Can a vitamin a drug boost platelets in Hard-to-Treat ITP?
NCT ID NCT07597395
First seen Jun 24, 2026 · Last updated Jun 27, 2026 · Updated 1 time
Summary
This study tests all-trans retinoic acid (ATRA), a form of vitamin A, in adults with immune thrombocytopenia (ITP) whose platelet counts stay low despite steroid treatment. About 192 participants will receive either ATRA or a placebo for 24 weeks. The goal is to see if ATRA can safely raise platelet counts to safer levels.
What this could mean
Our plain-language read of the trial. This is informational only, not medical advice or a prediction.
- Active substance
- all-trans retinoic acid (ATRA), also known as tretinoin
- What this could lead to
- If it works, this could provide a new treatment option for adults with immune thrombocytopenia (ITP) whose platelet counts remain low despite standard steroid therapy.
- What could go wrong
- This is a phase 3 trial, but it hasn't started recruiting yet. ATRA may not improve platelet counts more than placebo, and side effects like skin dryness or liver issues are possible.
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 3
Large-scale testing in a bigger group. Usually the last step before a treatment can be approved.
- Participants
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About 192 people
The number the study aims to enrol. It can still change while the study runs.
- Expected to start
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May 2026
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: * Primary ITP if aged ⩾18 years; * With an average of two platelet counts ⩾1 day apart of \<30×10\^9/L during screening and no single platelet count \>35×10\^9/L within 2 weeks before study treatment; * Patients who have previously received at least one first-line standard therapy for ITP (corticosteroid and/or intravenous immunoglobulin) with unsustained efficacy, relapse, intolerance to standard therapy, or insufficient response. Exclusion Criteria: * Pregnant or lactating women, and who were possibly pregnant, planning to become pregnant, or who had partners planning to become pregnant; * With active malignancy or a history of malignant tumor; * Having experienced severe bacterial, viral, fungal or parasitic infection within the past 4 weeks; * With a history of symptomatic herpes zoster infection within 12 weeks prior to screening; * Active or chronic HBV, HCV or HIV infection; * Evidence of active tuberculosis; or previous evidence of active tuberculosis without appropriate and documented treatment; or household contact with patients with active tuberculosis without appropriate and documented tuberculosis prophylaxis; * Receipt of live vaccines within the past 12 weeks, or planned live vaccination during the study period; * Prior ATRA therapy; * History of solid organ transplant or planned surgery; * Myelodysplastic syndrome, aplastic anemia or myelofibrosis; * Patients with other diseases were undergoing treatment with immunosuppressants; * Clinically significant thromboembolic events within the past 24 weeks, or ongoing anticoagulant treatment, who are deemed ineligible for the study by the investigator; * History or presence of myocardial infarction, unstable ischemic heart disease, stroke, or NYHA Class IV heart failure; * History or presence of cardiovascular, respiratory, hepatic, gastrointestinal, endocrine, neurological, neuropsychiatric or any other severe and/or unstable diseases that, in the opinion of the investigator, may pose an unacceptable risk with the investigational product or interfere with the interpretation of study data; * AST \> 2 times the upper limit of normal (ULN), ALT \> 2×ULN, TBIL ≥ 1.5×ULN; * WBC \< 2500/µL, neutrophil count \< 1200/µL, lymphocyte count \< 750/µL, hemoglobin \< 9 g/dL; * eGFR \< 50 mL/min/1.73m²; * Other patients deemed unsuitable for enrollment in this study by the investigator.
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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 places running it
1 site. The list below names each one and where it is.
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The official record
ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.
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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.
Contacts and locations
Locations
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Peking University Institute of hematology, People's Hospital
Beijing, China
More trials for these conditions
Other studies related to the condition(s) this trial covers.
- Real-World data may reveal which ITP treatments work best
- A diet tweak may boost platelets in a bleeding disorder
- What Trade-Offs are ITP patients willing to make for better treatment?
- Can a new drug calm the immune System's attack on blood cells?
- Experimental injection aims to boost platelets in chronic blood disorder
- New combo therapy aims to boost platelets in immune disorder