Corticosteroid-resistant or Relapsed ITPNCT04214951
A Study of Eltrombopag and Recombinant Human Thrombopoietin In Primary Immune Thrombocytopenia
An observational study of Corticosteroid-resistant or Relapsed ITP, run by Peking University People's Hospital at 1 site in China.
Trial summary
Thrombopoietin Receptor Agonists (TPO-ra) are novel treatments for patients with refractory Primary Immune Thrombocytopenia (ITP). Rh-TPO and eltrombopag increase the number of platelets through different mechanism. If there is cross-resistance between 2 drugs for the treatment of adult ITP is still no answer. Read more about this study
- Who is it for?
- Ages 18 to 75 years
- Conditions the study is for
- Corticosteroid-resistant or Relapsed ITP
- What is being tested?
- Eltrombopag and Recombinant human thrombopoietin (rh-TPO)
- How many people
- 100 participants
- Where
- 1 siteChina
- Study dates
- 1 Jan 2020to Dec 2022
- Phase
- Observational
- 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 18 to 75 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Study population: adult ITP patients
Who may be able to join
- 1.18 years or older
- 2.Primary ITP
- 3.Platelet count ≤ 30 × 109/l
- 4.Normal neutrophils, reticulocyte count, creatinine and liver enzyme values
- 5.Available follow-up of 2 months at least for each period
- 6.Failed initial glucocorticosteroid treatment
- 7.Unwillingness to accept splenectomy or failed splenectomy
- -
Who cannot join
- HIV, hepatitis B or C, Helicobacter pylori infection
- Malignancy
- Congenital or acquired immunologic deficit
- History of thrombosis plus two or more risk factors
- Nursing or pregnant women
- Abnormal liver and renal functions: AST/ALT/total bilirubin ≥1.5 × ULN, creatinine ≥1.5 mg/dl
- Severe heart and lung dysfunctions -
Trial locations and contacts
1 site in 1 country.
China1 site
Peking University Institute of Hematology
Beijing, Beijing Municipality, 100044
About this study
Official title: A Prospective Observational Study of Switching Eltrombopag and Recombinant Human Thrombopoietin In Primary Immune Thrombocytopenia
Thrombopoietin Receptor Agonists (TPO-ra) are novel treatments for patients with refractory Primary Immune Thrombocytopenia (ITP). Rh-TPO and eltrombopag increase the number of platelets through different mechanism. If there is cross-resistance between 2 drugs for the treatment of adult ITP is still no answer. The purpose of this study is to investigate the efficacy and safety of switching eltrombopag and Rh-TPO in adults with ITP.
Read the detailed description
Non-interventional study. Patients who fail previous steroids and receive rh-TPO and then switch to EPAG or vice versa will be enrolled. The reason for switch will be recorded. Patients in the rh-TPO group were given rh-TPO 300 U/kg once daily for 21 days, and those in the eltrombopag group were given eltrombopag 50mg once daily for 6 weeks. Rh-TPO and eltrombopag were terminated any time the platelet counts increased above 100 × 10^9/L in the rh-TPO group and 300 × 10^9/L in the eltrombopag group. The efficacy, safety, and patient/physician preference will be assessed and compared between the two agents.
- Conditions
- Corticosteroid-resistant or Relapsed ITP
- Being tested
- Eltrombopag, Recombinant human thrombopoietin (rh-TPO)
- Keywords
- Immune Thrombocytopenia, Recombinant human thrombopoietin, Eltrombopag, switching
- Medical subject headings
- Purpura, Thrombocytopenic, Idiopathic
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
EltrombopagDrug
Patients will be given eltrombopag 50mg once daily for 6 weeks.
Recombinant human thrombopoietin (rh-TPO)Drug
Patients will be given rh-TPO 300 U/kg once daily for 21 days.
| Study group | Role | What they receive |
|---|---|---|
| Recombinant human thrombopoietin (rh-TPO) group | Patients who fail previous steroids and eltrombopag and then switch to Rh-TPO will be enrolled. The reason for switch will be recorded. Patients will be given rh-TPO 300 U/kg once daily for 21 days. Rh-TPO will be terminated any time the platelet counts increased above 100 × 10^9/L. The efficacy, safety, and patient/physician preference will be assessed. Receives: Drug: Recombinant human thrombopoietin (rh-TPO) | |
| Eltrombopag group | Patients who fail previous steroids and rh-TPO and then switch to eltrombopag will be enrolled. The reason for switch will be recorded. Patients will be given eltrombopag 50mg once daily for 6 weeks. Eltrombopag will be terminated any time the platelet counts increased above 300× 10^9/L.The efficacy, safety, and patient/physician preference will be assessed. Receives: Drug: Eltrombopag |
What is being measured
The primary outcome is the main question the study is designed to answer.
