Clinical trials: Glanzmann Thrombasthenia
Glanzmann Thrombasthenia
Eptacog Beta in Glanzmann's (HeT_LFB-Strength-Study_FID531)
This study is evaluating an investigational drug, eptacog beta (EB), for the treatment and prevention of acute bleeding episodes in people with Glanzmann Thrombasthenia, a rare inherited bleeding disorder.
- Trial name
- STRENGTH (NCT07136857)
- Testing
- EPTACOG BETA
- Where
- United States
- Volunteers
- 6 people
- Sponsor
- Emory University
Glanzmann Thrombasthenia
A Phase 1/2 Study to Investigate Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Efficacy of HMB-001 in Glanzmann Thrombasthenia
The goal of this clinical trial is to Investigate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Efficacy of HMB-001 in Participants with Glanzmann Thrombasthenia.
- Trial name
- NCT06211634
- Testing
- HMB-001
- Where
- 17 sites in United States, Belgium and 4 more
- Volunteers
- 57 people
- Sponsor
- Hemab ApS
Congenital Bleeding Disorder, Glanzmann's Disease
Observational Registry of the Treatment of Glanzmann's Thrombasthenia
This observational registry is conducted in Europe, Asia, Africa and the United States of America (USA). The purpose of the registry is to evaluate the efficacy and safety of activated recombinant human factor VII (rFVIIa) during…
- Trial name
- NCT01476423
- Testing
- activated recombinant human factor VII
- Where
- 15 sites in United States, Algeria and 13 more
- Volunteers
- 218 people
- Sponsor
- Novo Nordisk A/S
Hemophilia A With Inhibitor, Hemophilia B With Inhibitor
Prospective, Non-interventional Study to Evaluate Immunogenicity of AryoSeven
This study evaluates the immunogenicity of the biosimilar rFVIIa (AryoSeven) in subjects receiving AryoSeven in real-life clinical practice.
- Conditions
- Hemophilia A With Inhibitor, Hemophilia B With Inhibitor, Factor VII Deficiency, Glanzmann's Thrombasthenia
- Trial name
- NCT03372993
- Where
- 2 sites in Iran
- Volunteers
- 200 people
- Sponsor
- AryoGen Pharmed Co.
Congenital Bleeding Disorder, Glanzmann's Disease
A Study to Evaluate Safety and Efficacy of NovoSeven® in Patients With Glanzmann's Thrombasthenia in Japan
This study is conducted in Asia. The aim of the study is to evaluate the safety and efficacy of NovoSeven® (activated recombinant factor VII, eptacog alfa (activated)) during bleeding episodes and for the prevention of bleeding…
- Trial name
- NCT01876745
- Testing
- eptacog alfa (activated)
- Where
- Japan
- Volunteers
- 4 people
- Sponsor
- Novo Nordisk A/S
Hemophilia, Thrombosis
ATHNdataset Registry
The Hemophilia Treatment Center (HTC) where you receive care is working with The American Thrombosis and Hemostasis Network (ATHN) to look at the quality of life of people with blood disorders and problems.
- Conditions
- Hemophilia, Thrombosis, Hemophilia A, Hemophilia B
- Trial name
- NCT06820515
- Where
- United States
- Volunteers
- 200,000 people
- Sponsor
- American Thrombosis and Hemostasis Network
Acquired Bleeding Disorder, Acquired Haemophilia
Bioequivalence of NovoSeven® and a NovoSeven® Formulation Stable at Room Temperature in Healthy Male Subjects
This trial is conducted in Europe. The aim of this trial is to demonstrate bio-equivalence between the marketed activated recombinant human factor VII (NovoSeven®) (CP-rFVIIa) and a new formulation stable at 25°C (VII25).
