Immune ThrombocytopeniaPhase 3NCT07597395
ATRA for Management of Primary ITP
A Phase 3 study testing all-trans retinoic acid (ATRA) in people with Immune Thrombocytopenia, run by Peking University People's Hospital at 1 site in China.
Trial summary
A multicenter, randomized, double-blind placebo-controlled study to report the efficacy and safety of all-trans etinoic acid compared to placebo for the treatment of adults with corticosteriod-resistant/relapsed primary immune thrombocytopenia (ITP). Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Immune Thrombocytopenia
- What is being tested?
- all-trans retinoic acid (ATRA)
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 192 participants
- Where
- 1 siteChina
- Study dates
- 30 May 2026to 30 Dec 2028
- Phase
- Phase 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
- 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.
Who cannot join
- 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;
Show all 18Show fewer
- 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.
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
Peking University Institute of hematology, People's Hospital
Beijing
Xiaohui Zhang, 861088326001, zhangxh@bjmu.edu.cn
About this study
Official title: All-trans Retinoic Acid for Management of Primary Immune Thrombocytopenia : a Randomized, Double-blind, Placebo-controlled Study
A multicenter, randomized, double-blind placebo-controlled study to report the efficacy and safety of all-trans etinoic acid compared to placebo for the treatment of adults with corticosteriod-resistant/relapsed primary immune thrombocytopenia (ITP).
Read the detailed description
The investigators are undertaking a parallel-group, multicenter, randomized controlled trial of 192 adults with corticosteriod-resistant/relapsed primary ITP. Patients were randomized to all-trans etinoic acid and placebo group. Platelet count, bleeding, and other symptoms were evaluated before and after treatment. Adverse events are also recorded throughout the study.
- Conditions
- Immune Thrombocytopenia
- Being tested
- all-trans retinoic acid (ATRA), Placebo
- Keywords
- Immune thrombocytopenia;, Corticosteriod resistance resistant/relaped, ATRA
- 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.
all-trans retinoic acid (ATRA)Drug
Also called tretinoin
10mg twice daily ×24 weeks
PlaceboDrug
10mg twice daily ×24 weeks
| Study group | Role | What they receive |
|---|---|---|
| ATRA | Experimental | ATRA 10mg twice daily Receives: Drug: all-trans retinoic acid (ATRA) |
| Placebo | Placebo comparator | Placebo 10mg twice daily Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Durable platelet response
Platelet count of ⩾50 × 10^9/L or between ⩾30 × 10^9/L and <50 × 10^9/L and at least doubled from baseline on at least four of six scheduled visits between weeks 14 and 24
Measured: Up to week 24
Secondary outcomes (14)
24-week overall response rate
From enrollment to the end of week 24, the proportion of patients with at least one platelet count of ≥50×10⁹/L or between 30 × 10^9/L and 50 × 10^9/L plus at least doubled from baseline
Measured: Up to week 24
12-week overall response rate
From enrollment to the end of week 12, the proportion of patients with at least one platelet count of ≥50×10⁹/L or between 30 × 10^9/L and 50 × 10^9/L plus at least doubled from baseline
Measured: Up to week 12
Consecutive Increased Platelet Counts (≥2 Consecutive PLT ≥ 30×10^9/L)
The proportion of participants who achieved platelet counts ≥ 30×10^9/L and at least doubled from baseline at two consecutive visits
Measured: Up to week 24
Consecutive Increased Platelet Counts (≥2 Consecutive PLT ≥ 50×10^9/L)
The proportion of patients with two consecutive platelet counts of 50×10⁹/L or more and doubling from the baseline count
Measured: Up to week 24
Quality of Life Score
ITP-patient assessment questionnaire (ITP-PAQ) was used to assess the HRQoL before and after treatment.
