Clinical trials: Congenital Bleeding Disorder
Hemophilia A, Hemophilia B
Pre-clinical Models for Mesenchymal Stem Cell Therapy in Hemophilic Arthropathy
Hemophilia is a constitutional coagulation disorder responsible for a hemorrhagic phenotype in patients from an early age. Hemarthrosis is one of the most frequent complications in hemophiliacs and leads to the development of…
- Conditions
- Hemophilia A, Hemophilia B, Arthropathy
- Trial name
- NCT05187936
- Where
- France
- Volunteers
- 200 people, healthy volunteers welcome
- Sponsor
- University Hospital, Montpellier
Hemophilia, Arthropathy of Knee
Evaluation of Static Postural Balance in Children With Hemophilia and Its Relationship With Joint Health
Hemophilia is a rare hematological disorder characterized by a partial or complete deficiency of clotting factor VIII or IX.Bleeding disorders mainly affect the musculoskeletal system.
- Conditions
- Hemophilia, Arthropathy of Knee, Arthropathy of Ankle or Foot
- Trial name
- NCT04406519
- Where
- Turkey (Türkiye)
- Volunteers
- 42 people, healthy volunteers welcome
- Sponsor
- Necmettin Erbakan University
Haemophilia
Inter Individual Variability in Initiation Pathway Activation and Regulation and Phenotypic Heterogeneity in Patients With Haemophilia A and B
Severe haemophilia A and B (SHA, SHB) are X - linked inherited bleeding disorders, characterised by factor VIII and IX levels of <1 IU/dL respectively.
- Trial name
- InPath (NCT03287999)
- Testing
- Thrombophilia screen, Initiation pathway analysis
- Where
- United Kingdom
- Volunteers
- 250 people, healthy volunteers welcome
- Sponsor
- Royal Free Hospital NHS Foundation Trust
Gastrointestinal Hemorrhage, Vascular Malformation
Efficacy Study of Thalidomide in Gastrointestinal Vascular Malformation Related Bleeding
Background: Repeated bleeding from gastrointestinal vascular malformations remains to be a major therapeutic challenge. Methods: The investigators performed a randomised, double-blind, placebo-controlled, single centre study to…
- Trial name
- NCT02754960
- Testing
- Thalidomide
- Where
- China
- Volunteers
- healthy volunteers welcome
- Sponsor
- Shanghai Jiao Tong University School of Medicine
Hemophilia A, Hemophilia B
Gaining Insight Into the Complexity of Pain in Patients With Haemophilia
Joint pain has been reported as a major problem in people with haemophilia (PwH). Therefore, haemophilia in adults seems clinically more related to a musculoskeletal disorder than a bleeding disorder, with many patients reporting…
- Trial name
- NCT05606640
- Testing
- Biopsychological pain assessment
- Where
- 2 sites in Belgium
- Volunteers
- 104 people, healthy volunteers welcome
- Sponsor
- Universiteit Antwerpen
Von Willebrand Disease (VWD), Acquired Von Willebrand Disease
Endothelial Colony-Forming Cells in Patients With VWD, AVWS and Healthy Subjects
The goal of this observational study is to learn how endothelial colony-forming cells (ECFCs) behave in people with von Willebrand disease (VWD), acquired von Willebrand syndrome (AVWS), and in healthy individuals.
- Trial name
- ECFCs/2022 (NCT07358013)
- Testing
- Blood sample collection for VWF measurements in plasma, Blood sample collection for ECFC isolation and characterization, Genetic testing of VWF
- Where
- Italy
- Volunteers
- 48 people, healthy volunteers welcome
- Sponsor
- Fondazione IRCCS Ca' Granda, Ospedale Maggiore Policlinico
Hemophilia A
Evaluation of the Training Program for Community Pharmacists for Dispensing Emicizumab (HEMLIBRA®) in France
Haemophilia A (HA) is a rare constitutional haemorrhagic disease whose drug management is based on the use of chronic lifelong replacement therapy.
