Immune ThrombocytopeniaPhase 2NCT03830749
Safety and Efficacy of Eltrombopag Plus Pulsed Dexamethasone for Subjects With Idiopathic Thrombocytopenic Purpura
A Phase 2 study testing Eltrombopag in people with Immune Thrombocytopenia, run by Humanity & Health Medical Group Limited at 1 site in Hong Kong.
Trial summary
Current first line treatments for immune thrombocytopenia (ITP) usually have transient effects and prolonged platelet response rate off therapy remains low. The aim is to evaluate whether a 12-week course of eltrombopag plus pulsed dexamethasone as first line therapy can increase the proportion of patients with… 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?
- Eltrombopag
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 53 participants
- Where
- 1 siteHong Kong
- Study dates
- 1 Jul 2018to 31 Mar 2023
- 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
- Subject has signed and dated a written informed consent.
- Adults (≥18 years) diagnosed with ITP according to the American Society for Hematology/British Committee for Standards in Haematology (ASH/BCSH) Guidelines. In addition, the peripheral blood smear should support the diagnosis of ITP with no evidence of other disease causative of thrombocytopenia (e.g., pseudo thrombocytopenia, myelofibrosis). The physical examination should be normal or at least not show signs suggestive of any disease likely to be associated with thrombocytopenia.
- No prior ITP treatment except platelet transfusions
- Subject has no intercurrent medical event, including evidence of any thrombosis.
- Normal prothrombin time (PT/INR) and activated partial thromboplastin time (aPTT), no history of hypercoagulable state.
- The following clinical chemistries must be within the normal reference range:
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- creatinine, ALT, AST, total bilirubin, total albumin and alkaline phosphatase.
- Subject is practicing an acceptable method of contraception (documented in chart).
- Subject is able to understand and comply with protocol requirements and instructions.
Who cannot join
- Any clinically relevant abnormality, other than ITP, identified on the screening examination, or any other medical condition or circumstance, which in the opinion of the investigator makes the subject unsuitable for participation in the study or suggests another diagnosis.
- History of active malignancy or on cancer therapy. Patients with a history of malignancy in complete remission for longer than 5 years are eligible
- History of arterial or venous thrombosis (stroke, transient ischemic attack, myocardial infarction, deep vein thrombosis or pulmonary embolism).
- ≥ two of the following risk factors: Factor V Leiden, hormone replacement therapy, systemic contraception containing estrogen, smoking, diabetes, hypercholesterolemia, medications for hypertension or cancer.
- Pre-existing cardiac disease (including congestive heart failure, and arrhythmia requiring treatment), or clinically significant findings on resting 12-lead ECG at screening.
- Female subjects who are nursing or pregnant (positive serum or urine β-human chorionic gonadotrophin (β-hCG) pregnancy test) at screening or pre-dose on Day 1.
- History of alcohol/drug abuse.
- Treatment with an investigational drug within 30 days or five half-lives (whichever is longer) preceding the first dose of study medication
Trial locations and contacts
1 site in 1 country.
About this study
Official title: A Pilot Study to Evaluate the Safety and Efficacy of Eltrombopag Plus Pulsed Dexamethasone as First Line Therapy for Subjects With Idiopathic Thrombocytopenic Purpura (ITP)
Current first line treatments for immune thrombocytopenia (ITP) usually have transient effects and prolonged platelet response rate off therapy remains low. The aim is to evaluate whether a 12-week course of eltrombopag plus pulsed dexamethasone as first line therapy can increase the proportion of patients with prolonged response. Diagnosis of ITP is established according to the American Society of Hematology guidelines. Eligible ITP subjects have platelet counts <30×109/L or counts <50×109/L and significant bleeding symptoms (WHO bleeding scale 2 or above). Subjects must have no prior ITP treatment except platelet transfusions. Treatment consists of eltrombopag 25-75 mg daily according to platelet response for 12 weeks plus pulsed dexamethasone, 40 mg daily for 4 consecutive days every 4 weeks for 1-3 courses. The primary endpoint is prolonged response rate which was defined as the proportion of enrolled subjects maintaining platelet counts >50×109/L for more than 6 months without any ITP therapy after completion of 12-week therapy.
- Conditions
- Immune Thrombocytopenia
- Being tested
- Eltrombopag
- 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
Eltrombopag Oral Tablet 25-75 mg daily for 12 weeks plus pulsed dexamethasone
| Study group | Role | What they receive |
|---|---|---|
| Single Arm | Experimental | Eltrombopag Oral Tablet 25-75 mg daily for 12 weeks plus pulsed dexamethasone Receives: Drug: Eltrombopag |
What is being measured
The primary outcome is the main question the study is designed to answer.
