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Active clinical trials for "beta-Thalassemia"

Results 51-60 of 249

A Long-term Follow-up Study in Subjects Who Received CTX001

Beta-ThalassemiaThalassemia6 more

This is a multi-site, observational study to evaluate the long-term safety and efficacy of CTX001 in subjects who received CTX001 in Study CTX001-111 (NCT03655678) or VX21-CTX001-141 (transfusion-dependent β-thalassemia [TDT] studies) or Study CTX001-121 (NCT03745287) or VX21-CTX001-151 (severe sickle cell disease [SCD] studies; NCT05329649).

Enrolling by invitation3 enrollment criteria

Safety and Efficacy Evaluation of γ-globin Reactivated Autologous Hematopoietic Stem Cells

Thalassemia Major

This is a single arm, open label, single-dose, phase 1/2 study in up to 5 participants with β-thalassemia major.The study will evaluate the safety and efficacy of the treatment with γ-globin reactivated autologous hematopoietic stem cells in subjects with β-thalassemia major.

Suspended21 enrollment criteria

Long - Term Follow Up of Sickle Cell Disease and Beta-thalassemia Subjects Previously Exposed to...

Blood and Lymphatic Diseases

Primary Objectives: Long-term safety of BIVV003 in participants with severe sickle cell disease (SCD) and ST- 400 in participants with transfusion-dependent beta-thalassemia (TDT) Secondary Objectives: Long-term efficacy of the biological treatment effect of BIVV003 in SCD Long-term efficacy of the clinical treatment effect of BIVV003 on SCD-related clinical events Long-term efficacy of the biological treatment effect of ST-400 in TDT Long-term efficacy of the clinical treatment effect of ST-400 in TDT

Enrolling by invitation4 enrollment criteria

Investigating the Mechanistic Effects of Mitapivat in Subjects With Sickle Cell Disease

Sickle Cell AnemiaSickle Cell Thalassemia6 more

Background: Sickle cell disease (SCD) is an inherited blood disorder. The disease affects the ability of red blood cells to carry oxygen; this in turn can injure organs including the heart, lungs, and kidneys. SCD can lead to serious illness and death. Treatments such as bone marrow transplants and gene therapies can cure SCD, but they are not widely available. Current drug treatments for SCD are not always effective. This natural history study will examine how a study drug (mitapivat) affects red blood cells in people with SCD. Objective: To learn how mitapivat affects red blood cells in people with SCD. Eligibility: People with SCD who are enrolled in the parent study, NIH protocol IRB001565-H. Design: Procedures for this study will be done during visits already scheduled for the parent study. Participants will have additional blood drawn during study visits. The additional amount will be about 3.5 teaspoons. Participants will undergo a test called near infrared spectroscopy (NIRS) up to 9 times. Probes will be placed on their skin. A blood pressure cuff will be placed on their arm. The cuff will be filled with air for up to 5 minutes and then released. Participants may be asked to breathe at a certain rate or to hold their breath during these measurements. NIRS measures oxygen levels, blood flow, and the makeup of skin and muscle. Researchers will draw additional information for this study from participants medical records.

Enrolling by invitation2 enrollment criteria

Evaluation of Heart Status in Patients of Beta Thalassemia Using Echocardiogram

Beta Thalassemia

Heart failure from myocardial iron deposition is a severe complication for patients with transfusion-dependent beta thalassemia . Increased cardiac iron content impacts the contractility of cardiomyocytes and can also lead to myocarditis, pericarditis, and arrhythmias. The severity of cardiac dysfunction depends on the amount of iron deposited in the myocardium.Echocardiogram is used as noninvasive method to observe heart status. The objective of this study is to evaluate the heart status in patients of Beta Thalassemia from southern China.

Recruiting2 enrollment criteria

Association of Hb F Level With Clinical Severity of Beta Thalassemia

Beta Thalassemia

The production of Hb F after birth is an important factor in modifying the clinical severity of beta thalassemia because an increased gamma-globin level will bind the additional a-globin and form Hb F. The objective of this project is to evaluate the association of Hb F level with phenotypic diversity of patients with beta thalassemia.

Recruiting2 enrollment criteria

Atrial Fibrillation in Beta-Thalassemia

Atrial FibrillationThalassemia

The study aims to evaluate the clinical, laboratory and instrumental differences that exist between beta-thalassemia patients with atrial fibrillation and those not affected by arrhythmia.

Recruiting7 enrollment criteria

Correlation Between Pulmonary Functions and Physical Fitness in Children With β-thalassemia

β-ThalassemiaPulmonary Function1 more

The aims of this study are: To determine the correlation between pulmonary functions and physical fitness in children with β-thalassemia. To differentiate between different types of B-thalassemia in both pulmonary functions and physical fitness

Recruiting9 enrollment criteria

A Single-Site Tissue Repository Providing Annotated Biospecimens for Approved Investigator-directed...

Age-Related Macular DegenerationAllergies45 more

To collect, preserve, and/or distribute annotated biospecimens and associated medical data to institutionally approved, investigator-directed biomedical research to discover and develop new treatments, diagnostics, and preventative methods for specific and complex conditions.

Recruiting8 enrollment criteria

The Prevelence of HBB c.93-21 G-A in β Thalassemia Patients

Beta-Thalassemia

To design an amplification-refractory mutation system (ARMS) for the DNA diagnosis of the IVS I-110 (G>A) [HBB:c.93-21G˃A] mutation. To detect the prevelence of the mutation among Assiut University Hospital patients. Phenotype/genotype correlation of the mutation.

Recruiting2 enrollment criteria
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