A Clinical Trial to Evaluate the Safety, Efficacy, and Pharmacokinetics of MegaLT Injection for Treating Refractory Thrombocytopenia Following Radiotherapy, Chemotherapy, or Transplantation.

NCT06534255 | Active Not Recruiting Phase 1

• 1 other identifier: MegaLT-2024
• Study Type: interventional
• Target: 10
• Locations: 1 country
• Sites: 1

Brief Summary

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

Conditions

Refractory Thrombocytopenia

Keywords

Refractory thrombocytopenia; Megakaryocyte

Outcome Measures

Primary Outcomes (5)

• 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 Outcomes (10)

• Overall response rate (ORR)

  1, 2 and 8 weeks after initial infusion

• Complete remission (CR) rate

  1, 2, 4 and 8 weeks after initial infusion

• partial response rate (PR)

  1, 2, 4 and 8 weeks after initial infusion

• Changes in platelet counts

  before treatment and at 1, 2, 4, and 8 weeks after treatment

• Median time of platelet count ≥ 50×10^9/L

  1 year after initial infusion

Study Arms (1)

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

Biological: MegaLT injection

Interventions

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.

MegaLT injection

Eligibility Criteria

• Age: 4 Years - 75 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• Age between 4 and 75 years (inclusive) at screening; gender is not restricted;
• Diagnosed with refractory thrombocytopenia following radiotherapy/chemotherapy or transplantation;
• Adequate organ function：Alanine aminotransferase (ALT) or Aspartate aminotransferase (AST) \< 2.5\*upper limit of normal (ULN); Serum creatinine \< 1.5\* ULN; Total bilirubin level \< 1.5\* ULN; Coagulation function: International Normalized Ratio (INR) ≤ 1.5\* ULN, Activated Partial Thromboplastin Time (APTT) ≤ 1.5\*ULN; Hemoglobin ≥ 60 g/L, or hemoglobin maintained at this level after transfusion; Absolute neutrophil count (ANC) ≥ 1.0×10\^9/L;
• Eastern Cooperative Oncology Group (ECOG) performance status of 0-2;
• Voluntarily participating in the clinical trial, with full understanding of the trial details and having signed the informed consent form.

You may not qualify if:

• Patients with any malignant tumor relapse;
• Pregnant or breastfeeding women;
• Life expectancy less than 3 months;
• Severe infections or severe concurrent conditions such as heart, liver, lung, kidney, neurological, or metabolic diseases;
• History of severe thrombotic events or known thrombotic risk factors. Exceptions: Participants for whom the potential benefits of the study, as determined by the investigator, outweigh the potential risks of thromboembolic events;
• Uncontrolled infectious or other serious diseases, including but not limited to infections (e.g., HIV positive), congestive heart failure, unstable angina, arrhythmias, psychiatric disorders, or conditions that limit the ability to meet study requirements or pose unpredictable risks as judged by the primary physician;
• Active hepatitis B or C infection;
• Refractory thrombocytopenia post-transplant with Grade III-IV acute graft-versus-host disease (GVHD) \[according to NIH standards\] or severe chronic GVHD (NIH 2014 standards);
• History of organ transplantation or planned organ transplantation (excluding hematopoietic stem cell transplantation);
• Participation in another clinical study within 30 days prior to the baseline visit, involving any investigational drug or device; observational studies are allowed;
• Any other conditions deemed by the investigator to make the participant unsuitable for the clinical trial.

Sponsors & Collaborators

• Anhui Provincial Hospital: lead

Study Sites (1)

The First Affiliated Hospital of University of Science and Technology of China (Anhui Provincial Hospital)

Hefei, Anhui, 230036, China

Location

MeSH Terms

Conditions

Thrombocytopenia

Condition Hierarchy (Ancestors)

Blood Platelet Disorders; Hematologic Diseases; Hemic and Lymphatic Diseases; Cytopenia

Study Officials

• Xiaoyu Zhu, Ph.D

  The First Affiliated Hospital of University of Science and Technology of China

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER GOV
• Responsible Party: SPONSOR

Model Details: 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.

Study Record Dates

• First Submitted: July 30, 2024
• First Posted: August 2, 2024
• Study Start: December 4, 2024
• Primary Completion: June 19, 2025
• Study Completion (Estimated): May 22, 2026
• Last Updated: April 14, 2026

Record last verified: 2026-04

Locations

CN (1)