- Conditions
- Acquired Bleeding Disorder, Acquired Haemophilia, Congenital Bleeding Disorder, Congenital FVII Deficiency
- Trial name
- NCT01561417
- Testing
- activated recombinant human factor VII
- Where
- France
- Volunteers
- 28 people
- Sponsor
- Novo Nordisk A/S
Glanzmann Thrombasthenia
Multinational Glanzmann Study
Glanzmann thrombasthenia is a rare autosomal recessive platelet disorder characterized by a lack of functional integrins alfaIIb or beta3 (glycoproteins IIb/IIIa).
- Trial name
- Glanzmann-NHS (NCT06204042)
- Volunteers
- 200 people
- Sponsor
- UMC Utrecht
Glanzmann Thrombasthenia
The Genetics and Functional Basis of Inherited Platelet, White Blood Cell, Red Blood Cell, and Blood Clotting Disorders.
Blood contains red blood cells, white blood cells, and platelets, as well as a fluid portion termed plasma. We primarily study blood platelets, but sometimes we also analyze the blood of patients with red blood cell disorders…
- Trial name
- NCT00230165
- Where
- United States
- Volunteers
- 60 people, healthy volunteers welcome
- Sponsor
- Rockefeller University
Hurler Syndrome, Fanconi Anemia
Bone Marrow Transplant With Abatacept for Non-Malignant Diseases
This is a single arm, phase I study to assess the tolerability of abatacept when combined with cyclosporine and mycophenolate mofetil as graft versus host disease prophylaxis in children undergoing unrelated hematopoietic stem…
- Conditions
- Hurler Syndrome, Fanconi Anemia, Glanzmann Thrombasthenia, Wiskott-Aldrich Syndrome
- Trial name
- NCT01917708
- Testing
- Abatacept
- Where
- United States
- Volunteers
- 10 people
- Sponsor
- Emory University
Glanzmann Thrombasthenia
The Experiences of People Who Live With Glanzmanns Thrombasthenia.
To understand the lived experiences of people with Glanzmanns Thrombasthenia
- Trial name
- NCT05315232
- Testing
- survey and interview
- Where
- United Kingdom
- Volunteers
- 122 people
- Sponsor
- Haemnet
Congenital Bleeding Disorder, Congenital FVII Deficiency
Observational Study on the Efficacy and Safety of NovoSeven® During "Real-life" Usage in Germany
This NON INTERVENTIONAL OBSERVATIONAL STUDY is conducted in Europe. The primary aim is to observe the haemostatic efficacy of NovoSeven® treatment during routine practice in German clinics.
- Conditions
- Congenital Bleeding Disorder, Congenital FVII Deficiency, Glanzmann's Disease, Acquired Bleeding Disorder
- Trial name
- WIRK (NCT00697320)
- Testing
- eptacog alfa (activated)
- Where
- Germany
- Volunteers
- 64 people
- Sponsor
- Novo Nordisk A/S
Glanzmann Thrombasthenia
Anti-αIIbβ3 Immunization in Glanzmann Thrombasthenia: Prevalence and Associated Risk Factors: Thrombasthenia Anti-αIIbβ3 Antibodies Study (TAAS)
This project aim to correlate risk factors (genetic, therapeutic and socio-demographic factors) to anti-αIIbβ3 antibodies formation following blood products transfusion (platelets or packed red cells) or pregnancy in a national…
- Trial name
- TAAS (NCT04595617)
- Testing
- Antibodies screening
- Where
- 7 sites in France
- Volunteers
- 28 people
- Sponsor
- University Hospital, Bordeaux
Sickle Cell Disease, Transfusion Dependent Alpha- or Beta- Thalassemia
Hematopoietic Stem Cell Transplant for High Risk Hemoglobinopathies
This is a study to collect the outcomes of stem cell transplantation for patients with hematologic diseases other than cancer.
