Immune ThrombocytopeniaPhase 2NCT04993885
Avatrombopag in the Treatment of Adult Immune Thrombocytopenia With Autoantibodies
A Phase 2 study testing Avatrombopag in people with Immune Thrombocytopenia and Autoantibodies, run by Institute of Hematology & Blood Diseases Hospital, China at 1 site in China.
Trial summary
This prospective, open-label, single-center, one-arm clinical trial aims at evaluating the efficacy and safety of avatrombopag in Chinese adult Immune Thrombocytopenia (ITP) patients with autoantibodies fail (due to intolerance or resistance) to eltrombopag or herombopag treatment. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Immune Thrombocytopenia, Autoantibodies, Evan Syndrome
- What is being tested?
- Avatrombopag
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 52 participants
- Where
- 1 siteChina
- Study dates
- 1 Sep 2021to 30 Sep 2025
- Phase
- Phase 2
- Are there results?
- Not yet: the study is under way
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
- The patients have provided written informed consent prior to enrollment.
- Men and women greater than or equal to 18 years of age.
- Diagnosed as ITP secondary to connective tissue diseases (including but not limited to systemic lupus erythematosus, Sjogren's syndrome and rheumatoid arthritis), primary ITP with positive antinuclear antibody but not up to the diagnostic criteria of connective tissue diseases, primary Evans syndrome, Evans syndrome secondary to connective tissue diseases, and primary ITP with positive Coomb's test but not up to the diagnostic criteria of Evans syndrome.
- Platelet count<30 ×10^9/L at screening.
- Patients who have previously failed to receive Eltrombopag or Herombopag [poor efficacy (Eltrombopag 75 mg/d or Herombopag 7.5 mg/D, at least 4 weeks), or the efficacy cannot be maintained], or who have contraindications, can not tolerate or refuse Eltrombopag or Herombopag treatment.
- Treatment for ITP (including but not limited to glucocorticoids, recombinant human thrombopoietin, and other thrombopoietin receptor agonists other than Avatrombopag) must be completed before enrollment, or the dose must be stable or in a phase of reduction within 2 weeks before enrollment.
- Effective contraceptive measures will be taken during the clinical trial.
Who cannot join
- Patients with active thyroid disease requiring treatment.
- Patients with any prior history of arterial or venous thrombosis within 3 months, and with any of the following risk factors: cancer, Factor V Leiden, ATIII deficiency, or patients who are using anticoagulants or antiplatelet drugs at the beginning of screening.
- Those who had received rituximab within 3 months.
- Patients who had failed to respond to the previous use of Avatrombopag (40mg once a day for more than 4 weeks).
- Subjects known to be allergic to Avatrombopag or any of its excipients.
- Patients who had received splenectomy within 3 months or have splenectomy plan within 3 months.
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- Patients with lupus encephalopathy or lupus nephritis.
- Patients with cataract.
- Patients with infectious fever (including but not limited to pulmonary infection) within 1 month or with active infection during screening.
- Existing hepatitis B virus, hepatitis C virus replication or HIV infection.
- Severe liver dysfunction (alanine aminotransferase or glutamic oxaloacetic transaminase > 3×ULN).
- Patients with severe cardiac or pulmonary dysfunction.
- Severe renal damage (creatinine clearance < 30 ml/min).
- There are surgical planners during the study.
- History of psychiatric disorder.
- Pregnant or lactating women or those planning to be pregnant during the trial.
- Patients with a history of drug/alcohol abuse (within 2 years before the study).
- Patients that had participated in other experimental researches within one month before enrollment.
- Any other circumstances that the investigator considers that the patient is not suitable to participate in the trial.
