EltrombopagPhase 3NCT03515096
Eltrombopag vs. rhTPO to Increase Platelet Level After HSCT
A Phase 3 study testing Eltrombopag and rhTPO in people with Eltrombopag and Hematopoietic Stem Cell Transplantation, run by Shenzhen Second People's Hospital at 1 site in China.
Trial summary
The primary objective is to compare the efficacy of eltrombopag vs rhTPO in complete response in patients after HSCT in China. This is a post-marketing, interventional, single-center, double-arm, prospective, open-label, non-inferior, randomized controlled study in adult patients with hematopoietic stem cell… Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Eltrombopag, Hematopoietic Stem Cell Transplantation, Thrombocytopenia
- What is being tested?
- Eltrombopag and rhTPO
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 98 participants
- Where
- 1 siteChina
- Study dates
- 1 Jul 2018to 1 Jul 2022
- Phase
- Phase 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 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Age ≥ 18;
- Patients who have undergone hematopoietic stem cell transplantation (HSCT);
- Patients who don't take any drug including Eltrombopag and rhTPO before hematopoietic stem cell transplantation (HSCT);
- Patients receiving either an autologous (Auto) or allogeneic (Allo) stem cell transplantation from a sibling,related donor, or matched unrelated donor are included;
- Agree to sign informed consent
Who cannot join
- Patients with thrombocytopenia causes by other reasons, such as drugs, cytomegalovirus or infection;
- Patients who had greater risk of thromboembolic disease within six months;
- Patients with a history of heart disease;
- Patients with severe organ dysfunction;
- Patients with other malignancies
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Eltrombopag vs. Recombinant Human Thrombopoietin to Increase Platelet Level After Hematopoietic Stem Cell Transplantation: a Non-inferiority, Open-label, Randomized-controlled Study
The primary objective is to compare the efficacy of eltrombopag vs rhTPO in complete response in patients after HSCT in China. This is a post-marketing, interventional, single-center, double-arm, prospective, open-label, non-inferior, randomized controlled study in adult patients with hematopoietic stem cell transplantation in China. Patients will be recruited consecutively from the study sites during the enrollment period. The enrolled patients will be given eltrombopag or rhTPO under the conditions of informed consent and frequent monitoring according to the clinical guideline.
Read the detailed description
Hematopoietic stem cell transplantation (HSCT) is an important method to treat malignant tumor of blood system, and promoting platelets after HSCT is one of the important factors to determine the success of transplantation.
Recently, the main effective method to treat thrombocytopenia after transplantation is to preventively transfuse platelets. However, this method can cause platelet transfusion related graft-versus-host disease(GVHD), increase the risk of reaction and infection associated with blood transfusion, easily produce platelet related and specific antibodies (HPA), which seriously affect the curative effect of platelet infusion and even render platelet transfusion invalid. Besides, study has shown that the effective rate of platelet transfusion is 30%-70%, and the application of platelets in clinic is limited because of the shortage of blood supply at present.
Recombinant human thrombopoietin (rhTPO) is a glycoprotein molecule modified by full-length glycosylation and is of a similar pharmacological effect with endogenous thrombopoietin (TPO). TPO is a specific cytokine of megakaryocyte, which can promote the proliferation and maturation of macronuclear progenitor cells by binding to the receptor c-mpl. In addition, the chinese State Food and Drug Administration (SFDA) approved rhTPO for the treatment of thrombocytopenia after tumor chemotherapy in 2006. The study shows that rhTPO can increase the mean value of platelet count in the low period of platelet, shorten the time of low platelet, and improve the peripheral blood platelet aggregation rate in the bone marrow depression period.
Eltrombopag is the first oral non-peptide receptor (TPO-R) agonist, which can induce the proliferation and maturation of macronuclear progenitor cells via the Janus kinase (JAK) /signal transducer and activator of transcription (STAT) pathway. In 2008, the U.S. FDA approved the use of eltrombopag for the treatment of chronic idiopathic thrombocytopenia, and it has been widely used in tumor patients with thrombocytopenia after chemotherapy. Tanaka et al. have proved that eltrombopag can improve the average elevation of the platelet count in the bone marrow depression period in patients with stem cell transplantation. Besides, within the 9 patients receiving transplantation, the complete response of patients with primary and secondary thrombocytopenia were 60% and 71% respectively.
