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Glanzmann Thrombasthenia clinical trials

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NCT ID: NCT06234813 Completed - Clinical trials for Glanzmann Thrombasthenia

Targeting TFPI With Concizumab to Improve Haemostasis in Glanzmann Thrombasthenia Patients: an in Vitro Study

GLAT
Start date: April 6, 2023
Phase: N/A
Study type: Interventional

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. The lack of this protein on the surface of platelets no longer allows these blood cells to bind to each other. This binding corresponds to the process of platelet aggregation. Generally, local measures will control nasal and superficial bleeding whereas platelet transfusions are used to control or prevent life-threatening. The main complication of this treatment is the risk of developing anti-αIIbβ3 antibodies directed against the absent protein and platelet transfusion therapy can become ineffective. Activated recombinant factor VII (rFVIIa) provides an alternative treatment for GT patients who develop such antibodies. However, this therapy has a short duration of efficacy, requiring repeated intravenous administrations every 2 to 3 hours. There is a new treatment, Concizumab, which has not yet been marketed. This treatment acts on TFPI (tissue factor pathway inhibitor). TFPI is a protein that occurs naturally in the body and prevents blood cells from binding to each other. Concizumab works by blocking TFPI, which may allow sufficient clotting to prevent bleeding. This treatment could replace recombinant activated factor VII (rFVIIa) because it has the advantage of a much longer duration of efficacy (about 3 days) and is administered subcutaneously.

NCT ID: NCT06211634 Recruiting - Clinical trials for Glanzmann Thrombasthenia

A Phase 1/2 Study to Investigate Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Efficacy of HMB-001 in Glanzmann Thrombasthenia

Start date: December 13, 2022
Phase: Phase 1/Phase 2
Study type: Interventional

The goal of this clinical trial is to Investigate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Efficacy of HMB-001 in Participants with Glanzmann Thrombasthenia. The main questions it aims to answer are: - Parts A, B, and C: To determine the safety and tolerability of HMB-001 - Part A: To establish the dose level(s) and dosing interval(s) of HMB-001 to be investigated in Parts B and C - Parts B and C: To estimate the ability of HMB-001 to prevent the number and severity of bleeds Part A will assess differing singular doses of HMB-001 in small groups of participants. The dose administered to a newly enrolled participant (or groups of participants) may only increase if analysis of data from previous dosing shows it is safe to do so. The planned duration of participation in Part A is approximately 6 months, which consists of a Screening Period, an optional Run-in Observation Period, and a follow-up period of 8 weeks. Part B is similar to Part A as it involves testing different dose levels of HMB-001 in small groups of participants. However, in Part B, HMB-001 is given multiple times over a 3-month period, either weekly, every 2 weeks, or every 4 weeks. Part B consists of a Screening Period, a Run-in Observation Period, a 3-month Treatment Period, and a Safety Follow-up following the last dose of HMB-001. Part C is open to participants from Part B and consists of approximately a 9-month Treatment Period and a Safety Follow-up following the last dose of HMB-001.

NCT ID: NCT06204042 Not yet recruiting - Clinical trials for Glanzmann Thrombasthenia

Multinational Glanzmann Study

Glanzmann-NHS
Start date: March 1, 2024
Phase:
Study type: Observational

Glanzmann thrombasthenia is a rare autosomal recessive platelet disorder characterized by a lack of functional integrins alfaIIb or beta3 (glycoproteins IIb/IIIa). The prevalence is variously reported to be between 1:200,000 to 1:1,000,000, with substantial geographic variation. The clinical phenotype is dominated by an increased mucocutaneous bleeding tendency. In absence of a primary bleeding prophylaxis, the current treatment of Glanzmann thrombasthenia is mainly focused on prevention or management of bleeding. However, as potential new therapies emerge, clinicians require unbiased, long-term safety and efficacy data for both current treatment and new therapies. We have designed this study to investigate genetic phenotype (ITGA2B and ITGB3 genes) and the prevalence of antibodies against human leucocyte antigen (HLA) and human platelet antigen (HPA), the latter two being a potential consequence of the current golden standard treatment: platelet transfusion. The results of this study will be merged with a longitudinal registry with retrospective and prospective data collection of clinical phenotype, haemorrhagic burden and bleeding management. Analysis of the data from the Glanzmann-NHS+ study and the registry will help us to get a better understanding of the clinical variation among participants with Glanzmann thrombasthenia. The ultimate goal is to accelerate improvement in the care of patients with Glanzmann thrombasthenia.

