ITP - Immune ThrombocytopeniaPhase 2/3NCT07278908
Avatrombopag Combined With All-trans Retinoic Acid in the Treatment of Primary Immune Thrombocytopenia
A Phase 2/3 study testing Avatrombopag+All trans retinoic acid and Avatrombopag in people with ITP - Immune Thrombocytopenia, run by Institute of Hematology & Blood Diseases Hospital, China.
Trial summary
This study is an open label, randomized controlled, multicenter clinical trial aimed at comparing the efficacy and safety of the combination of atorvastatin and all trans retinoic acid with atorvastatin alone in the treatment of first-line ineffective or recurrent immune thrombocytopenia. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- ITP - Immune Thrombocytopenia
- What is being tested?
- Avatrombopag+All trans retinoic acid and Avatrombopag
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 248 participants
- Where
- No sites listed yet
- Study dates
- Dec 2025to Dec 2028
- Phase
- Phase 2/3
- 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 patient signs an informed consent form;
- ≥ 18 years old;
- The patient's history of ITP is at least 3 months (persistent or chronic ITP);
- 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;
- First line treatment, such as ineffective hormone or immunoglobulin therapy, or ITP patients who relapse after treatment;
- 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;
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- Within the past year, there have been no heart diseases, including NYHA class III/IV congestive heart failure, drug-induced arrhythmias, or myocardial infarction;
- 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;
- 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;
- 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;
- 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;
- 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.
Who cannot join
- Individuals who are allergic to known ingredients or excipients in atorvastatin or all trans retinoic acid;
- 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;
- 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;
- There is bone marrow fibrosis with MF ≥ 2;
- 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;
- Previous ITP treatments, including rescue therapy with platelet transfusions, glucocorticoids, immunoglobulins, immunomodulators, etc., did not end within 2 weeks prior to enrollment;
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- 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;
- Pregnant or lactating women;
- History of alcohol/drug abuse within 12 months prior to screening or first dose;
- Previous studies have shown poor efficacy in the treatment of sufficient and sufficient doses of atorvastatin;
- Previous all trans retinoic acid had poor efficacy;
- 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);
- Life threatening bleeding (WHO bleeding score 3) or estimated need for salvage treatment before the first dose of treatment in patients;
- History of malignant tumors or accompanying malignant tumors;
- The researchers believe that there are any other circumstances that may prevent the subjects from completing the study or pose significant risks to them.
About this study
Official title: Efficacy and Safety of Avatrombopag Versus Avatrombopag Combined With All-trans Retinoic Acid in the Treatment of Primary Immune Thrombocytopenia: a Multicenter, Open-label, Randomized Controlled Study
This study is an open label, randomized controlled, multicenter clinical trial aimed at comparing the efficacy and safety of the combination of atorvastatin and all trans retinoic acid with atorvastatin alone in the treatment of first-line ineffective or recurrent immune thrombocytopenia.
The study is divided into a screening period (2 weeks), a treatment period (0-24 weeks), a reduction period (25-36 weeks), and a follow-up period (37-52 weeks), with a total of one year from the start of treatment to the end of follow-up (52 weeks).
- Conditions
- ITP - Immune Thrombocytopenia
- Being tested
- Avatrombopag+All trans retinoic acid, Avatrombopag
- Keywords
- Immune Thrombocytopenia, all trans retinoic acid, Avatrombopag
- 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.
Avatrombopag+All trans retinoic acidDrug
Avatrombopag, po, starting dose 20mg qd, later adjusted dose or frequency according to response; All trans retinoic acid, po, 10mg bid.
AvatrombopagDrug
Avatrombopag, po, starting dose 20mg qd, later adjusted dose or frequency according to response
| Study group | Role | What they receive |
|---|---|---|
| Avatrombopag+All trans retinoic acid | Experimental | Not described Receives: Drug: Avatrombopag+All trans retinoic acid |
| Avatrombopag | Active comparator | Not described Receives: Drug: Avatrombopag |
What is being measured
The primary outcome is the main question the study is designed to answer.