Response Rate at 6 Weeks After Switching
The percentage of patients who have reached platelet count ≥ 50×10^9/L at 6 weeks after switching.
Measured: 6 weeks
Secondary outcomes (5)
Treatments Associated Adverse Events
Adverse event/serious adverse event associated with study drugs during 6 weeks after switching
Measured: 6 weeks
Reasons of Switching
Reasons of switching eltrombopag and rh-TPO will be recorded, including lack of efficacy, patient preference, side effects, platelet count fluctuation
Measured: 6 weeks
Number of Participants With Bleeding Events
Number of participants with bleeding events of the two groups during 6 weeks after switching
Measured: 6 weeks
TOR (Time to Response)
The time to achieve platelet count ≥ 50×10^9/L after switching.
Measured: 6 weeks
DOR (Duration of Response)
The duration of achieving platelet count ≥ 50×10^9/L after switching.
Measured: 6 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted2 Jan 2020
- Study start1 Jan 2020
- Primary completion1 Aug 2022 (estimated)
- Study completionDec 2022 (estimated)
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 (13)Papers the sponsor has linked to this study.
- Other trials for Corticosteroid-resistant or Relapsed ITPStudies of the same condition, open and under way.
- More trials run by Peking University People's HospitalThe 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
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 100 participants (actual)
Sponsor and investigators
- Lead sponsor
- Peking University People's Hospital
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Xiao Hui Zhang, Peking University People's Hospital
- Investigators
- Xiao Hui Zhang, MD, Principal investigator, Peking University People's Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04214951
- Sponsor's protocol ID
- ZXH-ITP2019
- Status last verified
- Oct 2020
- First submitted
- 28 Dec 2019
- Last updated
- 20 Oct 2020
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
Neunert C, Lim W, Crowther M, Cohen A, Solberg L Jr, Crowther MA; American Society of Hematology. The American Society of Hematology 2011 evidence-based practice guideline for immune thrombocytopenia. Blood. 2011 Apr 21;117(16):4190-207. doi: 10.1182/blood-2010-08-302984. Epub 2011 Feb 16.
PubMed 21325604Provan D, Stasi R, Newland AC, Blanchette VS, Bolton-Maggs P, Bussel JB, Chong BH, Cines DB, Gernsheimer TB, Godeau B, Grainger J, Greer I, Hunt BJ, Imbach PA, Lyons G, McMillan R, Rodeghiero F, Sanz MA, Tarantino M, Watson S, Young J, Kuter DJ. International consensus report on the investigation and management of primary immune thrombocytopenia. Blood. 2010 Jan 14;115(2):168-86. doi: 10.1182/blood-2009-06-225565. Epub 2009 Oct 21.
PubMed 19846889Ghanima W, Godeau B, Cines DB, Bussel JB. How I treat immune thrombocytopenia: the choice between splenectomy or a medical therapy as a second-line treatment. Blood. 2012 Aug 2;120(5):960-9. doi: 10.1182/blood-2011-12-309153. Epub 2012 Jun 26.
PubMed 22740443Deutsch VR, Tomer A. Advances in megakaryocytopoiesis and thrombopoiesis: from bench to bedside. Br J Haematol. 2013 Jun;161(6):778-93. doi: 10.1111/bjh.12328. Epub 2013 Apr 18.
PubMed 23594368Kuter DJ. The biology of thrombopoietin and thrombopoietin receptor agonists. Int J Hematol. 2013 Jul;98(1):10-23. doi: 10.1007/s12185-013-1382-0. Epub 2013 Jul 3.
PubMed 23821332Bussel JB, Lakkaraja M. Thrombopoietic agents: there is still much to learn. Presse Med. 2014 Apr;43(4 Pt 2):e69-78. doi: 10.1016/j.lpm.2014.02.008. Epub 2014 Mar 27.
