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Thalassemia Major clinical trials

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NCT ID: NCT06137079 Recruiting - Iron Overload Clinical Trials

"Iron Overload and Endocrinological Diseases"

Start date: June 20, 2013
Phase:
Study type: Observational

Patients with hemochromatosis or Thalassemia develop progressive tissue and organs damages secondary to iron overload. Iron overload can result both from transfusional hemosiderosis and excess gastrointestinal iron absorption. Iron deposition in the heart, liver, and multiple endocrine glands results in severe damage to these organs, with variable degrees of endocrine and organ failure. Although patients with iron overload often present endocrine disorders, the pathogenetic mechanisms underlying endocrinopathies are not completely clear. In particular it is not elucidated if the spectrum of endocrinopathies could change with advancing age. All endocrinological comorbidities can develop from a primary damage of the target gland, from pituitary secondary failure or from both. The aim of this study is to investigate the prevalence of endocrinological diseases in adult patients with iron overload due to β-thalassemia or hemochromatosis and their impact on well-being and quality of life. The study design is a prospective cross-sectional clinical study. All subjects enrolled will be evaluated for the endocrine diseases. The study protocol will include data collection from family and patients' history of diseases, physical examination, hormonal assessment for all endocrine axes and instrumental examinations. The results will provide evidence on the prevalence of endocrine diseases in patients with iron overload and will add information to characterize the type and the degree of endocrine deficiencies, and on the pathogenic mechanisms involved, in order to individualize diagnostic and therapeutic approaches.

NCT ID: NCT06041620 Recruiting - Thalassemia Major Clinical Trials

Safety and Efficacy Evaluation of Autologous CRISPR-Cas12b Edited Hematopoietic Stem Cells

Start date: August 31, 2023
Phase: N/A
Study type: Interventional

This is a single-arm, open, single-injection exploratory clinical study with two transfusion-dependent β thalassemia (β-TDT) participants planned to enroll.

NCT ID: NCT06026839 Not yet recruiting - Acute Leukemia Clinical Trials

Longitudinal Study on the QoL of Pediatric Patients After HSCT and Its Influencing Factors

Start date: October 30, 2023
Phase:
Study type: Observational

Objective: To longitudinally track the dynamic changes in the survival quality of pediatric patients after hematopoietic stem cell transplantation at different time points within 1 year post-transplantation, analyze the influencing factors of survival quality at each time point, identify independent risk factors that can be intervened, provide reference for medical staff to recognize survival quality problems early, guide the dynamic management of clinical survival quality, and formulate continuation care management plans. Methods: This study adopted a repeated measurement study design. A total of 250 pediatric patients who underwent hematopoietic stem cell transplantation in three tertiary hospitals in Guangdong Province from August 2023 to December 2025 and met the research standards were selected as the research subjects. The "Childhood Health Assessment Questionnaire Transplant Module 3.0 Chinese Version" was used to evaluate the survival quality of the patients at six time points: 1 week before pre-treatment (T0), the day of stem cell infusion (T1), 1 month (T2), 3 months (T3), 6 months (T4), and 1 year (T5) after transplantation. Statistical methods for repeated measures were used to analyze the relevant information, and mixed-effect linear models were used to analyze the influencing factors of survival quality at the six time points, and to identify independent risk factors.

NCT ID: NCT05777733 Not yet recruiting - Thalassemia Major Clinical Trials

NAC Effect on Iron Overload and Blood Transfusion in β-thalassemia Major

Start date: March 23, 2024
Phase: Phase 1
Study type: Interventional

The effect of N_acetylcystein as an antioxidant on iron overload and frequency of blood transfusion in β-thalassemia major patients at Assiut Childern Hospital University And its cosubmitted for partial fulfillment of master degree in Pediatrics

NCT ID: NCT05462548 Recruiting - Thalassemia Major Clinical Trials

The Safety and Efficiency of Luspatercept in Chinese Adults With Transfusion Dependent β-thalassemia: a Real-world Study

Start date: July 2022
Phase: Phase 4
Study type: Interventional

This is a prospective, single-arm, open-label study. Twenty adult patients with transfusion-dependent β -thalassemia will be enrolled to receive Luspatercept with optimal supportive care, including blood transfusion and iron removal, based on the clinician's judgment and practice. The main objective of this study was to evaluate the efficacy and safety of Luspatercept in the treatment of adult patients with transfusion-dependent β -thalassaemia in Chinese clinical practice, and to provide evidence reference for subsequent clinical use.

NCT ID: NCT05444894 Recruiting - Hemoglobinopathies Clinical Trials

EDIT-301 for Autologous Hematopoietic Stem Cell Transplant (HSCT) in Participants With Transfusion-Dependent Beta Thalassemia (TDT)

Start date: April 29, 2022
Phase: Phase 1/Phase 2
Study type: Interventional

The purpose of this study is to evaluate the safety, tolerability, and efficacy of treatment with EDIT-301 in adult participants with Transfusion Dependent beta Thalassemia

NCT ID: NCT05442346 Suspended - Thalassemia Major Clinical Trials

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

Start date: December 25, 2023
Phase: N/A
Study type: Interventional

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.

NCT ID: NCT05303506 Not yet recruiting - Thalassemia Major Clinical Trials

Study on the Effect of Different Risk Factors on the Growth Parameters of Thalassemic Patients in Assiut (AUCH)

Start date: July 2, 2022
Phase:
Study type: Observational

To assess the effect of different risk factors on the growth parameters of thalassemic patients in Assiut University children Hospital (AUCH) In order to help in decreasing the morbidity and mortality resulting from iron overload and improving the quality of life for thalassemic patient

NCT ID: NCT04526405 Completed - Covid19 Clinical Trials

Frequency of COVID-19 Antibodies in Patients With Hereditary Hematological Diseases

ThalaCoV
Start date: July 14, 2020
Phase:
Study type: Observational

In Italy there are about 5000 patients with dependent transfusion thalassemia (source Italian Thalassemia and Hemoglobinopathies - SITE) and a smaller number, currently not definable, of patients with sickle cell anemia in chronic transfusion. A recent study in the Lombardy region identified the positivity of anti-Covid-19 antibodies in 4.5-7% of asymptomatic donors (Valenti L et al). As already known, a preliminary study conducted in Italy (Motta I et al, Hussain FA et al, Taher A et al) reported only 11 cases of symptomatic infection all with benign evolution. Currently there are 15 reported cases (12 thalassemias and 3 sickle cell anemias). 75% of the cases have been identified in Lombardy. Our hypothesis is that in a percentage of polytransfused patients a transmission of the virus may have developed that stimulated the production of protective antibodies. This could be an explanation of the low contagiousness and severity of the infection in polytransfused patients. Currently no data are available for this purpose. This study will be conducted in collaboration with the Microbiology Unit and involves the determination of SARS-CoV-2 antibodies (anti-s1 and s2) by CLIA method with a high sensitivity (94.7%) and specificity (98.5%).

NCT ID: NCT04523376 Active, not recruiting - Sickle Cell Disease Clinical Trials

Pilot Study PBSCT With TCRab Depletion For Hemoglobinopathies

Start date: May 14, 2020
Phase: N/A
Study type: Interventional

This is a single arm pilot study of peripheral stem cell transplantation (PSCT) with ex vivo t-cell receptor alpha beta+(TCRαβ+) T cell and cluster of differentiation 19+ beta (CD19+ B) cell depletion of unrelated donor (URD) grafts using the CliniMACS device in patients with sickle cell disease (SCD) and beta thalassemia major (BTM).