NCT ID: NCT05315232 Completed - Clinical trials for Glanzmann Thrombasthenia

The Experiences of People Who Live With Glanzmanns Thrombasthenia.

Start date: April 27, 2022
Phase:
Study type: Observational

To understand the lived experiences of people with Glanzmanns Thrombasthenia

NCT ID: NCT04595617 Completed - Clinical trials for Glanzmann Thrombasthenia

Anti-αIIbβ3 Immunization in Glanzmann Thrombasthenia: Prevalence and Associated Risk Factors: Thrombasthenia Anti-αIIbβ3 Antibodies Study (TAAS)

TAAS
Start date: January 6, 2021
Phase: N/A
Study type: Interventional

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 cohort of GT patients.

NCT ID: NCT04548791 Terminated - Clinical trials for Hemophilia A With Inhibitor

Study of Coagulation Factor VIIa Marzeptacog Alfa (Activated) in Subjects With Inherited Bleeding Disorders

Start date: May 17, 2021
Phase: Phase 1/Phase 2
Study type: Interventional

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.

NCT ID: NCT04119908 Recruiting - Clinical trials for Von Willebrand Diseases

Videomicroscopy for the Prediction of Bleeding in Constitutional Haemorrhagic Diseases

VIDEO-BLEED
Start date: May 24, 2023
Phase: N/A
Study type: Interventional

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 sensitive, reproducible and non-invasive technique, to assess whether sublingual capillary density is predictive of hemorrhagic risk for patients with von Willebrand disease.

NCT ID: NCT03333486 Active, not recruiting - Clinical trials for Acute Myeloid Leukemia

Fludarabine Phosphate, Cyclophosphamide, Total Body Irradiation, and Donor Stem Cell Transplant in Treating Patients With Blood Cancer

Start date: December 7, 2017
Phase: Phase 2
Study type: Interventional

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. Drugs used in chemotherapy, such as fludarabine phosphate and cyclophosphamide, work in different ways to stop the growth of cancer cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading. Radiation therapy uses high energy x-rays to kill cancer cells and shrink tumors. Giving chemotherapy and total-body irradiation before a donor peripheral blood stem cell transplant helps stop the growth of cells in the bone marrow, including normal blood-forming cells (stem cells) and cancer cells. It may also stop the patient's immune system from rejecting the donor's stem cells. When the healthy stem cells from a donor are infused into the patient they may help the patient's bone marrow make stem cells, red blood cells, white blood cells, and platelets. The donated stem cells may also replace the patient?s immune cells and help destroy any remaining cancer cells.

NCT ID: NCT02179359 Recruiting - Sickle Cell Disease Clinical Trials

Hematopoietic Stem Cell Transplant for High Risk Hemoglobinopathies

Start date: September 2, 2014
Phase: N/A
Study type: Interventional

This is a study to collect the outcomes of stem cell transplantation for patients with hematologic diseases other than cancer.

NCT ID: NCT01917708 Completed - Sickle Cell Disease Clinical Trials

Bone Marrow Transplant With Abatacept for Non-Malignant Diseases

Start date: January 2014
Phase: Phase 1
Study type: Interventional

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 cell transplant for serious non-malignant diseases as well as to assess the immunological effects of abatacept. Participants will be followed for 2 years.