Clinical Trials on Hereditary Complement Deficiency Diseases

Total 11037 results

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
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
CSL Behring

Completed

Postmarketing Immunogenicity Study in HAE Subjects Treated With Berinert

Hereditary Angioedema Types I and II

Bulgaria, Hungary, Poland, Romania
CSL Behring

Completed

A Study to Evaluate the Safety and Pharmacokinetics of Two Formulations of C1-esterase Inhibitor

Hereditary Angioedema Types I and II

Germany
CSL Behring

Completed

A Study to Evaluate the Clinical Efficacy and Safety of Subcutaneously Administered C1-esterase Inhibitor in the Prevention of Hereditary Angioedema

Hereditary Angioedema Types I and II

United States, Spain, Australia, Canada, Czechia, Hungary, Israel, Italy, Romania, United Kingdom
University Hospital, Catania

Unknown

Hereditary AngioEdema, Neurobiology and Psychopathology (HAENP)

Depression | Anxiety | Hereditary AngioEdema

Italy
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
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
Penn State University

Withdrawn

C1-INH Compared to Placebo at the Time of Prodromal Symptoms for Hereditary Angioedema (HAE) Exacerbation

Hereditary Angioedema
KalVista Pharmaceuticals, Ltd.

Terminated

A Trial to Evaluate the Efficacy and Safety of Different Doses of KVD824 for Prophylactic Treatment of HAE Type I or II (KVD824-201)

Angioedema, Hereditary, Types I and II

United States, Czechia, Germany, Hungary, Italy, North Macedonia, United Kingdom, Australia, Bulgaria, Canada, France, New Zealand, Puerto Rico
University Hospital, Grenoble

Completed

Assessment of the State of Health, Quality of Life and Expectations of Patients With Hereditary Angioedema (BESQAOH)

Hereditary Angioedema

France
Takeda
Takeda Development Center Americas, Inc.

Completed

A Study of Lanadelumab in Teenagers and Adults to Prevent Acute Attacks of Non-histaminergic Angioedema With Normal C1-Inhibitor (C1-INH)

Angioedema

United States, Spain, Netherlands, Hungary, Italy, Canada, Germany, France, Japan, Poland
Catalyst Biosciences

Terminated

Screening Study to Identify Patients With Complement Factor I Deficiencies

Previous Diagnosis With a Complement-mediated Disease and/or With Clinical Manifestations Reasonably Associated With Complement Factor I Deficiency

United States
Shire
Takeda Development Center Americas, Inc.

Completed

A Study of Long-Term Safety and Efficacy of Lanadelumab for Prevention of Acute Attacks of Non-histaminergic Angioedema With Normal C1-Inhibitor

Angioedema

United States, France, Netherlands, Spain, Hungary, Italy, Canada, Poland, Japan, Germany
CENTOGENE GmbH Rostock

Completed

Epidemiological Analysis for Hereditary Angioedema Disease (EHA)

Abdominal Pain | Functional Abdominal Pain

United Kingdom, Germany, Turkey, Poland, Italy, Japan
Stanley Jordan, MD
CSL Behring

Completed

Safety & Tolerability of Berinert® (C1 Inhibitor) Therapy to Prevent Rejection

Kidney Transplantation

United States
Pharming Technologies B.V.
University of Wisconsin, Madison

Withdrawn

Recombinant Human C1 Inhibitor for the Treatment of Early Antibody-Mediated Rejection in Renal Transplantation

Kidney Transplantation | Graft Rejection

United States
Applied Therapeutics, Inc.

Active, not recruiting

Pharmacodynamic EffIcacy and Clinical Benefit of AT 007 in Patients With Sorbitol Dehydrogenase (SORD) Deficiency (INSPIRE)

Hereditary Neuropathy Caused by SORD Deficiency

United States, United Kingdom, Czechia, Italy
Foundation For Rare Disease Research
Institute for Asthma & Allergy; Virant Diagnostics, Inc.; MedBio Reference Laboratories...

Not yet recruiting

Angioedema Biomarker Research Study

Angioedema | Urticaria | C1 Inhibitor Deficiency | Angioedemas, Hereditary | Mastocytosis | Indolent Systemic Mastocytosis | Systemic Mastocytoses | ACE Inhibitor-Induced Angioedema
T. van der Poll
ZonMw: The Netherlands Organisation for Health Research and Development; Prothya...

Terminated

C1-inhibitor in Allergic ASThma Patients (CAST)

Asthma

Netherlands
First Affiliated Hospital, Sun Yat-Sen University

Completed

The Predictive Value of Complement C3 in Patients With Advanced Gastric Cancer

Gastric Cancer | Surgery | Complement Component Deficiency

China
University Hospital, Lille
CSL Behring; Laboratoire français de Fractionnement et de Biotechnologies; Oc... and other collaborators

Terminated

Systematic Search for Primary Immunodeficiency in Adults With Infections (SPIDAC)

Pneumococcal Infections | Pneumonia, Bacterial | Meningitis, Bacterial | Otitis Media | Chronic Sinus Infection | Streptococcal Infection | Antibody Deficiency | Complement Deficiency | Neisseria Infections | Haemophilus Influenza

France
Oslo University Hospital

Completed

A Comparison Study of Bypassing Agent Therapy With and Without Tranexamic Acid in Haemophilia A Patients With Inhibitor (BPATXAS)

Hereditary Factor VIII Deficiency Disease With Inhibitor

Norway
Hoffmann-La Roche

Active, not recruiting

A Study to Evaluate the Safety, Efficacy, Pharmacokinetics and Pharmacodynamics of Emicizumab in Participants With Mild or Moderate Hemophilia A Without FVIII Inhibitors (HAVEN 6)

Hemophilia A | Mild Hereditary Factor VIII Deficiency Disease Without Inhibitor | Moderate Hereditary Factor VIII Deficiency Disease Without Inhibitor

United States, Netherlands, Belgium, France, Germany, South Africa, Spain, Canada, United Kingdom, Poland
Hoffmann-La Roche

Completed

An Observational Study to Evaluate Physical Activity, Bleeding Incidence and Health Related Quality of Life, in Participants With Haemophilia A Without Inhibitors Receiving Standard of Care Treatment (POWER)

Haemophilia A | Moderate Hereditary Factor VIII Deficiency Disease Without Inhibitor | Severe Hereditary Factor VIII Deficiency Disease Without Inhibitor

Italy
Scripps Health
J. Craig Venter Institute

Completed

Biomarker Monitoring in TP53 Mutation Carriers

Li-Fraumeni Syndrome | Hereditary Cancer Syndromes | TP53 Gene Germline Mutation Carrier

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
Apellis Pharmaceuticals, Inc.

Active, not recruiting

Phase III Study Assessing the Efficacy and Safety of Pegcetacoplan in Patients With C3 Glomerulopathy or Immune-Complex Membranoproliferative Glomerulonephritis (VALIANT)

C3G | IC-MPGN | C3 Glomerulopathy | C3 Glomerulonephritis | Complement 3 Glomerulopathy | Complement 3 Glomerulopathy (C3G) | Complement 3 Glomerulonephritis | Dense Deposit Disease | DDD | Membranoproliferative Glomerulonephritis | Membranoproliferative Glomerulonephritis (MPGN) | Immune Complex Membranoproliferative...

United States, Spain, France, Germany, United Kingdom, Netherlands, Brazil, Israel, Japan, Australia, Austria, Italy, Switzerland, Korea, Republic of, Czechia, Belgium, Argentina, Canada, Poland

Clinical Trials on Hereditary Complement Deficiency Diseases

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