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.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record

Search all trials

Trial summary

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
Are there results?: Yes, posted in the registrySummary in the doctors' view

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

Other trials in China

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 groups in NCT02487563
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.

Oct 201512 Apr 2019

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.
More trials run by The First Affiliated Hospital of Soochow 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 3
Primary purpose: Treatment
Allocation: Randomised
Intervention model: Parallel groups
Masking: None (open label)
Enrolment: 97 participants (actual)

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 3881142
Wang 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 21713321
Zhou 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 25566808
Tang 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

Want to know more?

The sponsor has not listed a contact. The official record may have more details.

Talk to your doctor first and take this page with you. Only the study team can confirm whether you can join.

Questions to ask the study team

What is the study trying to find out?
What would I need to do, and how often would I visit?
Could I receive a placebo instead of the treatment?
What are the possible risks and benefits for me?
Will it cost me anything? Are travel or time paid for?
Can I keep seeing my own doctor during the study?
What happens when the study ends?

See locations

RecruitingObservational study
A Study in Children and, Adults With Congenital Thrombotic Thrombocytopenic Purpura (cTTP) Treated With Adzynma
Thrombotic Thrombocytopenic Purpura (TTP)
RecruitingPhase 3
rhTPO for Thrombocytopenia in Chronic Liver Disease Patients Undergoing Elective Invasive Procedures.
Thrombocytopenia Associated With Chronic Liver Disease
RecruitingPhase 2
A Study to Assess the Tolerability of Ianalumab (VAY736) With Investigator's Choice Thrombopoietin Receptor Agonist (IC TPO-RA) in Patients With Primary Immune Thrombocytopenia (ITP)
Primary Immune Thrombocytopenia (ITP), Primary Evans Syndrome (ES)
Enrolling by invitationObservational study
Screening for Hematology Branch Protocols
Hematologic Disease and Disorders, Donors
RecruitingPhase 1/2
A Study of Pirtobrutinib in Participants With Immune Thrombocytopenia
Immune Thrombocytopenia (ITP)

All Thrombocytopenia trials

Study at a glance

From the registry record, last updated by the sponsor on 6 May 2020. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title: A Prospective Study of Patients With Isolated Thrombocytopenia Following Hematopoietic Stem Cell Transplantation
ClinicalTrials.gov ID: NCT02487563
Acronym / sponsor ID: SOOCHOW-HY-2015
Conditions: Thrombocytopenia, Hematologic Diseases
Keywords: Isolated thrombocytopenia, decitabine, rhTPO
Phase: Phase 3
Study type: Interventional
Recruitment status: Completed
Enrolment: 97 participants (actual)
Lead sponsor: The First Affiliated Hospital of Soochow UniversityUniversity, 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
Study start: Oct 2015
Primary completion: 12 Apr 2019 (actual)
Study completion: 12 Apr 2019 (actual)
First posted: 1 Jul 2015 (estimated)
Results: Posted 6 May 2020

Clinical summary

The research question as the sponsor registered it. This describes what the study plans to test, not what it found.

Detailed description

Study design

Primary purpose: Treatment
Allocation: Randomised
Intervention model: Parallel groups
Masking: None (open label)
Number of arms: 3

Arm	Type	Receives
Experimental Group 1	Experimental	Drug: Decitabine, Drug: rhTPODecitabine in combination with rhTPO.
Experimental Group 2	Experimental	Drug: DecitabineDecitabine
Control Group	Active comparator	Other: Conventional TreatmentConventional treatment except decitabine.

Who may qualify

Sex: All
Healthy volunteers: Not accepted
Age groups: Child, Adult, Older adult

Key inclusion criteria (4 listed)

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;

Key exclusion criteria (6 listed)

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;

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
DecitabineAlso: 5-Aza-2'-deoxycytidineDecitabine	Drug	Experimental Group 1, Experimental Group 2
rhTPOAlso: ThrombopoietinrhTPO	Drug	Experimental Group 1
Conventional Treatmentimmunoglobulin, glucocorticoid etc	Other	Control Group

Primary outcome

Number of Participants With Platelet Count Recovery
Time frame: Up to 4 weeks after the treatment
Platelet response refers to a sustained increase (stable or increasing level) of at least 30×10E9/L independent of transfusion for 3 days.

1 secondary outcome

Megakaryocyte Count
Time frame: Up to 4 weeks after the treatment

Results and safety

Results posted to the registry on 6 May 2020, as reported by the sponsor. Figures are copied from the record, not re-analysed. Full results on ClinicalTrials.gov.

Participant flow

Group	Started	Completed
Experimental Group 1	32	30
Experimental Group 2	33	30
Control Group	32	31

Primary outcome: Number of Participants With Platelet Count Recovery

Time frame: Up to 4 weeks after the treatment. Count of participants. Unit: Participants

Measure	Experimental Group 1 n = 30	Experimental Group 2 n = 30	Control Group n = 31
Value	20	22	6

1 secondary outcome is reported in the full results.

Adverse events

Time frame: Study period (4 weeks). Other adverse events reported at 1% frequency or more

Group	Serious adverse events	Other adverse events	Deaths
Experimental Group 1	4 of 30 (13.3%)	8 of 30 (26.7%)	1 of 30
Experimental Group 2	1 of 30 (3.3%)	5 of 30 (16.7%)	0 of 30
Control Group	3 of 31 (9.7%)	7 of 31 (22.6%)	1 of 31

Limitations and caveats (from the sponsor): Small number of patients, the diversity of patients, and the heterogeneous treatment options in Arm C.

Loading the posted results from ClinicalTrials.gov…

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 6 May 2020.
Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
Results publication: PubMed 3881142First 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.
Results publication: PubMed 21713321Wang 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.
Results publication: PubMed 25566808Zhou 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.
Derived from this study: PubMed 33646303Tang 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.
Individual participant dataSharing planned: No

Related trials for Thrombocytopenia

RecruitingObservational study
A Study in Children and, Adults With Congenital Thrombotic Thrombocytopenic Purpura (cTTP) Treated With Adzynma
Thrombotic Thrombocytopenic Purpura (TTP)
RecruitingPhase 3
rhTPO for Thrombocytopenia in Chronic Liver Disease Patients Undergoing Elective Invasive Procedures.
Thrombocytopenia Associated With Chronic Liver Disease
RecruitingPhase 2
A Study to Assess the Tolerability of Ianalumab (VAY736) With Investigator's Choice Thrombopoietin Receptor Agonist (IC TPO-RA) in Patients With Primary Immune Thrombocytopenia (ITP)
Primary Immune Thrombocytopenia (ITP), Primary Evans Syndrome (ES)
Enrolling by invitationObservational study
Screening for Hematology Branch Protocols
Hematologic Disease and Disorders, Donors
RecruitingPhase 1/2
A Study of Pirtobrutinib in Participants With Immune Thrombocytopenia
Immune Thrombocytopenia (ITP)

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.

Depei Wu, PhD,MD study chair

The First Affiliated Hospital of Soochow University

1 site in 1 country: see every site and its status.

Questions about this trial

What is this clinical trial studying?

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 4 inclusion and 6 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: finished, as of the sponsor's last update on 6 May 2020. 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 phase mean?

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?

Yes. Results are posted in the registry (first posted 6 May 2020). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Number of Participants With Platelet Count Recovery (time frame: Up to 4 weeks after the treatment). 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 NCT02487563, which also lists 4 linked publications.

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 6 May 2020.