Clinical Trials on Factor VIII Deficiency, Congenital

Total 543 results

Rocket Pharmaceuticals Inc.

Active, not recruiting

Gene Therapy for Pyruvate Kinase Deficiency (PKD)

Pyruvate Kinase Deficiency

Spain, United States
Agios Pharmaceuticals, Inc.

Completed

A Study Evaluating the Efficacy and Safety of AG-348 in Regularly Transfused Adult Participants With Pyruvate Kinase Deficiency (PKD)

Anemia, Hemolytic | Pyruvate Kinase Deficiency

United States, United Kingdom, Netherlands, Canada, Denmark, France, Italy, Ireland, Thailand
Cairo University

Completed

Patient Specific Intraoral Inverted-L Osteotomy Modified With Inferior Alveolar Nerve Relocation in Corrective Mandibular Surgery

Class III Malocclusion

Egypt
Damascus University

Completed

Treatment of Skeletal Class III Malocclusion Using Orthodontic Removable Traction Appliance

Class III Malocclusion

Syrian Arab Republic
Agios Pharmaceuticals, Inc.

Completed

Pyruvate Kinase Deficiency Global Longitudinal Registry Substudy of Protocol AG348-C-008

Pyruvate Kinase Deficiency

United States, Italy, Netherlands
CENTOGENE GmbH Rostock

Completed

Pyruvate Kinase Deficiency Epidemiological Study (PIECE) (PIECE)

Pyruvate Kinase Deficiency

United States
Agios Pharmaceuticals, Inc.

Completed

A Study to Evaluate Efficacy and Safety of AG-348 in Not Regularly Transfused Adult Participants With Pyruvate Kinase Deficiency (PKD)

Anemia, Hemolytic | Pyruvate Kinase Deficiency

United States, Spain, Switzerland, Turkey, Germany, France, United Kingdom, Japan, Netherlands, Brazil, Canada, Czechia, Denmark, Italy, Korea, Republic of, Thailand
Shahid Beheshti University

Unknown

Comparative Results of Maxillary Deficiency Treatment by Fixed Tongue Appliance and Fixed Facemask in Growing Patients

Maxillary Deficiency

Iran, Islamic Republic of
Shahid Beheshti University of Medical Sciences

Completed

Comparative Results of Maxillary Deficiency Treatment by Tongue Plate and Facemask in Growing Patients

Maxillary Deficiency

Iran, Islamic Republic of
National Center for Research Resources (NCRR)
James Whitcomb Riley Hospital for Children

Completed

Phase II Pilot Study of Granulocyte Colony-Stimulating Factor for Inherited Bone Marrow Failure Syndromes

Thrombocytopenia | Dyskeratosis Congenita | Fanconi's Anemia | Shwachman Syndrome
Rocket Pharmaceuticals Inc.

Not yet recruiting

A Phase 2 Clinical Trial to Evaluate the Efficacy of Gene Therapy for Pyruvate Kinase Deficiency

Pyruvate Kinase Deficiency

Spain, United States
Agios Pharmaceuticals, Inc.

Active, not recruiting

Extension Study of AG-348 in Adult Participants With Pyruvate Kinase Deficiency Previously Enrolled in AG-348-006 or AG348-C-007

Pyruvate Kinase Deficiency

United States, Spain, Japan, Canada, United Kingdom, Korea, Republic of, Ireland, Thailand, Italy, Turkey, Denmark, Brazil, France, Germany, Netherlands, Switzerland
Xiaofan Zhu

Available

Use Massive Parallel Sequencing and Exome Capture Technology to Sequence the Exome of Fanconi Anemia Children and Their Patents

Fanconi Anemia | Autosomal or Sex Linked Recessive Genetic Disease | Bone Marrow Hematopoiesis Failure, Multiple Congenital Abnormalities, and Susceptibility to Neoplastic Diseases. | Hematopoiesis Maintainance.

China
University Hospital Schleswig-Holstein

Withdrawn

Gene Polymorphisms Influencing Steroid Synthesis and Action

Disorders of Sex Development | Congenital Adrenal Hyperplasia | Adrenal Insufficiency | Congenital Adrenal Hypoplasia | Mineralocorticoid Deficiency | Intersex
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
First Affiliated Hospital, Sun Yat-Sen University

Recruiting

Safety and Efficacy of Aspirin in Stroke Patients With Glucose-6-phosphate Dehydrogenase Deficiency (SAST) (SAST)

Stroke | G6PD Deficiency

China
University of Baghdad

Recruiting

The Effectiveness of Modified 3D Printed Customized Facemask Appliance for Treatment of Class III Malocclusion

Jaw Protrusions

Iraq
Sydney Local Health District

Enrolling by invitation

Effects of Class III Elastics on Stability of Orthopaedic Class III Correction

Class III Malocclusion | Underbite

Australia
Assiut University

Unknown

Red Cell Distribution Width Index Versus Red Cell Distribution Width as Discriminating Guide for Iron Deficiency Anaemia and Beta Thalassemia Trait .

