Refractory ThrombocytopeniaPhase 1NCT06534255
A Clinical Trial to Evaluate the Safety, Efficacy, and Pharmacokinetics of MegaLT Injection for Treating Refractory Thrombocytopenia Following Radiotherapy, Chemotherapy, or Transplantation.
A Phase 1 study testing MegaLT injection in people with Refractory Thrombocytopenia, run by Anhui Provincial Hospital at 1 site in China.
Trial summary
A study to evaluate the safety, efficacy, and pharmacokinetics of MegaLT in treating refractory thrombocytopenia following radiotherapy, chemotherapy, or transplantation. Read more about this study
- Who is it for?
- Ages 4 to 75 years
- Conditions the study is for
- Refractory Thrombocytopenia
- What is being tested?
- MegaLT injection
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 10 participants
- Where
- 1 siteChina
- Study dates
- 4 Dec 2024to 22 May 2026
- Phase
- Phase 1
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 4 to 75 years
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- 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.
Who cannot join
- 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;
Show all 11Show fewer
- 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.
Trial locations and contacts
1 site in 1 country.
China1 site
The First Affiliated Hospital of University of Science and Technology of China (Anhui Provincial Hospital)
Hefei, Anhui, 230036
About this study
A study to evaluate the safety, efficacy, and pharmacokinetics of MegaLT in treating refractory thrombocytopenia following radiotherapy, chemotherapy, or transplantation.
Read the 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.
- Conditions
- Refractory Thrombocytopenia
- Being tested
- MegaLT injection
- Keywords
- Refractory thrombocytopenia, Megakaryocyte
- Medical subject headings
- Thrombocytopenia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
MegaLT injectionBiological
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.
| Study group | Role | What they receive |
|---|---|---|
| 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 Receives: Biological: MegaLT injection |
What is being measured
The primary outcome is the main question the study is designed to answer.
Adverse Event(AE)
Number of treatment-related adverse events as assessed by CTCAE v4.0
Measured: From the date of initial infusion to 1 year after initial infusion
Incidence of bleeding events
The incidence of bleeding events after initial infusion
Measured: 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
Measured: 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.
Measured: 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)
Measured: 4 weeks after initial infusion
Secondary outcomes (10)
Overall response rate (ORR)
Percentage of participants achieved Complete Response(CR) or Partial Response(PR)
Measured: 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
Measured: 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
Measured: 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
Measured: 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
Measured: 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
Measured: 1 year after initial infusion
Total platelet transfusion during 4 weeks of treatment
Total platelet transfusion during 4 weeks of treatment
Measured: 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
Measured: 1 year after initial infusion
erythroid reconstitution
Red blood count (RBC) engraftment time was defined as the first day of achieving a reticulocyte count greater than 1% for 3 consecutive days.
Measured: 1 year after initial infusion
Megakaryocyte levels in bone marrow smears
Megakaryocyte levels in bone marrow smears
Measured: before treatment and 2 and 4 weeks after treatment
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted2 Aug 2024
- Study start4 Dec 2024
- Primary completion19 Jun 2025
- Study completion22 May 2026
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Refractory ThrombocytopeniaStudies of the same condition, open and under way.
- More trials run by Anhui Provincial HospitalThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Model description
- 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.
- Masking
- None (open label)
- Enrolment
- 10 participants (actual)
Sponsor and investigators
- Lead sponsor
- Anhui Provincial Hospital
- Sponsor type
- Other government
- Responsible party
- Sponsor
- Investigators
- Xiaoyu Zhu, Ph.D, Principal investigator, The First Affiliated Hospital of University of Science and Technology of China
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06534255
- Sponsor's protocol ID
- MegaLT-2024
- Status last verified
- Jul 2026
- First submitted
- 30 Jul 2024
- Last updated
- 7 Jul 2026
- Results posted
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 7 Jul 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Clinical Trial to Evaluate the Safety, Efficacy, and Pharmacokinetics of MegaLT Injection for Treating Refractory Thrombocytopenia Following Radiotherapy, Chemotherapy, or Transplantation.