In China, studies showed that rhTPO could promote the recovery of platelet count, effectively reduce the infusion of platelet, decrease the risk of transplanting hemorrhage in acute leukemia patients after allo-HSCT. However, it is not clear that the effect of eltrombopag on improving platelet count after the treatment of HSCT. In addition, the comparison of efficacy and safety of eltrombopag vs rhTPO on platelet recovery after HSCT in clinical trial is not investigated. Therefore, it is of great significance to explore the efficacy and safety of eltrombopag and rhTPO in improving the recovery of platelet count after HSCT, which will provide a better method to recover platelet after HSCT.
The purpose of this study is to compare the efficacy of Eltrombopag vs rhTPO in complete response in patients after HSCT in China. To be more precise, Eltrombopag is taken 50 mg po daily from day 1 until platelet is completely effectively recovered after HSCT, while rhTPO is given by subcutaneous injection 15000 u daily from day 1 until platelet is completely effectively recovered after HSCT.
The primary endpoint was cumulative numbers of platelet engraftment (platelet recovery to ≥20 X 109/L for seven consecutive days without transfusion) on day 60 after transplantation
- Being tested
- Eltrombopag, rhTPO
- Keywords
- rhTPO, HSCT, Thrombopoietin
- Medical subject headings
- Thrombocytopenia, Jacobs syndrome
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
EltrombopagDrug
Also called Promacta, Revolade
Eltrombopag 50 mg; po; from day 1 to platelet is completely effective recovered after HSCT.
rhTPODrug
Also called no other name
rhTPO 15000 u; subcutaneously injection; from day 1 to platelet is completely effective recovered after HSCT.
| Study group | Role | What they receive |
|---|---|---|
| Eltrombopag | Experimental | Thrombopoietin- receptor (TPO-R) agonist Receives: Drug: Eltrombopag |
| rhTPO | Placebo comparator | Recombinant human thrombopoietin (rhTPO) Receives: Drug: rhTPO |
What is being measured
The primary outcome is the main question the study is designed to answer.
The cumulative numbers of platelet engraftment (platelet recovery to ≥20 X 109/L for seven consecutive days without transfusion) on day 60 after transplantation
Measured: From the start of study treatment (Day 1) to day 60
Secondary outcomes (4)
Number of Participants (Responders) Achieving a Platelet Count >=30×10^9/L On Day 21 After HSCT
On days 21 after HSCT, the number of participants (responders) with platelet count >=30x10^9/L and no platelet infusion for 7 days were compared between treatments
Measured: From the start of study treatment (Day 1) to days 21
Number of Participants (Responders) Achieving a Platelet Count >=100×10^9/L On Day 21 After HSCT
On days 21 after HSCT, the number of participants (responders) with platelet count >=100x10^9/L and no platelet infusion for 7 days were compared between treatments
Measured: From the start of study treatment (Day 1) to days 21
Number of Participants With Bleeding events as Assessed Using the predefined bleeding scoring system.
The predefined bleeding scoring system is a measure of bleeding severity with the following score: score 0 = no bleeding, score 1= occult blood in body secretions (detected by heme-positive dipstick), mild petechiae, or minimal vaginal spotting, score 2= minor bleeding that does not require red blood cells (RBC) transfusions over routine transfusion needs (epistaxis, vaginal bleeding, mild hematemesis, melena, and mild hematuria) 3 = hemorrhage causing rapid decrease in hematocrit level, necessitating one or more units of RBCs per day beyond routine transfusion needs, and score 4 = Life-threatening hemorrhage, defined as either massive bleeding causing severe hemodynamic compromise or bleeding into vital organ (e.g., intracranial hemorrhage, pericardial, or diffuse alveolar hemorrhage).
Measured: From the start of study treatment (Day 1) to days 90
Number of Participants With Any Adverse Event (AE) or Serious Adverse Event (SAE)
An AE is defined as any unfavorable and unintended sign (including an abnormal laboratory finding), symptom, or disease (new or exacerbated) temporally associated with the use of a medicinal product whether or not considered related to the medicinal product.A SAE is any untoward medical occurrence that, at any dose results in death, is life-threatening, requires hospitalization or prolongation of existing hospitalization, is a congenital anomaly/birth defect or is associated with protocol specified liver injury and impaired liver function or is any protocol specific AEs.
