Clinical Trials on Congenital FVII Deficiency

Total 17620 results

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
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
CSL Behring

Completed

Study of the Pharmacokinetics and Safety of Recombinant Factor VIIa Fusion Protein (rVIIa-FP, CSL689) in Patients With Congenital Factor VII Deficiency

Congenital Coagulation Factor VII Deficiency

Netherlands, Norway
Novo Nordisk A/S

Completed

Observational Study on the Efficacy and Safety of NovoSeven® During "Real-life" Usage in Germany (WIRK)

Congenital Bleeding Disorder | Acquired Bleeding Disorder | Acquired Haemophilia | Congenital FVII Deficiency | Glanzmann's Disease

Germany
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
Centers for Disease Control and Prevention

Completed

Limb Loss Self-Management Program

Congenital or Acquired Limb Deficiency

United States
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
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
Novo Nordisk A/S

Completed

Bioequivalence of NovoSeven® and a NovoSeven® Formulation Stable at Room Temperature in Healthy Male Subjects

Healthy | Congenital Bleeding Disorder | Haemophilia A With Inhibitors | Haemophilia B With Inhibitors | Acquired Bleeding Disorder | Acquired Haemophilia | Congenital FVII Deficiency | Glanzmann's Disease

France
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
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
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
Northwestern University

Completed

The Effects of Metreleptin in Congenital Leptin Deficiency

Congenital Leptin Deficiency (Disorder)

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
University of Minnesota

Recruiting

Treatment of LSCD With DM

Limbal Stem-cell Deficiency | Congenital Aniridia

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
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
AryoGen Pharmed Co.

Completed

Study Comparing the Pharmacokinetic of Biosimilar Eptacog Alfa With Novoseven®, in Patients With Congenital Factor VII Deficiency

Factor VII Deficiency

Iran, Islamic Republic of
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
Cure CMD
Congenital Muscle Disease International Registr

Withdrawn

LAMA2-related Muscular Dystrophy Brain Study

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

United States
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

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 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

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
University of L'Aquila
TRIB s.r.l.

Completed

Treatment of Inherited Factor VII Deficiency (STER)

Factor VII Deficiency

Italy
Hydrocephalus and Neuroscience Institute

Completed

Salt-FA to Increase Folate Levels

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

United States
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
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, Grenoble

Recruiting

Pediatric and Adult Registry for Patients With ARThrogryposis (PARART)

Arthrogryposis Multiplex Congenita (AMC)

France
University Hospital, Grenoble

Not yet recruiting

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

Arthrogryposis Multiplex Congenita (AMC)
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
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
Rigshospitalet, Denmark
Ultragenyx Pharmaceutical Inc

Completed

The Effect of Triheptanoin on Fatty Acid Oxidation and Exercise Tolerance in Patients With Glycogenoses

Tarui Disease | Debrancher Deficiency | GYG1 DEFICIENCY

Denmark
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
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
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
Agios Pharmaceuticals, Inc.

Terminated

Pyruvate Kinase (PK) Deficiency Global Longitudinal Registry: Patient-Reported Outcomes (PRO) (PKD)

Anemia | Anemia, Hemolytic | Pyruvate Kinase Deficiency | Pyruvate Kinase Deficiency Anemia

United States, United Kingdom, Italy, Spain, Netherlands
Rigshospitalet, Denmark

Unknown

Fat and Sugar Metabolism During Exercise in Patients With Metabolic Myopathy

Carbohydrate Metabolism, Inborn Errors | Metabolism, Inborn Errors | Lipid Metabolism, Inborn Errors | Glycogen Storage Disease Type II | Glycogen Storage Disease Type V | VLCAD Deficiency | Glycogen Storage Disease Type III | Phosphoglycerate Kinase Deficiency | Neutral Lipid Storage Disease | Carnitine... and other conditions

Denmark
Takeda

Active, not recruiting

A Study of TAK-662 for Japanese Patients With Congenital Protein C Deficiency

Congenital Protein C Deficiency

Japan

Clinical Trials on Congenital FVII Deficiency

Sponsors and Collaborators

Medical Conditions

Drug Interventions

CROs by country

CROs in Algeria

Conditions

Rare Diseases

Drug Interventions

Dietary Supplements

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