Immune Thrombocytopenic PurpuraPhase 2NCT01652599
Eltrombopag and High-dose Dexamethasone as First Line Treatment for IT
A Phase 2 study testing Eltrombopag and dexamethasone in people with Immune Thrombocytopenic Purpura, run by Hospital Universitario Dr. Jose E. Gonzalez at 1 site in Mexico.
Trial summary
The purpose of this study is to determine the response rate and response duration with the combination of eltrombopag and high-dose dexamethasone Read more about this study
- Who is it for?
- Ages 18 to 85 years
- Conditions the study is for
- Immune Thrombocytopenic Purpura
- What is being tested?
- Eltrombopag and dexamethasone
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 30 participants
- Where
- 1 siteMexico
- Study dates
- Jun 2012to Nov 2013
- Phase
- Phase 2
- 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 to 85 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Clinically confirmed immune thrombocytopenic (IT) Platelet count less than 30,000/mm3 on two occasions. Platelets >30000/mm3 with bleeding.
- Less than seven days taking corticosteroids
- Normal to increased numbers of megakaryocytes on bone marrow examination in patients ≥ 60 years
- Subject is ≥ 18 years
- Subject has signed and dated written informed consent.
- No sepsis or fever
Show all 12Show fewer
- No active infection requiring therapy
- No active chronic viral infection
- HIV negative
- Not pregnant or nursing
- Negative pregnancy test
- Fertile patients must use effective contraception
Who cannot join
- Performance status above or equal to 2.
- Previous treatment with eltrombopag
- Immunosuppressive treatment within the last month
- Previous splenectomy
- Presence of malignant haematological disease
- Connective tissue disease
Show all 9Show fewer
- Autoimmune hemolytic anemia
- Pregnancy and lactation
- Not willing to participate in the study.
Trial locations and contacts
1 site in 1 country.
Mexico1 site
Hospital Universitario "Dr. Jose E. Gonzalez" UANL
Monterrey, Nuevo León, 64460
About this study
Official title: Eltrombopag and High-dose Dexamethasone as First Line Treatment for Immune Thrombocytopenia
The purpose of this study is to determine the response rate and response duration with the combination of eltrombopag and high-dose dexamethasone
Read the detailed description
Immune Thrombocytopenia is an autoimmune disorder characterized by formation of autoantibodies against platelet antigens leading platelet destruction.
Corticosteroids increase the platelet count in about 80 percent of patients.However, many patients have a relapse when the dose of corticosteroid is reduced. Debilitating side effects are common in patients who require long-term corticosteroid therapy to maintain the platelet count. Eltrombopag, it is a small molecule agonist of the c-mpl (TpoR) receptor, which is the physiological target of the hormone thrombopoietin, has been shown to be effectively raise the platelet count in adult patients (aged 18 years and over) who have had their spleen removed or where splenectomy is not an option and have received prior treatment with corticosteroids or immunoglobulins, and these medicines did not work (refractary ITP). There are a few case reports where eltrombopag was an option as first line treatment for IT.
The purpose of this study is to determine the response rate and response duration with the combination of eltrombopag (50mg PO once a day for 4 weeks) and high-dose dexamethasone (40mg PO days 1,2,3,4) in untreated adult patients immune thrombocytopenic or in patients with less than 7 days of treatment with corticosteroids.
A complete platelet response is defined as an increase in platelet counts to >150×109/L on two consecutive occasions. A partial response is defined as an increase in the platelet count to between 50 and 150×109/L on two consecutive occasions, 1 week apart. Duration of response is considered from the day of the initial administration to the first time of relapse (platelet count <30×109/L)or to time of analysis.
At the end of the first 5 weeks, the patients will followed by 6 months every month.
- Conditions
- Immune Thrombocytopenic Purpura
- Being tested
- Eltrombopag and dexamethasone
- Keywords
- Eltrombopag, Dexamethasone, High-dose, ITP
- 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.
Eltrombopag and dexamethasoneDrug
Also called Revolade
Eltrombopag 50 mg PO days 5-32 Dexamethasone 40 mg PO days 1-4
| Study group | Role | What they receive |
|---|---|---|
| Eltrombopag and dexamethasone | Experimental | Not described Receives: Drug: Eltrombopag and dexamethasone |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of patients with sustained response after 6 months
Number of patients with complete response at month 6
Measured: 6 months
Secondary outcomes (2)
Number of patients with complete response at month 6
Number of patients with platelet count at least 150x109/L, 6 months after therapy
Measured: month 6
Bleeding
Number of patients with bleeding complication therapy
Measured: month 6
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted30 Jul 2012 (estimated)
- Study startJun 2012
- Primary completionJun 2013
- Study completionNov 2013
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (3)Papers the sponsor has linked to this study.
