Megakaryocytic Progenitor Cells for Prophylaxis and Treatment of Thrombocytopenia

Phase 2

• Conditions: Hematological Diseases; Thrombocytopenia
• Interventions: Biological: MPs; Drug: thrombopoietin (TPO) and interleukin-11

• Registration Number: NCT02241031
• Lead Sponsor: Nanfang Hospital, Southern Medical University

Brief Summary

The purpose of this study is to evaluate the efficacy of ex vivo generated megakaryocytic progenitor cells (MPs) in prophylaxis and treatment of thrombocytopenia caused by chemotherapy in patients with acute leukemia (AL).

Detailed Description

Thrombocytopenia is a common and potentially fatal complication of chemotherapy and hematopoietic stem cell transplantation. Owing to the short storage time and increased demand of platelets from unrelated donors, a constant shortage in the supply of platelets has become an important medical and society challenge. Therefore, investigation of alternative sources of platelets would be beneficial.

Hematopoietic stem cells (HSCs) can be used to generate functional megakaryocytic progenitors (MPs), megakaryocytes, and platelets on a large scale. Functional MPs and platelets have successfully been produced in vitro from CD34+ hematopoietic cells from bone marrow, cord blood, and peripheral blood. Several studies have reported that transplantation of in vitro auto-producing MPs can promote platelet recovery after high-dose therapy and HSC transplantation.

Umbilical cord blood is an abundant source of HSCs. In vitro large scale production of MPs from cord blood could represent an effective platelet substitute. Theoretically, the additional transplantation of ex vivo generated progenitor and post-progenitor cells might lead to the production of sufficient numbers of mature functional cells within a few days after transplantation.

Study Timeline

• Status Verified: 2014-09
• Study Start: 2014-09
• Study First Submitted: 2014-09-13
• Study First Submitted QC: 2014-09-15
• Last Update Submitted: 2014-09-15
• Study First Posted: 2014-09-16
• Last Update Posted: 2014-09-16
• Primary Completion: 2016-09
• Study Completion: 2016-12

Recruitment & Eligibility

• Status: UNKNOWN
• Sex: All
• Target Recruitment: 250

Inclusion Criteria

• age：14-65 years
• achieve complete remission of acute leukemia
• the first course of consolidation chemotherapy
• ECOG grades 0 or 1
• expected survival time ≥ three months
• Subjects (or their legally acceptable representatives) must have signed an informed consent document.

Exclusion Criteria

• cardiac dysfunction (particularly congestive heart failure), hepatic abnormalities (bilirubin ≥ 3 mg/dL, aminotransferase> 2 times the upper limit of normal), renal dysfunction (creatinine clearance rate < 30 mL/min)
• Any abnormality in a vital sign (e.g., heart rate, respiratory rate, or blood pressure)
• Patients with any conditions not suitable for the trial (investigators' decision)

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
MPs	MPs	MPs will be intravenously infused within 48 hours after chemotherapy. During the study period, patients can receive platelet infusion but can not receive platelet stimulating factors.
Non-MPs	thrombopoietin (TPO) and interleukin-11	Patients can receive platelet infusion but can not receive platelet stimulating factors.

Primary Outcome Measures

Name	Time	Method
Platelet recovery after infusion of MPs	3 months	Platelet recovery after infusion of MPs includes the time from infusion to platelet count≥20×10\^9/L,50×10\^9/L,100×10\^9/L.

Secondary Outcome Measures

Name	Time	Method
frequency of platelet infusion	3 months
incidence of acute toxicity	3 month	Acute toxicity mainly involves the heart,live and kidney.

Trial Locations

Locations (1)

Department of Hematology,Nanfang Hospital, Southern Medical University; 🇨🇳 Guangzhou, Guangdong, China