V122I or T60A Mutation

Polyneuropathy | Hereditary Transthyretin-mediated (ATTRv) Amyloidosis

United States
Eidos Therapeutics, a BridgeBio company

Withdrawn

Efficacy and Safety of Acoramidis (AG10) in Subjects With Transthyretin Amyloid Polyneurophathy (ATTRibute-PN)

Transthyretin-Related (ATTR) Familial Amyloid Polyneuropathy
Eidos Therapeutics, a BridgeBio company

Withdrawn

Efficacy and Safety of AG10 in Subjects With Transthyretin Amyloid Polyneurophathy (ATTRibute-PN)

Transthyretin-Related (ATTR) Familial Amyloid Polyneuropathy

United States
Ionis Pharmaceuticals, Inc.

Completed

Efficacy and Safety of Inotersen in Familial Amyloid Polyneuropathy

Amyloidosis | Familial Amyloid Polyneuropathy | FAP | TTR | Transthyretin

New Zealand, United States, Spain, Argentina, Brazil, France, Germany, Italy, Portugal, United Kingdom
Alnylam Pharmaceuticals

Completed

The Study of an Investigational Drug, Revusiran (ALN-TTRSC), for the Treatment of Transthyretin (TTR)-Mediated Amyloidosis in Patients Whose Disease Has Continued to Worsen Following Liver Transplant

ATTR Amyloidosis | Transthyretin (TTR)-Mediated Amyloidosis | Familial Amyloidotic Polyneuropathy (FAP) | Familial Amyloid Neuropathies

France, Germany, Portugal, United Kingdom, Sweden, Spain
Boston University
National Institute of Neurological Disorders and Stroke (NINDS); Food and Drug...

Completed

The Effect of Diflunisal on Familial Amyloidosis

Familial Amyloid Polyneuropathy | Familial Amyloidosis

United States, Italy, Japan, Sweden, United Kingdom
SOM Innovation Biotech SA
Hospital Vall d'Hebron

Completed

Study of SOM0226 in Familial Amyloid Polyneuropathy

Familial Amyloid Polyneuropathy (FAP)

Spain
Pfizer

Completed

The Effect Of Tafamidis For The Transthyretin Amyloid Polyneuropathy Patients With V30M Or Non-V30M Transthyretin

Transthyretin Familial Amyloid Polyneuropathy

Japan
Pfizer

Completed

An Extension of Study Fx-005 Evaluating Long-Term Safety And Clinical Outcomes Of Fx-1006A In Patients With Transthyretin Amyloid Polyneuropathy

Familial Amyloid Polyneuropathy | ATTR-PN

Sweden, Brazil, Germany, Argentina, France, Portugal
Pfizer

Completed

Safety and Efficacy Study of Fx-1006A in Patients With Familial Amyloidosis

Familial Amyloid Polyneuropathy

United Kingdom, Spain, United States, Argentina, Brazil, France, Germany, Portugal, Sweden
Leiden University Medical Center
The Dutch Brain Foundation (funding)

Not yet recruiting

Stimulating Amyloid Clearance in Cerebral Amyloid Angiopathy (Clear-Brain)

Cerebral Amyloid Angiopathy
Alnylam Pharmaceuticals

Recruiting

A Phase 2 Trial of ALN-APP in Patients With Cerebral Amyloid Angiopathy (cAPPricorn-1)

Cerebral Amyloid Angiopathy

Canada
Washington University School of Medicine
Alzheimer's Association; Eisai Inc.

Not yet recruiting

DIAN-TU Amyloid Removal Trial (ART) in Dominantly Inherited Alzheimer's Disease (DIAN-TU)

Dementia | Alzheimer's Disease | Alzheimer's Disease, Familial

United States, Australia, United Kingdom
John Paul II Hospital, Krakow

Recruiting

New Biomarkers and Plasma Prothrombotic Potential in Cardiac Transthyretin Amyloidosis

Transthyretin Amyloidosis

Poland
AstraZeneca

Recruiting

Transthyretin Amyloidosis Cardiomyopathy in Patients With HFpEF in Russia (TETRAMER)

Transthyretin Amyloidosis Cardiomyopathy, Heart Failure With Preserved Ejection Fraction

Russian Federation
Steen Hvitfeldt Poulsen
Novo Nordisk A/S

Recruiting

Phenotyping and Characterization of wtATTR-CM (TRACE 1)

Quality of Life | Transthyretin Amyloidosis | Transthyretin Amyloid Cardiomyopathy | Wild-Type Transthyretin-Related (ATTR)Amyloidosis