- ClinicalTrials.gov ID
- NCT06534255
- Acronym / sponsor ID
- MegaLT-2024
- Conditions
- Refractory Thrombocytopenia
- Keywords
- Refractory thrombocytopenia, Megakaryocyte
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 10 participants (actual)
- Lead sponsor
- Anhui Provincial HospitalOther government
- Study start
- 4 Dec 2024 (actual)
- Primary completion
- 19 Jun 2025 (actual)
- Study completion
- 22 May 2026 (actual)
- First posted
- 2 Aug 2024 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
- Model description
- 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.
| Arm | Type | Receives |
|---|---|---|
| MegaLT injection | Experimental | Biological: MegaLT injectionDosage 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 |
Who may qualify
- Ages
- 4 to 75 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (5 listed)
- 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.
Key exclusion criteria (11 listed)
- 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;
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| MegaLT injectionThree 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. | Biological | MegaLT injection |
Primary outcome
Adverse Event(AE)
Time frame: From the date of initial infusion to 1 year after initial infusion
Number of treatment-related adverse events as assessed by CTCAE v4.0
Incidence of bleeding events
Time frame: From the date of initial infusion to 1 year after initial infusion
The incidence of bleeding events after initial infusion
The cumulative incidence and grade of graft-versus-host disease (GVHD) including acute and chronic GVHD
Time frame: From the date of initial infusion to 1 year after initial infusion
The occurrence incidence and grade of GVHD after transplantation
The probability of GVHD-free, relapse-free survival(GRFS)
Time frame: From the date of initial infusion to 1 year after initial infusion
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.
Overall response rate (ORR)
Time frame: 4 weeks after initial infusion
Percentage of participants achieved Complete response(CR) or Partial response(PR)
10 secondary outcomes
Overall response rate (ORR)
Time frame: 1, 2 and 8 weeks after initial infusion
Complete remission (CR) rate
Time frame: 1, 2, 4 and 8 weeks after initial infusion
partial response rate (PR)
Time frame: 1, 2, 4 and 8 weeks after initial infusion
Changes in platelet counts
Time frame: before treatment and at 1, 2, 4, and 8 weeks after treatment
Median time of platelet count ≥ 50×10^9/L
Time frame: 1 year after initial infusion
Median time of platelet count ≥ 100×10^9/L
Time frame: 1 year after initial infusion
Total platelet transfusion during 4 weeks of treatment
Time frame: 4 weeks after initial infusion
neutrophil reconstitution
Time frame: 1 year after initial infusion
erythroid reconstitution
Time frame: 1 year after initial infusion
Megakaryocyte levels in bone marrow smears
Time frame: before treatment and 2 and 4 weeks after treatment
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 7 Jul 2026.
Related trials for Refractory Thrombocytopenia
Active, not recruitingObservational study
Observational Study on Eltrombopag in Hypocellular Myelodysplastic Neoplasms
Myelodysplastic Neoplasm, Myelodysplastic Syndrome
RecruitingPhase 1
RN1201 Injection in the Treatment of Antibody-Mediated Diseases
Refractory Immune-mediated Platelet Transfusion Refractoriness, Relapsed or Refractory Immune Thrombocytopenia
Not yet recruitingPhase 1
Allogeneic CD19/BCMA-Targeted CAR-γδT Cell Therapy: Safety and Preliminary Pharmacodynamics in Relapsed/Refractory Autoimmune Diseases
Refractory/Relapsed Systemic Lupus Erythematosus, Refractory / Relapsed / Progressive Systemic Sclerosis
Recruiting
Contribution of Anti-platelet Antibodies Identified With MAIPA Assay in the Demonstration of the Auto-immune Character of a Thrombocytopenia at Diagnosis
Thrombocytopenia, Immune Thrombocytopenia
RecruitingPhase 2
The Investigate Efficacy and Safety Evaluation of Bortezomib in Patients With Relapsed/Refractory Immune Thrombocytopenia
Relapsed/Refractory Immune Thrombocytopenia
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Xiaoyu Zhu, Ph.D principal investigator
The First Affiliated Hospital of University of Science and Technology of China
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing MegaLT injection in people with Refractory Thrombocytopenia, run by Anhui Provincial Hospital at 1 site in China.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 5 inclusion and 11 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 7 Jul 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in China. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Adverse Event(AE) (time frame: From the date of initial infusion to 1 year after initial infusion). The study lists 5 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 7 Jul 2026; AzTrials last refreshed its copy on 7 Nov 2026.