Measured: From the start of study treatment (Day 1) to days 90
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted3 May 2018
- Study start1 Jul 2018
- Primary completion2 Aug 2021
- Study completion1 Jul 2022
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 EltrombopagStudies 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
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 98 participants (actual)
Sponsor and investigators
- Lead sponsor
- Shenzhen Second People's Hospital
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- XIN DU, MD, Principal investigator, Second People's Hospital of Shenzhen
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03515096
- Sponsor's protocol ID
- 201803070044
- Status last verified
- Jun 2022
- First submitted
- 22 Apr 2018
- Last updated
- 27 Jul 2022
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
Wen B, Zhang X, Chen S, Fan J, Yang S, Cai Y, Wang P, Zhang Q, Gu Q, Du X. Oral eltrombopag versus subcutaneous recombinant human thrombopoietin for promoting platelet engraftment after allogeneic stem cell transplantation: A prospective, non-inferiority, randomized controlled trial. Hematol Oncol. 2022 Oct;40(4):777-786. doi: 10.1002/hon.3017. Epub 2022 May 18.
PubMed 35554955
Study at a glance
From the registry record, last updated by the sponsor on 27 Jul 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Eltrombopag vs. Recombinant Human Thrombopoietin to Increase Platelet Level After Hematopoietic Stem Cell Transplantation: a Non-inferiority, Open-label, Randomized-controlled Study
- ClinicalTrials.gov ID
- NCT03515096
- Acronym / sponsor ID
- 201803070044
- Keywords
- rhTPO, HSCT, Thrombopoietin
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 98 participants (actual)
- Lead sponsor
- Shenzhen Second People's HospitalUniversity, hospital or other
- Study start
- 1 Jul 2018 (actual)
- Primary completion
- 2 Aug 2021 (actual)
- Study completion
- 1 Jul 2022 (actual)
- First posted
- 3 May 2018 (actual)
- 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 primary objective is to compare the efficacy of eltrombopag vs rhTPO in complete response in patients after HSCT in China. This is a post-marketing, interventional, single-center, double-arm, prospective, open-label, non-inferior, randomized controlled study in adult patients with hematopoietic stem cell transplantation in China. Patients will be recruited consecutively from the study sites during the enrollment period. The enrolled patients will be given eltrombopag or rhTPO under the conditions of informed consent and frequent monitoring according to the clinical guideline.
Detailed description
Hematopoietic stem cell transplantation (HSCT) is an important method to treat malignant tumor of blood system, and promoting platelets after HSCT is one of the important factors to determine the success of transplantation.
Recently, the main effective method to treat thrombocytopenia after transplantation is to preventively transfuse platelets. However, this method can cause platelet transfusion related graft-versus-host disease(GVHD), increase the risk of reaction and infection associated with blood transfusion, easily produce platelet related and specific antibodies (HPA), which seriously affect the curative effect of platelet infusion and even render platelet transfusion invalid. Besides, study has shown that the effective rate of platelet transfusion is 30%-70%, and the application of platelets in clinic is limited because of the shortage of blood supply at present.
Recombinant human thrombopoietin (rhTPO) is a glycoprotein molecule modified by full-length glycosylation and is of a similar pharmacological effect with endogenous thrombopoietin (TPO). TPO is a specific cytokine of megakaryocyte, which can promote the proliferation and maturation of macronuclear progenitor cells by binding to the receptor c-mpl. In addition, the chinese State Food and Drug Administration (SFDA) approved rhTPO for the treatment of thrombocytopenia after tumor chemotherapy in 2006. The study shows that rhTPO can increase the mean value of platelet count in the low period of platelet, shorten the time of low platelet, and improve the peripheral blood platelet aggregation rate in the bone marrow depression period.
Eltrombopag is the first oral non-peptide receptor (TPO-R) agonist, which can induce the proliferation and maturation of macronuclear progenitor cells via the Janus kinase (JAK) /signal transducer and activator of transcription (STAT) pathway. In 2008, the U.S. FDA approved the use of eltrombopag for the treatment of chronic idiopathic thrombocytopenia, and it has been widely used in tumor patients with thrombocytopenia after chemotherapy. Tanaka et al. have proved that eltrombopag can improve the average elevation of the platelet count in the bone marrow depression period in patients with stem cell transplantation. Besides, within the 9 patients receiving transplantation, the complete response of patients with primary and secondary thrombocytopenia were 60% and 71% respectively.
