Clinical Trials on Hereditary Amyloidosis

Total 3115 results

Mathew S. Maurer
National Heart, Lung, and Blood Institute (NHLBI); Yale University; Boston Medical... and other collaborators

Recruiting

Screening for Cardiac Amyloidosis With Nuclear Imaging for Minority Populations (SCAN-MP)

Amyloid Cardiomyopathy, Transthyretin-Related

United States
Pfizer

Active, not recruiting

A Study to Learn About the Study Medicine Called Tafamidis 61mg in People Diagnosed With Transthyretin Amyloid Cardiomyopathy (ATTR-CM)

Transthyretin Amyloid Cardiomyopathy

Korea, Republic of
Pfizer

Completed

A Study to Assess the Safety and Efficacy Of Tafamidis In Chinese Participants With Transthyretin Amyloid Cardiomyopathy (ATTR-CM)

Transthyretin Amyloid Cardiomyopathy

China
Byron Lam
National Eye Institute (NEI)

Active, not recruiting

Safety Study of an Adeno-associated Virus Vector for Gene Therapy of Leber's Hereditary Optic Neuropathy (LHON)

Leber's Hereditary Optic Neuropathy

United States
Santhera Pharmaceuticals

Completed

RHODOS Follow-up Single-visit Study (RHODOS-OFU)

Leber's Hereditary Optic Neuropathy

Canada, Germany, United Kingdom
National Eye Institute (NEI)

Completed

Studies in Families With Corneal Dystrophy or Other Inherited Corneal Diseases

Corneal Dystrophies, Hereditary | Corneal Disease

United States, China, Italy
University Hospital, Toulouse

Terminated

Transthyretin Cardiac Amyloidosis in Patients With Idiopathic Carpal Tunnel Syndrome Referred for Release Surgery (PRATHYC)

Carpal Tunnel Syndrome | Transthyretin Amyloidosis

France
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
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
Aarhus University Hospital Skejby
Regional Hospital Holstebro

Completed

Prevalence of Wild Type Transthyretin Cardiac Amyloidosis in Patients Operated for Idiopathic Carpal Tunnel Syndrome (CACTuS)

Carpal Tunnel Syndrome | Amyloidosis Cardiac

Denmark
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
University of Leipzig

Active, not recruiting

Native T1 CMR Imaging for Diagnosis of Cardiac Amyloidosis (CMR for CA)

Heart Failure NYHA Class II | Heart Failure NYHA Class III | Heart Failure With Preserved Ejection Fraction | Hypertrophy, Left Ventricular | Heart Failure NYHA Class IV | Cardiac Amyloidosis | Heart Failure With Mid Range Ejection Fraction

Germany
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
Boston Children's Hospital
CureAP4 Foundation

Recruiting

Registry and Natural History Study for Early Onset Hereditary Spastic Paraplegia (HSP)

Hereditary Spastic Paraplegia | SPG50 | SPG47 | SPG51 | SPG52 | AP4-related Hereditary Spastic Paraplegia | Early Onset Hereditary Spastic Paraplegia | SPG4 | SPG3A | SPG15 | SPG11

United States
University of Rochester
Livionex Inc

Completed

A Pilot Study to Assess the Preliminary Efficacy of EDTA Eye Drops on Band Keratopathy

Corneal Dystrophy, Band-Shaped

United States
Centre Hospitalier Universitaire de Besancon

Completed

Long Term Cornea Graft Survival Study (BELIEvE)

Keratoconus | Fuchs' Endothelial Dystrophy

France
University Hospital, Rouen

Completed

Comparison of Two Techniques in Achieving Corneal Graft

Fuch's Endothelial Dystrophy

France
Avellino Labs USA, Inc.

Unknown

Study of the Prevalence of TGFBI Corneal Dystrophies

Corneal Dystrophy
Maastricht University Medical Center

Completed

Fuchs' Torsional Phaco Study

Fuchs' Endothelial Dystrophy | Phacoemulsification

Netherlands
Barbara Ann Karmanos Cancer Institute

Completed

COVID-19 VAX Booster Dosing in Patients With Hematologic Malignancies

Multiple Myeloma | AL Amyloidosis | Chronic Lymphocytic Leukemia

United States
Eye Hospital Pristina Kosovo

Enrolling by invitation

Fresh Corneal Lenticule Implantation in Macular Corneal Distrophy With Relex Smile Surgery

Macular Corneal Dystrophy

Kosovo
Cornea Research Foundation of America

Completed

Trial of Netarsudil for Prevention of Corticosteroid-induced Intraocular Pressure Elevation

Fuchs' Endothelial Dystrophy | Bullous Keratopathy

United States
Glostrup University Hospital, Copenhagen

Unknown

Air Versus SF6 for Descemet's Membrane Endothelial Keratoplasty (DMEK)

Fuchs' Endothelial Dystrophy | Bullous Keratopathy

Denmark
University Hospital, Bordeaux

Completed

Neuropathy in Patients Monitored for Wild-type TTR Cardiac Amyloidosis (Non-mutated) (N-SAC)

Wild-type Amyloid Cardiopathy

France
Takeda

Not yet recruiting

A Study of Lanadelumab (Takhzyro) and Icatibant (Firazyr®) in Persons With HAE in China

Hereditary Angioedema (HAE)
CSL Behring

Recruiting

CSL312_3003 Safety and Pharmacokinetic Study in Subjects 2 to 11 Years of Age With Hereditary Angioedema

Hereditary Angioedema (HAE)

United States, Israel
Takeda

Recruiting

A Study of Lanadelumab in Teenagers and Adults With Hereditary Angioedema (HAE)

Hereditary Angioedema (HAE)

United Kingdom
Takeda

Recruiting

A Survey of Lanadelumab in Participants With Hereditary Angioedema

Hereditary Angioedema (HAE)

Japan
Takeda

Recruiting

A Study With Lanadelumab in Persons With Hereditary Angioedema (HAE) in Poland (CHOPIN)

Hereditary Angioedema (HAE)

Poland
Shire
Takeda Development Center Americas, Inc.

