Clinical trial NCT06534255: A Clinical Trial to Evaluate the Safety, Efficacy, and Pharmacokinetics of MegaLT Injection for Treating Refractory Thrombocytopenia Following Radiotherapy, Chemotherapy, or Transplantation.
Official title: A Clinical Trial to Evaluate the Safety, Efficacy, and Pharmacokinetics of MegaLT Injection for Treating Refractory Thrombocytopenia Following Radiotherapy, Chemotherapy, or Transplantation.
Study type: INTERVENTIONAL
Overall status: RECRUITING
Phase: NA
Conditions: Refractory Thrombocytopenia
Keywords: Refractory thrombocytopenia
Megakaryocyte
Lead sponsor: Anhui Provincial Hospital
Sponsor class: OTHER_GOV
First posted: 2024-08-02
Enrollment: 10
Sex: ALL
Minimum age (years): 4
Maximum age (years): 75
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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

Detailed description: 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.

Eligibility criteria: Inclusion Criteria:

* 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.

Exclusion Criteria:

* 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.

Primary outcomes:
measure: Adverse Event(AE)
timeFrame: From the date of initial infusion to 1 year after initial infusion
measure: Incidence of bleeding events
timeFrame: From the date of initial infusion to 1 year after initial infusion
measure: The cumulative incidence and grade of graft-versus-host disease (GVHD) including acute and chronic GVHD
timeFrame: From the date of initial infusion to 1 year after initial infusion
measure: The probability of GVHD-free, relapse-free survival(GRFS)
timeFrame: From the date of initial infusion to 1 year after initial infusion
measure: Overall response rate (ORR)
timeFrame: 4 weeks after initial infusion
Secondary outcomes:
measure: Overall response rate (ORR)
timeFrame: 1, 2 and 8 weeks after initial infusion
measure: Complete remission (CR) rate
timeFrame: 1, 2, 4 and 8 weeks after initial infusion
measure: partial response rate (PR)
timeFrame: 1, 2, 4 and 8 weeks after initial infusion
measure: Changes in platelet counts
timeFrame: before treatment and at 1, 2, 4, and 8 weeks after treatment
measure: Median time of platelet count ≥ 50×10^9/L
timeFrame: 1 year after initial infusion
measure: Median time of platelet count ≥ 100×10^9/L
timeFrame: 1 year after initial infusion
measure: Total platelet transfusion during 4 weeks of treatment
timeFrame: 4 weeks after initial infusion
measure: neutrophil reconstitution
timeFrame: 1 year after initial infusion
measure: erythroid reconstitution
timeFrame: 1 year after initial infusion
measure: Megakaryocyte levels in bone marrow smears
timeFrame: before treatment and 2 and 4 weeks after treatment
Other outcomes:
Locations:
city: Hefei
country: China
facility: The First Affiliated Hospital of University of Science and Technology of China (Anhui Provincial Hospital)
state: Anhui
Study officials:
affiliation: The First Affiliated Hospital of University of Science and Technology of China
name: Xiaoyu Zhu, Ph.D
role: PRINCIPAL_INVESTIGATOR
References: