Health Trials

β-thalassemia Major

2

Review clinical trials related to β-thalassemia Major. Use filters to narrow results by trial status, phase, treatment, biological sex and sponsor.

Condition / disease
Location
Status: Not yet recruiting

A Phase 2 Safety and Efficacy Study Evaluating CS-101 in Participants With β-Thalassemia Major

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

Participants needed: 20
Trial details
Phase: Phase 2Age: 12-35Biological sex: AllType: InterventionalSponsor: CorrectSequence Therapeutics Co., LtdUpdated: Mar 24, 2026Locations: 3
Eligibility criteria

Voluntarily signed informed consent. Male or female participants aged 12 to 35 y... [+4]

Received other investigational products or other experimental interventions with... [+18]

Read more
Status: Recruiting

Safety and Efficacy of Gene Modified Autologous Hematopoietic Stem Cells to Treat Transfusion-dependent β-thalassemia

This study will be intented to evaluate the safety, tolerability, and engraftment efficacy after myeloablative preconditioning and transplantation of autologous CD34+ hematopoietic stem cells transduced with a lentiviral vector encoding the human βA-T87Q-globin gene in patients with transfusion-dependent (TDT) β-thalassemia.

Participants needed: 10
Trial details
Age: 6-35Biological sex: AllType: InterventionalSponsor: Shanghai BDgene Co., Ltd.Updated: Jun 14, 2024Locations: 1
Eligibility criteria

The tumor genes chip detection results about acute leukemia and myeloid tumor ge... [+5]

Availability of voluntary, fully HLA-matched hematopoietic cell donors, unless r... [+17]

Read more