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New combo aims to free ITP patients from lifelong medication
NCT ID NCT07278908
First seen Jun 24, 2026 · Last updated Jun 27, 2026 · Updated 1 time
Summary
This study tests whether adding all-trans retinoic acid (ATRA) to the drug avatrombopag can help adults with immune thrombocytopenia (ITP) maintain normal platelet levels without needing ongoing treatment. About 248 participants who have not responded well to first-line therapy or have relapsed will be randomly assigned to receive either avatrombopag alone or avatrombopag plus ATRA for up to 24 weeks, followed by a tapering period. The main goal is to see how many people can stop all treatment and still have safe platelet counts at one year.
What this could mean
Our plain-language read of the trial. This is informational only, not medical advice or a prediction.
- Active substance
- avatrombopag and all-trans retinoic acid
- What this could lead to
- If it works, this combination could help more people with ITP stop taking medication for a longer time, reducing the need for ongoing treatment.
- What could go wrong
- This is an early-to-mid stage trial with only 248 people, so results may not apply to everyone. The combination may cause side effects like headache, nausea, or liver issues, and it's not yet proven to be better than avatrombopag alone.
This is an AI summary of the original study and may miss details. Read our disclaimer.
Study facts
What this study's own registry entry says, in plain language.
- Phase
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Phase 2/3
Runs two stages together: whether the treatment works, then large-scale confirmation.
- Participants
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About 248 people
The number the study aims to enrol. It can still change while the study runs.
- Expected to start
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Dec 2025
An estimate. Start dates often move.
- Expected to finish
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Dec 2028
An estimate. End dates often move.
- Lead sponsor
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Other sponsor
The registry's catch-all category, for sponsors it does not file as a company, a government agency, or a research network.
Who can take part
This study's own entry requirements. Only the study team can say for certain whether you qualify.
- Ages
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18 years and older
- Sex
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Anyone
- Healthy volunteers
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Not accepted
This study is not open to healthy volunteers. The entry requirements below say who it is open to.
Show the full entry requirements Hide the full entry requirements
Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.
Inclusion Criteria: 1. The patient signs an informed consent form; 2. ≥ 18 years old; 3. The patient's history of ITP is at least 3 months (persistent or chronic ITP); 4. The platelet count of the subject within 24 hours before the first administration of the study drug is less than 30 × 10\^9/L; during the screening period, platelet count is measured at least twice (at least 1 week apart), with an average platelet count of less than 30 × 10\^9/L and no platelet count greater than 35 × 10\^9/L; 5. First line treatment, such as ineffective hormone or immunoglobulin therapy, or ITP patients who relapse after treatment; 6. Receive concomitant therapy drugs (including stable doses of glucocorticoids within the past month, stable doses of azathioprine, danazol, cyclosporine A, or mycophenolate mofetil within the past three months) for treatment; TPO receptor agonists should be discontinued at least 2 weeks before enrollment; Patients receiving anti-CD20 treatment should discontinue the medication six months prior to enrollment; Patients who underwent splenectomy were enrolled six months after the completion of the surgery; 7. Within the past year, there have been no heart diseases, including NYHA class III/IV congestive heart failure, drug-induced arrhythmias, or myocardial infarction; 8. Laboratory tests of coagulation function showed that the prothrombin time (PT/INR) and activated partial thromboplastin time (APTT) values did not exceed 20% of the normal reference range; No history of coagulation abnormalities except for ITP; 9. White blood cell count, absolute neutrophil count, and hemoglobin are within the normal range in the laboratory, with no abnormalities other than ITP, except for the following situations: a) Hemoglobin: If the anemia is clearly caused by iron deficiency anemia (excessive loss of blood related to thrombocytopenia) due to ITP, the subject's hemoglobin level below the lower limit of normal values can be selected for the study based on the researcher's judgment; c) Absolute neutrophil count ≥ 1.5 × 10\^9/L can be included in the group; 10. The main organ function is good, that is, the liver and kidney function is good before treatment, AST and ALT are ≤ 1.5 times the upper limit of normal (ULN), total bilirubin is ≤ 1.5 times ULN, and serum creatinine is ≤ 1.5 times ULN; 11. The subjects adopted approved contraceptive methods. Female participants must be infertile (hysterectomy, bilateral salpingectomy, bilateral tubal ligation, or more than 1 year after menopause) or have the ability to conceive but have used study approved contraceptive methods during the entire study period until the end of the follow-up period, 2 weeks before the first dose; Women with fertility must have a negative serum pregnancy test within 24 hours prior to the first dose of medication; 12. The subjects fully understand and are able to comply with the requirements of the research protocol, and are willing to complete the study as planned. Exclusion Criteria: 1. Individuals who are allergic to known ingredients or excipients in atorvastatin or all trans retinoic acid; 2. Accept medication maintenance therapy (including but not limited to aspirin, clopidogrel, and/or nonsteroidal anti-inflammatory drugs NSAIDs) or anticoagulants that affect platelet function or quantity; 3. Accompanied by autoimmune hemolytic anemia, or various secondary or hereditary thrombocytopenia; Such as leukemia, lymphoma, multiple myeloma, aplastic anemia, myelodysplastic syndrome, Evans syndrome, common variant immunodeficiency, systemic lupus erythematosus, cirrhosis, antiphospholipid antibody syndrome, pseudothrombocytopenia, drug-induced thrombocytopenia (such as quinine, heparin, antimicrobial drugs, antiepileptic drugs, etc.), etc; 4. There is bone marrow fibrosis with MF ≥ 2; 5. Participate in other research drug studies (including vaccine studies) or be exposed to other research drugs (TPO receptor agonists or salvage treatments) within 4 weeks or 5 half lives (whichever is longer) before the first use of the medication; 6. Previous ITP treatments, including rescue therapy with platelet transfusions, glucocorticoids, immunoglobulins, immunomodulators, etc., did not end within 2 weeks prior to enrollment; 7. The subject has a history of any arterial/venous thrombosis (including stroke, transient ischemic attack, myocardial infarction, deep vein thrombosis, or pulmonary embolism) within the past year; 8. Pregnant or lactating women; 9. History of alcohol/drug abuse within 12 months prior to screening or first dose; 10. Previous studies have shown poor efficacy in the treatment of sufficient and sufficient doses of atorvastatin; 11. Previous all trans retinoic acid had poor efficacy; 12. All laboratory or clinical evidence of HIV infection and active hepatitis during screening. Laboratory tests during the screening period indicate active hepatitis C infection or hepatitis B infection. (hepatitis B reference: HBsAg positive and HBV DNA ≥ 500 IU/ml; Hepatitis C reference: HCV antibody positive and HCV virus copy number\>upper limit of normal); 13. Life threatening bleeding (WHO bleeding score 3) or estimated need for salvage treatment before the first dose of treatment in patients; 14. History of malignant tumors or accompanying malignant tumors; 15. The researchers believe that there are any other circumstances that may prevent the subjects from completing the study or pose significant risks to them.
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
How to take part
Only the study team decides who joins. These are the ways to reach them.
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The official record
The full official record for this study. This one lists no contact details, but it is the first place any would appear.
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A doctor treating you
A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.
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Other studies related to the condition(s) this trial covers.
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