Clinical Trials on Amyloidosis, Hereditary

Total 3125 results

Shire

Completed

Observational Safety Study for KALBITOR (Ecallantide) in the Treatment of Acute Attacks of Hereditary Angioedema

Hereditary Angioedema (HAE)

United States
Shire

Completed

Phase 1 Study to Assess the Safety, Tolerability, and Pharmacokinetics of Recombinant Human C1 Esterase Inhibitor in Healthy Adult Subjects

Hereditary Angioedema (HAE)

United States
Shire

Completed

Study of C1 Inhibitor (Human) for the Prevention of Angioedema Attacks and Treatment of Breakthrough Attacks in Japanese Subjects With Hereditary Angioedema (HAE)

Hereditary Angioedema (HAE)

Japan
Shire

Completed

EDEMA2: Evaluation of DX-88's Effect in Mitigating Angioedema

Hereditary Angioedema (HAE)
Shire
Dyax Corp.

Completed

A Single Increasing Dose Study to Assess Safety and Tolerability of DX-2930 in Healthy Subjects

Hereditary Angioedema (HAE)

United States
National Cancer Institute (NCI)

Completed

Phase I-II Multiple-Dose Safety and Efficacy Study of a Selective Inhibitor of Cyclooxygenase - 2 (SC-58635) in Hereditary Non-Polyposis Colorectal Cancer (HNPCC) Patients and Carriers

Colorectal Neoplasm | Hereditary Nonpolyposis

United States
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
Vaderis Therapeutics AG

Recruiting

Assess Safety and Efficacy of VAD044 in HHT Patients

Hereditary Hemorrhagic Telangiectasia (HHT)

United States, Spain, France, Belgium, Netherlands, Italy
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
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
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
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
University Hospital, Bordeaux

Completed

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

Wild-type Amyloid Cardiopathy

France
Peking University People's Hospital

Recruiting

Daratumumab, Bortezomib, Cyclophosphamide, and Dexamethasone Therapy for Patients With MIDD

Monoclonal Gammopathy of Renal Significance

China
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
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
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
Medical University of Vienna

Completed

Assessment of Corneal Graft Attachment in Patients With Fuchs Endothelial Corneal Dystrophy Following DMEK Using Ultra-high Resolution OCT

Fuchs' Endothelial Dystrophy

Austria
Centre Hospitalier Régional Metz-Thionville

Completed

Predictive Factors of Good Results After Primary Descemet's Membrane Endothelial Keratoplasty (DMEK) (GOODRMEK)

Endothelial Corneal Dystrophy
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
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
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
University Hospital, Grenoble

Recruiting

Status of Dental Care Practices in Patients With Hereditary Angioedema (PRADA)

Angio Edema Hereditary

France
Takeda

Recruiting

A Survey of Icatibant in Pediatric Participants With Hereditary Angioedema

Hereditary Angioedema (HAE)

Japan

Clinical Trials on Amyloidosis, Hereditary

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