- Other trials for Immune Thrombocytopenic PurpuraStudies 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 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 30 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Hospital Universitario Dr. Jose E. Gonzalez
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, David Gomez Almaguer, Hospital Universitario Dr. Jose E. Gonzalez
- Investigators
- David Gomez-Almaguer, MD, Principal investigator, Hospital Universitario Dr. Jose E. Gonzalez
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01652599
- Sponsor's protocol ID
- HE12-010
- Status last verified
- Jan 2014
- First submitted
- 26 Jul 2012
- Last updated
- 7 Jan 2014
- Results posted
- No
- Data monitoring committee
- No
Publications
Bussel JB, Provan D, Shamsi T, Cheng G, Psaila B, Kovaleva L, Salama A, Jenkins JM, Roychowdhury D, Mayer B, Stone N, Arning M. Effect of eltrombopag on platelet counts and bleeding during treatment of chronic idiopathic thrombocytopenic purpura: a randomised, double-blind, placebo-controlled trial. Lancet. 2009 Feb 21;373(9664):641-8. doi: 10.1016/S0140-6736(09)60402-5.
PubMed 19231632Jenkins JM, Williams D, Deng Y, Uhl J, Kitchen V, Collins D, Erickson-Miller CL. Phase 1 clinical study of eltrombopag, an oral, nonpeptide thrombopoietin receptor agonist. Blood. 2007 Jun 1;109(11):4739-41. doi: 10.1182/blood-2006-11-057968. Epub 2007 Feb 27.
PubMed 17327409Gomez-Almaguer D, Herrera-Rojas MA, Jaime-Perez JC, Gomez-De Leon A, Cantu-Rodriguez OG, Gutierrez-Aguirre CH, Tarin-Arzaga L, Hernandez-Reyes J, Ruiz-Arguelles GJ. Eltrombopag and high-dose dexamethasone as frontline treatment of newly diagnosed immune thrombocytopenia in adults. Blood. 2014 Jun 19;123(25):3906-8. doi: 10.1182/blood-2014-01-549360. Epub 2014 May 6.
PubMed 24802773
Study at a glance
From the registry record, last updated by the sponsor on 7 Jan 2014. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Eltrombopag and High-dose Dexamethasone as First Line Treatment for Immune Thrombocytopenia
- ClinicalTrials.gov ID
- NCT01652599
- Acronym / sponsor ID
- HE12-010
- Conditions
- Immune Thrombocytopenic Purpura
- Keywords
- Eltrombopag, Dexamethasone, High-dose, ITP
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 30 participants (estimated)
- Lead sponsor
- Hospital Universitario Dr. Jose E. GonzalezUniversity, hospital or other
- Study start
- Jun 2012
- Primary completion
- Jun 2013 (actual)
- Study completion
- Nov 2013 (actual)
- First posted
- 30 Jul 2012 (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 determine the response rate and response duration with the combination of eltrombopag and high-dose dexamethasone
Detailed description
Immune Thrombocytopenia is an autoimmune disorder characterized by formation of autoantibodies against platelet antigens leading platelet destruction.
Corticosteroids increase the platelet count in about 80 percent of patients.However, many patients have a relapse when the dose of corticosteroid is reduced. Debilitating side effects are common in patients who require long-term corticosteroid therapy to maintain the platelet count. Eltrombopag, it is a small molecule agonist of the c-mpl (TpoR) receptor, which is the physiological target of the hormone thrombopoietin, has been shown to be effectively raise the platelet count in adult patients (aged 18 years and over) who have had their spleen removed or where splenectomy is not an option and have received prior treatment with corticosteroids or immunoglobulins, and these medicines did not work (refractary ITP). There are a few case reports where eltrombopag was an option as first line treatment for IT.
The purpose of this study is to determine the response rate and response duration with the combination of eltrombopag (50mg PO once a day for 4 weeks) and high-dose dexamethasone (40mg PO days 1,2,3,4) in untreated adult patients immune thrombocytopenic or in patients with less than 7 days of treatment with corticosteroids.