PubMed 24680422Wormann B. Clinical indications for thrombopoietin and thrombopoietin-receptor agonists. Transfus Med Hemother. 2013 Oct;40(5):319-25. doi: 10.1159/000355006. Epub 2013 Sep 11.
PubMed 24273485Wang S, Yang R, Zou P, Hou M, Wu D, Shen Z, Lu X, Li Y, Chen X, Niu T, Sun H, Yu L, Wang Z, Zhang Y, Chang N, Zhang G, Zhao Y. A multicenter randomized controlled trial of recombinant human thrombopoietin treatment in patients with primary immune thrombocytopenia. Int J Hematol. 2012 Aug;96(2):222-8. doi: 10.1007/s12185-012-1124-8. Epub 2012 Jun 30.
PubMed 22753022Erickson-Miller CL, Delorme E, Tian SS, Hopson CB, Landis AJ, Valoret EI, Sellers TS, Rosen J, Miller SG, Luengo JI, Duffy KJ, Jenkins JM. Preclinical activity of eltrombopag (SB-497115), an oral, nonpeptide thrombopoietin receptor agonist. Stem Cells. 2009 Feb;27(2):424-30. doi: 10.1634/stemcells.2008-0366.
PubMed 19038790Erhardt JA, Erickson-Miller CL, Aivado M, Abboud M, Pillarisetti K, Toomey JR. Comparative analyses of the small molecule thrombopoietin receptor agonist eltrombopag and thrombopoietin on in vitro platelet function. Exp Hematol. 2009 Sep;37(9):1030-7. doi: 10.1016/j.exphem.2009.06.011. Epub 2009 Jul 24.
PubMed 19631713Gonzalez-Porras JR, Godeau B, Carpenedo M. Switching thrombopoietin receptor agonist treatments in patients with primary immune thrombocytopenia. Ther Adv Hematol. 2019 May 9;10:2040620719837906. doi: 10.1177/2040620719837906. eCollection 2019.
PubMed 31156798Cantoni S, Carpenedo M, Mazzucconi MG, De Stefano V, Carrai V, Ruggeri M, Specchia G, Vianelli N, Pane F, Consoli U, Artoni A, Zaja F, D'adda M, Visentin A, Ferrara F, Barcellini W, Caramazza D, Baldacci E, Rossi E, Ricco A, Ciminello A, Rodeghiero F, Nichelatti M, Cairoli R. Alternate use of thrombopoietin receptor agonists in adult primary immune thrombocytopenia patients: A retrospective collaborative survey from Italian hematology centers. Am J Hematol. 2018 Jan;93(1):58-64. doi: 10.1002/ajh.24935. Epub 2017 Nov 9.
PubMed 28983953Cai X, Fu H, Zhao X, Lu J, Jiang Q, Chang Y, Huang X, Zhang X. Switching between eltrombopag and recombinant human thrombopoietin in patients with immune thrombocytopenia: an observational study. Chin Med J (Engl). 2022 Oct 5;135(19):2344-2350. doi: 10.1097/CM9.0000000000002346.
PubMed 36535011
Study at a glance
From the registry record, last updated by the sponsor on 20 Oct 2020. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Prospective Observational Study of Switching Eltrombopag and Recombinant Human Thrombopoietin In Primary Immune Thrombocytopenia
- ClinicalTrials.gov ID
- NCT04214951
- Acronym / sponsor ID
- ZXH-ITP2019
- Conditions
- Corticosteroid-resistant or Relapsed ITP
- Keywords
- Immune Thrombocytopenia, Recombinant human thrombopoietin, Eltrombopag, switching
- Study type
- Observational
- Recruitment status
- Unknown status
- Enrolment
- 100 participants (actual)
- Lead sponsor
- Peking University People's HospitalUniversity, hospital or other
- Study start
- 1 Jan 2020 (actual)
- Primary completion
- 1 Aug 2022 (estimated)
- Study completion
- Dec 2022 (estimated)
- First posted
- 2 Jan 2020 (actual)
- Results
- Posted 20 Oct 2020
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
Thrombopoietin Receptor Agonists (TPO-ra) are novel treatments for patients with refractory Primary Immune Thrombocytopenia (ITP). Rh-TPO and eltrombopag increase the number of platelets through different mechanism. If there is cross-resistance between 2 drugs for the treatment of adult ITP is still no answer. The purpose of this study is to investigate the efficacy and safety of switching eltrombopag and Rh-TPO in adults with ITP.