Recruiting

Firazyr® Patient Registry (Icatibant Outcome Survey - IOS)

Hereditary Angioedema (HAE)

France, Austria, Australia, Spain, Czechia, Italy, United Kingdom, Germany, Israel, Ireland, Brazil, Denmark, Greece, Sweden
Shire
Takeda Development Center Americas, Inc.

Completed

A Study of Lanadelumab in Persons With Hereditary Angioedema (HAE) Type I or II in North America (EMPOWER)

Hereditary Angioedema (HAE)

Canada, United States, Puerto Rico
Takeda

Completed

A Study in Teenagers and Adults With Hereditary Angioedema (HAE) Type I or Type II Who Use Lanadelumab as Long-Term Prophylaxis (INTEGRATED)

Hereditary Angioedema (HAE)

France, Germany, Austria, Greece
Shire

Completed

Efficacy Study of DX-88 (Ecallantide) to Treat Acute Attacks of Hereditary Angioedema (HAE)

Hereditary Angioedema (HAE)

United States, Jordan, Canada
Shire
Dyax Corp.

Completed

Efficacy and Safety Study of DX-2930 to Prevent Acute Angioedema Attacks in Patients With Type I and Type II HAE

Hereditary Angioedema (HAE)

United States, Canada, Germany, Italy, Jordan, Puerto Rico, United Kingdom
Shire

Withdrawn

Study to Evaluate Ecallantide in Paediatric Patients With Acute Attacks of Hereditary Angioedema

Hereditary Angioedema (HAE)
BioCryst Pharmaceuticals

Completed

Study of BCX7353 as a Treatment for Attacks of Hereditary Angioedema

Hereditary Angioedema (HAE)

Austria, Denmark, France, Germany, Hungary, Israel, Italy, North Macedonia, Poland, Romania, Switzerland, United Kingdom
Shire

Completed

A European Post-Authorisation Observational Study Of Patients With Hereditary Angioedema

Hereditary Angioedema (HAE)

Germany, Spain, United Kingdom, France, Italy, Belgium
CSL Behring
Chiltern International Inc.

Completed

Patient Registry Study of Berinert® in Normal Clinical Practice

Includes: Hereditary Angioedema

United States, Denmark, Germany, Switzerland
Oregon Health and Science University
Ionis Pharmaceuticals, Inc.

Recruiting

carDIo-ttranSSfOrm nucLEar Imaging Study (DISSOLvE)

Amyloidosis | Transthyretin Amyloid Cardiomyopathy

United States
University of Trieste
Pfizer

Unknown

Unmasking the Prevalence of AC in an Unselected Echocardiographic Population (AC-TIVE)

Amyloid Cardiomyopathy

Italy
Shanghai Vitalgen BioPharma Co., Ltd.

Not yet recruiting

Safety and Tolerability of VGR-R01 for Patients With Bietti Crystalline Dystrophy

Bietti Crystalline Dystrophy

China
Beijing Tongren Hospital

Recruiting

Safety and Tolerability of VGR-R01 in Patients With Bietti Crystalline Dystrophy

Bietti Crystalline Dystrophy

China
Unity Health Toronto
Sunnybrook Health Sciences Centre; University of Pittsburgh; Duke University; Barrow... and other collaborators

Active, not recruiting

Doxycycline for Hereditary Hemorrhagic Telangiectasia (HHT)

Hereditary Hemorrhagic Telangiectasia (HHT)

Canada
Imperial College London

Completed

Frequency of Common Medical Conditions in People With and Without HHT

Hereditary Hemorrhagic Telangiectasia (HHT)

United Kingdom
University Hospital, Essen

Completed

Symptoms and Treatment Results in Hereditary Hemorrhagic Telangiectasia

Hereditary Haemorrhagic Telangiectasia (HHT)

Germany
Octapharma

Recruiting

Study of IV Human Plasma-derived C1 Esterase Inhibitor Concentrate in Patients With Congenital C1-INH Deficiency for Treatment and Pre-procedure Preventing of Acute Hereditary Angioedema Attacks

Acute Hereditary Angio Edema

Romania
Hospital Universitari Vall d'Hebron Research Institute
Hospital Universitario La Paz

Not yet recruiting

Identification and Characterization of Genetic Variants in Hereditary Angioedema (GENOMAEH_01)

Hereditary Angioedema With C1 Esterase Inhibitor Deficiency

Spain
First Affiliated Hospital of Fujian Medical University

Recruiting

PCSK9 Inhibitor Treatment for Patients With SPG5

Hereditary Spastic Paraplegia Type 5

China
CSL Behring

Completed

Postmarketing Immunogenicity Study in HAE Subjects Treated With Berinert

Hereditary Angioedema Types I and II

Bulgaria, Hungary, Poland, Romania
Santhera Pharmaceuticals

Completed

Study to Assess Efficacy,Safety and Tolerability of Idebenone in the Treatment of Leber's Hereditary Optic Neuropathy (RHODOS)

Leber's Hereditary Optic Neuropathy

Canada, Germany, United Kingdom

Clinical Trials on Hereditary Amyloidosis

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