Clinical Trials on Elliptocytosis, Hereditary

Total 2620 results

KalVista Pharmaceuticals, Ltd.

Completed

Phase 1 Crossover Study in Healthy Subjects to Evaluate the PK Profile of KVD824 Following Single and Multiple Doses of Modified Release (MR) Formulations

Hereditary Angioedema

United Kingdom
Shire
Halozyme Therapeutics

Completed

A Study to Evaluate the Safety and Pharmacology of Subcutaneous Administration of CINRYZE With Recombinant Human Hyaluronidase

Hereditary Angioedema

United States
Shire

Completed

A Study to Evaluate the Safety, Pharmacokinetics, and Pharmacodynamics of Subcutaneous CINRYZE Administration

Hereditary Angioedema

United States
Shire

Completed

Subcutaneous Treatment With Icatibant for Acute Attacks of Hereditary Angioedema

Angioedema

United States
Shire

Completed

Open-Label C1 Esterase Inhibitor (C1INH-nf) for the Treatment of Acute Hereditary Angioedema (HAE) Attacks (CHANGE 2)

Hereditary Angioedema

United States
Shire

Completed

Pharmacokinetics of C1 Esterase Inhibitor in Hereditary Angioedema Subjects

Hereditary Angioedema

United States
KalVista Pharmaceuticals, Ltd.

Completed

A Single Dose Safety, Tolerability, Pharmacokinetic and Food Effect Study of KVD900 in Healthy Volunteers

Hereditary Angioedema

United Kingdom
BioCryst Pharmaceuticals

Completed

A Relative Bioavailability Study of Two Formulations of BCX7353

Hereditary Angioedema

United Kingdom
Pharming Technologies B.V.

Completed

A Phase 2 HAE Prophylaxis Study With Recombinant Human C1 Inhibitor

Hereditary Angioedema

United States, Canada, Czechia, Italy, Macedonia, The Former Yugoslav Republic of, Romania, Serbia
BioCryst Pharmaceuticals

Completed

An Open-label Drug-Drug Interaction Study to Evaluate the Effect of BCX7353 on Cytochrome P450 Enzyme Activity Using Probe Substrates

Hereditary Angioedema

United Kingdom
Massachusetts General Hospital
Shire Human Genetic Therapies, Inc.

Completed

Efficacy, Safety and Tolerability of Icatibant for the Treatment of HAE (IHA)

Hereditary Angioedema

United States
BioCryst Pharmaceuticals

Completed

First-in-Human Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of BCX7353 in Healthy Western and Japanese Volunteers

Hereditary Angioedema

United Kingdom
Pharming Technologies B.V.

Completed

Efficacy, Safety and Immunogenicity Study of Recombinant Human C1 Inhibitor for the Treatment of Acute HAE Attacks

Hereditary Angioedema

United States, Bulgaria, Canada, Hungary, Israel, Italy, Macedonia, The Former Yugoslav Republic of, Poland, Romania, Serbia, South Africa
CSL Behring

Completed

C1 Esterase Inhibitor in Hereditary Angioedema (HAE)(Extension Study)

Hereditary Angioedema

United States, Canada
CSL Behring

Completed

Human C1 Esterase Inhibitor (C1-INH) in Subjects With Acute Abdominal or Facial Hereditary Angioedema (HAE) Attacks

Hereditary Angioedema

Bulgaria, United States, Poland, Spain, Hungary, United Kingdom, Russian Federation, Macedonia, The Former Yugoslav Republic of, Argentina, Australia, Canada, Czech Republic, Israel, Romania, Sweden
BioCryst Pharmaceuticals

Completed

Study to Determine How BCX4161 is Metabolized and Eliminated by the Body

Hereditary Angioedema

United Kingdom
Radboud University Medical Center
ZonMw: The Netherlands Organisation for Health Research and Development

Unknown

Implementation of a New Strategy to Identify HNPCC Patients

Colorectal Neoplasms | Hereditary Nonpolyposis Colorectal Cancer

Netherlands
KalVista Pharmaceuticals, Ltd.

Recruiting

PK Subtrial in Adolescent Patients With HAE Type I or II Participating in the KVD900-302 Trial

Hereditary Angioedema

United States, Bulgaria, France, Greece, Israel, Netherlands, Spain, United Kingdom, Australia, Germany, South Africa, Slovakia, Austria, New Zealand, Romania, Canada, Japan
KalVista Pharmaceuticals, Ltd.