The prolonged response rate
Defined as proportion of patients with platelet counts > 50,000/μl at 6 months after completion of therapy and free of ITP rescue therapy
Measured: 6 months after completion of therapy
Secondary outcomes (3)
Time to relapse
Defined as the interval from completion of 3 months treatment to platelet count<30,000/ul and restarting of ITP therapy including but not limited to platelet transfusion, IVIG, corticosteroids, immunosuppressive drugs, rituximab
Measured: 6 months after completion of therapy
Early response rate
Defined as proportion of patients requiring less than 3 courses of pulsed dexamethasone to maintain platelet counts > 150,000/ul
Measured: 6 months after completion of therapy
Health related quality of life
Assessed by SV36 HRQoL questionnaires
Measured: 6 months after completion of therapy
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted5 Feb 2019
- Study start1 Jul 2018
- Primary completion31 Dec 2022
- Study completion31 Mar 2023
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Immune ThrombocytopeniaStudies 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
- 53 participants (actual)
Sponsor and investigators
- Lead sponsor
- Humanity & Health Medical Group Limited
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Gregory Cheng, PhD, MD, Principal investigator, Humanity & Health Research Centre
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03830749
- Sponsor's protocol ID
- HHRC_ITP
- Status last verified
- Feb 2024
- First submitted
- 1 Feb 2019
- Last updated
- 28 Feb 2024
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 28 Feb 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Pilot Study to Evaluate the Safety and Efficacy of Eltrombopag Plus Pulsed Dexamethasone as First Line Therapy for Subjects With Idiopathic Thrombocytopenic Purpura (ITP)
- ClinicalTrials.gov ID
- NCT03830749
- Acronym / sponsor ID
- HHRC_ITP
- Conditions
- Immune Thrombocytopenia
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 53 participants (actual)
- Lead sponsor
- Humanity & Health Medical Group LimitedUniversity, hospital or other
- Study start
- 1 Jul 2018 (actual)
- Primary completion
- 31 Dec 2022 (actual)
- Study completion
- 31 Mar 2023 (actual)
- First posted
- 5 Feb 2019 (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.
Current first line treatments for immune thrombocytopenia (ITP) usually have transient effects and prolonged platelet response rate off therapy remains low. The aim is to evaluate whether a 12-week course of eltrombopag plus pulsed dexamethasone as first line therapy can increase the proportion of patients with prolonged response. Diagnosis of ITP is established according to the American Society of Hematology guidelines. Eligible ITP subjects have platelet counts <30×109/L or counts <50×109/L and significant bleeding symptoms (WHO bleeding scale 2 or above). Subjects must have no prior ITP treatment except platelet transfusions. Treatment consists of eltrombopag 25-75 mg daily according to platelet response for 12 weeks plus pulsed dexamethasone, 40 mg daily for 4 consecutive days every 4 weeks for 1-3 courses. The primary endpoint is prolonged response rate which was defined as the proportion of enrolled subjects maintaining platelet counts >50×109/L for more than 6 months without any ITP therapy after completion of 12-week therapy.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Single Arm | Experimental | Drug: EltrombopagEltrombopag Oral Tablet 25-75 mg daily for 12 weeks plus pulsed dexamethasone |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (9 listed)
- Subject has signed and dated a written informed consent.
- Adults (≥18 years) diagnosed with ITP according to the American Society for Hematology/British Committee for Standards in Haematology (ASH/BCSH) Guidelines. In addition, the peripheral blood smear should support the diagnosis of ITP with no evidence of other disease causative of thrombocytopenia (e.g., pseudo thrombocytopenia, myelofibrosis). The physical examination should be normal or at least not show signs suggestive of any disease likely to be associated with thrombocytopenia.
- No prior ITP treatment except platelet transfusions
- Subject has no intercurrent medical event, including evidence of any thrombosis.
- Normal prothrombin time (PT/INR) and activated partial thromboplastin time (aPTT), no history of hypercoagulable state.
Key exclusion criteria (8 listed)
- Any clinically relevant abnormality, other than ITP, identified on the screening examination, or any other medical condition or circumstance, which in the opinion of the investigator makes the subject unsuitable for participation in the study or suggests another diagnosis.
- History of active malignancy or on cancer therapy. Patients with a history of malignancy in complete remission for longer than 5 years are eligible
- History of arterial or venous thrombosis (stroke, transient ischemic attack, myocardial infarction, deep vein thrombosis or pulmonary embolism).
- ≥ two of the following risk factors: Factor V Leiden, hormone replacement therapy, systemic contraception containing estrogen, smoking, diabetes, hypercholesterolemia, medications for hypertension or cancer.
- Pre-existing cardiac disease (including congestive heart failure, and arrhythmia requiring treatment), or clinically significant findings on resting 12-lead ECG at screening.
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 |
|---|---|---|
| EltrombopagEltrombopag Oral Tablet 25-75 mg daily for 12 weeks plus pulsed dexamethasone | Drug | Single Arm |
Primary outcome
The prolonged response rate
Time frame: 6 months after completion of therapy
Defined as proportion of patients with platelet counts > 50,000/μl at 6 months after completion of therapy and free of ITP rescue therapy
3 secondary outcomes
Time to relapse
Time frame: 6 months after completion of therapy
Early response rate
Time frame: 6 months after completion of therapy
Health related quality of life
Time frame: 6 months after completion of therapy
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 28 Feb 2024.
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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.
Gregory Cheng, PhD, MD principal investigator
Humanity & Health Research Centre
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 in people with Immune Thrombocytopenia, run by Humanity & Health Medical Group Limited at 1 site in Hong Kong.
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 9 inclusion and 8 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 28 Feb 2024. 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 Hong Kong. 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?
The prolonged response rate (time frame: 6 months after completion of therapy). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 2024.