Clinical trials: Hemophilia, Classic
Trisomy 13 Syndrome, Arthrogryposis Congenita Multiplex With Intestinal Atresia
Palliative Care Needs of Children With Rare Diseases and Their Families
The palliative care needs of family caregivers of children with rare diseases and their children are largely unmet, including the need for support to prepare for future medical decision making.
- Conditions
- Trisomy 13 Syndrome, Arthrogryposis Congenita Multiplex With Intestinal Atresia, Asparagine Synthetase Deficiency, CHARGE Syndrome
- Trial name
- FACE-Rare (NCT06938542)
- Testing
- Family Centered pediatric palliative care for family caregivers of children with rare diseases.
- Where
- United States
- Volunteers
- 480 people, healthy volunteers welcome
- Sponsor
- Children's National Research Institute
Immune Thrombocytopenia, Thrombotic Thrombocytopenic Purpura
Longitudinal Cohort of Thrombosis and Hemostasis Diseases
This is a multicenter, prospective, longitudinal, observational cohort study to investigate thrombosis and hemostasis diseases in Chinese patients.
- Conditions
- Immune Thrombocytopenia, Thrombotic Thrombocytopenic Purpura, Hemophilia A, Acquired, Disseminated Intravascular Coagulation
- Trial name
- NCT06727669
- Where
- 5 sites in China
- Volunteers
- 3,000 people, healthy volunteers welcome
- Sponsor
- Peking University People's Hospital
Hemophilia
Assessment of Proprioceptive and Functional Characteristics in Patients With Hemophilia
Analyze and evaluate the skeletal muscle of patients affected with hemophilia in the Region of Murcia. Describe the periarticular muscular deficit of the lower limbs, depending on the degree of injury: acute and chronic, or…
- Trial name
- HE-BALANCE (NCT02165592)
- Where
- Spain
- Volunteers
- 60 people, healthy volunteers welcome
- Sponsor
- Universidad Católica San Antonio de Murcia
Hemophilia
Risk Models to Optimise Prophylaxis Schedules in Children With Haemophilia
The MOrPH study is designed to identify optimal prophylaxis schedules for children with haemophilia. This involves development of combined pharmacokinetic and pharmacodynamic models.
- Trial name
- MOrPH (NCT02585635)
- Volunteers
- 9 people, healthy volunteers welcome
- Sponsor
- Neuroscience Research Australia
Hemophilia, Hemophilia A
Viscoelastic Properties of Lower Extremity Muscles in Patients With Hemophilia
The aim of our study is to evaluate the viscoelastic properties of lower extremity muscles in patients with hemophilia A and hemophilia B secondarily to compare them with their healthy peers.
- Conditions
- Hemophilia, Hemophilia A, Hemophilia B
- Trial name
- NCT05981313
- Where
- Turkey (Türkiye)
- Volunteers
- 40 people, healthy volunteers welcome
- Sponsor
- Hasan Kalyoncu University
Severe Hemophilia A Without Inhibitor
A Study on the Bone-health Effectiveness of Applying Recombinant Factor VIII Fc (rFVIIIFc) to Patients With Hemophilia A (Prototype A)
Study Objectives* 1. Provide a systematic evaluation of the treatment outcomes in patients with hemophilia A 2. Emphasize the importance and clinical benefits of rFVIII-Fc in joint and bone health. 3.
- Trial name
- NCT05981274
- Testing
- serum biomarker.
- Where
- Taiwan
- Volunteers
- 45 people, healthy volunteers welcome
- Sponsor
- Tri-Service General Hospital (TSGH)
Hemophilia A, Hemophilia A, Severe
Hemophilia A Research Program
This study longitudinally observes the intergenerational (mother-child) continuum in hemophilia A from pregnancy through early childhood.
- Conditions
- Hemophilia A, Hemophilia A, Severe, Factor VIII (FVIII), FVIII Deficiency
- Trial name
- HARP (NCT07414511)
- Where
- United States
- Volunteers
- 500 people, healthy volunteers welcome
- Sponsor
- University of Washington
Hemophilia, Healthy Children
Comparison of Pulmonary Function, Exercise Capacity, and Physical Activity Level Between Children With Hemophilia and Healthy Controls
Hemophilia is a rare X-linked congenital bleeding disorder characterized by deficiency of clotting factor VIII (hemophilia A) or deficiency of factor IX (hemophilia B) with complex diagnosis and management.
- Trial name
- NCT06636565
- Testing
- No intervention
- Where
- Turkey (Türkiye)
- Volunteers
- 34 people, healthy volunteers welcome
- Sponsor
- Hacettepe University
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
Hemophilia A
Comparison of Different Prophylaxis Regimens for Moderate to Severe Hemophilia A Pediatric Patients
Title: Comparison of different prophylaxis regimens for hemophilia A pediatric patients Principal Investigator: Assistant Professor Darintr Sosothikul, MD Research Question: Does different factor VIII dosage effect outcome of…
- Trial name
- NCT02727647
- Testing
- FVIII
- Where
- Thailand
- Volunteers
- 16 people, healthy volunteers welcome
- Sponsor
- Chulalongkorn University
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
Hemophilia
Are There Differences Between Carriers of Haemophilia A and B?