Recruiting

An Open-label Extension Trial to Evaluate the Long-term Safety of KVD900 for On-Demand Treatment of Angioedema Attacks in Adolescent and Adult Patients With Hereditary Angioedema (HAE)

Hereditary Angioedema

United States, Bulgaria, France, Greece, Hungary, Israel, Italy, Netherlands, New Zealand, North Macedonia, Poland, Spain, United Kingdom, Australia, Germany, South Africa, Austria, Portugal, Slovakia, Romania, Canada, Saudi Arabia, Japa...
Pharming Technologies B.V.
US Hereditary Angioedema Association

Completed

Patient Registry to Evaluate the Real-world Safety of Ruconest®

Hereditary Angioedema

United States
Imperial College London

Completed

Diet and Hereditary Haemorrhagic Telangiectasia

Hereditary Haemorrhagic Telangiectasia (HHT)

United Kingdom
BioCryst Pharmaceuticals

Active, not recruiting

Berotralstat Treatment in Children With Hereditary Angioedema (APeX-P)

Hereditary Angioedema | Pediatric

Spain, Austria, Canada, France, Germany, Israel, Italy, Poland, Romania, United Kingdom
Ionis Pharmaceuticals, Inc.

Active, not recruiting

An Extension Study of Donidalorsen (IONIS-PKK-LRx) in Participants With Hereditary Angioedema

Hereditary Angioedema

Netherlands, United States
KalVista Pharmaceuticals, Ltd.

Completed

A Phase III, Crossover Trial Evaluating the Efficacy and Safety of KVD900 for On-Demand Treatment of Angioedema Attacks in Adolescent and Adult Patients With Hereditary Angioedema (HAE)

Hereditary Angioedema

United States, Australia, Bulgaria, Canada, France, Germany, Greece, Hungary, Israel, Italy, Japan, Netherlands, New Zealand, North Macedonia, Poland, Portugal, Puerto Rico, Romania, Slovakia, Spain, United Kingdom
Takeda

Completed

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

Angioedemas, Hereditary

Argentina
BioCryst Pharmaceuticals

Completed

Efficacy and Safety Study of BCX7353 as an Oral Treatment for the Prevention of Attacks in HAE (APeX-2)

Hereditary Angioedema | HAE

United States, Austria, Canada, Czechia, France, Germany, Hungary, North Macedonia, Romania, Spain, United Kingdom
Shire

Completed

A Study to Evaluate the Safety and Effect of Escalating Doses of CINRYZE

Hereditary Angioedema

United States
Shire
Jerini AG

Completed

EASSI - Evaluation of the Safety of Self-Administration With Icatibant (EASSI)

Hereditary Angioedema

Spain, Argentina, Austria, Denmark, France, Germany, Israel, Italy, Switzerland, United Kingdom
University Hospital Tuebingen

Completed

Studying Cognition in SPG4

Cognitive Impairment | Hereditary Spastic Paraplegia

Germany
BioCryst Pharmaceuticals

Completed

A Drug-Drug Interaction Study to Evaluate Drug Transporter Interactions

Hereditary Angioedema

United Kingdom
Universitaire Ziekenhuizen KU Leuven

Completed

I-Scan For Colon Polyp Detection In HNPCC

Lynch Syndrome | Hereditary Non-polyposis Colon Carcinoma | HNPCC

Belgium
Assistance Publique - Hôpitaux de Paris

Completed

A Call Center During HAE Attacks (SOS HAE) (SOS AOH)

Hereditary Angioedema

France
National Health Research Institutes, Taiwan
Chang Gung Memorial Hospital

Unknown

Hereditary Nonpolyposis Colorectal Cancer in Taiwan-Related Genetic Study and Clinical Applications

Hereditary Nonpolyposis Colorectal Cancer

Taiwan
BioCryst Pharmaceuticals

Completed

12-Week Safety and Efficacy Study of BCX4161 as an Oral Prophylaxis Against HAE Attacks (OPuS-2)

Hereditary Angioedema | HAE

France, United Kingdom, Germany, United States, Belgium, Hungary, Italy, Canada
Johann Wolfgang Goethe University Hospital
CSL Behring; University of Milan; PharmaPart; Clinical trial center Rhine-Main; ZKI... and other collaborators

Completed

Berinert P Study of Subcutaneous Versus Intravenous Administration (PASSION)

Hereditary Angioedema

Germany
Oslo University Hospital

Unknown

DSAEK- Postoperative Positioning and Transplant Dislocation

Corneal Dystrophies, Hereditary | Fuchs' Endothelial Dystrophy | Corneal Transplantation | Descemet Stripping Automated Endothelial Keratoplasty

Norway
University of Pennsylvania
AstraZeneca; Ionis Pharmaceuticals, Inc.