Microcytic Hypochromic Anemia
Laboratoires Vivacy

Recruiting

Study of a Novel Hyaluronic Acid Based Gel for Volume Deficiency in the Mid-face (FaceHyal) (FaceHyal)

Aging | Aesthetics | Volume Deficiency in the Mid-Face

France
University of California, San Francisco
National Cancer Institute (NCI)

Completed

Donor Stem Cell Transplant in Treating Young Patients With Myelodysplastic Syndrome, Leukemia, Bone Marrow Failure Syndrome, or Severe Immunodeficiency Disease

Myelodysplastic Syndromes | Leukemia | Congenital Amegakaryocytic Thrombocytopenia | Severe Congenital Neutropenia

United States
National Center for Research Resources (NCRR)
University of North Carolina

Completed

Phase I/II Study of Monoclonal Factor IX Concentrate for Factor IX Deficiency

Hemophilia B | Factor IX Deficiency
Rennes University Hospital

Completed

Evaluation of Antithrombin Deficiency in Patients of Intensive Care Unit Placed on Extracorporeal Membrane Oxygenation (ATECMO)

Severe Cardiac Insufficiency

France
Mostafa Hassanein, Msc

Completed

Patients With Acl Injury and Varus Knee Will Undergo Acl Reconstruction Alone, or Combined With High Tibial Osteotomy in the Same Session.

ACL Injury | Knee Deformity | ACL - Anterior Cruciate Ligament Deficiency | Varus Deformity of Tibia (Disorder)

Egypt
dr. Laura C. G. de Graaff-Herder

Not yet recruiting

Growing up With the Young Endocrine Support System (YESS!) (YESS)

Congenital Adrenal Hyperplasia | Hypogonadotropic Hypogonadism | Growth Hormone Deficiency | Turner Syndrome | Addison's Disease | Klinefelter Syndrome | Combined Pituitary Hormone Deficiency | Androgen Insensitivity Syndrome | Thyroid Dysgenesis

Spain, United Kingdom, Belgium, Netherlands
National Heart, Lung, and Blood Institute (NHLBI)

Completed

Cord Blood Stem Cell Transplantation Study (COBLT)

Neoplasms | Myelodysplastic Syndromes | Hematologic Diseases | Leukemia | Severe Combined Immunodeficiency | Fanconi Anemia | Anemia, Aplastic | Hematopoietic Stem Cell Transplantation
Bezmialem Vakif University

Recruiting

The Effect of Splint Usage in Laterognathic Cl III Orthognathic Surgery Patients (TMJ)

Class III Malocclusion | Skeletal Malocclusion | Laterognathia

Turkey
Bezmialem Vakif University

Completed

Determining the Presence of RAP in Orthognathic Patients and Its Effect on Root Resorption by Biomarkers (RAP)

Root Resorption | Class III Malocclusion

Turkey
Institut National de la Santé Et de la Recherche...

Recruiting

MPS (RaDiCo Cohort) (RaDiCo-MPS)

Mucopolysaccharidosis I | Mucopolysaccharidosis II | Mucopolysaccharidosis VI | Mucopolysaccharidosis III | Mucopolysaccharidosis IV | Mucopolysaccharidosis VII | Multiple Sulfatase Deficiency Disease | Mucopolysaccharidosis IX

France
Diurnal Limited
National Institutes of Health Clinical Center (CC)

Completed

Comparison of Two Forms of Hydrocortisone in Patients With Congenital Adrenal Hyperplasia

Adrenogenital Syndrome | Congenital Adrenal Hyperplasia | 21-Hydroxylase Deficiency

United States
University Hospital Freiburg

Completed

Assessing Immune Function in Young Patients With Cytopenia That Did Not Respond to Treatment

Myelodysplastic Syndromes | Fanconi Anemia | Dyskeratosis Congenita | Pearson Marrow-pancreas Syndrome | Shwachman-diamond Syndrome

Spain, Germany, Switzerland, Austria, Netherlands, Italy, Czech Republic, Belgium, Denmark, Ireland
Southern Medical University, China

Unknown

Efficacy of Yinzhihuang Oral Liquid on Indirect Bilirubin of Neonates With Glucose-6-phosphate Dehydrogenase Deficiency

Neonatal Hyperbilirubinemia

China
Al-Azhar University

Recruiting

Effects of Conventional Versus Skeletally Anchored Facemask in Treatment of the Prepubertal Skeletal Class III Patients