Measured: Up to week 24
Adverse Events
The rate of participants with adverse events
Measured: Up to week 28
Complete response rate
The proportion of patients with a platelet count of ≥ 100×10^9/L in absence of bleeding at any time
Measured: Up to week 24
Duration of response
Number of weeks with platelet count of ≥50 × 10^9/L or between 30 × 10^9/L and 50 × 10^9/L plus at least doubled from baseline
Measured: Up to 24 weeks
Time to response
Time from treatment initiation to first platelet count reaching ≥30x10^9/L and doubling from the baseline count in 0-24 weeks
Measured: Up to week 24
Initial response
Platelet count ≥30×10^9/L and at least doubling baseline at day 28
Measured: Up to week 4
Peak platelet count
The peak platelet count without rescue treatment
Measured: Up to week 24
Bleeding events
Bleeding incidence and severity per WHO bleeding score in 0-12 weeks and 0-24 weeks
Measured: 0-12 weeks and 0-24 weeks
Rescue treatment
Proportion of patients receiving predefined rescue treatment
Measured: Up to week 24
Reduced or discontinued concomitant therapy
Proportion of patients with reduced or discontinued baseline concomitant anti-ITP therapy
Measured: Up to week 24
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted19 May 2026
- Study start30 May 2026 (estimated)
- Primary completion30 Dec 2027 (estimated)
- Study completion30 Dec 2028 (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 Peking University People's HospitalThe 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 3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 192 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Peking University People's Hospital
- Sponsor type
- University, hospital or other
- Collaborators
- Peking University First Hospital, Beijing Friendship Hospital, Beijing Tongren Hospital, Peking University Third Hospital, Beijing Hospital
- Responsible party
- Principal investigator, Xiao Hui Zhang, Peking University People's Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07597395
- Sponsor's protocol ID
- 2025PHD007-001
- Status last verified
- May 2026
- First submitted
- 13 May 2026
- Last updated
- 19 May 2026
- 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 19 May 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- All-trans Retinoic Acid for Management of Primary Immune Thrombocytopenia : a Randomized, Double-blind, Placebo-controlled Study
- ClinicalTrials.gov ID
- NCT07597395
- Acronym / sponsor ID
- 2025PHD007-001
- Conditions
- Immune Thrombocytopenia
- Keywords
- Immune thrombocytopenia;, Corticosteriod resistance resistant/relaped, ATRA
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Not yet recruiting
- Enrolment
- 192 participants (estimated)
- Lead sponsor
- Peking University People's HospitalUniversity, hospital or other
- Collaborators
- Peking University First Hospital, Beijing Friendship Hospital, Beijing Tongren Hospital, Peking University Third Hospital, Beijing Hospital
- Study start
- 30 May 2026 (estimated)
- Primary completion
- 30 Dec 2027 (estimated)
- Study completion
- 30 Dec 2028 (estimated)
- First posted
- 19 May 2026 (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.
A multicenter, randomized, double-blind placebo-controlled study to report the efficacy and safety of all-trans etinoic acid compared to placebo for the treatment of adults with corticosteriod-resistant/relapsed primary immune thrombocytopenia (ITP).
Detailed description
The investigators are undertaking a parallel-group, multicenter, randomized controlled trial of 192 adults with corticosteriod-resistant/relapsed primary ITP. Patients were randomized to all-trans etinoic acid and placebo group. Platelet count, bleeding, and other symptoms were evaluated before and after treatment. Adverse events are also recorded throughout the study.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| ATRA | Experimental | Drug: all-trans retinoic acid (ATRA)ATRA 10mg twice daily |
| Placebo | Placebo comparator | Drug: PlaceboPlacebo 10mg twice daily |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (3 listed)
- 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.
Key exclusion criteria (18 listed)
- 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;
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 |
|---|---|---|
| all-trans retinoic acid (ATRA)Also: tretinoin10mg twice daily ×24 weeks | Drug | ATRA |
| Placebo10mg twice daily ×24 weeks | Drug | Placebo |
Primary outcome
Durable platelet response
Time frame: Up to week 24
Platelet count of ⩾50 × 10^9/L or between ⩾30 × 10^9/L and <50 × 10^9/L and at least doubled from baseline on at least four of six scheduled visits between weeks 14 and 24
14 secondary outcomes
24-week overall response rate
Time frame: Up to week 24
12-week overall response rate
Time frame: Up to week 12
Consecutive Increased Platelet Counts (≥2 Consecutive PLT ≥ 30×10^9/L)
Time frame: Up to week 24
Consecutive Increased Platelet Counts (≥2 Consecutive PLT ≥ 50×10^9/L)
Time frame: Up to week 24
Quality of Life Score
Time frame: Up to week 24
Adverse Events
Time frame: Up to week 28
Complete response rate
Time frame: Up to week 24
Duration of response
Time frame: Up to 24 weeks
Time to response
Time frame: Up to week 24
Initial response
Time frame: Up to week 4
Peak platelet count
Time frame: Up to week 24
Bleeding events
Time frame: 0-12 weeks and 0-24 weeks
Rescue treatment
Time frame: Up to week 24
Reduced or discontinued concomitant therapy
Time frame: Up to week 24
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 19 May 2026.
- 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.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing all-trans retinoic acid (ATRA) in people with Immune Thrombocytopenia, run by Peking University People's Hospital 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 3 inclusion and 18 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 19 May 2026. 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. Some participants in this study may receive a placebo. 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 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?
Contact Xiaohui Zhang, Prof. (861088326001, zhangxh@bjmu.edu.cn), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Durable platelet response (time frame: Up to week 24). 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 19 May 2026.