Active, not recruiting

Early Detection of Neuropathy in ATTRv (EDONA)

Hereditary Amyloidosis, Transthyretin-Related

United States
Cornea Associates of Texas

Active, not recruiting

Safety & Effectiveness of the PXL-Platinum 330 System for CXL Using Riboflavin Solution

Keratoconus | Corneal Ectasia | Pellucid Marginal Corneal Degeneration | Corneal Degeneration

United States
Aalborg University Hospital
Aarhus University Hospital; ARCEDI Biotech

Completed

Cell-based Non-invasive Prenatal Testing as an Alternative to Invasive Chorionic Villus Sampling

Hereditary Diseases

Denmark
Centre Hospitalier Universitaire Dijon

Unknown

Genomic Screening for Hereditary Erythrocytosis and Related Diseases (GENRED)

Hereditary Erythrocytosis/Idiopathic Erythrocytosis

France
Michigan State University

Recruiting

Family Health Histories: Creating a Culturally Tailored Tool to Reduce Health Disparities in the Black Community (FHH)

Hereditary Diseases

United States
ProQR Therapeutics

Withdrawn

Study With QR-504a to Evaluate Safety, Tolerability & Corneal Endothelium Molecular Biomarker(s) in Subjects With FECD3 (Fuchs Focus)

Corneal Diseases | Fuchs Endothelial Corneal Dystrophy | FECD3 | LRS | Descemet's Membrane Disorder

United Kingdom
Singapore Eye Research Institute

Unknown

Safety and Efficacy of Tissue Engineered Endothelial Keratoplasty (TE-EK)

Fuchs' Endothelial Dystrophy | Bullous Keratopathy | Mild to Moderate Corneal Endothelial Decompensation | Post-surgical Corneal Decompensation (Irreversible)

Singapore
The Cleveland Clinic
National Eye Institute (NEI)

Completed

PIONEER: Intraoperative and Perioperative OCT Study (PIONEER)

Retinal Detachment | Epiretinal Membrane | Fuchs Endothelial Dystrophy | Macular Hole

United States
Murdoch Childrens Research Institute
Melbourne Health; Austin Health; Royal Brisbane and Women's Hospital; The University... and other collaborators

Completed

Mi-iron - Moderately Increased Iron - is Reducing Iron Overload Necessary? (Mi-iron)

Hereditary Haemochromatosis

Australia
Children's Hospital of Fudan University

Recruiting

The China Neonatal Genomes Project (CNGP)

Genetic Predisposition to Disease | Newborn | Defect, Congenital | Hereditary Disease

China
University of Texas Southwestern Medical Center

Terminated

The Dallas Hereditary Spherocytosis Cohort Study

Hereditary Spherocytosis
Massachusetts Eye and Ear Infirmary

Recruiting

Assessment of Corneal Endothelial Function Following Hypoxic Stress

Fuchs' Endothelial Corneal Dystrophy

United States
Dr. Rebecca Schule
German Federal Ministry of Education and Research; German Center for Neurodegenerative...

Recruiting

Phenotypes, Biomarkers and Pathophysiology in Hereditary Spastic Paraplegias and Related Disorders (HSP-PBP)

Hereditary Spastic Paraplegia

Austria, Germany, Italy
Wageningen University
Dutch Cancer Society; Wereld Kanker Onderzoek Fonds; Funding: Biobanking and...

Recruiting

The GEOLynch Cohort Study (GEOLynch)

Neoplasms | Colorectal Neoplasms | Lynch Syndrome | Hereditary Nonpolyposis Colorectal Cancer

Netherlands

Clinical Trials on Elliptocytosis, Hereditary

Sponsors and Collaborators

Medical Conditions

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CROs by country

CROs in Zambia

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Rare Diseases

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