ThrombocytopeniaPhase 3NCT02487563
Prospective Study of Patients With Thrombocytopenia Following HSCT
A Phase 3 study testing Decitabine, rhTPO and Conventional Treatment in people with Thrombocytopenia and Hematologic Diseases, run by The First Affiliated Hospital of Soochow University at 1 site in China.
Trial summary
Isolated thrombocytopenia is a common and severe complication of HSCT, which often leads to an increased risk of life-threatening hemorrhage, frequent requirement of platelet transfusions and extended hospital stays, representing a challenging clinical problem. Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Thrombocytopenia, Hematologic Diseases
- What is being tested?
- Decitabine, rhTPO and Conventional Treatment
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 97 participants
- Where
- 1 siteChina
- Study dates
- Oct 2015to 12 Apr 2019
- Phase
- Phase 3
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 Any age
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Platelet count ≤ 30 × 109/L persistently at day 60 post-HSCT or later;
- Neutrophil and hemoglobin were well recovered;
- Full donor chimerism was achieved;
- No response to conventional treatments (e.g. thrombopoietin, immunoglobulin, glucocorticoid alone or in combination) for a duration of at least 4 weeks;
Who cannot join
- Patients with malignancy relapse;
- Active infections;
- Grade Ⅲ-Ⅳ acute GVHD or severe chronic GVHD according to National Institute of Health criteria;
- Severe organ damage;
- Thrombosis requiring treatment;
- Received decitabine following the current transplantation.
Trial locations and contacts
1 site in 1 country.
China1 site
The First affiliated Hospital of SooChow University
Suzhou, Jiangsu, 215000
About this study
Official title: A Prospective Study of Patients With Isolated Thrombocytopenia Following Hematopoietic Stem Cell Transplantation
Isolated thrombocytopenia is a common and severe complication of HSCT, which often leads to an increased risk of life-threatening hemorrhage, frequent requirement of platelet transfusions and extended hospital stays, representing a challenging clinical problem. Current treatments for thrombocytopenia after HSCT are frequently unsatisfactory in platelet recovery and for preventing potentially fatal bleeding complications. Therefore, it is urgent to explore an effective therapy to improve the outcomes of thrombocytopenia after HSCT. Previous studies have demonstrated that decitabine, a hypomethylating agent, may reduce platelet transfusions in myelodysplastic syndrome (MDS) patients. The investigators conducted an prospective clinical trial to evaluate the safety and efficiency of rhTPO and decitabine in the treatment of thrombocytopenia following HSCT.
Read the detailed description
Isolated thrombocytopenia is a frequent and severe complication of hematopoietic stem cell transplantation (HSCT). It often leads to an increased risk of life-threatening hemorrhage, frequent requirement of platelet transfusions and extended hospital stays, representing a challenging clinical problem. Current treatments for thrombocytopenia after HSCT, including thrombopoietin, interleukin-11, immunoglobulin, methylprednisolone and rituximab, are frequently unsatisfactory in platelet recovery. Therefore, it is urgent to explore an effective therapy to improve the outcomes of thrombocytopenia after HSCT. Thrombopoietin (TPO) is a cytokine that drives thrombopoiesis by stimulating the differentiation of stem cells into megakaryocytes and promoting megakaryocyte proliferation and polyploidization. Decitabine was approved for the treatment of myelodysplastic syndrome (MDS) as a DNA methylation inhibitors. Studies in vitro show that decitabine enhances platelet release and megakaryocyte maturation. Here, the investigators performed a prospective clinical trial, in order to investigate the safety and efficiency of rhTPO and decitabine in the treatment of thrombocytopenia following HSCT.
