Immune ThrombocytopeniaPhase 2NCT04289207
Phase II Trial of Romiplostim With Danazol in Patients With Eltrombopag-resistant Immune Thrombocytopenia
A Phase 2 study testing Romiplostim and danazol in people with Immune Thrombocytopenia, run by Seoul National University Hospital.
Trial summary
In this study, we tried to demonstrate clinical benefit of additive danazol to romiplostim therapy in patients with eltrombopag-resistant immune thrombocytopenia Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Immune Thrombocytopenia
- What is being tested?
- Romiplostim and danazol
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 55 participants
- Where
- No sites listed yet
- Study dates
- 1 Mar 2020to 1 Mar 2021
- 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 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- aged over 18 years
- diagnosed with immune thrombocytopenia (bone marrow examination is not necessary)
- failed to achieve platelet count over 50X10^9/L with eltrombopag
- ECOG performance status 0,1,2
- available to obtain informed consent
Who cannot join
- Hepatitis B or C carriers
- HIV positive patients
- diagnosed with systemic lupus erythematosus or other autoimmune disorders
- unable to intake orally or absorb through gastrointestinal tract
- pregnant or breast-feeding
- diagnosed with uncontrolled seizure or other neuropsychiatric disorders
Show all 14Show fewer
- diagnosed with clinically significant cardiovascular events within 6 months or dyspnea on exertion evaluated to New York Heart Association Functional Classification III or IV
- diagnosed with clinically significant cerebrovascular disorders
- previously diagnosed or treated with thromboembolism
- current treating malignant diseases
- currently accompanied by uncontrolled infection or active bleeding
- with blood test results as follows; total bilirubin > 2xUNL(upper normal limit), AST/ALT > 1.5xUNL, creatinine > 1.5xUNL, glomerular filtration rate < 30ml/min/1.73m^2
- registered to other clinical trials for treatment of immune thrombocytopenia
- judged to be inappropriate for clinical trial by doctor in charge
Trial locations and contacts
No sites are listed yet.
About this study
In this study, we tried to demonstrate clinical benefit of additive danazol to romiplostim therapy in patients with eltrombopag-resistant immune thrombocytopenia
Read the detailed description
The patients with eltrombopag-resistant immune thrombocytopenia are treated with romiplostim and danazol. The efficacy and toxicity of treatment will be evaluated.
- Conditions
- Immune Thrombocytopenia
- Being tested
- Romiplostim and danazol
- 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.
Romiplostim and danazolDrug
Treatment with romiplostim and danazol
| Study group | Role | What they receive |
|---|---|---|
| Romiplostim and danazol | Experimental | Treatment group (romiplostim and danazol) Receives: Drug: Romiplostim and danazol |
What is being measured
The primary outcome is the main question the study is designed to answer.
Response rate
proportion of patients with platelet count over 50x10^9/L
Measured: week 8
Secondary outcomes (3)
Time to best response
time interval between initiation of treatment and best response
Measured: week 24
Response duration
period of time with platelet count over 50x10^9/L
Measured: week 24
Effects on quality of life
changes in EORTC QLQ-C30 scores
Measured: week 24
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted28 Feb 2020
- Study start1 Mar 2020 (estimated)
- Primary completion1 Sep 2020 (estimated)
- Study completion1 Mar 2021 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (4)Papers the sponsor has linked to this study.
- Other trials for Immune ThrombocytopeniaStudies of the same condition, open and under way.
- More trials run by Seoul National University 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
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 55 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Seoul National University Hospital
- Sponsor type
- University, hospital or other
- Collaborators
- Kyowa Kirin Korea Co., Ltd.
- Responsible party
- Principal investigator, Youngil Koh, Seoul National University Hospital
- Investigators
- Youngil Koh, Principal investigator, Seoul National University Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04289207
- Sponsor's protocol ID
- 0620200130
- Status last verified
- Feb 2020
- First submitted
- 25 Feb 2020
- Last updated
- 28 Feb 2020
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
Cooper N, Bussel J. The pathogenesis of immune thrombocytopaenic purpura. Br J Haematol. 2006 May;133(4):364-74. doi: 10.1111/j.1365-2141.2006.06024.x.
PubMed 16643442Bussel JB, Kuter DJ, Pullarkat V, Lyons RM, Guo M, Nichol JL. Safety and efficacy of long-term treatment with romiplostim in thrombocytopenic patients with chronic ITP. Blood. 2009 Mar 5;113(10):2161-71. doi: 10.1182/blood-2008-04-150078. Epub 2008 Nov 3.
PubMed 18981291Wong RSM, Saleh MN, Khelif A, Salama A, Portella MSO, Burgess P, Bussel JB. Safety and efficacy of long-term treatment of chronic/persistent ITP with eltrombopag: final results of the EXTEND study. Blood. 2017 Dec 7;130(23):2527-2536. doi: 10.1182/blood-2017-04-748707. Epub 2017 Oct 17.
