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.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
10
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.
The First Affiliated Hospital of University of Science and Technology of China (Anhui Provincial Hospital)
Hefei, Anhui, China
Adverse Event(AE)
Number of treatment-related adverse events as assessed by CTCAE v4.0
Time frame: From the date of initial infusion to 1 year after initial infusion
Incidence of bleeding events
The incidence of bleeding events after initial infusion
Time frame: 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
Time frame: 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.
Time frame: 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)
Time frame: 4 weeks after initial infusion
Overall response rate (ORR)
Percentage of participants achieved Complete Response(CR) or Partial Response(PR)
Time frame: 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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Time frame: 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
Time frame: 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
Time frame: 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
Time frame: 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
Time frame: 1 year after initial infusion
Total platelet transfusion during 4 weeks of treatment
Total platelet transfusion during 4 weeks of treatment
Time frame: 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
Time frame: 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.
Time frame: 1 year after initial infusion
Megakaryocyte levels in bone marrow smears
Megakaryocyte levels in bone marrow smears
Time frame: before treatment and 2 and 4 weeks after treatment