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

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

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.

Study Overview

• Status: Not yet recruiting
• Conditions: Thrombocytopenia; Hematologic Malignacies; Platelet Recovery After Umbilical Cord Blood Transplantation
• Intervention / Treatment: Biological: MegaLT Injection

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.

• Study Type: Interventional
• Enrollment (Estimated): 7
• Phase: Early Phase 1

Contacts and Locations

• Study Contact: Name: Xiaoyu Zhu, MD, PhD; Phone Number: +86-551-62282931; Email: xiaoyuz@ustc.edu.cn
• Study Contact Backup: Name: Aijie Huang, MD; Email: huangaijie@mail.ustc.edu.cn

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Adult; Older Adult
• Accepts Healthy Volunteers: No

Description

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.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm

Intervention / Treatment

Experimental: MegaLT Injection; 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.

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 considered if necessary according to the protocol.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	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

Secondary Outcome Measures

Outcome Measure	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	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

Other Outcome Measures

Outcome Measure	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	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

Collaborators and Investigators

• Sponsor: Anhui Provincial Hospital

Study record dates

Study Major Dates

• Study Start (Estimated): July 31, 2026
• Primary Completion (Estimated): December 31, 2026
• Study Completion (Estimated): June 30, 2027

Study Registration Dates

• First Submitted: July 4, 2026
• First Submitted That Met QC Criteria: July 9, 2026
• First Posted (Actual): July 10, 2026

Study Record Updates

• Last Update Posted (Actual): July 10, 2026
• Last Update Submitted That Met QC Criteria: July 9, 2026
• Last Verified: July 1, 2026

More Information

Terms related to this study

• Keywords: Platelet Engraftment; Thrombocytopenia; Platelet Recovery; Umbilical Cord Blood Transplantation; MegaLT Injection
• Additional Relevant MeSH Terms: Cytopenia; Hematologic Diseases; Blood Platelet Disorders; Hemic and Lymphatic Diseases; Thrombocytopenia
• Other Study ID Numbers: MegaLT-2026

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No