In China, studies showed that rhTPO could promote the recovery of platelet count, effectively reduce the infusion of platelet, decrease the risk of transplanting hemorrhage in acute leukemia patients after allo-HSCT. However, it is not clear that the effect of eltrombopag on improving platelet count after the treatment of HSCT. In addition, the comparison of efficacy and safety of eltrombopag vs rhTPO on platelet recovery after HSCT in clinical trial is not investigated. Therefore, it is of great significance to explore the efficacy and safety of eltrombopag and rhTPO in improving the recovery of platelet count after HSCT, which will provide a better method to recover platelet after HSCT.
The purpose of this study is to compare the efficacy of Eltrombopag vs rhTPO in complete response in patients after HSCT in China. To be more precise, Eltrombopag is taken 50 mg po daily from day 1 until platelet is completely effectively recovered after HSCT, while rhTPO is given by subcutaneous injection 15000 u daily from day 1 until platelet is completely effectively recovered after HSCT.
The primary endpoint was cumulative numbers of platelet engraftment (platelet recovery to ≥20 X 109/L for seven consecutive days without transfusion) on day 60 after transplantation
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Eltrombopag | Experimental | Drug: EltrombopagThrombopoietin- receptor (TPO-R) agonist |
| rhTPO | Placebo comparator | Drug: rhTPORecombinant human thrombopoietin (rhTPO) |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (5 listed)
- Age ≥ 18;
- Patients who have undergone hematopoietic stem cell transplantation (HSCT);
- Patients who don't take any drug including Eltrombopag and rhTPO before hematopoietic stem cell transplantation (HSCT);
- Patients receiving either an autologous (Auto) or allogeneic (Allo) stem cell transplantation from a sibling,related donor, or matched unrelated donor are included;
- Agree to sign informed consent
Key exclusion criteria (5 listed)
- Patients with thrombocytopenia causes by other reasons, such as drugs, cytomegalovirus or infection;
- Patients who had greater risk of thromboembolic disease within six months;
- Patients with a history of heart disease;
- Patients with severe organ dysfunction;
- Patients with other malignancies
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 |
|---|---|---|
| EltrombopagAlso: Promacta, RevoladeEltrombopag 50 mg; po; from day 1 to platelet is completely effective recovered after HSCT. | Drug | Eltrombopag |
| rhTPOAlso: no other namerhTPO 15000 u; subcutaneously injection; from day 1 to platelet is completely effective recovered after HSCT. | Drug | rhTPO |
Primary outcome
The cumulative numbers of platelet engraftment (platelet recovery to ≥20 X 109/L for seven consecutive days without transfusion) on day 60 after transplantation
Time frame: From the start of study treatment (Day 1) to day 60
4 secondary outcomes
Number of Participants (Responders) Achieving a Platelet Count >=30×10^9/L On Day 21 After HSCT
Time frame: From the start of study treatment (Day 1) to days 21
Number of Participants (Responders) Achieving a Platelet Count >=100×10^9/L On Day 21 After HSCT
Time frame: From the start of study treatment (Day 1) to days 21
Number of Participants With Bleeding events as Assessed Using the predefined bleeding scoring system.
Time frame: From the start of study treatment (Day 1) to days 90
Number of Participants With Any Adverse Event (AE) or Serious Adverse Event (SAE)
Time frame: From the start of study treatment (Day 1) to days 90
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 27 Jul 2022.
- Derived from this study: PubMed 35554955Wen B, Zhang X, Chen S, Fan J, Yang S, Cai Y, Wang P, Zhang Q, Gu Q, Du X. Oral eltrombopag versus subcutaneous recombinant human thrombopoietin for promoting platelet engraftment after allogeneic stem cell transplantation: A prospective, non-inferiority, randomized controlled trial. Hematol Oncol. 2022 Oct;40(4):777-786. doi: 10.1002/hon.3017. Epub 2022 May 18.
- Individual participant dataSharing planned: No
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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.
XIN DU, MD principal investigator
Second People's Hospital of Shenzhen
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Eltrombopag and rhTPO in people with Eltrombopag and Hematopoietic Stem Cell Transplantation, run by Shenzhen Second People's Hospital 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 5 inclusion and 5 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 27 Jul 2022. 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. Some participants in this study may receive a placebo. 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?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
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?
The cumulative numbers of platelet engraftment (platelet recovery to ≥20 X 109/L for seven consecutive days without transfusion) on day 60 after transplantation (time frame: From the start of study treatment (Day 1) to day 60). 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 NCT03515096, 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 27 Jul 2022.