PubMed 29042367Khellaf M, Viallard JF, Hamidou M, Cheze S, Roudot-Thoraval F, Lefrere F, Fain O, Audia S, Abgrall JF, Michot JM, Dauriac C, Lefort S, Gyan E, Niault M, Durand JM, Languille L, Boutboul D, Bierling P, Michel M, Godeau B. A retrospective pilot evaluation of switching thrombopoietic receptor-agonists in immune thrombocytopenia. Haematologica. 2013 Jun;98(6):881-7. doi: 10.3324/haematol.2012.074633. Epub 2013 Feb 26.
PubMed 23445876
Study at a glance
From the registry record, last updated by the sponsor on 28 Feb 2020. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Phase II Trial of Romiplostim With Danazol in Patients With Eltrombopag-resistant Immune Thrombocytopenia
- ClinicalTrials.gov ID
- NCT04289207
- Acronym / sponsor ID
- 0620200130
- Conditions
- Immune Thrombocytopenia
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 55 participants (estimated)
- Lead sponsor
- Seoul National University HospitalUniversity, hospital or other
- Collaborators
- Kyowa Kirin Korea Co., Ltd.
- Study start
- 1 Mar 2020 (estimated)
- Primary completion
- 1 Sep 2020 (estimated)
- Study completion
- 1 Mar 2021 (estimated)
- First posted
- 28 Feb 2020 (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.
In this study, we tried to demonstrate clinical benefit of additive danazol to romiplostim therapy in patients with eltrombopag-resistant immune thrombocytopenia
Detailed description
The patients with eltrombopag-resistant immune thrombocytopenia are treated with romiplostim and danazol. The efficacy and toxicity of treatment will be evaluated.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Romiplostim and danazol | Experimental | Drug: Romiplostim and danazolTreatment group (romiplostim and danazol) |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (5 listed)
- aged over 18 years
- diagnosed with immune thrombocytopenia (bone marrow examination is not necessary)
- failed to achieve platelet count over 50X10^9/L with eltrombopag
- ECOG performance status 0,1,2
- available to obtain informed consent
Key exclusion criteria (14 listed)
- Hepatitis B or C carriers
- HIV positive patients
- diagnosed with systemic lupus erythematosus or other autoimmune disorders
- unable to intake orally or absorb through gastrointestinal tract
- pregnant or breast-feeding
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 |
|---|---|---|
| Romiplostim and danazolTreatment with romiplostim and danazol | Drug | Romiplostim and danazol |
Primary outcome
Response rate
Time frame: week 8
proportion of patients with platelet count over 50x10^9/L
3 secondary outcomes
Time to best response
Time frame: week 24
Response duration
Time frame: week 24
Effects on quality of life
Time frame: week 24
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 28 Feb 2020.
- Background: PubMed 16643442Cooper N, Bussel J. The pathogenesis of immune thrombocytopaenic purpura. Br J Haematol. 2006 May;133(4):364-74. doi: 10.1111/j.1365-2141.2006.06024.x.
- Background: PubMed 18981291Bussel JB, Kuter DJ, Pullarkat V, Lyons RM, Guo M, Nichol JL. Safety and efficacy of long-term treatment with romiplostim in thrombocytopenic patients with chronic ITP. Blood. 2009 Mar 5;113(10):2161-71. doi: 10.1182/blood-2008-04-150078. Epub 2008 Nov 3.
- Background: PubMed 29042367Wong RSM, Saleh MN, Khelif A, Salama A, Portella MSO, Burgess P, Bussel JB. Safety and efficacy of long-term treatment of chronic/persistent ITP with eltrombopag: final results of the EXTEND study. Blood. 2017 Dec 7;130(23):2527-2536. doi: 10.1182/blood-2017-04-748707. Epub 2017 Oct 17.
- Background: PubMed 23445876Khellaf M, Viallard JF, Hamidou M, Cheze S, Roudot-Thoraval F, Lefrere F, Fain O, Audia S, Abgrall JF, Michot JM, Dauriac C, Lefort S, Gyan E, Niault M, Durand JM, Languille L, Boutboul D, Bierling P, Michel M, Godeau B. A retrospective pilot evaluation of switching thrombopoietic receptor-agonists in immune thrombocytopenia. Haematologica. 2013 Jun;98(6):881-7. doi: 10.3324/haematol.2012.074633. Epub 2013 Feb 26.
- 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.
Youngil Koh principal investigator
Seoul National University Hospital
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Romiplostim and danazol in people with Immune Thrombocytopenia, run by Seoul National University Hospital.
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 14 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 28 Feb 2020. Status can change between updates, so confirm with the study contact before making plans.
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?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Youngil Koh (82-02-2072-7217, go01@snu.ac.kr), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Response rate (time frame: week 8). 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 NCT04289207, 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 28 Feb 2020.