This study aims to develop a systematic genetic screening strategy for (potential) female carriers of haemophilia by identifying as many female carriers as possible within the families of haemophilia patients regularly followed…
- Trial name
- NCT05217992
- Testing
- Genetic assessment (hemophilia testing)
- Where
- Belgium
- Volunteers
- 900 people, healthy volunteers welcome
- Sponsor
- Cliniques universitaires Saint-Luc- Université Catholique de Louvain
Hemophilia A
Health-Related Quality of Life Outcomes and Bleeding Rates Among Patients With Severe Hemophilia A on Emicizumab
The main purposes of this study are to determine if use of emicizumab prophylaxis treatment reduces number of bleeding episodes, if it improves quality of life of individuals and if improves arthropathy in persons with hemophilia…
- Trial name
- ML43518 (NCT06104826)
- Testing
- Emicizumab
- Where
- United States
- Volunteers
- 50 people, healthy volunteers welcome
- Sponsor
- Newark Beth Israel Medical Center
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
Hemophilia A, Hemophilia B
Global Hemostatic Methods in Hemophilia and Von Willebrand's Disease
Patients with hemophilia who have the same level of deficient factor(s) may express different severity of clinical presentation and bleeding tendency.
- Conditions
- Hemophilia A, Hemophilia B, Von Willebrand's Disease
- Trial name
- GHMHW (NCT02061033)
- Where
- Sweden
- Volunteers
- 180 people, healthy volunteers welcome
- Sponsor
- Karolinska University Hospital
Hemophilia A, Hemophilia B
Gait Evaluation in Haemophiliac Patients
Joint damage secondary to recurrent haemarthroses and chronic synovitis represents the commonest clinical manifestation of haemophilia.
- Trial name
- GAIT_GENERAL (NCT00824798)
- Where
- Belgium
- Volunteers
- 31 people, healthy volunteers welcome
- Sponsor
- Cliniques universitaires Saint-Luc- Université Catholique de Louvain
Severe Hemophilia A Without Inhibitor, Joint Bleed
Comparison of Outcomes Between Low Dose Emicizumab and Factor VIII in Clinically Severe Hemophilia A
To outcome between low dose Emicizumab and low dose prophylaxis with FVIII concentration To study pharmacokinetic, side effect of low dose Emicizumab
- Trial name
- NCT06155955
- Testing
- "Emicizumab", "HEMLIBRA®"
- Where
- Thailand
- Volunteers
- 15 people, healthy volunteers welcome
- Sponsor
- Chulalongkorn University
Congenital Adrenal Hyperplasia, Familial Hyperinsulinemic Hypoglycemia 1
Baby Detect : Genomic Newborn Screening
Newborn screening (NBS) is a global initiative of systematic testing at birth to identify babies with pre-defined severe but treatable conditions.
- Conditions
- Congenital Adrenal Hyperplasia, Familial Hyperinsulinemic Hypoglycemia 1, Phosphoglucomutase 1 Deficiency, Maturity Onset Diabetes of the Young
- Trial name
- BabyDetect (NCT05687474)
- Where
- Belgium
- Volunteers
- 6,824 people, healthy volunteers welcome
- Sponsor
- Centre Hospitalier Universitaire de Liege
Spinal Muscular Atrophy, Fragile X Syndrome
Early Check: Expanded Screening in Newborns
Early Check provides voluntary screening of newborns for a selected panel of conditions. The study has three main objectives: 1) develop and implement an approach to identify affected infants, 2) address the impact on infants and…
- Conditions
- Spinal Muscular Atrophy, Fragile X Syndrome, Fragile X - Premutation, Duchenne Muscular Dystrophy
- Trial name
- NCT03655223
- Testing
- Confirmatory Testing
- Where
- United States
- Volunteers
- 30,000 people, healthy volunteers welcome
- Sponsor
- RTI International
Coronavirus Infection (COVID-19), Pulmonary Arterial Hypertension
Pharmacokinetics, Pharmacodynamics, and Safety Profile of Understudied Drugs Administered to Children Per Standard of Care (POPS)
The study investigators are interested in learning more about how drugs, that are given to children by their health care provider, act in the bodies of children and young adults in hopes to find the most safe and effective dose…
- Conditions
- Coronavirus Infection (COVID-19), Pulmonary Arterial Hypertension, Urinary Tract Infections in Children, Hypertension
- Trial name
- POPS or POP02 (NCT04278404)
- Testing
- The POP02 study is collecting bodily fluid samples (i.e., whole blood, effluent samples) of children prescribed the following drugs of interest per standard of care:
- Where
- 52 sites in United States, Canada
- Volunteers
- 5,000 people, healthy volunteers welcome
- Sponsor
- Duke University