Clinical Trials on Congenital FXIII Deficiency

Total 17594 results

Novo Nordisk A/S

Completed

Safety of Monthly Recombinant Factor XIII Replacement Therapy in Subjects With Congenital Factor XIII Deficiency: An Extension to Trial F13CD-1725 (mentor™2)

Congenital Bleeding Disorder | Congenital FXIII Deficiency

France, United States, Israel, Spain, Switzerland, Germany, Canada, Japan, United Kingdom, Italy, Finland, Austria
Novo Nordisk A/S

Completed

Safety of a Single Intravenous Dose of Recombinant Factor XIII in Children With Congenital FXIII A-subunit Deficiency (mentor™4)

Congenital Bleeding Disorder | Congenital FXIII Deficiency

United States, Israel, United Kingdom
Novo Nordisk A/S

Enrolling by invitation

A Study of Safety and Effectiveness of NovoThirteen® (rFXIII) During Treatment of Congenital FXIII Deficiency in Japan

Congenital Bleeding Disorder | Congenital FXIII Deficiency

Japan
Novo Nordisk A/S

Completed

Use of rFXIII in Treatment of Congenital FXIII Deficiency, a Prospective Multi-centre Observational Study (mentor™6)

Congenital Bleeding Disorder | Congenital FXIII Deficiency

Spain, United States, Canada, Italy, United Kingdom, Hungary
Novo Nordisk A/S

Completed

Safety and Efficacy of Monthly Replacement Therapy With Recombinant Factor XIII (rFXIII) in Paediatric Subjects With Congenital Factor XIII A-subunit Deficiency (mentor™5)

Congenital Bleeding Disorder | Congenital FXIII Deficiency

United Kingdom, Israel, United States
Novo Nordisk A/S

Completed

Safety and Pharmacokinetics of Recombinant Factor XIII in Healthy Volunteers

Healthy | Congenital Bleeding Disorder | Congenital FXIII Deficiency

United Kingdom
Novo Nordisk A/S

Completed

Safety and Pharmacokinetics of Recombinant Factor XIII Administration in Healthy Volunteers

Healthy | Congenital Bleeding Disorder | Congenital FXIII Deficiency

United Kingdom
Novo Nordisk A/S

Completed

Comparison of a Blood Clotting Drug (Recombinant Factor XIII) Produced by Two Different Manufacturers in Healthy Male Subjects

Healthy | Congenital Bleeding Disorder | Congenital FXIII Deficiency

United Kingdom
Novo Nordisk A/S

Completed

Safety and Pharmacokinetics of Recombinant Factor XIII in Patients With Congenital Factor Xlll Deficiency

Congenital Bleeding Disorder | Congenital FXIII Deficiency

United States
Novo Nordisk A/S

Completed

Evaluation of Recombinant Factor XIII for Prevention of Bleeding in Patients With FXIII Inherited Deficiency

Congenital Bleeding Disorder | Congenital FXIII Deficiency

United States, Israel, Austria, Spain, Switzerland, Germany, Canada, France, United Kingdom, Italy, Finland
Patricia Duque González

Completed

Impact of Acquired FXIII Deficiency on Morbidity and Mortality

Acquired Factor XIII Deficiency Disease

Spain
Baxalta now part of Shire

Completed

ADVATE 2 mL Post-Authorization Safety Surveillance (PASS)

Hemophilia A | Congenital Factor VIII (FVIII) Deficiency

United Kingdom, France, Germany, Hungary
Spark Therapeutics

Completed

Dose-finding Study of SPK-8016 Gene Therapy in Patients With Hemophilia A to Support Evaluation in Individuals With FVIII Inhibitors

Hematologic Diseases | Blood Coagulation Disorders, Inherited | Coagulation Protein Disorders | Hemorrhagic Disorders | Genetic Diseases, Inborn | Genetic Diseases, X-Linked | Gene Therapy | Blood Coagulation Disorder | Gene Transfer | Adeno-Associated Virus (AAV) | Factor VIII (FVIII) | Factor VIII (FVIII)... and other conditions

United States
Centers for Disease Control and Prevention

Completed

Limb Loss Self-Management Program

Congenital or Acquired Limb Deficiency

United States
QOL Medical, LLC

Completed

Adult Pilot Study for Reference Ranges and Optimal Cut-offs for the Sucrose Challenge Test and Sucrose Breath Test

Congenital Sucrase-Isomaltase Deficiency

United States
National Cardiovascular Center Harapan Kita Hospital...

Unknown

Thiamine Influenced on Substrate Energy Effectiveness in Indonesian Children Undergoing Cardiopulmonary Bypass

Cardiopulmonary Bypass | Congenital Heart Disease | Thiamine Deficiency

Indonesia
Children's Hospital of Eastern Ontario
McGill University; Ottawa Hospital Research Institute; The Ottawa Hospital; Children...

