Active clinical trials for "Thalassemia"

This study will evaluate pediatric patients with malignant or non-malignant blood cell disorders who are having a blood stem cell transplant depleted of T cell receptor (TCR) alfa and beta cells that comes from a partially matched family donor. The study will assess whether immune cells, called T cells, from the family donor, that are specially grown in the laboratory and given back to the patient along with the stem cell transplant can help the immune system recover faster after transplant. As a safety measure these T cells have been programmed with a self-destruct switch so that they can be destroyed if they start to react against tissues (Graft versus host disease).

This is a clinical research trial in which a novel preparatory regimen was developed for bone marrow transplant (BMT) which eliminates the primary obstacle to transplant, the lack of a matched sibling donor. It is believed this regimen is sufficiently efficacious and sufficiently gentle to apply to patients with sickle cell anemia and related disorders. It is proposed to characterize the efficacy and toxicity of this regimen in high risk patients with sickle cell anemia using criteria for patient selection that have been accepted in prior BMT trials in patients with sickle cell disease, specifically only the subset of patients whose prior clinical behavior indicates that they are at high risk for serious morbidity and early mortality. In addition, it is proposed to characterize the pathophysiology of a consistent febrile response seen in the haploidentical BMT regimen the investigators have developed at Thomas Jefferson University (TJU). The primary goal of this study is to determine the response rate to a reduced intensity conditioning regimen which consists of fludarabine, cytarabine, low dose total body irradiation and cyclophosphamide in patients with severe sickle cell anemia.

The primary efficacy endpoint of this interventional study was to evaluate the number of patients achieving a complete response (CR), defined as patients switching from intensive deferasirox -DFO treatment, at any time point during the 24 months of study, to deferasirox monotherapy based on improvement in the cardiac magnetic resonance imaging (MRI) T2* value to >10ms, and continue to maintain their MRI T2* to values >10 msec.

The purpose of this study is to find out if using a lower dose of chemotherapy before stem cell transplantation can cure patients of sickle cell anemia or thalassemia while causing fewer severe side effects than conventional high dose chemotherapy with transplantation.

Primary Objective Define the pharmacokinetics of liquid-formulated HU in infants (9 months to <2 years) Assess the relative bioavailability of HU "sprinkles" compared to capsules in children and adolescents (≥2 to 18 years). Secondary Objective: Compare PK parameters in infants versus older children on this study and those from our previous "Pharmacokinetics and Bioavailability of a Liquid Formulation of Hydroxyurea in Pediatric Patients with Sickle Cell Anemia" (NCT01506544) trial. Exploratory Objectives: Capture information regarding the taste of HU sprinkles using palatability questionnaire. This trial is an open label, single center assessment of the pharmacokinetics of two formulations of hydroxyurea (HU) designed to (1) determine the pharmacokinetic profile of a liquid formulation in infants and to (2) determine the bioavailability of "sprinkles", a novel method of administration for older children. The study aims to generate data to facilitate FDA approval for HU in children and potentially validate a new mode of administration ("sprinkles") that will optimize access and adherence for children in the US and globally.

The major goal of this study is to determine the risks and benefits of bone marrow transplants in patients with severe thalassemia or sickle cell disease. Participation in this project will be for two years.

The major goal of this study is to determine the risks and benefits of stem cell transplants in combination with a newer, less toxic conditioning chemotherapy treatment in patients with severe sickle cell disease (SCD) or sickle hemoglobin variants (hemoglobin SC or hemoglobin SB0/+), or homozygous b0/+ thalassemia or severe B0/+ thalassemia variants. Participation in this project will be for one year, with follow up evaluations done every 6 months thereafter for 10 years or until participants are 18 years old.

Thalassemia syndromes are a heterogeneous group of single gene disorders, inherited in an autosomal recessive manner ,prevalent among all ethnic groups and in almost every country around the world . Once a child has been diagnosed as thalassemia, he has to take lifelong treatment , where cure is not attainable and treatment may be prolonged . It is a life-threatening and life-limiting condition that affects the patient clinically and psychologically, so Health-related Quality of Life (HRQOL) is likely to be an essential outcome for these patients. Quality of life in thalassemic children such as : Repeated visits to hospitals for regular blood transfusion, cost of chelation therapy, repeated laboratory tests for monitoring therapy and for early detection of any complications. also life-long costly therapy along with poor quality of life will have adverse impact on the family. A better understanding of the factors associated with HRQOL among children with thalassemia could have a direct effect on the development of more suitable clinical, counselling and social support programs to enhance treatment outcomes. Cognitive dysfunction was Reported either due to the disease or its treatment ,frequent school absences, frequent hospitalizations, and physical and social restrictions lead to cognitive dysfunction . This neurological involvement in thalassemic children is primarily silent, with subclinical manifestations that can only be detected by cognitive assessment tests.

This study will evaluate the use of reduced intensity conditioning regimen in patients with high risk hemoglobinopathy Sickle Cell and B-Thalassemia Major in combination with standard immunosuppressive medications, followed by a routine stem cell transplant in order to assess whether or not it is as effective as myeloablative high dose chemotherapy and transplant.

This is a Phase 3, double-blind, randomized, placebo-controlled, multicenter study to determine the efficacy and safety of luspatercept (ACE-536) plus Best supportive care (BSC) versus placebo plus BSC in adults who require regular red blood cell transfusion due to (β)-thalassemia. The study is divided into the following periods: Historical Period, Screening/Run-in Period, Double-blind Treatment Period (48 weeks), Double-blind Long-term Treatment Period, (at the investigator's discretion an additional 48 weeks), Open-Label Phase post unblinding and upon Data Monitoring Committee positive recommendation Post-treatment Follow-up Period