Denmark
Intellia Therapeutics
Regeneron Pharmaceuticals

Recruiting

MAGNITUDE: A Phase 3 Study of NTLA-2001 in Participants With Transthyretin Amyloidosis With Cardiomyopathy (ATTR-CM)

Transthyretin Amyloidosis (ATTR) With Cardiomyopathy

United States, Australia, United Kingdom, New Zealand, Canada
Martin Dichgans

Recruiting

High Frequency Imaging in Cerebral Amyloid Angiopathy (HIFI-CAA)

Cerebral Amyloid Angiopathy

Germany
UK Kidney Association

Recruiting

National Registry of Rare Kidney Diseases (RaDaR)

Vasculitis | AL Amyloidosis | Tuberous Sclerosis | Fabry Disease | Cystinuria | Focal Segmental Glomerulosclerosis | IgA Nephropathy | Bartter Syndrome | Pure Red Cell Aplasia | Membranous Nephropathy | Atypical Hemolytic Uremic Syndrome | Autosomal Dominant Polycystic Kidney Disease | Cystinosis | Nephronophthisis | BK Nephropathy and other conditions

United Kingdom
Rigshospitalet, Denmark

Recruiting

Polyneuropathy, Impairments and Physical Activity - The PolyImPAct Study (PolyImPAct)

Polyneuropathies | Diabetic Polyneuropathy | Chronic Inflammatory Demyelinating Polyneuropathy (CIDP) | POEMS Syndrome | Multifocal Motor Neuropathy | Charcot-Marie-Tooth | hATTR Amyloidosis | Vasculitic Neuropathy | Idiopathic Neuropathy

Denmark
Virginia Commonwealth University

Not yet recruiting

Southeastern ATTR Amyloidosis Consortium: SEATTRAC Family Registry

Amyloidosis, Hereditary

United States
Universitair Ziekenhuis Brussel

Recruiting

Prevalence of Wild-type TTR Cardiac Amyloidosis in Patients With Polyneuropathy of Unknown Cause.

Amyloidosis | Polyneuropathy

Belgium
Fondazione Policlinico Universitario Agostino Gemelli...

Recruiting

Exploring Biomarkers in Hereditary Transthyretin Amyloidosis (ELBA)

Hereditary Transthyretin Amyloidosis

Italy
Fondazione Policlinico Universitario Agostino Gemelli...

Recruiting

Prospective Evaluation of NfL as a Biomarker in ATTRv (NfLInATTRv)

Amyloidosis, Hereditary

Italy
Katarzyna Holcman

Recruiting

The Regional Scintigraphic DPD Uptake in Cardiac Transthyretin Amyloidosis. (AMYLOIDOZA)

Transthyretin Amyloidosis

Poland
University Hospital Inselspital, Berne

Recruiting

MRI-markers to Monitor Small Vessel Disease Dynamics in the Prognosis of Small Vessel Disease-associated, Cerebrovascular Events (MRI-PRO-SVD)

Cerebral Small Vessel Diseases | Intracerebral Hemorrhage | CAA - Cerebral Amyloid Angiopathy

Switzerland
University Hospital Inselspital, Berne

Recruiting

Prognosis of Cerebral Small Vessel Disease - a Prospective Cohort Study (PRO-SVD)

Cerebral Amyloid Angiopathy | Cadasil | Small Vessel Cerebrovascular Disease | CAA - Cerebral Amyloid Angiopathy

Switzerland
Amydis Inc.
National Institutes of Health (NIH); National Institute on Aging (NIA)

Recruiting

A Study of AMDX-2011P in Participants With CAA

Cerebral Amyloid Angiopathy

United States
Centre Hospitalier Universitaire de Liege
Sanofi; Takeda; University of Liege; Orchard Therapeutics; Centre Hospitalier Régional... and other collaborators

Recruiting

Baby Detect : Genomic Newborn Screening

Congenital Adrenal Hyperplasia | Hemophilia A | Hemophilia B | Mucopolysaccharidosis I | Mucopolysaccharidosis II | Cystic Fibrosis | Alpha 1-Antitrypsin Deficiency | Sickle Cell Disease | Fanconi Anemia | Chronic Granulomatous Disease | Wilson Disease | Severe Congenital Neutropenia | Ornithine Transcarbamylase... and other conditions

Belgium
Leiden University Medical Center

Recruiting

Antibiotics Against Amyloid Angiopathy (BATMAN)

Cerebral Amyloid Angiopathy

Netherlands

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