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A study to evaluate the safety, efficacy, and pharmacokinetics of MegaLT in treating refractory thrombocytopenia following radiotherapy, chemotherapy, or transplantation.
A single-center, open-label, dose-escalation study to assess the safety, efficacy, and pharmacokinetics of MegaLT for treating refractory thrombocytopenia following radiotherapy, chemotherapy, or transplantation.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| MegaLT injection | Experimental | Dosage Form: ex vivo three-dimensional induced umbilical cord blood mononuclear cells differentiated megakaryocyte injection (MegaLT injection). Administration Route: Intravenous Infusion Regimen: Single or Multiple Infusions |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| MegaLT injection | Biological | Three dose groups were set: 1×10^6/kg, 5×10^6/kg, and 1×10^7/kg. The dose escalation was carried out in ascending order using a "3+3" design. To ensure participant safety, enrollment followed a "1+2" rule. Specifically, the first participant in each dose group received the cell infusion and was observed for 14 days. If no dose-limiting toxicity (DLT) was observed, the remaining two participants could then be enrolled and receive cell therapy at the same dose level. |
| Measure | Description | Time Frame |
|---|---|---|
| Adverse Event(AE) | Number of treatment-related adverse events as assessed by CTCAE v4.0 | From the date of initial infusion to 1 year after initial infusion |
| Incidence of bleeding events | The incidence of bleeding events after initial infusion | From the date of initial infusion to 1 year after initial infusion |
| The cumulative incidence and grade of graft-versus-host disease (GVHD) including acute and chronic GVHD | The occurrence incidence and grade of GVHD after transplantation | From the date of initial infusion to 1 year after initial infusion |
| The probability of GVHD-free, relapse-free survival(GRFS) | The composite endpoint of GRFS was defined as the first events occurring after transplantation among Grade III to IV aGVHD, moderate to severe cGVHD, relapse, or death for any reason. | From the date of initial infusion to 1 year after initial infusion |
| Overall response rate (ORR) | Percentage of participants achieved Complete response(CR) or Partial response(PR) | 4 weeks after initial infusion |
| Measure | Description | Time Frame |
|---|---|---|
| Overall response rate (ORR) | Percentage of participants achieved Complete Response(CR) or Partial Response(PR) | 1, 2 and 8 weeks after initial infusion |
| Complete remission (CR) rate | CR was defined as a platelet count ≥ 50×10^9/L without platelet transfusions for 7 consecutive days |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Xiaoyu Zhu, Ph.D | The First Affiliated Hospital of University of Science and Technology of China | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| The First Affiliated Hospital of University of Science and Technology of China (Anhui Provincial Hospital) | Hefei | Anhui | 230036 | China |
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Subjects will receive cell infusions sequentially according to their enrollment order, with three planned dose levels of: 1×10⁶/kg, 5×10⁶/kg, and 1×10⁷/kg.
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| 1, 2, 4 and 8 weeks after initial infusion |
| partial response rate (PR) | PR was defined as a platelet count < 50×10^9/L but above the baseline level at enrollment, with a sustained increase for 7 consecutive days without platelet transfusions | 1, 2, 4 and 8 weeks after initial infusion |
| Changes in platelet counts | Changes in platelet counts before treatment and at 1, 2, 4, and 8 weeks after treatment | before treatment and at 1, 2, 4, and 8 weeks after treatment |
| Median time of platelet count ≥ 50×10^9/L | Platelet count ≥50×10^9/L for 3 consecutive days without platelet transfusions for 7 consecutive days | 1 year after initial infusion |
| Median time of platelet count ≥ 100×10^9/L | Platelet count ≥50×10^9/L for 3 consecutive days without platelet transfusions for 7 consecutive days | 1 year after initial infusion |
| Total platelet transfusion during 4 weeks of treatment | Total platelet transfusion during 4 weeks of treatment | 4 weeks after initial infusion |
| neutrophil reconstitution | Neutrophil engraftment time was defined as the first of three consecutive days during which the neutrophil count was at least 0.5×10^9/L | 1 year after initial infusion |
| erythroid reconstitution | Red blood count (RBC) engraftment time was defined as the first day of achieving a reticulocyte count greater than 1% for 3 consecutive days. | 1 year after initial infusion |
| Megakaryocyte levels in bone marrow smears | Megakaryocyte levels in bone marrow smears | before treatment and 2 and 4 weeks after treatment |
| ID | Term |
|---|---|
| D013921 | Thrombocytopenia |
| ID | Term |
|---|---|
| D001791 | Blood Platelet Disorders |
| D006402 | Hematologic Diseases |
| D006425 | Hemic and Lymphatic Diseases |
| D000095542 | Cytopenia |
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