Clinical Trials on Hereditary Autoinflammatory Diseases

Total 379801 results

Astria Therapeutics, Inc.

Enrolling by invitation

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

Hereditary Angioedema

United States, Canada
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
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
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
Nanoscope Therapeutics Inc.

Enrolling by invitation

Non-Interventional Long Term Follow-up Study of Participants Previously Enrolled in the RESTORE Study (REMAIN)

Eye Diseases | Retinal Degeneration | Retinal Diseases | Retinitis Pigmentosa | Retinitis | Eye Diseases, Hereditary | Retinal Dystrophies

United States, Puerto Rico
Amgen

Recruiting

A Study of Apremilast in Children With Oral Ulcers Associated With Behçet's Disease or Juvenile Psoriatic Arthritis

Juvenile Psoriatic Arthritis | Behçet's Disease

Spain, Turkey, Israel, Greece
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
Radboud University Medical Center
Jacques and Gloria Gossweiler Foundation

Completed

Improving Gait Adaptability in Hereditary Spastic Paraplegia (Move-HSP)

Hereditary Spastic Paraplegia | Strumpell Disease

Netherlands
BioCryst Pharmaceuticals

Completed

A Long Term Safety Study of BCX7353 in Hereditary Angioedema (APeX-S)

Hereditary Angioedema | Prophylaxis | HAE

Austria, Denmark, France, Germany, Hungary, Israel, Italy, North Macedonia, Poland, Switzerland, United Kingdom, United States, Australia, Hong Kong, Korea, Republic of, New Zealand, Serbia, Slovakia, South Africa, Spain
Pharming Technologies B.V.

Completed

A Study of the Safety and Immunogenicity of Repeated rhC1INH Administration (OPERA)

Hereditary Angioedema | Genetic Disorders

Netherlands, Romania
Pharming Technologies B.V.

Completed

Recombinant Human C1 Inhibitor for the Treatment of Acute Attacks in Patients With Hereditary Angioedema

Genetic Disorders

Netherlands
Pharming Technologies B.V.

Completed

Recombinant Human C1 Inhibitor for the Treatment of Acute Attacks in Patients With Hereditary Angioedema

Hereditary Angioedema | Angioneurotic Edema

Netherlands
Pharming Technologies B.V.

Completed

Recombinant Human C1 Inhibitor for the Treatment of Acute Attacks in Patients With Hereditary Angioedema

Hereditary Angioedema | Genetic Disorders | Angioneurotic Edema

Netherlands, Romania
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
BioCryst Pharmaceuticals

Approved for marketing

Oral Berotralstat Expanded Access Program

Hereditary Angioedema | Prophylaxis | HAE
University Hospital, Grenoble

Terminated

Determination of Specific Biomarkers of Acute Attack of Angioedema Within Pediatric Population (BRADYKID)

Healthy Volunteers | Hereditary Angioedema

France
Pharming Technologies B.V.

Completed

Recombinant Human C1 Inhibitor for the Treatment of Acute Attacks in Patients With Hereditary Angioedema

Genetic Disorders

Netherlands
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
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
London North West Healthcare NHS Trust

Completed

Magnification Narrow Band Imaging Colonoscopy for Hereditary Non-Polyposis Colorectal Cancer Surveillance

Colorectal Neoplasms, Hereditary Nonpolyposis

United Kingdom
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
University of California, San Francisco
National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK); University... and other collaborators

Completed

Clinical Diagnosis of Acute Porphyria

Acute Intermittent Porphyria (AIP) | Hereditary Coproporphyria (HCP) | Variegate Porphyria (VP)

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
Genomic Prediction Inc.

Active, not recruiting

Embryo Health Study (EHS)

Infertility | Hereditary Diseases

United States
Istituto Ortopedico Rizzoli

Completed

Height, Ulnar Length and Forearm Function in Multiple Hereditary Exostoses

Exostoses, Multiple Hereditary

Italy
University Hospital Tuebingen

Completed

Physiotherapy in Hereditary Spastic Paraplegia

Hereditary Spastic Paraplegia

Germany
State Scientific Centre of Coloproctology, Russian...
The Loginov MCSC MHD; Pirogov National Medical and Surgical Center; Moscow City... and other collaborators

Recruiting

Prospective Multicenter Registry Study to Assess the Frequency of Lynch Syndrome Among Patients With Colorectal Cancer (MSIRus22)

Colorectal Cancer | Lynch Syndrome | Hereditary Colorectal Cancer | MSI

Russian Federation
University of British Columbia
Children's & Women's Health Centre of British Columbia; Vancouver Foundation; Circle of Care

Terminated

Establishing the Genetic Profile of Multiple Hereditary Exostoses (HME) in Families of BC

Exostoses, Multiple Hereditary

Canada
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
St George's, University of London

Terminated

Intrauterine Levonorgestrel and Observation or Observation Alone in Preventing Atypical Endometrial Hyperplasia and Endometrial Cancer in Women With Hereditary Non-Polyposis Colorectal Cancer or Lynch Syndrome

Endometrial Cancer | Hereditary Non-polyposis Colon Cancer (hmsh2, hmlh1, hpms1, hpms2)

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
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

Clinical Trials on Hereditary Autoinflammatory Diseases

Sponsors and Collaborators

Medical Conditions

Drug Interventions

CROs by country

CROs in Dominican Republic

Conditions

Rare Diseases

Drug Interventions

Dietary Supplements

Sponsor/Collaborators

Locations