Clinical Trials on Hereditary Retinoblastoma

Total 2643 results

Shire

Completed

Open-Label C1 Esterase Inhibitor (C1INH-nf) for the Prevention of Acute Hereditary Angioedema (HAE) Attacks (CHANGE 3)

Hereditary Angioedema

United States
AO GENERIUM

Completed

Safety and Pharmacokinetics of GNR-038 in Healthy Volunteers

Hereditary Angioedema

Russian Federation
BioCryst Pharmaceuticals

Terminated

Open-label, Long-term Safety Study of Avoralstat in Subjects With Hereditary Angioedema (OPuS-4)

Hereditary Angioedema | HAE

France, Belgium
BioCryst Pharmaceuticals

Completed

Safety and Efficacy of Oral BCX4161 as a Prophylactic Treatment for HAE (OPuS-1)

Hereditary Angioedema

Germany, United Kingdom
BioCryst Pharmaceuticals

Completed

A Study of the Relative Bioavailability of a New Formulation of BCX4161 and the Effect of Food on BCX4161

Hereditary Angioedema

United Kingdom
National Cancer Institute (NCI)

Completed

Rebeccamycin Analogue in Treating Children With Solid Tumors or Non-Hodgkin's Lymphoma

Sarcoma | Lymphoma | Unspecified Childhood Solid Tumor, Protocol Specific | Brain and Central Nervous System Tumors | Neuroblastoma | Retinoblastoma

United States, Canada, Puerto Rico, Australia, Netherlands, New Zealand, Switzerland
Case Western Reserve University
National Eye Institute (NEI); Jaeb Center for Health Research

Recruiting

Impact of Donor Diabetes on DMEK Success and Endothelial Cell Loss (DEKS)

Fuchs' Endothelial Dystrophy | Corneal Endothelial Decompensation

United States
Alcon Research

Enrolling by invitation

Air Optix® Night and Day® Aqua Therapeutic Wear

Corneal Edema | Bullous Keratopathy | Corneal Dystrophy | Entropion | Corneal Erosion

United States
Cornea Associates of Texas

Completed

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

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

United States
Aerie Pharmaceuticals

Completed

A Phase 2 Study Evaluating the Safety and Efficacy of Netarsudil Ophthalmic Solution in Patients With Corneal Edema Due to Fuchs Corneal Dystrophy (Fuchs)

Corneal Edema

United States
Duke University
National Cancer Institute (NCI)

Completed

Mobile Health Case Management System for Reducing Pediatric Treatment Abandonment

Burkitt Lymphoma | Retinoblastoma | Diffuse Large B-cell Lymphoma

Tanzania
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
Astria Therapeutics, Inc.

Recruiting

A Study of STAR-0215 in Participants With Hereditary Angioedema

Hereditary Angioedema

United States, United Kingdom, Canada, Germany, Bulgaria, Czechia
ADARx Pharmaceuticals, Inc.

Recruiting

Safety, Tolerability, PK, PD of ADX-324 in Healthy Volunteers and Hereditary Angioedema Patients

Hereditary Angioedema

Australia
Pharming Technologies B.V.

Recruiting

C1 Inhibitor Registry in the Treatment of Hereditary Angioedema (HAE) Attacks

Hereditary Angioedema

Germany, Bulgaria, Croatia, Czechia, France, Hungary, Italy, North Macedonia, Poland, Slovakia, Norway, Slovenia, Sweden
BioCryst Pharmaceuticals

Active, not recruiting

Open-label Berotralstat Access to HAE Patients Previously Enrolled in Berotralstat Studies (APeX-A)

Hereditary Angioedema | HAE

France, North Macedonia, Poland, Korea, Republic of, Slovakia, South Africa, Czechia
Intellia Therapeutics

Active, not recruiting

NTLA-2002 in Adults With Hereditary Angioedema (HAE) (NTLA-2002)

Hereditary Angioedema

France, New Zealand, Germany, United Kingdom, Netherlands, Australia
CSL Behring

Active, not recruiting

Long-term Safety and Efficacy of CSL312 (Garadacimab) in the Prophylactic Treatment of Hereditary Angioedema Attacks

Hereditary Angioedema

Australia, Canada, United States, New Zealand, Germany, Hong Kong, Netherlands, Taiwan, Czechia, Israel, Hungary, Japan, Russian Federation, Spain
Ionis Pharmaceuticals, Inc.

Available

Donidalorsen Expanded Access Program for Patients With Hereditary Angioedema

Hereditary Angioedema
Istituti Clinici Scientifici Maugeri SpA

Completed

Autonomic Nervous System Profile in Hereditary Angioedema (ANS-HAE)

Hereditary Angioedema

Italy
Astria Therapeutics, Inc.

Enrolling by invitation

A Long-term Study of STAR-0215 in Participants With Hereditary Angioedema

Hereditary Angioedema

United States, Canada
Ionis Pharmaceuticals, Inc.

Completed

A Study to Assess the Clinical Efficacy of Donidalorsen (Also Known as IONIS-PKK-LRx and ISIS 721744) in Participants With Hereditary Angioedema

Hereditary Angioedema

United States, Netherlands
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
University Hospital, Strasbourg, France

Terminated

Development of New Prenatal Diagnostic Tests From Maternal Blood

Hereditary Diseases

France
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
Oslo University Hospital

Unknown

DSAEK- Postoperative Positioning and Transplant Dislocation

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

Norway
National Cancer Institute (NCI)

Completed

Cixutumumab in Treating Patients With Relapsed or Refractory Solid Tumors

Recurrent Neuroblastoma | Recurrent Osteosarcoma | Recurrent Childhood Rhabdomyosarcoma | Recurrent Adult Soft Tissue Sarcoma | Recurrent Wilms Tumor and Other Childhood Kidney Tumors | Previously Treated Childhood Rhabdomyosarcoma | Recurrent Childhood Soft Tissue Sarcoma | Adult Rhabdomyosarcoma | Neuroectodermal Tumor and other conditions

United States, Canada, Australia
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
National Cancer Institute (NCI)

Recruiting

Comprehensive Omics Analysis of Pediatric Solid Tumors and Establishment of a Repository for Related Biological Studies

Sarcoma | Neuroblastoma | Retinoblastoma | Renal Cancer | Endocrine Tumors

United States
Masonic Cancer Center, University of Minnesota

Terminated

MT2004-30: Tomotherapy for Solid Tumors

Sarcoma | Kidney Cancer | Brain and Central Nervous System Tumors | Retinoblastoma | Liver Cancer

United States
University Hospital, Antwerp
Belgium Health Care Knowledge Centre

Recruiting

The Belgian Endothelial Surgical Transplant of the Cornea (BESTCornea)

Corneal Edema | Fuchs' Endothelial Dystrophy | Bullous Keratopathy | Pseudophakic Bullous Keratopathy | Corneal Endothelial Disorder

Belgium
Massachusetts General Hospital
Harvard Medical School (HMS and HSDM)

Recruiting

A Study to Test the Diagnostic Potential of Brillouin Microscopy for Corneal Ectasia

Keratoconus | Ectasia | Fuchs' Endothelial Dystrophy | Crosslinking

United States

Clinical Trials on Hereditary Retinoblastoma

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