A complete platelet response is defined as an increase in platelet counts to >150×109/L on two consecutive occasions. A partial response is defined as an increase in the platelet count to between 50 and 150×109/L on two consecutive occasions, 1 week apart. Duration of response is considered from the day of the initial administration to the first time of relapse (platelet count <30×109/L)or to time of analysis.
At the end of the first 5 weeks, the patients will followed by 6 months every month.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Eltrombopag and dexamethasone | Experimental | Drug: Eltrombopag and dexamethasone |
Who may qualify
- Ages
- 18 to 85 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (12 listed)
- Clinically confirmed immune thrombocytopenic (IT) Platelet count less than 30,000/mm3 on two occasions. Platelets >30000/mm3 with bleeding.
- Less than seven days taking corticosteroids
- Normal to increased numbers of megakaryocytes on bone marrow examination in patients ≥ 60 years
- Subject is ≥ 18 years
- Subject has signed and dated written informed consent.
Key exclusion criteria (9 listed)
- Performance status above or equal to 2.
- Previous treatment with eltrombopag
- Immunosuppressive treatment within the last month
- Previous splenectomy
- Presence of malignant haematological disease
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 |
|---|---|---|
| Eltrombopag and dexamethasoneAlso: RevoladeEltrombopag 50 mg PO days 5-32 Dexamethasone 40 mg PO days 1-4 | Drug | Eltrombopag and dexamethasone |
Primary outcome
Number of patients with sustained response after 6 months
Time frame: 6 months
Number of patients with complete response at month 6
2 secondary outcomes
Number of patients with complete response at month 6
Time frame: month 6
Bleeding
Time frame: month 6
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 7 Jan 2014.
- Background: PubMed 19231632Bussel JB, Provan D, Shamsi T, Cheng G, Psaila B, Kovaleva L, Salama A, Jenkins JM, Roychowdhury D, Mayer B, Stone N, Arning M. Effect of eltrombopag on platelet counts and bleeding during treatment of chronic idiopathic thrombocytopenic purpura: a randomised, double-blind, placebo-controlled trial. Lancet. 2009 Feb 21;373(9664):641-8. doi: 10.1016/S0140-6736(09)60402-5.
- Background: PubMed 17327409Jenkins JM, Williams D, Deng Y, Uhl J, Kitchen V, Collins D, Erickson-Miller CL. Phase 1 clinical study of eltrombopag, an oral, nonpeptide thrombopoietin receptor agonist. Blood. 2007 Jun 1;109(11):4739-41. doi: 10.1182/blood-2006-11-057968. Epub 2007 Feb 27.
- Derived from this study: PubMed 24802773Gomez-Almaguer D, Herrera-Rojas MA, Jaime-Perez JC, Gomez-De Leon A, Cantu-Rodriguez OG, Gutierrez-Aguirre CH, Tarin-Arzaga L, Hernandez-Reyes J, Ruiz-Arguelles GJ. Eltrombopag and high-dose dexamethasone as frontline treatment of newly diagnosed immune thrombocytopenia in adults. Blood. 2014 Jun 19;123(25):3906-8. doi: 10.1182/blood-2014-01-549360. Epub 2014 May 6.
Related trials for Immune Thrombocytopenic Purpura
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)
RecruitingPhase 1/2
A Study of Pirtobrutinib in Participants With Immune Thrombocytopenia
Immune Thrombocytopenia (ITP)
RecruitingPhase 3
A Study of Mezagitamab in Adults With Chronic Primary Immune Thrombocytopenia
Immune Thrombocytopenic Purpura (ITP)
RecruitingPhase 2
Study to Assess the Safety and Tolerability of Tafasitamab in Adult Participants With Primary Autoimmune Blood Cell Disorders
Immune Thrombocytopenia
RecruitingPhase 3
A Follow-up Study of Mezagitamab in Adults With Chronic Primary Immune Thrombocytopenia
Immune Thrombocytopenic Purpura (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.
David Gomez-Almaguer, MD principal investigator
Hospital Universitario Dr. Jose E. Gonzalez
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Eltrombopag and dexamethasone in people with Immune Thrombocytopenic Purpura, run by Hospital Universitario Dr. Jose E. Gonzalez at 1 site in Mexico.
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 12 inclusion and 9 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 7 Jan 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 Mexico. 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 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
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?
Number of patients with sustained response after 6 months (time frame: 6 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 NCT01652599, which also lists 3 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 7 Jan 2014.