Completed

Prevention of Vitamin D Deficiency Following Pediatric CHD Surgery: a Phase II Dose Evaluation Randomized Controlled Trial Comparing Usual Care With a High Dose Pre-operative Supplementation Regimen Based on the Institute of Medicine Daily Upper Tolerable Intake Level (HICCUPS 2)

Vitamin D Deficiency | Heart Defects, Congenital | Thoracic Surgery | Pediatric Disorders

Canada
Queen Fabiola Children's University Hospital

Recruiting

Iron Deficiency in Pediatric Heart Surgery

Iron Deficiency Anemia | Congenital Heart Disease in Children | Cardiac Surgical Procedures | Iron Deficiencies

Belgium
CSL Behring

Completed

A Study of the Use of Factor XIII Concentrate in Patients With Inherited FXIII Deficiency

Factor XIII Deficiency

United States, Spain
CSL Behring

Completed

A Study of Factor XIII Concentrate in Subjects With Congenital Factor XIII Deficiency

Factor XIII Deficiency

United States, Spain
CSL Behring

Completed

An Open Enrollment Study of Factor XIII Concentrate in Subjects With Congenital Factor XIII Deficiency

Factor XIII Deficiency

United States
Northwestern University

Completed

The Effects of Metreleptin in Congenital Leptin Deficiency

Congenital Leptin Deficiency (Disorder)

United States
University of Minnesota

Recruiting

Treatment of LSCD With DM

Limbal Stem-cell Deficiency | Congenital Aniridia

United States
QOL Medical, LLC
Johns Hopkins University; Massachusetts General Hospital; Baylor College of Medicine and other collaborators

Completed

Congenital Sucrase-Isomaltase Deficiency (CSID) Genetic Prevalence Study (GPS) (CSID GPS)

Congenital Sucrase-isomaltase Deficiency (CSID)

United States
Maggie's Pearl, LLC

Active, not recruiting

Oral Epalrestat Therapy in Pediatric Subjects With PMM2-CDG

Pmm2-CDG | Phosphomannomutase 2 Deficiency | Phosphomannomutase 2 Congenital Disorder of Glycosylation | Phosphomannomutase II Congenital Disorder of Glycosylation | Phosphomannomutase II Deficiency

United States
Pfizer

Completed

Post-Marketing Surveillance To Observe Safety And Efficacy Of Xyntha Solofuse Prefilled Syringe

Factor VIII Deficiency, Congenital | Hemophilia A, Congenital | Factor 8 Deficiency, Congenital | Autosomal Hemophilia A | Classic Hemophilia
Children's Hospital of Orange County
CSL Behring

Completed

Clinical Trial of Factor XIII (FXIII) Concentrate

Hemophilia | Factor XIII Deficiency

United States
Klinikum St. Georg gGmbH

Completed

Wound Healing Abnormalities in Major Abdominal Surgery (INWOUND)

Impaired Wound Healing | Factor XIII Deficiency

Germany
University of Alberta

Withdrawn

Initiation of Resuscitation While Attached to the Cord With Congenital Heart Disease (INSPIRE-CHD)

Hypoplastic Left Heart Syndrome | Congenital Heart Defect | TGA - Transposition of Great Arteries | Congential Absence of Heart Structure | Congenital Malformation of Heart, Unspecified | Hypoplastic Right Heart Syndrome | Congential Malformations of Aortic Valve | Congential Malformations of Great...

Canada
Universitas Sumatera Utara

Recruiting

The Role of Thiamine After Transcatheter Closure in Children With Left-to-Right Shunt Congenital Heart Disease

Atrial Septal Defect | Congenital Heart Disease | Patent Ductus Arteriosus | Ventricular Septal Defect | Thiamine Deficiency

Indonesia
QOL Medical, LLC

Recruiting

7-Day Trial of Sucraid for Alleviating CSID Symptoms in Subjects With Low, Moderate, and Normal Sucrase Levels

Congenital Sucrase-Isomaltase Deficiency | CSID | Sucrase Isomaltase Deficiency

United States
University of Alabama at Birmingham

Completed

Feasibility, Acceptability and Directional Signal Effect on Blood Folate Levels of Iodized Salt Fortified With Folic Acid: Clinical Study (FASALT)

Folic Acid Deficiency | Neural Tube Defects | Fortification

United States
Octapharma

Recruiting

Efficacy of Atenativ in Patients With Congenital Antithrombin Deficiency Undergoing Surgery or Delivery

Congenital Antithrombin Deficiency

Spain, Italy, United States, Serbia, France, Hungary, Austria, Germany, United Kingdom, Israel
Kyowa Hakko Kirin UK, Ltd.

