Clinical trials: Congenital Bleeding Disorder
Premature Infants, Intracranial Hemorrhages
Effect of Erythropoietin in Premature Infants on White Matter Lesions and Neurodevelopmental Outcome
Preterm and very preterm infants are at risk of developing encephalopathy of prematurity and long-term neurodevelopmental delay.
- Conditions
- Premature Infants, Intracranial Hemorrhages, Periventricular Leukomalacia, Cerebral Palsy
- Trial name
- NCT03110341
- Testing
- Erythropoietin, Normal saline
- Where
- China
- Volunteers
- 400 people
- Sponsor
- First Affiliated Hospital Xi'an Jiaotong University
Haemophilia
Multidisciplinary Evaluation of Patients With Hemophilia
Multidisciplinary assessment of the physical, functional and psychosocial alterations in patients with hemophilia around the country.
- Trial name
- HOLISTIC (NCT02198430)
- Where
- Spain
- Volunteers
- 104 people
- Sponsor
- Universidad de Murcia
Postpartum Hemorrhage, Neonatal Asphyxia
Saving Lives at Birth in Uganda: Building and Sustaining Capacity of Frontline Health Workers - A Program Evaluation
The study aims are to evaluate the impact of supportive follow-up strategies for a novel twinned training and capacity-sustaining program among frontline health workers (providers) attending facility-based births in remote and…
- Trial name
- NCT03254628
- Testing
- Helping Mothers Survive - Bleeding After Birth training, Helping Babies Breathe, Mentor
- Volunteers
- 3,440 people
- Sponsor
- Jhpiego
Hemophilia A
Effects of Emicizumab vs. Factor VIII Prophylaxis on Joint and Bone Health in Severe Hemophilia A
The investigators propose to study longitudinal joint and bone density changes in patients with severe Hemophilia A. Per current standard of care, most patients are on prophylactic FVIII replacement therapy intravenously several…
- Trial name
- EmiMSK (NCT04131036)
- Testing
- assessment of joint health and bone density
- Where
- 4 sites in United States
- Volunteers
- 40 people
- Sponsor
- Washington Institute for Coagulation
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
Hereditary Cystatin C Amyloid Angiopathy (HCCAA), Hemorrhage Brain
Safety, Tolerability and Efficacy of NPI-001 in Patients With Hereditary Cystatin C Amyloid Angiopathy (HCCAA)
The study aims to measure safety, tolerability and biomarker-based efficacy of NPI-001 (AT-001) in Subjects with Hereditary Cystatin C Amyloid Angiopathy (HCCAA).
- Conditions
- Hereditary Cystatin C Amyloid Angiopathy (HCCAA), Hemorrhage Brain, Hemorrhage Cerebral
- Trial name
- HCCAA (NCT07716865)
- Testing
- NACA
- Where
- Iceland
- Volunteers
- 15 people
- Sponsor
- Arctic Therapeutics
Severe Hemophilia A
Study to Investigate the Long-term Efficacy and Safety of Human-cl rhFVIII in Previously Treated Patients (PTPs)
The purpose of the study is to study the long-term efficacy, safety and tolerability of Human-cl rhFVIII in previously treated patients with severe hemophilia A.
- Trial name
- NCT01341912
- Testing
- Human-cl rhFVIII
- Where
- 2 sites in United States, Bulgaria
- Volunteers
- 3 people
- Sponsor
- Octapharma
Autoimmune Diseases, Lupus Erythematosus, Systemic
Comparative Autoantibody and Immunologic Cell Marker Study
The purpose of this study is to compare immune phenotype, function, and specificity of B lymphocytes from different developmental stages in autoimmune patients to B cells from infectious disease patients and healthy controls.
- Conditions
- Autoimmune Diseases, Lupus Erythematosus, Systemic, Communicable Diseases
- Trial name
- NCT02422875
- Where
- 3 sites in United States
- Volunteers
- 2,500 people, healthy volunteers welcome
- Sponsor
- Emory University
Trauma, Bleeding Disorder
Early Administration of Fibrinogen in Polytraumatized Patients With Hypofibrinogenemia: a Randomized Feasibility Trial
This is a randomized feasibility trial conducted with severe trauma patients. At admission patients presented hypofibrinogenemia, hypotension and tachycardia.
- Conditions
- Trauma, Bleeding Disorder, Fibrinogen; Deficiency, Acquired
- Trial name
- NCT02864875
- Testing
- Fibrinogen concentrate
- Where
- Brazil
- Volunteers
- 32 people
- Sponsor
- University of Sao Paulo General Hospital
Severe Haemophilia A
Assess the Safety and Efficacy of Individually Tailored Prophylaxis With Human-cl rhFVIII in Patients With Severe Haemophilia A
To compare the number of breakthrough bleeds under tailored prophylaxis with Human cell line recombinant factor FVIII (Human-cl rhFVIII) with the historical bleeding rate from patients who received Human-cl rhFVIII as on demand…
- Trial name
- NCT01863758
- Testing
- Human-cl rhFVIII
- Where
- 20 sites in Austria, Bulgaria and 6 more
- Volunteers
- 66 people
- Sponsor
- Octapharma
Hemophilia B
A Phase 1/2 Study of SHP648, an Adeno-Associated Viral Vector for Gene Transfer in Hemophilia B Subjects
The purpose of this study is to evaluate the safety and dose escalation of SHP648 an adeno-associated viral vector for gene transfer in hemophilia B participants.