Maxillary Deficiency | Maxillary Retrusion

Egypt
University of Alexandria

Recruiting

Mandibular Reconstruction With Axially Vascularised Bone Substitutes

Mandibular Deficiency

Egypt
Columbia University

Unknown

Risk-Adapted Allogeneic Stem Cell Transplantation For Mixed Donor Chimerism In Patients With Non-Malignant Diseases

Severe Combined Immunodeficiency | Fanconi Anemia | Bone Marrow Failure | Osteopetrosis

United States
Taner OZTURK, DDS, MS

Completed

The Effect of Orthopedic Face Mask Use on Head Posture and Pharyngeal Airway

Class III Malocclusion | Hyoid Bone

Turkey
National Medical Research Center for Children's...

Recruiting

Vitamin D Status and Bone Metabolism Status in Children With Congenital Epidermolysis Bullosa

Vitamin D Deficiency | Osteoporosis | Osteopenia | Phosphorus and Calcium Disorders | Bullosa Epidermolysis

Russian Federation
Universitas Padjadjaran
Hasan Sadikin General Hospital

Completed

Immunomodulatory Vitamin D in Thalassemia (ThalTB)

Vitamin D Deficiency | Tuberculosis | Thalassemia

Indonesia
Children's Hospital Los Angeles
Lucile Packard Children's Hospital

Terminated

Related Hematopoietic Stem Cell Transplantation (HSCT) for Genetic Diseases of Blood Cells

Metabolic Diseases | Stem Cell Transplantation | Chronic Granulomatous Disease | Bone Marrow Transplantation | Thalassemia | Wiskott-Aldrich Syndrome | Genetic Diseases | Peripheral Blood Stem Cell Transplantation | Pediatrics | Diamond-Blackfan Anemia | Allogeneic Transplantation | Combined Immune Deficiency | X-linked Lymphoproliferative Disease
Ferring Pharmaceuticals

Completed

Evaluation of Overall Compliance and Duration of Zomacton® Treatment With the Zomajet® Needle-free Device (ZOMAJET)

Turner's Syndrome | Human Growth Hormone Deficiency

France
Cairo University

Unknown

Evaluation of the Effects of Flat and Modified Inclined Fixed Anterior Bite Planes on Mandibular Position in Growing Females With Skeletal Class 2 Malocclusion Due to Mandibular Deficiency and Deep Bite

Skeletal Class 2

Egypt
Zhejiang University

Unknown

Iodine Status in Pregnant Women and Their Newborns: is Congenital Hypothyroidism Related to Iodine Deficiency in Pregnancy?

Pregnancy | Congenital Hypothyroidism | Iodine Deficiency

China
Federal Research Institute of Pediatric Hematology...

Not yet recruiting

Total Lymphoid Irradiation Pre-HSCT in Severe Congenital Neutropenia

Severe Congenital Neutropenia | GATA2 Deficiency

Russian Federation
Lucas Otieno Tina, MD MSc
GlaxoSmithKline; Strathmore University

Completed

Zinc Supplementation in Children With Sickle Cell Disease in Western Kenya

Sickle Cell Disease | Zinc Deficiency | Infection
CENTOGENE GmbH Rostock

Completed

Biomarker for Hereditary AngioEdema Disease (BioHAE)

Hereditary Angioedema | Hereditary Angioedema Type I | Hereditary Angioedema Type II | C1 Esterase Inhibitor Deficiency | HAE | Angio Edema | C4 Deficiency | Hereditary Angioedema Type III

Turkey, Armenia, Georgia, India, Peru, Poland, Romania
Baylor College of Medicine
Shire

Active, not recruiting

Low VW Activity in Adolescent HMB (Low VWF)

Von Willebrand Factor Deficiency

United States
Azienda Ospedaliero-Universitaria Careggi

Not yet recruiting

Intraoral Device vs Facial Mask for Class III Treatment

Class III Malocclusion | Dentofacial Anomalies, Including Malocclusion
McGill University
Ethiopian Health and Nutrition Research Institute; Micronutrient Initiative

Completed

The Effects of Iodized Salt on Cognitive Development in Ethiopia

Goiter | Iodine Deficiency | Cretinism

Canada
University of Utah
The Hospital for Sick Children; Boston Children's Hospital

Completed

Vascular Health and Risk Factors in Children With Down Syndrome

Atherosclerosis | Down Syndrome | Cardiovascular Risk Factor

United States
Cairo University

Unknown

Effect of Modified Twin Block in Skeletal Class II Growing Females With Mandibular Deficiency

Class II Division 1 Malocclusion

Clinical Trials on Factor VIII Deficiency, Congenital

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