Clinical trial NCT02241031: Megakaryocytic Progenitor Cells for Prophylaxis and Treatment of Thrombocytopenia
Official title: Megakaryocytic Progenitor Cells for Prophylaxis and Treatment of Thrombocytopenia in Patients With Acute Leukemia Receiving Chemotherapy
Study type: INTERVENTIONAL
Overall status: UNKNOWN
Phase: PHASE2
PHASE3
Conditions: Hematological Diseases
Thrombocytopenia
Keywords: Megakaryocytic Progenitor Products
Hematological Diseases
Thrombocytopenia
Lead sponsor: Nanfang Hospital, Southern Medical University
Sponsor class: OTHER
Collaborators: name: Guangdong Provincial People's Hospital
name: Third Affiliated Hospital, Sun Yat-Sen University
name: Guangzhou General Hospital of Guangzhou Military Command
name: Guangzhou First People's Hospital
name: Sun Yat-Sen Memorial Hospital of Sun Yat-Sen University
name: Southern Medical University, China
name: Peking University People's Hospital
name: Huazhong University of Science and Technology
name: Sun Yat-sen University
name: Academy Military Medical Science, China
First posted: 2014-09-16
Enrollment: 250
Sex: ALL
Minimum age (years): 14
Maximum age (years): 65
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

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.

Eligibility criteria: 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)

Primary outcomes:
measure: Platelet recovery after infusion of MPs
timeFrame: 3 months
Secondary outcomes:
measure: frequency of platelet infusion
timeFrame: 3 months
measure: incidence of acute toxicity
timeFrame: 3 month
Other outcomes:
Locations:
city: Guangzhou
country: China
facility: Department of Hematology,Nanfang Hospital, Southern Medical University
state: Guangdong
Study officials:
affiliation: Nanfang Hospital, Southern Medical University
name: Qifa Liu, MD
role: PRINCIPAL_INVESTIGATOR
References:
citation: Xi J, Zhu H, Liu D, Nan X, Zheng W, Liu K, Shi W, Chen L, Lv Y, Yan F, Li Y, Xie X, Wang Y, Yue W, Xu X, Wei X, Zhu J, Huang X, Pei X. Infusion of megakaryocytic progenitor products generated from cord blood hematopoietic stem/progenitor cells: results of the phase 1 study. PLoS One. 2013;8(2):e54941. doi: 10.1371/journal.pone.0054941. Epub 2013 Feb 4.