Clinical Research Directory

Sonrotoclax Plus Azacitidine in Patients With MDS-IB2

This study will evaluate the safety and preliminary effectiveness of sonrotoclax combined with azacitidine in adults aged 18-70 years with newly diagnosed myelodysplastic neoplasms with increased blasts-2 (MDS-IB2). Sonrotoclax blocks BCL-2, a protein that helps cancer cells survive. Combining sonrotoclax with azacitidine may improve disease control. This study aims to determine whether the combination can induce remission with an acceptable safety profile, facilitate allogeneic stem cell transplantation in suitable patients, and improve long-term clinical outcomes. This multicenter, open-label study has two sequential parts. Phase Ib will evaluate different sonrotoclax doses and identify the recommended dose for phase II. Phase II will further assess remission rates and safety at the selected dose. All participants will receive sonrotoclax plus azacitidine; there is no randomized control group. Participants will undergo regular blood tests, bone marrow examinations, and monitoring for side effects. Suitable participants may proceed to transplantation based on disease response and clinical condition. Those who are not proceeding to transplantation and continue to benefit may continue treatment according to the protocol. Follow-up will assess the duration of remission, disease progression, subsequent treatment, survival, and quality of life.

A Study Testing an Improved Dose of UM171 to Help Make Cord Blood Transplants More Effective and Safe.

This clinical study is testing a new way to improve stem cell transplants for adults with high-risk blood cancers, such as leukemia or myelodysplasia, who do not have a suitable donor. The transplant uses stem cells from umbilical cord blood that have been expanded in the lab using a molecule called UM171. Previous studies showed that UM171 helps these cells grow and work better, leading to faster blood count recovery and fewer complications. In this study, researchers are testing whether increasing the dose of UM171 during the lab expansion process can make the transplant less toxic. The hypothesis is that using a higher dose of UM171 to expand cord blood stem cells will help patients recover blood counts faster after transplant by improving the growth and function of the cells. This may lead to better immune recovery, fewer infections, shorter hospital stays, and improved overall outcomes. Only seven patients will be enrolled, and they will be followed for one year after their transplant.