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
China1 site, 1 recruiting
- Recruiting
Institute of Hematology & Blood Diseases Hospital
Tianjin, Tianjin Municipality, 300020
Rongfeng Fu, MD, +862223909009, furongfeng@ihcams.ac.cn
Lei Zhang, MD, +862223909240, zhanglei1@ihcams.ac.cn
About this study
Official title: Efficacy and Safety of Avatrombopag in the Treatment of Adult Immune Thrombocytopenia With Autoantibodies Fail to Eltrombopag or Herombopag Treatment: a Single-center, Prospective, One-arm Clinical Trial
This prospective, open-label, single-center, one-arm clinical trial aims at evaluating the efficacy and safety of avatrombopag in Chinese adult Immune Thrombocytopenia (ITP) patients with autoantibodies fail (due to intolerance or resistance) to eltrombopag or herombopag treatment.
Read the detailed description
This prospective, open-label, single-center, one-arm clinical trial aims at evaluating the efficacy and safety of avatrombopag in Chinese adult ITP patients with autoantibodies fail (due to intolerance or resistance) to eltrombopag or herombopag treatment.
The subjects include ITP secondary to connective tissue diseases (including but not limited to systemic lupus erythematosus, Sjogren's syndrome and rheumatoid arthritis), primary ITP with positive antinuclear antibody but not up to the diagnostic criteria of connective tissue diseases, primary Evans syndrome, Evans syndrome secondary to connective tissue diseases, and primary ITP with positive Coomb's test but not up to the diagnostic criteria of Evans syndrome.
Fifty-two eligible subjects will be enrolled in this study. The dose will be adjusted according to the platelet count during the period from week 1 to week 12.
- Being tested
- Avatrombopag
- Medical subject headings
- Purpura, Thrombocytopenic, Idiopathic, Evans Syndrome, Connective Tissue Diseases
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
AvatrombopagDrug
The subjects will receive avatrombopag treatment with an initial dose of 40mg once a day. Platelet counts will be obtained weekly during the first 4 weeks and then every 2 weeks until week 12 after treatment. The dose adjustment range is 20 mg per week to 40 mg per day to maintain the platelet level between 30×10^9/L and 200×10^9/L. If the platelet count does not reach to 30×10^9/L after taking avatrombopag 40mg once a day for 4 consecutive weeks, the treatment will be stopped.
| Study group | Role | What they receive |
|---|---|---|
| Treatment group | Experimental | Fifty-two subjects will be enrolled with the indicated treatment dose of avatrombopag Receives: Drug: Avatrombopag |
What is being measured
The primary outcome is the main question the study is designed to answer.
Platelet response
Percentage of participants achieving a platelet count >=30×10^9/L and at least doubling of the baseline count within 12 weeks of treatment.
Measured: From the start of study treatment (Day 1) up to the end of week 12.
Secondary outcomes (14)
Platelet response
Proportion of subjects who achieve response (R) within 1, 2, 4 and 8 weeks of treatment.
Measured: From the start of study treatment (Day 1) up to the end of week 12.
Platelet response
Proportion of subjects who achieve complete response (CR) within 4, 8 and 12 weeks of treatment.
Measured: From the start of study treatment (Day 1) up to the end of week 12.
Duration of platelet response
Proportion of subjects with a platelet count >=30×10^9/L for at least 4 consecutive weeks during the 12 week treatment period without remedial treatment.
Measured: From the start of study treatment (Day 1) up to the end of week 12.
Platelet response
Percentage of participants achieving a platelet count >=50×10^9/L within 12 weeks of treatment.
Measured: From the start of study treatment (Day 1) up to the end of week 12.
Time to platelet response
Time to response is defined as time from the start of treatment to the first time of achieving a platelet count >= 30×10^9/L and at least doubling of the baseline count during the whole 12 weeks.
Measured: From the start of study treatment (Day 1) up to the end of week 12.
Duration of platelet response
Total duration of time a participant with a response of R.
Measured: From the start of study treatment (Day 1) up to the end of week 12.
Proportion of patients receiving remedial treatment.
Proportion of patients receiving remedial treatment.
Measured: From the start of study treatment (Day 1) up to the end of week 12.
Bleeding score
The incidence and grade of bleeding symptoms according to the World Health Organization Bleeding Scale.
Measured: From the start of study treatment (Day 1) up to the end of week 12.
Changes of disease activity index in patients with systemic lupus erythematosus
The proportion of subjects with improvement of disease activity index in patients with systemic lupus erythematosus according to the SLEDAI standard.
