Megakaryocytic Progenitor Cells for Prophylaxis and Treatment of Thrombocytopenia

Nanfang Hospital, Southern Medical University

Status and phase: Unknown; Phase 3; Phase 2

Conditions: Thrombocytopenia; Hematological Diseases

Treatments: Biological: MPs; Drug: thrombopoietin (TPO) and interleukin-11

Study type: Interventional

Funder types: Other; Industry

Identifiers

NCT02241031

MPs-AL-2014

Details and patient eligibility

About

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).

Full 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.

Enrollment: 250 estimated patients

Sex: All

Ages: 14 to 65 years old

Volunteers: No Healthy Volunteers

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)

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: None (Open label)

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250 participants in 2 patient groups

MPs

Experimental group

Description:

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.

Treatment:

Biological: MPs

Non-MPs

Active Comparator group

Description:

Patients can receive platelet infusion but can not receive platelet stimulating factors.

Treatment:

Drug: thrombopoietin (TPO) and interleukin-11