Completed

A Study of KW-3357 in Congenital Antithrombin Deficiency

Congenital Antithrombin Deficiency

France, Germany, Italy, Sweden, United Kingdom
Cure CMD
Congenital Muscle Disease International Registr

Withdrawn

LAMA2-related Muscular Dystrophy Brain Study

LAMA2-MD (Merosin Deficient Congenital Muscular Dystrophy, MDC1A)

United States
Laboratoire français de Fractionnement et de Biotechnologies

Completed

Human Fibrinogen Concentrate (FGTW) in Pediatric Patients With Congenital Fibrinogen Deficiency

Hypofibrinogenemia, Congenital | Afibrinogenemia, Congenital

France, Lebanon, Morocco, Turkey
The University of Hong Kong
Queen Mary Hospital, Hong Kong

Not yet recruiting

Effect of Folic Acid Supplementation in Pregnant Women Having Thalassaemia Trait

Thalassemia | Folic Acid Deficiency Anemia
Novo Nordisk A/S

Completed

Observational Study on Safety and Efficacy of NovoSeven® in Subjects With Congenital FVII Deficiency

Congenital Bleeding Disorder | Congenital FVII Deficiency

Japan
Novo Nordisk A/S

Completed

Treatment of Congenital Factor VII Deficiency (F7CONDEF)

Congenital Bleeding Disorder | Congenital FVII Deficiency

Slovakia, Israel, Spain, India, Iran, Islamic Republic of, Pakistan, Turkey, Germany, Serbia, Italy, Greece, Thailand, France, United States, Hong Kong, Venezuela
Hydrocephalus and Neuroscience Institute

Completed

Salt-FA to Increase Folate Levels

Folic Acid Deficiency | Neural Tube Defects | Spina Bifida | Anencephaly-Spina Bifida

United States
Biotest
Accovion GmbH; ICON plc; SYNLAB Analytics and Services Germany GmbH; Phoenix Clinical... and other collaborators

Completed

Pharmacokinetic, Efficacy and Safety of BT524 in Patients With Congenital Fibrinogen Deficiency

Congenital Afibrinogenemia | Congenital Hypofibrinogenemia

Bulgaria, Egypt, Germany, Lebanon, Tunisia
University Hospital, Clermont-Ferrand

Terminated

VISIODOL: Validation of the VISIODOL® Scale (VISIODOL)

Visual Deficiency | Congenital Blindness | Acquired Blindness

France
Prometic Biotherapeutics, Inc.

Completed

A Study of Prometic Plasminogen IV Infusion in Subjects With Hypoplasminogenemia

Hypoplasminogenemia | Congenital Plasminogen Deficiency

United States, Norway
University Hospital, Grenoble

Not yet recruiting

Technical Feasibility Study of Ultrasound Muscle Imaging in Antenatal Ultrasound (FetUS)

Arthrogryposis Multiplex Congenita (AMC)
University Hospital, Grenoble

Recruiting

Pediatric and Adult Registry for Patients With ARThrogryposis (PARART)

Arthrogryposis Multiplex Congenita (AMC)

France
Steno Diabetes Center Copenhagen
The Novo Nordisk Foundation Center for Basic Metabolic Research; Department... and other collaborators

Completed

The Effect on Metabolism, Food Intake and Preferences of a Knockout Gene Variant Involved in Carbohydrate Metabolism

Diabetes Mellitus, Type 2 | Metabolic Disease | Sucrase Isomaltase Deficiency | Sucrose Intolerance Congenital

Greenland
Jerry Vockley, MD, PhD
Ultragenyx Pharmaceutical Inc

No longer available

Compassionate Use of Triheptanoin (C7) for Inherited Disorders of Energy Metabolism

Barth Syndrome | Mitochondrial Trifunctional Protein Deficiency | Very Long-chain acylCoA Dehydrogenase (VLCAD) Deficiency | Carnitine Palmitoyltransferase Deficiencies (CPT1, CPT2) | Long-chain Hydroxyacyl-CoA Dehydrogenase Deficiency | Glycogen Storage Disorders | Pyruvate Carboxylase Deficiency... and other conditions

United States
Eva Morava-Kozicz
Children's Hospital of Philadelphia; Seattle Children's Hospital

Not yet recruiting

A Study of GlcNAc on Tear Production in NGLY1-CDDG

NGLY1 Deficiency

United States
Boston Children's Hospital

Terminated

Safety and Efficacy Trial of Danazol in Patients With Fanconi Anemia or Dyskeratosis Congenita

Fanconi Anemia | Dyskeratosis Congenita

United States
Agios Pharmaceuticals, Inc.

Active, not recruiting

A Study to Evaluate the Efficacy and Safety of Mitapivat in Pediatric Participants With Pyruvate Kinase Deficiency (PKD) Who Are Regularly Transfused, Followed by a 5-Year Extension Period (ACTIVATE-KidsT)

Pediatric Pyruvate Kinase Deficiency | Pediatric Hemolytic Anemia

United States, Spain, Denmark, Turkey, Netherlands, Czechia, United Kingdom, Germany, Canada, Switzerland

Clinical Trials on Congenital FXIII Deficiency

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