Measured: From the start of study treatment (Day 1) up to the end of week 12.
The improvement of symptoms
The proportion of subjects with improvement of symptoms including skin symptom, joint pain, dry mouth and dry eyes.
Measured: From the start of study treatment (Day 1) up to the end of week 12.
Improvement in immune indexes
The proportion of subjects with improvement immune indexes including antinuclear antibody, extractable nuclear antigens spectrum and Coomb's test.
Measured: From the start of study treatment (Day 1) up to the end of week 12.
Discontinuation rate of glucocorticoids
The proportion of subjects with discontinuation use of glucocorticoids.
Measured: From the start of study treatment (Day 1) up to the end of week 12.
Functional assessment of chronic illness therapy-fatigue
In all participants, functional assessment of chronic illness therapy-fatigue questionnaire will be used to assess the health related quality of life before and after treatment.
Measured: From the start of study treatment (Day 1) up to the end of week 12.
ITP-Patient Assessment Questionnaire
In all participants, ITP-Patient Assessment Questionnaire will be used to assess the health related quality of life before and after treatment.
Measured: From the start of study treatment (Day 1) up to the end of week 12.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted6 Aug 2021
- Study start1 Sep 2021
- Primary completion31 Jul 2024
- Study completion30 Sep 2025 (estimated)
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.
- More trials run by Institute of Hematology & Blood Diseases Hospital, ChinaThe 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
- 52 participants (estimated)
Sponsor and investigators
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Lei Zhang, M.D., Principal investigator, Institute of Hematology & Blood Diseases Hospital, China
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04993885
- Sponsor's protocol ID
- IIT2021033
- Status last verified
- Feb 2025
- First submitted
- 3 Aug 2021
- Last updated
- 24 Feb 2025
- Results posted
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 24 Feb 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Efficacy and Safety of Avatrombopag in the Treatment of Adult Immune Thrombocytopenia With Autoantibodies Fail to Eltrombopag or Herombopag Treatment: a Single-center, Prospective, One-arm Clinical Trial
- ClinicalTrials.gov ID
- NCT04993885
- Acronym / sponsor ID
- IIT2021033
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 52 participants (estimated)
- Lead sponsor
- Institute of Hematology & Blood Diseases Hospital, ChinaUniversity, hospital or other
- Study start
- 1 Sep 2021 (actual)
- Primary completion
- 31 Jul 2024 (actual)
- Study completion
- 30 Sep 2025 (estimated)
- First posted
- 6 Aug 2021 (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.
This prospective, open-label, single-center, one-arm clinical trial aims at evaluating the efficacy and safety of avatrombopag in Chinese adult Immune Thrombocytopenia (ITP) patients with autoantibodies fail (due to intolerance or resistance) to eltrombopag or herombopag treatment.
Detailed description
This prospective, open-label, single-center, one-arm clinical trial aims at evaluating the efficacy and safety of avatrombopag in Chinese adult ITP patients with autoantibodies fail (due to intolerance or resistance) to eltrombopag or herombopag treatment.
The subjects include ITP secondary to connective tissue diseases (including but not limited to systemic lupus erythematosus, Sjogren's syndrome and rheumatoid arthritis), primary ITP with positive antinuclear antibody but not up to the diagnostic criteria of connective tissue diseases, primary Evans syndrome, Evans syndrome secondary to connective tissue diseases, and primary ITP with positive Coomb's test but not up to the diagnostic criteria of Evans syndrome.
Fifty-two eligible subjects will be enrolled in this study. The dose will be adjusted according to the platelet count during the period from week 1 to week 12.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Treatment group | Experimental | Drug: AvatrombopagFifty-two subjects will be enrolled with the indicated treatment dose of avatrombopag |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (7 listed)
- The patients have provided written informed consent prior to enrollment.
- Men and women greater than or equal to 18 years of age.