- Trial name
- PASODOBLEDEMI1 (NCT05449197)
- Testing
- HEMOPHAR e-learning program
- Where
- France
- Volunteers
- 363 people, healthy volunteers welcome
- Sponsor
- Hospices Civils de Lyon
Hemophilia A
Manual Therapy in Hemophilic Arthropathy of the Ankle
Introduction: Haemophilic ankle arthropathy manifests as functional (deficit in muscle strength, mobility and proprioception), intra-articular degenerative alterations and chronic pain.
- Trial name
- NCT06816056
- Testing
- Manual Therapy
- Where
- Spain
- Volunteers
- 13 people, healthy volunteers welcome
- Sponsor
- Investigación en Hemofilia y Fisioterapia
Congenital Bleeding Disorder, Haemophilia A
A Multi-centre, Open Labelled, Multiple Dosing Trial Investigating Safety, Pharmacokinetics and Pharmacodynamics of NNC 0172-2021 Administered Subcutaneously to Healthy Male Subjects and Haemophilia Subjects
This trial is conducted in Europe. The aim of this trial is to investigate safety, pharmacokinetics (the exposure of the trial drug in the body) and pharmacodynamics (the effect of the investigated drug on the body) of NNC…
- Conditions
- Congenital Bleeding Disorder, Haemophilia A, Healthy
- Trial name
- explorer™2 (NCT01631942)
- Testing
- NNC172-2021
- Where
- 6 sites in Austria, France and 3 more
- Volunteers
- 4 people, healthy volunteers welcome
- Sponsor
- Novo Nordisk A/S
Hemophilia Arthropathy
Pain in Patients With Congenital Coagulopathies
Introduction. The development of joint hemorrhages in patients with congenital coagulopathies favor the development of an intra-articular, degenerative and progressive lesion (hemophilic arthropathy).
- Trial name
- He-PainSit (NCT03499522)
- Testing
- Observational group
- Volunteers
- 80 people, healthy volunteers welcome
- Sponsor
- Real Fundación Victoria Eugenia
Hemophilia
EPOCH-Economics and Patient Outcomes in China in Haemophilia
The overarching goal of the EPOCH project is to: Support a broad and robust needs assessment in the haemophilia space in China Measure the variability in the needs and care provision Generate outcome data (health economic…
- Trial name
- EPOCH (NCT07454239)
- Where
- China
- Volunteers
- 500 people, healthy volunteers welcome
- Sponsor
- Institute of Hematology & Blood Diseases Hospital, China
Congenital Bleeding Disorder, Congenital FXIII Deficiency
Safety and Pharmacokinetics of Recombinant Factor XIII in Healthy Volunteers
This trial was conducted in Europe. The aim of this trial was to investigate safety and pharmacokinetics of escalating single doses of catridecacog (recombinant factor XIII, rFXIII) in healthy volunteers.
- Conditions
- Congenital Bleeding Disorder, Congenital FXIII Deficiency, Healthy
- Trial name
- NCT01847989
- Testing
- catridecacog
- Where
- United Kingdom
- Volunteers
- 50 people, healthy volunteers welcome
- Sponsor
- Novo Nordisk A/S
Haemophilia, Arthropathy
Biomechanical Determinants and Patterns Associated to the Pathophysiological Cascade of Ankle Arthropathy in Children With Haemophilia: Non-invasive In-vivo Measurement of Foot Joints in Children With Haemophilia During Gait.