Detailed description
Non-interventional study. Patients who fail previous steroids and receive rh-TPO and then switch to EPAG or vice versa will be enrolled. The reason for switch will be recorded. Patients in the rh-TPO group were given rh-TPO 300 U/kg once daily for 21 days, and those in the eltrombopag group were given eltrombopag 50mg once daily for 6 weeks. Rh-TPO and eltrombopag were terminated any time the platelet counts increased above 100 × 10^9/L in the rh-TPO group and 300 × 10^9/L in the eltrombopag group. The efficacy, safety, and patient/physician preference will be assessed and compared between the two agents.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Recombinant human thrombopoietin (rh-TPO) group | – | Drug: Recombinant human thrombopoietin (rh-TPO)Patients who fail previous steroids and eltrombopag and then switch to Rh-TPO will be enrolled. The reason for switch will be recorded. Patients will be given rh-TPO 300 U/kg once daily for 21 days. Rh-TPO will be terminated any time the platelet counts increased above 100 × 10^9/L. The efficacy, safety, and patient/physician preference will be assessed. |
| Eltrombopag group | – | Drug: EltrombopagPatients who fail previous steroids and rh-TPO and then switch to eltrombopag will be enrolled. The reason for switch will be recorded. Patients will be given eltrombopag 50mg once daily for 6 weeks. Eltrombopag will be terminated any time the platelet counts increased above 300× 10^9/L.The efficacy, safety, and patient/physician preference will be assessed. |
Who may qualify
- Ages
- 18 to 75 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
- Study population
- adult ITP patients
Key inclusion criteria (8 listed)
- 1.18 years or older
- 2.Primary ITP
- 3.Platelet count ≤ 30 × 109/l
- 4.Normal neutrophils, reticulocyte count, creatinine and liver enzyme values
- 5.Available follow-up of 2 months at least for each period
Key exclusion criteria (7 listed)
- HIV, hepatitis B or C, Helicobacter pylori infection
- Malignancy
- Congenital or acquired immunologic deficit
- History of thrombosis plus two or more risk factors
- Nursing or pregnant women
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 |
|---|---|---|
| EltrombopagPatients will be given eltrombopag 50mg once daily for 6 weeks. | Drug | Eltrombopag group |
| Recombinant human thrombopoietin (rh-TPO)Patients will be given rh-TPO 300 U/kg once daily for 21 days. | Drug | Recombinant human thrombopoietin (rh-TPO) group |
Primary outcome
Response Rate at 6 Weeks After Switching
Time frame: 6 weeks
The percentage of patients who have reached platelet count ≥ 50×10^9/L at 6 weeks after switching.
5 secondary outcomes
Treatments Associated Adverse Events
Time frame: 6 weeks
Reasons of Switching
Time frame: 6 weeks
Number of Participants With Bleeding Events
Time frame: 6 weeks
TOR (Time to Response)
Time frame: 6 weeks
DOR (Duration of Response)
Time frame: 6 weeks
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 20 Oct 2020.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Background: PubMed 21325604Neunert C, Lim W, Crowther M, Cohen A, Solberg L Jr, Crowther MA; American Society of Hematology. The American Society of Hematology 2011 evidence-based practice guideline for immune thrombocytopenia. Blood. 2011 Apr 21;117(16):4190-207. doi: 10.1182/blood-2010-08-302984. Epub 2011 Feb 16.
- Background: PubMed 19846889Provan D, Stasi R, Newland AC, Blanchette VS, Bolton-Maggs P, Bussel JB, Chong BH, Cines DB, Gernsheimer TB, Godeau B, Grainger J, Greer I, Hunt BJ, Imbach PA, Lyons G, McMillan R, Rodeghiero F, Sanz MA, Tarantino M, Watson S, Young J, Kuter DJ. International consensus report on the investigation and management of primary immune thrombocytopenia. Blood. 2010 Jan 14;115(2):168-86. doi: 10.1182/blood-2009-06-225565. Epub 2009 Oct 21.