- Diagnosed as ITP secondary to connective tissue diseases (including but not limited to systemic lupus erythematosus, Sjogren's syndrome and rheumatoid arthritis), primary ITP with positive antinuclear antibody but not up to the diagnostic criteria of connective tissue diseases, primary Evans syndrome, Evans syndrome secondary to connective tissue diseases, and primary ITP with positive Coomb's test but not up to the diagnostic criteria of Evans syndrome.
- Platelet count<30 ×10^9/L at screening.
- Patients who have previously failed to receive Eltrombopag or Herombopag [poor efficacy (Eltrombopag 75 mg/d or Herombopag 7.5 mg/D, at least 4 weeks), or the efficacy cannot be maintained], or who have contraindications, can not tolerate or refuse Eltrombopag or Herombopag treatment.
Key exclusion criteria (19 listed)
- Patients with active thyroid disease requiring treatment.
- Patients with any prior history of arterial or venous thrombosis within 3 months, and with any of the following risk factors: cancer, Factor V Leiden, ATIII deficiency, or patients who are using anticoagulants or antiplatelet drugs at the beginning of screening.
- Those who had received rituximab within 3 months.
- Patients who had failed to respond to the previous use of Avatrombopag (40mg once a day for more than 4 weeks).
- Subjects known to be allergic to Avatrombopag or any of its excipients.
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 |
|---|---|---|
| AvatrombopagThe subjects will receive avatrombopag treatment with an initial dose of 40mg once a day. Platelet counts will be obtained weekly during the first 4 weeks and then every 2 weeks until week 12 after treatment. The dose adjustment range is 20 mg per week to 40 mg per day to maintain the platelet level between 30×10^9/L and 200×10^9/L. If the platelet count does not reach to 30×10^9/L after taking avatrombopag 40mg once a day for 4 consecutive weeks, the treatment will be stopped. | Drug | Treatment group |
Primary outcome
Platelet response
Time frame: From the start of study treatment (Day 1) up to the end of week 12.
Percentage of participants achieving a platelet count >=30×10^9/L and at least doubling of the baseline count within 12 weeks of treatment.
14 secondary outcomes
Platelet response
Time frame: From the start of study treatment (Day 1) up to the end of week 12.
Platelet response
Time frame: From the start of study treatment (Day 1) up to the end of week 12.
Duration of platelet response
Time frame: From the start of study treatment (Day 1) up to the end of week 12.
Platelet response
Time frame: From the start of study treatment (Day 1) up to the end of week 12.
Time to platelet response
Time frame: From the start of study treatment (Day 1) up to the end of week 12.
Duration of platelet response
Time frame: From the start of study treatment (Day 1) up to the end of week 12.
Proportion of patients receiving remedial treatment.
Time frame: From the start of study treatment (Day 1) up to the end of week 12.
Bleeding score
Time frame: From the start of study treatment (Day 1) up to the end of week 12.
Changes of disease activity index in patients with systemic lupus erythematosus
Time frame: From the start of study treatment (Day 1) up to the end of week 12.
The improvement of symptoms
Time frame: From the start of study treatment (Day 1) up to the end of week 12.
Improvement in immune indexes
Time frame: From the start of study treatment (Day 1) up to the end of week 12.
Discontinuation rate of glucocorticoids
Time frame: From the start of study treatment (Day 1) up to the end of week 12.
Functional assessment of chronic illness therapy-fatigue
Time frame: From the start of study treatment (Day 1) up to the end of week 12.
ITP-Patient Assessment Questionnaire
Time frame: From the start of study treatment (Day 1) up to the end of week 12.
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 24 Feb 2025.
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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.
Lei Zhang, M.D. principal investigator
Institute of Hematology & Blood Diseases Hospital, China
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 Avatrombopag in people with Immune Thrombocytopenia and Autoantibodies, run by Institute of Hematology & Blood Diseases Hospital, China 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 7 inclusion and 19 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: looking for participants now, as of the sponsor's last update on 24 Feb 2025. 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.
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?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Rongfeng Fu, M.D. (+862223909009, furongfeng@ihcams.ac.cn), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Platelet response (time frame: From the start of study treatment (Day 1) up to the end of week 12.). 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 24 Feb 2025.