- Conditions
- Thrombocytopenia, Hematologic Diseases
- Being tested
- Decitabine, rhTPO, Conventional Treatment
- Keywords
- Isolated thrombocytopenia, decitabine, rhTPO
- Medical subject headings
- Thrombocytopenia, Hematologic Diseases
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
DecitabineDrug
Also called 5-Aza-2'-deoxycytidine
Decitabine
rhTPODrug
Also called Thrombopoietin
rhTPO
Conventional TreatmentOther
immunoglobulin, glucocorticoid etc
| Study group | Role | What they receive |
|---|---|---|
| Experimental Group 1 | Experimental | Decitabine in combination with rhTPO. Receives: Drug: Decitabine; Drug: rhTPO |
| Experimental Group 2 | Experimental | Decitabine Receives: Drug: Decitabine |
| Control Group | Active comparator | Conventional treatment except decitabine. Receives: Other: Conventional Treatment |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of Participants With Platelet Count Recovery
Platelet response refers to a sustained increase (stable or increasing level) of at least 30×10E9/L independent of transfusion for 3 days.
Measured: Up to 4 weeks after the treatment
Secondary outcomes (1)
Megakaryocyte Count
The total number of megakaryocytes as well as the platelet-shedding megakaryocytes of bone marrow smears (per cm2) was counted and cross-checked by blinded observers.
Measured: Up to 4 weeks after the treatment
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted1 Jul 2015 (estimated)
- Study startOct 2015
- Primary completion12 Apr 2019
- Study completion12 Apr 2019
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (4)Papers the sponsor has linked to this study.
- Other trials for ThrombocytopeniaStudies of the same condition, open and under way.
- 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 3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 97 participants (actual)
Sponsor and investigators
- Lead sponsor
- The First Affiliated Hospital of Soochow University
- Sponsor type
- University, hospital or other
- Collaborators
- Peking University People's Hospital, Nanfang Hospital, Southern Medical University, Ruijin Hospital, Union Hospital, Tongji Medical College, Huazhong University of Science and Technology, Qilu Hospital of Shandong University, Children's Hospital of Soochow University, Fujian Medical University Union Hospital, Hebei Yanda Ludaopei Hospital
- Responsible party
- Sponsor
- Investigators
- Depei Wu, PhD,MD, Study chair, The First Affiliated Hospital of Soochow University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02487563
- Sponsor's protocol ID
- SOOCHOW-HY-2015
- Status last verified
- Apr 2020
- First submitted
- 21 Jun 2015
- Last updated
- 6 May 2020
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- Individual data sharing
- No
Publications
First LR, Smith BR, Lipton J, Nathan DG, Parkman R, Rappeport JM. Isolated thrombocytopenia after allogeneic bone marrow transplantation: existence of transient and chronic thrombocytopenic syndromes. Blood. 1985 Feb;65(2):368-74.
PubMed 3881142Wang J, Yi Z, Wang S, Li Z. The effect of decitabine on megakaryocyte maturation and platelet release. Thromb Haemost. 2011 Aug;106(2):337-43. doi: 10.1160/TH10-11-0744. Epub 2011 Jun 28.
PubMed 21713321Zhou H, Hou Y, Liu X, Qiu J, Feng Q, Wang Y, Zhang X, Min Y, Shao L, Liu X, Li G, Li L, Yang L, Xu S, Ni H, Peng J, Hou M. Low-dose decitabine promotes megakaryocyte maturation and platelet production in healthy controls and immune thrombocytopenia. Thromb Haemost. 2015 May;113(5):1021-34. doi: 10.1160/TH14-04-0342. Epub 2015 Jan 8.
PubMed 25566808Tang Y, Chen J, Liu Q, Chu T, Pan T, Liang J, He XF, Chen F, Yang T, Ma X, Wu X, Hu S, Cao X, Hu X, Hu J, Liu Y, Qi J, Shen Y, Ruan C, Han Y, Wu D. Low-dose decitabine for refractory prolonged isolated thrombocytopenia after HCT: a randomized multicenter trial. Blood Adv. 2021 Mar 9;5(5):1250-1258. doi: 10.1182/bloodadvances.2020002790.
PubMed 33646303