A Clinical Trial to Evaluate the Safety, Efficacy, and Pharmacokinetics of MegaLT Injection for Treating Refractory Thrombocytopenia Following Radiotherapy, Chemotherapy, or Transplantation.
NCT Number: NCT06534255
Status: NOT_YET_RECRUITING
Study Type: INTERVENTIONAL
Phases: NA
Conditions: Refractory Thrombocytopenia
Interventions: BIOLOGICAL: MegaLT injection
Sponsor: Anhui Provincial Hospital
Sex: ALL
Age: CHILD, ADULT, OLDER_ADULT
Enrollment: 10
Study Design: Allocation: NA|Intervention Model: SINGLE_GROUP|Masking: NONE|Primary Purpose: TREATMENT
Start Date: 2024-08-01
Completion Date: 2026-12-31
Locations: The First Affiliated Hospital of University of Science and Technology of China (Anhui Provincial Hospital), Hefei, Anhui, 230036, China

Summary:
A study to evaluate the safety, efficacy, and pharmacokinetics of MegaLT in treating refractory thrombocytopenia following radiotherapy, chemotherapy, or transplantation.

Primary Outcome Measures: 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
Secondary Outcome Measures: 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, 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, 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