Efficacy and Safety of MegaLT Injection for Platelet Recovery After Umbilical Cord Blood Transplantation

Official title

Study of the Efficacy and Safety of MegaLT Injection for Promoting Platelet Recovery After Umbilical Cord Blood Transplantation

Overview

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.

Detailed description

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.

Interventions

• Biological MegaLT Injection
  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 consider

Primary outcome measures

• Time to Platelet Engraftment [Time frame: 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 [Time frame: At day 28 after umbilical cord blood transplantation]

Secondary outcome measures (5)

• Platelet Recovery by Day 28 [Time frame: At day 28 after umbilical cord blood transplantation]
• Time to Platelet Count Thresholds [Time frame: 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 [Time frame: From MegaLT Injection to 4 weeks after injection]
• Neutrophil Engraftment by Day 28 [Time frame: At day 28 after umbilical cord blood transplantation]
• Megakaryocyte Level in Bone Marrow [Time frame: At 4 weeks after MegaLT Injection]

Eligibility criteria

Inclusion criteria

• Age ≥18 years, male or female.
• Patients with a definite diagnosis of hematologic malignancy who are undergoing umbilical cord blood transplantation.
• Patients at day 14 after umbilical cord blood transplantation.
• ECOG performance status ≤2.
• Patients who voluntarily agree to participate in this clinical trial, fully understand the study content, and sign the informed consent form.

Exclusion criteria

• Patients with relapse of any malignant tumor.
• Pregnant or lactating women.
• Patients with severe infection or severe cardiac, hepatic, pulmonary, renal, neurologic, or metabolic disease.
• Patients with a history of severe thrombotic events or known risk factors for thrombosis. Exception: participants for whom the investigator determines that the potential benefit of study participation outweighs the potential risk of thromboembolic events.
• Patients with uncontrolled infectious disease or other serious diseases, including but not limited to infection such as HIV positivity, congestive heart failure, unstable angina, cardiac arrhythmia, psychosis, social circumstances that would limit compliance with study requirements, or any condition that the treating physician considers may pose unpredictable risks.
• Patients with active hepatitis B or hepatitis C.
• Patients who have previously received organ transplantation or are planning to receive organ transplantation, except for hematopoietic stem cell transplantation.
• Patients who participated in another clinical study and used any investigational drug or device within 30 days before the baseline visit. Participation in observational studies is allowed.
• Patients considered by the investigator to be unsuitable for participation in this clinical trial for any other reason.

Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.

Healthy volunteers: No

Study design

Allocation

N/A

Model

Single group

Masking

Open label

Primary purpose

Treatment

Study locations

Center list to be confirmed — check the primary protocol.

Identifiers

NCT: NCT07696767 · MegaLT-2026

Primary sources (government registries)

• ClinicalTrials.gov (US NIH)

View this study on ClinicalTrials.gov ↗