Transfusion-dependent Beta-Thalassemia

The primary objective of this study is to compare the effect of mitapivat versus placebo on transfusion burden in pediatric participants with α- or β-transfusion-dependent thalassemia.

This is a prospective, dual-centre, single dose, Phase IIb, single arm, open label study. The proposed clinical trial involves a single infusion of autologous HSPCs genetically modified with the GLOBE lentiviral vector, using an improved transduction protocol in 9 patients affected by transfusion dependent Beta-Thalassemia. Four study phases are foreseen: 1. Screening phase, during which the conditions required by the clinical protocol for patients' inclusion/exclusion will be assessed after the signature of the informed consents/assents. Patients will be recruited from the two participating sites: IRCCS Ospedale San Raffaele (OSR), Department of Pediatric Immunohematology and adult Hematology (OSR Stem Cells Programme) (Milan) and IRCCS Ospedale Pediatrico Bambino Gesù (OPBG), Department of Haematology, Oncology and Gene and Cell Therapy (Rome). 2. Baseline phase, carried from the end of the screening phase to the day before the start of the conditioning regimen. 3. Treatment phase, from the first day of conditioning regimen until DP administration. 4. Follow-up phase: from DP administration until 2 years follow-up.

This is a non-randomized, open-label, single-dose study. The aim of this study is to evaluate the safety and efficacy of the treatment with lentiviral vector encoding βA-T87Q-globin gene transduced autologous hematopoietic stem cells transfusion in subjects with transfusion-dependent β-thalassemia.

This is a non-randomized, open label, single-dose study in up to 41 participants with β-thalassemia major. The goal of this clinical trial is to evaluate the safety and efficacy of KL003 cell injection in subjects with β-thalassemia major.

This study is a single-arm, open-label, dose-escalation trial, planning to enroll 3-9 patients with transfusion-dependent β-thalassemia, aimed at assessing the safety and tolerability of a single-dose of YOLT-204 in patients with transfusion-dependent β-thalassemia; to preliminarily evaluate the impact of a single -dose of YOLT-204 on the levels of fetal hemoglobin in the plasma

Plasticizers are chemicals commonly found in many everyday items, from food packaging to medical equipment. Although they are pervasive in our daily lives, researchers still don't have a clear picture of their long-term effects on human health. Evidence suggests that these substances might disrupt various biological functions such as the immune system, the balance of gut bacteria, hormone regulation, and brain processes. While some studies have linked plasticizer exposure to health issues, definitive data from human studies are still lacking. The PEACH study aims to bridge these knowledge gaps by investigating how plasticizers affect human health. The study focuses on understanding how these chemicals are absorbed, distributed, and accumulated in the body across different groups of patients. The investigators are particularly interested in how plasticizers influence gut microbiota and the functionality of immune cells, as well as their effects on neurotransmitters involved in brain function. A combination of patient data, systems biology, and laboratory models will be used to thoroughly assess the biological impacts of plasticizers. Advanced techniques such as mass spectrometry will aid in studying toxicokinetic properties, sequencing technologies will be used to examine immune effects, and radiouptake assays will be employed to explore interactions with neurotransmitter transport. This comprehensive methodology will provide new insights into the effects of both short-term and long-term exposure to plasticizers. The PEACH study introduces innovative methods to the field, aiming to create a robust model for understanding how plasticizer compounds behave in the human body. It employs state-of-the-art techniques to assess the dynamics of these chemicals, marking a significant advancement in environmental health research.

The investigate will conduct a cohort study to compare the growth and development, metabolism, lifestyle behavior, and health-related quality of life among three groups: children with transfusion-dependent β-thalassemia (TDT) who have received gene therapy, TDT children with lifelong supportive therapy and healthy children.

This is a single-arm, open label, single-dose study in subjects with transfusion dependent β-thalassaemia. The study will evaluate the safety and efficacy of autologous CD34+ Human Hematopoietic Stem Cells that was transduced with LentiRed Lentivrial vector.