- Trial name
- NCT04394286
- Testing
- SHP648
- Where
- 2 sites in Spain, Turkey (Türkiye)
- Volunteers
- 2 people
- Sponsor
- Baxalta now part of Shire
Hemophilia A
Safety and Efficacy Study of a Recombinant and Protein-Free Factor VIII (rAHF-PFM) in Hemophilia A Patients Undergoing Surgery
The purpose of this study is to evaluate whether rAHF-PFM is safe and effective in the treatment of hemophilia A patients undergoing surgery.
- Trial name
- NCT00157105
- Testing
- Antihemophilic factor, recombinant, manufactured protein-free
- Where
- 10 sites in United States
- Volunteers
- 59 people
- Sponsor
- Baxalta now part of Shire
Giant Cell Arteritis, Takayasu's Arteritis
Reproductive Health in Men and Women With Vasculitis
The purpose of this study is to learn about reproductive health, including fertility and pregnancies, in people with vasculitis.
- Conditions
- Giant Cell Arteritis, Takayasu's Arteritis, Polyarteritis Nodosa, Wegener's Granulomatosis
- Trial name
- NCT02176070
- Where
- United States
- Volunteers
- 467 people
- Sponsor
- University of South Florida
Intracranial Hemorrhage, Traumatic, Intracranial Hemorrhage, Spontaneous
FFP Versus PCC in Intracranial Hemorrhage
The goal of this study will be to determine whether PCC confers any benefits over FFP in traumatic and spontaneous intracranial hemorrhage with respect to multiple factors including time to correction, absolute international…
- Trial name
- NCT02429453
- Testing
- Four Factor Prothrombin Complex Concentrate, Fresh Frozen Plasma
- Where
- United States
- Sponsor
- University of Utah
Hemophilia, Arthropathy
Observational Study of Long-Term Joint Health Outcome in Hemophilia Patient
Patients with severe hemophilia A can experience repeated bleeding into the same joint (ie, a target joint; most frequently in the ankle, knee, and elbow), which can contribute to hemophilic arthropathy and, over time, ultimately…
- Trial name
- NCT04618237
- Where
- Taiwan
- Volunteers
- 35 people
- Sponsor
- Taipei Medical University Hospital
Hemophilia A
Evaluation of a Standardized Protocol for Thrombin Generation Assay
This study is aims at determining the inter laboratory variation when using the thrombin generation assay calibrated automated thrombogram (TGA CAT). It is thus, not a clinical trial in its usual meaning.
- Trial name
- NCT03313531
- Testing
- Thrombin Generation Assay, Treatment with recombinant coagulation factor
- Where
- Sweden
- Volunteers
- 9 people, healthy volunteers welcome
- Sponsor
- Lund University
Hemophilia A, Hemophilia B
Web-based Application for the Population Pharmacokinetic Service - Phase 1
The aims of this trials are: 1. to collect published and unpublished individual classic pharmacokinetic data (individual patient data from independent investigators and pharmaceutical companies) 2.
- Trial name
- WAPPS (NCT02061072)
- Testing
- Population pharmacokinetic estimation
- Where
- Canada
- Volunteers
- 400 people
- Sponsor
- McMaster University
Idiopathic Pulmonary Fibrosis
23andMe IPF Research Study
The long term goal of this study is to increase genetic understanding of IPF to enable the development of an effective drug for IPF that can improve the lives of those living with the condition.
- Trial name
- IPF (NCT05028894)
- Testing
- No intervention
- Where
- United States
- Volunteers
- 690 people
- Sponsor
- 23andMe, Inc.
Congenital Bleeding Disorder, Haemophilia A
To Quantify the Range of Main Psychosocial Factors Affecting Patients and Caregivers in Their Daily Lives
This survey is conducted in South America. The purpose is to identify the key psychosocial issues affecting patients with haemophilia.
- Conditions
- Congenital Bleeding Disorder, Haemophilia A, Haemophilia B, Haemophilia A With Inhibitors
- Trial name
- HERO (NCT02241694)
- Testing
- No treatment given
- Where
- Brazil
- Volunteers
- 200 people
- Sponsor
- Novo Nordisk A/S
Gene Therapy, Hemophilia B
Needs Assessment of Knowledge, Beliefs, and Attitudes of Patients With Hemophilia B About Gene Therapy
Gene therapy is a paradigm-shifting treatment for hemophilia B patients, particularly in resource-limited countries where factor availability remains low.
- Trial name
- NCT05044845
- Testing
- Interview
- Where
- 6 sites in United States, Nepal and 4 more
- Volunteers
- 150 people
- Sponsor
- St. Jude Children's Research Hospital