Clinical Trials on Congenital Fibrinogen Deficiency in United Kingdom

Total 8202 results

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
St. George's Hospital, London

Completed

Indexed Aortic Area in Bicuspid Aortic Valve Aortopathy

Thoracic Aortic Aneurysm | Bicuspid Cardiac Valve

United Kingdom
Wave Life Sciences Ltd.

Recruiting

A Phase 1 Research Study to Evaluate Safety, Tolerability, and Pharmacokinetics of WVE-006 in Healthy Participants With Wild-type AAT Expression (RestorAATion-1) (RestorAATion-1)

Alpha-1 Antitrypsin Deficiency

United Kingdom
Takeda

Completed

Calcichew D3 Preference Study in Participants Eligible for Calcium and Vitamin D Supplementation.

Calcium and Vitamin D Deficiencies

United Kingdom, Germany
Wirral University Teaching Hospital NHS Trust

Completed

Measures to Increase the Effectiveness of Fast Track Colorectal Clinics for Iron Deficiency Anaemia.

Colorectal Cancer

United Kingdom
K2M, Inc.

Withdrawn

Comparing Pedicle Screw Systems for the Treatment of Adolescent Paediatric Spine Deformity

Adolescent Idiopathic Scoliosis

United Kingdom
University College, London
Moorfields Eye Hospital NHS Foundation Trust; Targeted Genetics Corporation

Completed

Safety Study of RPE65 Gene Therapy to Treat Leber Congenital Amaurosis

Retinal Degeneration

United Kingdom
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
Ultragenyx Pharmaceutical Inc

Completed

A Study of UX007 (Triheptanoin) in Participants With Long-Chain Fatty Acid Oxidation Disorders (LC-FAOD)

Long-chain Fatty Acid Oxidation Disorders (LC-FAOD) | Very Long Chain Acyl-CoA Dehydrogenase (VLCAD) Deficiency | Trifunctional Protein (TFP) Deficiency | Carnitine Palmitoyltransferase (CPT II) Deficiency | Longchain 3-hydroxy-acyl-CoA Dehydrogenase (LCHAD) Deficiency

United States, United Kingdom
Hussain Kazi

Completed

Metal Ions in Ceramic on Metal Total Hip Arthroplasty (COM)

Osteoarthritis | Rheumatoid Arthritis | Avascular Necrosis | Developmental Dysplasia of the Hip

United Kingdom
BioCryst Pharmaceuticals

Completed

A Drug-Drug Interaction Study to Evaluate Drug Transporter Interactions

Hereditary Angioedema

United Kingdom
Prometic Biotherapeutics, Inc.

Completed

A Study of Prometic Plasminogen IV Infusion in Subjects With Hypoplasminogenemia

Hypoplasminogenemia | Congenital Plasminogen Deficiency

United States, Norway
University College, London
Society for Cardiothoracic Surgery in Great Britain & Ireland; British Congenital...

Unknown

National Congenital Heart Disease Audit

Congenital Heart Disease (CHD)

United Kingdom
Great Ormond Street Hospital for Children NHS Foundation...

Unknown

Gene Therapy for X-linked Severe Combined Immunodeficiency (SCID-X1)

X-linked Severe Combined Immunodeficiency

United Kingdom
Emory University
Children's Healthcare of Atlanta

Terminated

Alefacept and Allogeneic Hematopoietic Stem Cell Transplantation

Sickle Cell Disease | Fanconi Anemia | Thalassemia | Severe Congenital Neutropenia | Wiskott-Aldrich Syndrome | Diamond-Blackfan Anemia | Severe Aplastic Anemia | Glanzmann Thrombasthenia | Leukocyte Adhesion Deficiency | Dyskeratosis-congenita | Chediak-Higashi Syndrome | Chronic-granulomatous Disease | Schwachman-Diamond...

United States
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
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
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
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 Scientific Corporation

Completed

ECG Signal Collection From Long QT Syndrome, Wide QRS Complexes, Heart Failure, and Cardiac Resynchronization Patients

Heart Failure | Long QT Syndrome | Wide QRS Complexes | Cardiac Resynchronisation

United Kingdom
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
NHS Lothian
University of Edinburgh

Recruiting

Nexus Evaluation Primary Trident II Uncemented Shell (NEPTUNE)

Hip Dysplasia | Hip Osteoarthritis | Hip Arthropathy | Arthritis of Hip

United Kingdom
St George's, University of London
SPD Development Company Limited

Suspended

Endocrine and Menstrual Disturbances in Women With Polycystic Ovary Syndrome (PCOS) (EMDPCOS)

Obesity | Polycystic Ovary Syndrome | Vitamin D Deficiency | Insulin Resistance

United Kingdom
University of Aberdeen
NHS Grampian

Unknown

PET-detected Myocardial Inflammation is a Characteristic of Cardiac Sarcoid But Not of ARVC

Sarcoidosis | Arrhythmogenic Right Ventricular Cardiomyopathy

United Kingdom
Takeda

Recruiting

A Study in Adults With Hereditary Angioedema (HAE) Who Currently Receive Icatibant at Home

Hereditary Angioedema (HAE)

United Kingdom
University of Aberdeen
NHS Grampian

Recruiting

Scottish Advanced Fetal Research Study (SAFeR)

Life Style | Fetal Development | Human | Fetal Developmental Abnormality

United Kingdom
King's College Hospital NHS Trust

Unknown

RCT of Steroids Following Kasai Portoenterostomy for Biliary Atresia.

Biliary Atresia

United Kingdom
Rhythm Pharmaceuticals, Inc.