- Background: PubMed 22740443Ghanima W, Godeau B, Cines DB, Bussel JB. How I treat immune thrombocytopenia: the choice between splenectomy or a medical therapy as a second-line treatment. Blood. 2012 Aug 2;120(5):960-9. doi: 10.1182/blood-2011-12-309153. Epub 2012 Jun 26.
- Background: PubMed 23594368Deutsch VR, Tomer A. Advances in megakaryocytopoiesis and thrombopoiesis: from bench to bedside. Br J Haematol. 2013 Jun;161(6):778-93. doi: 10.1111/bjh.12328. Epub 2013 Apr 18.
- Background: PubMed 23821332Kuter DJ. The biology of thrombopoietin and thrombopoietin receptor agonists. Int J Hematol. 2013 Jul;98(1):10-23. doi: 10.1007/s12185-013-1382-0. Epub 2013 Jul 3.
- Background: PubMed 24680422Bussel JB, Lakkaraja M. Thrombopoietic agents: there is still much to learn. Presse Med. 2014 Apr;43(4 Pt 2):e69-78. doi: 10.1016/j.lpm.2014.02.008. Epub 2014 Mar 27.
- Background: PubMed 24273485Wormann B. Clinical indications for thrombopoietin and thrombopoietin-receptor agonists. Transfus Med Hemother. 2013 Oct;40(5):319-25. doi: 10.1159/000355006. Epub 2013 Sep 11.
- Background: PubMed 22753022Wang S, Yang R, Zou P, Hou M, Wu D, Shen Z, Lu X, Li Y, Chen X, Niu T, Sun H, Yu L, Wang Z, Zhang Y, Chang N, Zhang G, Zhao Y. A multicenter randomized controlled trial of recombinant human thrombopoietin treatment in patients with primary immune thrombocytopenia. Int J Hematol. 2012 Aug;96(2):222-8. doi: 10.1007/s12185-012-1124-8. Epub 2012 Jun 30.
- Background: PubMed 19038790Erickson-Miller CL, Delorme E, Tian SS, Hopson CB, Landis AJ, Valoret EI, Sellers TS, Rosen J, Miller SG, Luengo JI, Duffy KJ, Jenkins JM. Preclinical activity of eltrombopag (SB-497115), an oral, nonpeptide thrombopoietin receptor agonist. Stem Cells. 2009 Feb;27(2):424-30. doi: 10.1634/stemcells.2008-0366.
- Background: PubMed 19631713Erhardt JA, Erickson-Miller CL, Aivado M, Abboud M, Pillarisetti K, Toomey JR. Comparative analyses of the small molecule thrombopoietin receptor agonist eltrombopag and thrombopoietin on in vitro platelet function. Exp Hematol. 2009 Sep;37(9):1030-7. doi: 10.1016/j.exphem.2009.06.011. Epub 2009 Jul 24.
- Background: PubMed 31156798Gonzalez-Porras JR, Godeau B, Carpenedo M. Switching thrombopoietin receptor agonist treatments in patients with primary immune thrombocytopenia. Ther Adv Hematol. 2019 May 9;10:2040620719837906. doi: 10.1177/2040620719837906. eCollection 2019.
- Background: PubMed 28983953Cantoni S, Carpenedo M, Mazzucconi MG, De Stefano V, Carrai V, Ruggeri M, Specchia G, Vianelli N, Pane F, Consoli U, Artoni A, Zaja F, D'adda M, Visentin A, Ferrara F, Barcellini W, Caramazza D, Baldacci E, Rossi E, Ricco A, Ciminello A, Rodeghiero F, Nichelatti M, Cairoli R. Alternate use of thrombopoietin receptor agonists in adult primary immune thrombocytopenia patients: A retrospective collaborative survey from Italian hematology centers. Am J Hematol. 2018 Jan;93(1):58-64. doi: 10.1002/ajh.24935. Epub 2017 Nov 9.
- 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.
Xiao Hui Zhang, MD principal investigator
Peking University People's Hospital
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Corticosteroid-resistant or Relapsed ITP, run by Peking University 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 8 inclusion and 7 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 20 Oct 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.
Are results available for this study?
Yes. Results are posted in the registry (first posted 20 Oct 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?
Response Rate at 6 Weeks After Switching (time frame: 6 weeks). 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 NCT04214951, which also lists 13 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 20 Oct 2020.