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Beta Thalassemia Major clinical trials

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NCT ID: NCT06466304 Recruiting - Clinical trials for Beta Thalassemia Major

Inspiratory Muscle Training in Children With Beta Thalasemia

Start date: June 6, 2024
Phase: N/A
Study type: Interventional

respiratory complications are very prevalent in children with beta thalasemia major (BTM), so choosing inspiratory muscle trianing to decrease of prevent it and to improve pulmoanry funntions is important

NCT ID: NCT06291961 Recruiting - Clinical trials for Beta-Thalassemia Major

A Safety and Efficacy Study Evaluating CS-101 in Subjects With β-Thalassemia Major

Start date: April 18, 2024
Phase: Phase 1
Study type: Interventional

The goal of this open label, single-arm clinical study is to learn about the safety and efficacy of CS-101 in treating patients with β-thalassemia major anemia.

NCT ID: NCT04776850 Withdrawn - Sickle Cell Disease Clinical Trials

Pre-transplant Immunosuppression and Donor Stem Cell Transplant for the Treatment of Severe Hemoglobinopathies

Start date: December 29, 2020
Phase: Early Phase 1
Study type: Interventional

This clinical trial studies the effect of pre-transplant immunosuppression (PTIS) and donor stem cell transplant in treating patients with severe blood diseases (hemoglobinopathies). PTIS helps prepare the body for the transplant and lowers the risk of developing graft versus host disease (GVHD). Hematopoietic cells are found in the bone marrow and produce blood cells. Hematopoietic cell transplantation (HCT) injects healthy hematopoietic cells into the body to support blood cell production. PTIS and HCT may help to control severe hemoglobinopathies.

NCT ID: NCT04353986 Recruiting - HCV Infection Clinical Trials

PK of SOF/LED in HCV - Infected Adolescents With Haematological Disorders

Start date: June 11, 2018
Phase: Phase 3
Study type: Interventional

This is a prospective, controlled, open-label, pharmacokinetic study. This study aims at studying the PK of sofosbuvir, ledipasvir and sofosbuvir metabolite (GS-331007) in HCV infected children with hematological Disorders. to develop predictive pharmacokinetic model for the 3 moieties in the studied population. In this study, patients in both treatment groups will receive 12 weeks of treatment with a fixed-dose combination tablet containing 400 mg of sofosbuvir and 90 mg of ledipasvir(SOF/LED) orally, once daily with food.

NCT ID: NCT04092205 Completed - Clinical trials for Beta Thalassemia Major

Phase 2a Pilot Study of NBMI Treatment in Patients With Beta Thalassemia Major, Requiring Iron Chelation

EMERA007
Start date: November 21, 2019
Phase: Phase 2
Study type: Interventional

A pilot study to explore safety and efficacy of NBMI treatment in patients with Beta Thalassemia Major requiring iron chelation Investigational product: NBMI (N1,N3-bis(2-mercaptoethyl) isophthalamide), INN: Emeramide Indication: Beta Thalassemia Major

NCT ID: NCT03947632 Completed - Clinical trials for Beta Thalassemia Major

Anthropometric Measurements in Children Having Transfusion-dependent Beta Thalassemia

Start date: January 1, 2013
Phase:
Study type: Observational [Patient Registry]

The aim of this study was to determine the anthropometric measurements in transfusion-dependent β-thalassemia children in Pakistan. The secondary aim was to correlate serum ferritin with the physical growth

NCT ID: NCT03653338 Recruiting - Sickle Cell Anemia Clinical Trials

T-Cell Depleted Alternative Donor Bone Marrow Transplant for Sickle Cell Disease (SCD) and Other Anemias

Start date: August 2, 2018
Phase: Phase 1/Phase 2
Study type: Interventional

The purpose of this study is to evaluate what effect, if any, mismatched unrelated volunteer donor and/or haploidentical related donor stem cell transplant may have on severe sickle cell disease and other transfusion dependent anemias. By using mismatched unrelated volunteer donor and/or haploidentical related donor stem cells, this study will increase the number of patients who can undergo a stem cell transplant for their specified disease. Additionally, using a T-cell depleted approach should reduce the incidence of graft-versus-host disease which would otherwise be increased in a mismatched transplant setting.

NCT ID: NCT03276455 Not yet recruiting - Clinical trials for Beta Thalassemia Major

Gene Therapy for Beta-Thalassemia Major Using Autologous Hematopoietic Stem Cell Genetically Modified

Start date: September 15, 2017
Phase: Phase 1/Phase 2
Study type: Interventional

This is a single group, open label study in 10 subjects who are 8 years of age or older with beta-thalassemia major. The objective of this study is to evaluate the safety and efficacy of autologous hematopoietic stem cell transduced with lentiviral vector for the treatment of beta-thalassemia major.

NCT ID: NCT03101423 Active, not recruiting - Clinical trials for Beta Thalassemia Major

Monitoring of Chimerism After Transplantation in Patients With β Thalassemia Major and the Treatment Strategies for the Reduction of Chimerism

Start date: August 1, 2016
Phase: N/A
Study type: Interventional

Hematopoietic stem cell transplantation is currently the only way to cure thalassemia, one of its main obstacles is the rejection after transplantation, chimerism continued to decline, which eventually lead to transplant failure. chimerism is a key indicator of the succession of immune response, which is a key indicator for predicting the failure of hematopoietic stem cell transplantation and provides an important basis for early detection of rejection. Transplantation of continuous chimerism can detect early unstable chimeras and rejection.The chimerism rates after transplantation were continuously monitored using fluorescence labeled multiplex PCR amplification of short tandem repeats (STR-PCR) ,and then follow our STR different rates for early interventional therapy to prevent further reduction in chimerism leading to lead to graft failure.

NCT ID: NCT02744560 Completed - Clinical trials for Beta Thalassemia Major

Effect of Spirulina on Liver Iron Concentration in Beta Thalassemic Children With Hepatitis C

Start date: March 2015
Phase: N/A
Study type: Interventional

Thalassemics can develop liver fibrosis because of iron overload and hepatitis C infection. The latter is the main risk factor for liver fibrosis in transfusion dependent thalassemics. Excess liver iron is clearly recognized as a co factor for the development of advanced fibrosis in patients with hepatitis virus C infection. Magnetic resonance imaging represents the most available noninvasive technique to assess the level of iron in the liver.there is evidence that suggests Spirulina may help to protect against liver damage, cirrhosis and liver failure in those with chronic liver disease.