ClinicalTrials.gov record NCT04993885
Avatrombopag in the Treatment of Adult Immune Thrombocytopenia With Autoantibodies
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
AI summaryWhat this study is about, in plain languageShow ▾Hide ▴
AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.
This open-label, single-arm Phase 2 trial evaluates avatrombopag in adults with immune thrombocytopenia (ITP) and autoantibodies who failed or cannot take eltrombopag or herombopag. About 52 participants will start at 40 mg once daily with dose adjustments guided by platelet counts over a 12-week treatment period.
What it is studying. The study tests whether avatrombopag raises platelet counts and its safety in adults with ITP and autoantibodies who previously failed, could not tolerate, or cannot take eltrombopag or herombopag. The main measure is the percentage reaching platelet ≥30×10^9/L and at least doubling baseline within 12 weeks; secondary measures include timing and duration of response, bleeding, disease activity in connective tissue disease, immune markers, steroid use, and quality-of-life.
What the researchers hope to learn. Scientifically, the study aims to assess avatrombopag's efficacy (primary: rate of platelet response by 12 weeks) and safety in this patient group. Secondary goals include timing and duration of platelet responses, rates of complete responses, bleeding frequency and severity, changes in connective tissue disease activity, symptom and immune-index changes, glucocorticoid discontinuation, and quality-of-life measures.
What this study is about
The study's own summary, as published on ClinicalTrials.gov. Underlined terms have a short explanation.
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.
Interventions being studied
Being studied in this trial does not mean the treatment or procedure is approved or available for this condition.
- Drug
- Avatrombopag
Eligibility
The criteria exactly as listed on ClinicalTrials.gov. Only the study team can say whether they apply to you.
Inclusion criteria
- 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.
Exclusion criteria
- 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.
- 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.
Study description and what participation involves
The study's detailed description, as published on ClinicalTrials.gov.
Detailed description from the registry
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.
AI summaryWhat participation may involve, in plain languageShow ▾Hide ▴
AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.
Participants receive avatrombopag starting at 40 mg once daily for up to 12 weeks with dose adjustments (changes of 20 mg/week within a 20–40 mg/day range) to keep platelets between 30×10^9/L and 200×10^9/L. Platelet counts are checked weekly for the first 4 weeks, then every 2 weeks through week 12; if platelets do not reach 30×10^9/L after 4 weeks at 40 mg/day, treatment is stopped. Study visits include blood tests for platelet counts and immune markers, assessments of bleeding and disease activity (SLEDAI for people with SLE), symptom checks, and questionnaires (fatigue and ITP‑Patient Assessment Questionnaire) before and after treatment.
How the study is structured. Phase 2, interventional, open-label, single-group trial (no placebo or blinding). All participants receive avatrombopag starting at 40 mg once daily; platelet counts are checked weekly for the first 4 weeks and then every 2 weeks up to week 12. Doses are adjusted to keep platelet counts between 30×10^9/L and 200×10^9/L; treatment is stopped if platelets do not reach 30×10^9/L after 4 weeks at 40 mg. Planned enrollment is 52 participants.
Questions you could ask
These questions are suggestions for a conversation with the study team or your own clinician, not advice.
- The listed sites are in China. Is any of them reachable for me, and does the study help with travel?
- Which of the eligibility criteria would need to be checked with tests before I could join?
More on Evans syndrome
Important dates
- Study start
- Sep 1, 2021
- Primary completion
- Jul 31, 2024
- Study completion
- Sep 30, 2025 (estimated)
- Target enrollment
- 52 participants (estimated)
- Last updated on registry
- Feb 24, 2025
- Data verified
- Oct 10, 2026
Sponsor and research team
The lead sponsor funds and oversees the trial. A pharmaceutical or biotech company sponsor typically means the study is testing that company's drug or device; a hospital, university, or government agency usually indicates investigator-initiated or publicly funded research.
- Lead sponsor
- Institute of Hematology & Blood Diseases Hospital, China
Central study contact
If you're interested in participating, the central contact can answer general questions about the study — including eligibility requirements and how to find a recruiting site near you. Actual eligibility is determined by the site's medical team.
- Rongfeng Fu, M.D. 📞 +862223909009 ✉ furongfeng@ihcams.ac.cn
- Lei Zhang, M.D. 📞 +862223909240 ✉ zhanglei1@ihcams.ac.cn
Participating sites (1)
- Institute of Hematology & Blood Diseases HospitalRecruiting
Tianjin, Tianjin Municipality, 300020 · China
- Rongfeng Fu, MD · +862223909009 · furongfeng@ihcams.ac.cn
- Lei Zhang, MD · +862223909240 · zhanglei1@ihcams.ac.cn
Terms used on this page · explanations by MedicalUpdates.ai
- open-label
- No masking is used, so the assigned intervention is not deliberately hidden from participants or study staff.
- written informed consent
- The process of explaining a study, including its risks and what participation involves, so that a person can decide freely whether to take part. Consent is usually documented in writing, and a participant can choose to leave the study later.
- enrollment
- The number of participants a study plans to include, or has included. 'Estimated' means the planned number; 'actual' means the number who joined.
- at screening
- The checks done before enrolment to see whether a person qualifies, such as tests, scans or a review of medical history.
- contraindications
- A reason a particular test or treatment should not be used for a person, for example a metal implant that rules out an MRI scan.
- glucocorticoids
- Steroid medicines, such as prednisone, that reduce inflammation. Studies often require a stable dose at entry or measure whether the study treatment allows the dose to be reduced.
- ULN
- The highest value of a blood test that a laboratory considers normal. Criteria such as '≤ 3 × ULN' mean the result must be no more than three times that value.
- creatinine clearance
- A measure of kidney function, calculated from a blood creatinine level together with age, weight and sex.
- Inclusion Criteria
- The conditions a person must meet to be considered for the study, for example a confirmed diagnosis, an age range or a test result.
- Exclusion Criteria
- The conditions that rule a person out even if they meet the inclusion criteria, for example other illnesses, certain medicines or pregnancy.
The study record on this page is reproduced from ClinicalTrials.gov, a database of the U.S. National Library of Medicine, where it was submitted by the study sponsor. MedicalUpdates.ai does not own this content, is not affiliated with or endorsed by ClinicalTrials.gov, NIH, or the U.S. Government, and does not verify the record. Plain-language notes, the glossary and the questions above are added by this site; text in the boxes marked "AI summary" was written by an AI model and may contain errors. Registry records change over time. Check the original record for the current version and discuss eligibility or participation with a qualified healthcare professional.
← Back to clinical trials