Treatment Free Rate at week 52
Measured: up to 52 weeks
Secondary outcomes (9)
At weeks 12 and 24 without rescue treatment, the proportion of subjects with platelet levels reaching CR, R, and ≥ 50 × 10^9/L, respectively
Measured: up to 12 and 24 weeks
Treatment Free Rate at week 12 after discontinuation of treatment
Measured: up to 12 weeks after discontinuation of treatment
The proportion of subjects whose platelet levels reached CR and R at 12 weeks after discontinuation of treatment
Measured: up to 12 weeks after discontinuation of treatment
The time from the start of treatment to the first platelet count ≥ 50 × 10^9/L
From date of randomization until the date of first documented platelet count ≥ 50 × 10^9/L, assessed up to 52 weeks
Measured: up to 52 weeks
The time required from the start of treatment to the first platelet count increasing by at least 2 times compared to the baseline platelet count
From date of randomization until the date of first documented platelet count increasing by at least 2 times compared to the baseline platelet count, assessed up to 52 weeks
Measured: up to 52 weeks
The number of times subjects received rescue therapy during treatment and follow-up, and the proportion of subjects who received at least one rescue therapy
Measured: up to 52 weeks
World Health Organization(WHO) bleeding scoring criteria
score range: 0-4; higher scores mean a worse outcome
Measured: up to 52 weeks
The Functional Assessment ot Chronic Illness Therapy-Fatigure(FACITF) score
score range: 0-52; higher scores mean a worse outcome
Measured: up to 52 weeks
Adverse event
Measured: up to 52 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted12 Dec 2025
- Study startDec 2025 (estimated)
- Primary completionDec 2028 (estimated)
- Study completionDec 2028 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for ITP - 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 2/3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 248 participants (estimated)
Sponsor and investigators
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07278908
- Sponsor's protocol ID
- IIT2025121
- Status last verified
- Dec 2025
- First submitted
- 17 Nov 2025
- Last updated
- 12 Dec 2025
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 12 Dec 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Efficacy and Safety of Avatrombopag Versus Avatrombopag Combined With All-trans Retinoic Acid in the Treatment of Primary Immune Thrombocytopenia: a Multicenter, Open-label, Randomized Controlled Study
- ClinicalTrials.gov ID
- NCT07278908
- Acronym / sponsor ID
- IIT2025121
- Conditions
- ITP - Immune Thrombocytopenia
- Keywords
- Immune Thrombocytopenia, all trans retinoic acid, Avatrombopag
- Phase
- Phase 2/3
- Study type
- Interventional
- Recruitment status
- Not yet recruiting
- Enrolment
- 248 participants (estimated)
- Lead sponsor
- Institute of Hematology & Blood Diseases Hospital, ChinaUniversity, hospital or other
- Study start
- Dec 2025 (estimated)
- Primary completion
- Dec 2028 (estimated)
- Study completion
- Dec 2028 (estimated)
- First posted
- 12 Dec 2025 (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 study is an open label, randomized controlled, multicenter clinical trial aimed at comparing the efficacy and safety of the combination of atorvastatin and all trans retinoic acid with atorvastatin alone in the treatment of first-line ineffective or recurrent immune thrombocytopenia.
The study is divided into a screening period (2 weeks), a treatment period (0-24 weeks), a reduction period (25-36 weeks), and a follow-up period (37-52 weeks), with a total of one year from the start of treatment to the end of follow-up (52 weeks).
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Avatrombopag+All trans retinoic acid | Experimental | Drug: Avatrombopag+All trans retinoic acid |
| Avatrombopag | Active comparator | Drug: Avatrombopag |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (12 listed)
- The patient signs an informed consent form;
- ≥ 18 years old;
- The patient's history of ITP is at least 3 months (persistent or chronic ITP);
- 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;
- First line treatment, such as ineffective hormone or immunoglobulin therapy, or ITP patients who relapse after treatment;
Key exclusion criteria (15 listed)
- Individuals who are allergic to known ingredients or excipients in atorvastatin or all trans retinoic acid;
- 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;
- 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;
- There is bone marrow fibrosis with MF ≥ 2;
- 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;
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 |
|---|---|---|
| Avatrombopag+All trans retinoic acidAvatrombopag, po, starting dose 20mg qd, later adjusted dose or frequency according to response; All trans retinoic acid, po, 10mg bid. | Drug | Avatrombopag+All trans retinoic acid |
| AvatrombopagAvatrombopag, po, starting dose 20mg qd, later adjusted dose or frequency according to response | Drug | Avatrombopag |
Primary outcome
Treatment Free Rate at week 52
Time frame: up to 52 weeks
9 secondary outcomes
At weeks 12 and 24 without rescue treatment, the proportion of subjects with platelet levels reaching CR, R, and ≥ 50 × 10^9/L, respectively
Time frame: up to 12 and 24 weeks
Treatment Free Rate at week 12 after discontinuation of treatment
Time frame: up to 12 weeks after discontinuation of treatment
The proportion of subjects whose platelet levels reached CR and R at 12 weeks after discontinuation of treatment
Time frame: up to 12 weeks after discontinuation of treatment
The time from the start of treatment to the first platelet count ≥ 50 × 10^9/L
Time frame: up to 52 weeks
The time required from the start of treatment to the first platelet count increasing by at least 2 times compared to the baseline platelet count
Time frame: up to 52 weeks
The number of times subjects received rescue therapy during treatment and follow-up, and the proportion of subjects who received at least one rescue therapy
Time frame: up to 52 weeks
World Health Organization(WHO) bleeding scoring criteria
Time frame: up to 52 weeks
The Functional Assessment ot Chronic Illness Therapy-Fatigure(FACITF) score
Time frame: up to 52 weeks
Adverse event
Time frame: up to 52 weeks
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 12 Dec 2025.
- 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.
The record lists no contact. Use the official record to reach the sponsor.
Questions about this trial
What is this clinical trial studying?
A Phase 2/3 study testing Avatrombopag+All trans retinoic acid and Avatrombopag in people with ITP - Immune Thrombocytopenia, run by Institute of Hematology & Blood Diseases Hospital, 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 12 inclusion and 15 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: opening soon, as of the sponsor's last update on 12 Dec 2025. 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 combined 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.
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
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?
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?
Treatment Free Rate at week 52 (time frame: up to 52 weeks). 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 12 Dec 2025.