Haemophilia is a rare X chromosome-linked coagulation disorder resulting from a congenital deficiency or absence of circulating factor VIII (Haemophilia A) or factor IX (Haemophilia B).As a consequence, patients with haemophilia…
- Trial name
- 3DMKFM (NCT02229331)
- Testing
- Gait analysis
- Volunteers
- 37 people, healthy volunteers welcome
- Sponsor
- Universitaire Ziekenhuizen KU Leuven
Cerebral Small Vessel Diseases, Cadasil
Taiwan Associated Genetic and Nongenetic Small Vessel Disease
The TAG-SVD enrolled patients with clinical and neuroimaging features of cerebral small vessel disease (CSVD). All enrolled patients will receive next-generation sequence (NGS) with probes designed to target five candidate CSVD…
- Conditions
- Cerebral Small Vessel Diseases, Cadasil, HTRA1-Related Autosomal Dominant Cerebral Angiopathy, COL4A1-Related Brain Small Vessel Disease With Haemorrhage
- Trial name
- TAG-SVD (NCT05473637)
- Testing
- MRI
- Where
- Taiwan
- Volunteers
- 500 people, healthy volunteers welcome
- Sponsor
- National Taiwan University Hospital
Bleeding Disorder, Von Willebrand Disease (VWD)
The Bleeding Assessment Tool (BAT)
This study looks at whether adding simple family history questions to a standard bleeding questionnaire can better identify children who truly have mild bleeding disorders.
- Trial name
- BAT Protocol (NCT07560007)
- Where
- United States
- Volunteers
- 1,000 people, healthy volunteers welcome
- Sponsor
- Children's Hospital Medical Center, Cincinnati
Infant, Newborn, Infant, Low Birth Weight
The Effects of Erythropoietin (EPO) on the Transfusion Requirements of Very Low Birth Weight Infants
This study tested the safety and efficacy of transfusing erythropoietin (Epo) and iron in infants of <1,250g birth weight. For infants 401-1,000g birth weight, we tested whether early erythropoietin (Epo) and iron therapy would…
- Conditions
- Infant, Newborn, Infant, Low Birth Weight, Infant, Small for Gestational Age, Infant, Premature
- Trial name
- EPO (NCT01203514)
- Testing
- Erythropoietin
- Where
- 9 sites in United States
- Volunteers
- 318 people, healthy volunteers welcome
- Sponsor
- NICHD Neonatal Research Network
Physical Activity
Validation of Objective Measures of Place-Based Physical Activity
Usability of the PALMS system, validity of the PALMS system and added value to an existing cohort study. Hypothesis: Addition of GPS device and use of PALMS system will allow researchers to better examine place-based…
- Trial name
- NCT01886261
- Where
- United States
- Volunteers
- 144 people, healthy volunteers welcome
- Sponsor
- University of California, San Diego
Coagulopathy, Congenital Heart Disease
FFP Usage in Pediatric CV Surgery
When small children undergo heart surgery with the heart-lung machine, in the past, blood products to help the blood clot such as Fresh Frozen Plasma (FFP) was routinely used in children under 10kg.
- Trial name
- NCT05819788
- Where
- Canada
- Volunteers
- 431 people, healthy volunteers welcome
- Sponsor
- University of Alberta
Hemophilia, Step
Validity and Reliability of the Two-minute Step Test in Hemophilia
The aim of our study was to evaluate the psychometric properties of the 2-minute step test in hemophilia patients, assess its intra-rater and inter-rater reliability, and evaluate its convergent validity and construct validity…
- Conditions
- Hemophilia, Step, Reliability and Validity, Hemophilia A and B
- Trial name
- NCT07425327
- Where
- Turkey (Türkiye)
- Volunteers
- 31 people, healthy volunteers welcome
- Sponsor
- Hasan Kalyoncu University
Vitamin D Deficiency, Sarcopenia
Vitamin D Deficiency and Body Composition in the Patients With Hemophilia
Hemophilia is associated with osteoporosis and increased prevalence of low bone mineral density (BMD). Many clinical risk factors have been reported, such as hemophilic arthropathy, reduced physical activity, decreased sun…
- Conditions
- Vitamin D Deficiency, Sarcopenia, Osteoporosis, Body Composition
- Trial name
- NCT05517070
- Where
- Taiwan
- Volunteers
- 80 people, healthy volunteers welcome
- Sponsor
- Tsung-Ying Li