- Conditions
- Sickle Cell Disease, Transfusion Dependent Alpha- or Beta- Thalassemia, Diamond Blackfan Anemia, Paroxysmal Nocturnal Hemoglobinuria
- Trial name
- NCT02179359
- Testing
- Reduced Toxicity Ablative Regimen, Reduced Intensity Preparative Regimen, Myeloablative Preparative Regimen
- Where
- United States
- Volunteers
- 38 people
- Sponsor
- Masonic Cancer Center, University of Minnesota
Accelerated Phase Chronic Myelogenous Leukemia, BCR-ABL1 Positive, Acute Leukemia in Remission
Fludarabine Phosphate, Cyclophosphamide, Total Body Irradiation, and Donor Stem Cell Transplant in Treating Patients With Blood Cancer
This phase II trial studies how well fludarabine phosphate, cyclophosphamide, total body irradiation, and donor stem cell transplant work in treating patients with blood cancer.
- Conditions
- Accelerated Phase Chronic Myelogenous Leukemia, BCR-ABL1 Positive, Acute Leukemia in Remission, Acute Lymphoblastic Leukemia, Acute Myeloid Leukemia
- Trial name
- NCT03333486
- Testing
- Cyclophosphamide, Fludarabine Phosphate, Laboratory Biomarker Analysis
- Where
- United States
- Volunteers
- 31 people
- Sponsor
- Roswell Park Cancer Institute
Von Willebrand Diseases, Glanzmann Thrombasthenia
Videomicroscopy for the Prediction of Bleeding in Constitutional Haemorrhagic Diseases
In Willebrand disease, there is currently no test available to identify non-invasively patients with a high risk of bleeding from angiodysplasias The study propose to use a sublingual capillary bed analysis by video-microscopy, a…
- Trial name
- VIDEO-BLEED (NCT04119908)
- Testing
- Sublingual videomicroscopy, blood sample
- Where
- France
- Volunteers
- 400 people
- Sponsor
- University Hospital, Lille
Glanzmann Thrombasthenia
Targeting TFPI With Concizumab to Improve Haemostasis in Glanzmann Thrombasthenia Patients: an in Vitro Study
Glanzmann thrombasthenia is a rare genetic disorder caused by the absence or the dysfunction of the main receptor present on the surface of platelets, integrin αIIbβ3 or GPIIb-IIIa.
- Trial name
- GLAT (NCT06234813)
- Testing
- Clot formation in whole blood under flow in a microfluidic flow chamber coated with tissue factor and collagen, : PRP viscoelastic changes under clot formation measured by thromboelastometry using RoTEM, Thrombin Generation Assay (TGA) in PRP using TF trigger
- Where
- France
- Volunteers
- 20 people, healthy volunteers welcome
- Sponsor
- University Hospital, Bordeaux
Thalassemia, Sickle Cell Disease
Alefacept and Allogeneic Hematopoietic Stem Cell Transplantation
Allogeneic blood and marrow transplantation remains the only viable cure for children who suffer from many serious non-malignant hematological diseases. Transplantation, however, carries a high risk of fatal complications.
- Conditions
- Thalassemia, Sickle Cell Disease, Glanzmann Thrombasthenia, Wiskott-Aldrich Syndrome
- Trial name
- NCT01319851
- Testing
- Alefacept
- Where
- United States
- Volunteers
- 3 people
- Sponsor
- Emory University
Factor VII Deficiency, Glanzmann Thrombasthenia
Study of Coagulation Factor VIIa Marzeptacog Alfa (Activated) in Subjects With Inherited Bleeding Disorders
The purpose of the trial is to evaluate the PK, bioavailability, PD, efficacy and safety of MarzAA for on demand treatment and control of bleeding episodes in adult subjects with inherited bleeding disorders.
- Conditions
- Factor VII Deficiency, Glanzmann Thrombasthenia, Hemophilia A With Inhibitor
- Trial name
- NCT04548791
- Testing
- Coagulation Factor VIIa variant
- Where
- 20 sites in United States, India and 3 more
- Volunteers
- 19 people
- Sponsor
- Catalyst Biosciences