Hematological DiseasesPhase 2/3NCT02241031
Megakaryocytic Progenitor Cells for Prophylaxis and Treatment of Thrombocytopenia
A Phase 2/3 study testing MPs and thrombopoietin (TPO) and interleukin-11 in people with Hematological Diseases and Thrombocytopenia, run by Nanfang Hospital, Southern Medical University at 1 site in China.
Trial 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). Read more about this study
- Who is it for?
- Ages 14 to 65 years
- Conditions the study is for
- Hematological Diseases, Thrombocytopenia
- What is being tested?
- MPs and thrombopoietin (TPO) and interleukin-11
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 250 participants
- Where
- 1 siteChina
- Study dates
- Sep 2014to Dec 2016
- Phase
- Phase 2/3
- 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 14 to 65 years
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- 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.
Who cannot join
- 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 locations and contacts
1 site in 1 country.
China1 site, 1 recruiting
- Recruiting
Department of Hematology,Nanfang Hospital, Southern Medical University
Guangzhou, Guangdong, 510515
Ren Lin, MD, +86-020-62787883, lansinglinren@hotmail.com
Qifa Liu, MD
About this study
Official title: Megakaryocytic Progenitor Cells for Prophylaxis and Treatment of Thrombocytopenia in Patients With Acute Leukemia Receiving Chemotherapy
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).
Read the 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.
- Conditions
- Hematological Diseases, Thrombocytopenia
- Being tested
- MPs, thrombopoietin (TPO) and interleukin-11
- Keywords
- Megakaryocytic Progenitor Products, Hematological Diseases, Thrombocytopenia
- Medical subject headings
- Hematologic Diseases, Thrombocytopenia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
MPsBiological
MPs are generated from cord blood using a combination of cytokines.
thrombopoietin (TPO) and interleukin-11Drug
Platelet stimulating factors include thrombopoietin (TPO) and interleukin-11 and so on.
| Study group | Role | What they receive |
|---|---|---|
| MPs | Experimental | 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. Receives: Biological: MPs |
| Non-MPs | Active comparator | Patients can receive platelet infusion but can not receive platelet stimulating factors. Receives: Drug: thrombopoietin (TPO) and interleukin-11 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Platelet recovery after infusion of MPs
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.
Measured: 3 months
Secondary outcomes (2)
frequency of platelet infusion
Measured: 3 months
incidence of acute toxicity
Acute toxicity mainly involves the heart,live and kidney.
Measured: 3 month
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted16 Sep 2014 (estimated)
- Study startSep 2014
- Primary completionSep 2016 (estimated)
- Study completionDec 2016 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Hematological DiseasesStudies of the same condition, open and under way.
- More trials run by Nanfang Hospital, Southern Medical UniversityThe 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 2/3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 250 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Nanfang Hospital, Southern Medical University
- Sponsor type
- University, hospital or other
- Collaborators
- Guangdong Provincial People's Hospital, Third Affiliated Hospital, Sun Yat-Sen University, Guangzhou General Hospital of Guangzhou Military Command, Guangzhou First People's Hospital, Sun Yat-Sen Memorial Hospital of Sun Yat-Sen University, Southern Medical University, China, Peking University People's Hospital, Huazhong University of Science and Technology, Sun Yat-sen University, Academy Military Medical Science, China
- Responsible party
- Principal investigator, Qifa Liu, Nanfang Hospital, Southern Medical University
- Investigators
- Qifa Liu, MD, Principal investigator, Nanfang Hospital, Southern Medical University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02241031
- Sponsor's protocol ID
- MPs-AL-2014
- Status last verified
- Sep 2014
- First submitted
- 13 Sep 2014
- Last updated
- 16 Sep 2014
- Results posted
- No
- Data monitoring committee
- Yes
Publications
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.
PubMed 23390507
Study at a glance
From the registry record, last updated by the sponsor on 16 Sep 2014. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Megakaryocytic Progenitor Cells for Prophylaxis and Treatment of Thrombocytopenia in Patients With Acute Leukemia Receiving Chemotherapy
- ClinicalTrials.gov ID
- NCT02241031
- Acronym / sponsor ID
- MPs-AL-2014
- Conditions
- Hematological Diseases, Thrombocytopenia
- Keywords
- Megakaryocytic Progenitor Products, Hematological Diseases, Thrombocytopenia
- Phase
- Phase 2/3
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 250 participants (estimated)
- Lead sponsor
- Nanfang Hospital, Southern Medical UniversityUniversity, hospital or other
- Collaborators
- Guangdong Provincial People's Hospital, Third Affiliated Hospital, Sun Yat-Sen University, Guangzhou General Hospital of Guangzhou Military Command, Guangzhou First People's Hospital, Sun Yat-Sen Memorial Hospital of Sun Yat-Sen University, Southern Medical University, China, Peking University People's Hospital, Huazhong University of Science and Technology, Sun Yat-sen University, Academy Military Medical Science, China
- Study start
- Sep 2014
- Primary completion
- Sep 2016 (estimated)
- Study completion
- Dec 2016 (estimated)
- First posted
- 16 Sep 2014 (estimated)
- 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.
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 design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| MPs | Experimental | Biological: MPsMPs 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 | Active comparator | Drug: thrombopoietin (TPO) and interleukin-11Patients can receive platelet infusion but can not receive platelet stimulating factors. |
Who may qualify
- Ages
- 14 to 65 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (6 listed)
- 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
Key exclusion criteria (3 listed)
- 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)
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 |
|---|---|---|
| MPsMPs are generated from cord blood using a combination of cytokines. | Biological | MPs |
| thrombopoietin (TPO) and interleukin-11Platelet stimulating factors include thrombopoietin (TPO) and interleukin-11 and so on. | Drug | Non-MPs |
Primary outcome
Platelet recovery after infusion of MPs
Time frame: 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.
2 secondary outcomes
frequency of platelet infusion
Time frame: 3 months
incidence of acute toxicity
Time frame: 3 month
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 16 Sep 2014.
- Background: PubMed 23390507Xi 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.
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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.
Qifa Liu, MD principal investigator
Nanfang Hospital, Southern Medical University
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2/3 study testing MPs and thrombopoietin (TPO) and interleukin-11 in people with Hematological Diseases and Thrombocytopenia, run by Nanfang Hospital, Southern Medical University 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 6 inclusion and 3 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: status not confirmed, as of the sponsor's last update on 16 Sep 2014. 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 combined phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Ren Lin, MD (+86-020-62787883, lansinglinren@hotmail.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Platelet recovery after infusion of MPs (time frame: 3 months). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT02241031, which also lists 1 linked publication.
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 16 Sep 2014.