Completed

Phase 3 Crossover Trial of Two Formulations of Setmelanotide in Patients With Specific Gene Defects in the MC4R Pathway

Bardet-Biedl Syndrome | POMC Deficiency

Netherlands, United States, Germany, Canada, Puerto Rico, United Kingdom
University of Edinburgh

Completed

Assessment of Risk in Thoracic Aortopathy Using 18F-Sodium Fluoride (AoRTAS)

Bicuspid Aortic Valve | Aortic Dissection | Thoracic Aortic Aneurysm

United Kingdom
University of Bath
University of Nottingham

Active, not recruiting

Physiological and Sensory Responses to Prolonged Fasting in Humans (PRO-FAST)

Adiposity | Metabolic Disturbance | Fasting | Energy Supply; Deficiency

United Kingdom
University of Alabama at Birmingham
Grifols Biologicals, LLC

Completed

Post-bypass Prophylactic IVIG in Infants and Neonates

Congenital Heart Disease | Hypogammaglobulinemia

United States
University College, London
Medical Research Council; Moorfields Eye Hospital NHS Foundation Trust

Recruiting

RAFT - Clinical Trial of RAFT for Aniridia Related Keratopathy (RAFT)

Aniridia

United Kingdom
University of Liverpool

Recruiting

An Exploratory Study of Arginine Supplementation and the Postoperative Immune REsponse (ASPIRE)

Surgery | Preterm | Nutritional Deficiency | Immune System and Related Disorders

United Kingdom
Ethicare GmbH

Unknown

Follow-up Safety Trial in Children With Chronic Heart Failure Therapy Receiving Orodispersible Minitablets of Enalapril (LENA-WP10)

Heart Failure | Dilated Cardiomyopathy | Congenital Heart Disease

Austria, United Kingdom, Netherlands, Serbia, Hungary
Merck KGaA, Darmstadt, Germany
Merck Serono S.A., Geneva

Completed

First Year Growth Response Associated Genetic Markers Validation Phase IV Open-label Study in Growth Hormone Deficient and Turner Syndrome Pre-pubertal Children: the PREDICT Pharmacogenetics Validation Study

Turner Syndrome | Idiopathic Growth Hormone Deficiency

Spain, France, Canada, United Kingdom, Czech Republic, Argentina, Germany, Italy, Sweden
DePuy International

Terminated

A Randomised Single Centre Study to Compare the Long-term Performance of 4 Designs of the DePuy Ultima LX Stem in Primary Total Hip Replacement

Osteoarthritis | Avascular Necrosis | Post-traumatic Arthritis | Congenital Hip Dysplasia | Slipped Capital Femoral Epiphysis | Traumatic Femoral Fractures | Nonunion of Femoral Fractures | Collagen Disorder

United Kingdom
University of Nottingham
Nottingham University Hospitals NHS Trust

Recruiting

Functional and Structural Lung Imaging in Chronic Obstructive Pulmonary Disease (FASTCOPD)

Chronic Obstructive Pulmonary Disease | Alpha 1-Antitrypsin Deficiency

United Kingdom
University of Oxford
British Heart Foundation; National Institute for Health Research, United Kingdom

Completed

Iron Status and Hypoxic Pulmonary Vascular Responses

Pulmonary Arterial Hypertension | Iron Deficiency | Lung Hypoxia

United Kingdom
Adnan Sharif

Active, not recruiting

Frailty Intervention Trial in End-Stage Patients on Dialysis (FITNESS)

Physical Activity | Hemodialysis Complication | Nutritional Deficiency

United Kingdom
KalVista Pharmaceuticals, Ltd.

Completed

A Single and Multiple Doses Safety, Tolerability, Pharmacokinetics and Food Effect Study of KVD824 in Healthy Volunteers

Hereditary Angioedema

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
University of Leicester

Recruiting

Real-time MRI for Evaluating Cardiac Volumetry

Left Ventricle Abnormality

United Kingdom
DePuy International

Terminated

A Two Centre Study to Assess the Stability and Long-term Performance of the C-Stem™ AMT in a Total Primary Hip Replacement

Osteoarthritis | Rheumatoid Arthritis | Avascular Necrosis | Post-traumatic Arthritis | Congenital Hip Dysplasia | Slipped Capital Femoral Epiphysis | Collagen Disorders | Traumatic Femoral Fractures | Nonunion of Femoral Fractures

United Kingdom
Z Factor Limited
Hammersmith Medicines Research; Centessa Pharmaceuticals plc

Terminated

A Single Ascending and Repeated Dose Study of Oral ZF874 in Healthy Volunteers and PiXZ Subjects

Alpha1 Anti-Trypsin Deficiency

United Kingdom
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
BioCryst Pharmaceuticals

Completed

Study to Determine How BCX4161 is Metabolized and Eliminated by the Body

Hereditary Angioedema

United Kingdom
Inozyme Pharma
IQVIA Biotech

Active, not recruiting

Evaluation of Safety, Tolerability, and Efficacy of INZ-701 in Adults With ABCC6 Deficiency Causing PXE

Pseudoxanthoma Elasticum | Generalized Arterial Calcification of Infancy | ATP-Binding Cassette Subfamily C Member 6 Deficiency

United States, United Kingdom
Premaitha Health

Completed

Clinical Evaluation of the IONA Test for Non-invasive Pre Natal Screening in Twin Pregnancies (TWIN)

Down Syndrome | Patau Syndrome | Edward's Syndrome

United Kingdom
DePuy International

Terminated

Multi-centre Study to Assess the Long-term Performance of the Deltamotion Cup System in Primary Hip Replacement Surgery

Osteoarthritis | Rheumatoid Arthritis | Avascular Necrosis | Post-traumatic Arthritis | Congenital Hip Dysplasia | Traumatic Femoral Fractures

India, United Kingdom

Clinical Trials on Congenital Fibrinogen Deficiency in United Kingdom

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