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This is a single-arm, early safety and feasibility exploratory study to evaluate the efficacy and safety of MegaLT Injection in promoting platelet recovery after umbilical cord blood transplantation. Adults with malignant hematologic diseases who undergo umbilical cord blood transplantation will receive a single intravenous infusion of MegaLT Injection on day 14 after transplantation. Two dose levels will be evaluated sequentially, and platelet recovery, platelet transfusion requirements, and safety outcomes will be assessed.
This study is designed as a single-arm, early safety and feasibility exploratory study of MegaLT Injection in patients undergoing umbilical cord blood transplantation. Eligible participants will receive MegaLT Injection by intravenous infusion on day 14 after transplantation. The study includes two sequential dose levels: 1×10^6/kg and 2.5×10^6/kg. Three participants are planned for each dose level, with a total planned enrollment of 7 participants after accounting for an approximately 10% dropout rate. The study will evaluate platelet engraftment, platelet recovery, platelet transfusion requirements, hematopoietic recovery, and safety outcomes including adverse events, bleeding events, thromboembolic events, graft-versus-host disease, and transplant-related mortality.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| MegaLT Injection | Experimental | Participants will receive MegaLT Injection by intravenous infusion on day 14 after umbilical cord blood transplantation. Dose escalation will proceed sequentially from 1×10^6/kg to 2.5×10^6/kg. Each participant will receive one assigned cell dose; multiple infusions may be considered if necessary according to the protocol. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| MegaLT Injection | Biological | MegaLT Injection is prepared from eligible umbilical cord blood cells through directed expansion and quality control to obtain a megakaryocyte cell product. Participants will receive MegaLT Injection by intravenous infusion on day 14 after umbilical cord blood transplantation. The assigned dose will be 1×10^6/kg or 2.5×10^6/kg, with sequential dose escalation from the low-dose level to the high-dose level. Each participant will receive one assigned cell dose; multiple infusions may be considered if necessary according to the protocol. |
| Measure | Description | Time Frame |
|---|---|---|
| Time to Platelet Engraftment | Platelet engraftment is defined as the first day of 7 consecutive days with platelet count ≥20×10^9/L without platelet transfusion. | From umbilical cord blood transplantation until documented platelet engraftment or the participant's last platelet assessment, assessed up to 6 months following MegaLT infusion. |
| Platelet Engraftment by Day 28 | Cumulative incidence of platelet engraftment by day 28 after transplantation, defined as 7 consecutive days with platelet count ≥20×10^9/L without platelet transfusion. | At day 28 after umbilical cord blood transplantation |
| Measure | Description | Time Frame |
|---|---|---|
| Platelet Recovery by Day 28 | Cumulative rate of platelet recovery by day 28 after transplantation, defined as platelet count ≥50×10^9/L for 7 consecutive days without platelet transfusion. | At day 28 after umbilical cord blood transplantation |
| Time to Platelet Count Thresholds |
| Measure | Description | Time Frame |
|---|---|---|
| Adverse Events | Incidence of adverse events after MegaLT Injection, including clinical symptoms and laboratory abnormalities. Adverse events will be assessed according to the Common Terminology Criteria for Adverse Events version 5.0 when applicable. | From MegaLT Injection to 6 months after injection |
| Bleeding Events |
Inclusion Criteria:
Exclusion Criteria:
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| Name | Role | Phone | Extension | |
|---|---|---|---|---|
| Xiaoyu Zhu, MD, PhD | Contact | +86-551-62282931 | xiaoyuz@ustc.edu.cn | |
| Aijie Huang, MD | Contact | huangaijie@mail.ustc.edu.cn |
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Median time to achieve platelet counts of ≥20×10^9/L, ≥50×10^9/L, and ≥100×10^9/L without platelet transfusion. |
| From umbilical cord blood transplantation until documented platelet count recovery at each threshold or the participant's last platelet assessment, assessed up to 6 months following MegaLT infusion. |
| Platelet Transfusion Volume | Total amount of platelet transfusion received by participants within 4 weeks after MegaLT Injection. | From MegaLT Injection to 4 weeks after injection |
| Neutrophil Engraftment by Day 28 | Cumulative incidence of neutrophil engraftment by day 28 after transplantation, defined as an absolute neutrophil count ≥0.5×10^9/L for 3 consecutive days. | At day 28 after umbilical cord blood transplantation |
| Megakaryocyte Level in Bone Marrow | Megakaryocyte level assessed by bone marrow smear 4 weeks after MegaLT Injection. | At 4 weeks after MegaLT Injection |
Incidence of bleeding events after MegaLT Injection, assessed using the WHO bleeding assessment scale. |
| From MegaLT Injection to 6 months after injection |
| Transplant-Related Complications and Mortality | Incidence of transplant-related complications and transplant-related mortality within 100 days after umbilical cord blood transplantation. | From umbilical cord blood transplantation to 100 days after transplantation |
| Graft-Versus-Host Disease | Incidence and severity of acute graft-versus-host disease and chronic graft-versus-host disease after MegaLT Injection. | From MegaLT Injection to 6 months after injection |
| Severe GVHD-Free, Relapse-Free Survival | Survival without severe graft-versus-host disease or relapse after MegaLT Injection. | From MegaLT Injection to 6 months after injection |
| Thromboembolic Events | Incidence and severity of thromboembolic events after MegaLT Injection, including deep vein thrombosis and pulmonary embolism. Relevant indicators such as D-dimer will be dynamically monitored. | From MegaLT Injection to 6 months after injection |
| ID | Term |
|---|---|
| D013921 | Thrombocytopenia |
| ID | Term |
|---|---|
| D001791 | Blood Platelet Disorders |
| D006402 | Hematologic Diseases |
| D006425 | Hemic and Lymphatic Diseases |
| D000095542 | Cytopenia |
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