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Purpura, Thrombocytopenic, IdiopathicPhase 3NCT05438875

The Combination of ATRA and Eltrombopag as the Treatment of Steroid-resistant/Relapse ITP Based on MSC-C5b-9

A Phase 3 study testing all-trans retinoic acid and Eltrombopag in people with Purpura, Thrombocytopenic, Idiopathic, run by Peking University People's Hospital at 5 sites in China.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
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Trial summary

A Prospective, Randomized, Open-Label, Multicenter Clinical Trial study to compare the efficacy and safety of ATRA plus eltrombopag compared to eltrombopag monotherapy in the treatment of steroid-resistant/relapsed immune thrombocytopenia (ITP). Read more about this study

Who is it for?
Ages 18 years and older
Conditions the study is for
Purpura, Thrombocytopenic, Idiopathic
What is being tested?
all-trans retinoic acid and Eltrombopag
Type of treatment
Pill or oral medicine
Could I receive a placebo?
No, everyone receives an active treatment
How many people
96 participants
Where
5 sitesChina
Study dates
12 Oct 2022to 30 Nov 2024
Phase
Phase 3
Are there results?
None posted in the registry

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

  • 1. Isolated thrombocytopenia (platelet count <30 × 109/L); 2. age > 18 years; 3. normal white blood cells and red blood cells on bone marrow examination; 4. increased number of megakaryocytes (bone marrow examination was performed in all patients except for myelofibrosis or other conditions that can cause thrombocytopenia disease); 5. the spleen was normal in size; 6. Eastern Cooperative Oncology Group status score (ECOG score) ≤ 2; 7. ineffective or relapsed after at least 1 course of full-dose full-course hormone therapy; 8. Failure of prior ITP therapy (eg, hormones, splenectomy, and cyclosporine) and at least 4 weeks from enrollment.

Who cannot join

  • 1. Secondary ITP such as drug-related thrombocytopenia; 2. thrombocytopenia due to viral infection (HIV, hepatitis B virus, or hepatitis C virus); 3. severe cardiac, renal, hepatic, or respiratory insufficiency; 4. severe immunodeficiency; 5. pregnancy or lactation; 6. myelodysplasia or Myelofibrosis; 7. history of malignancy; 8. ongoing immunosuppressive therapy for other diseases; 9. patients previously treated with eltrombopag were excluded from this study.

Trial locations and contacts

5 sites in 1 country.

5 sites in 1 country

China5 sites

  • Peking University Insititute of Hematology, Peking University People's Hospital

    Beijing, Beijing Municipality, 100010

  • Beijing Tongren hospital

    Beijing, Beijing Municipality

  • Department of Hematology, Beijing Friendship Hospital, Capital Medical University

    Beijing

  • Department of Hematology, Beijing Hospital

    Beijing

  • Department of Hematology, Senior Department of Hematology, the Fifth Medical Center of PLA General Hospital

    Beijing

Other trials in China

About this study

Official title: The Combination of ATRA and Eltrombopag as the Treatment Strategy for Glucocorticoid-Resistant/Relapsed ITP Based on the Stratification of the New Biomarker MSC-C5b-9: A Prospective, Randomized, Open-Label, Multicenter Clinical Trial

A Prospective, Randomized, Open-Label, Multicenter Clinical Trial study to compare the efficacy and safety of ATRA plus eltrombopag compared to eltrombopag monotherapy in the treatment of steroid-resistant/relapsed immune thrombocytopenia (ITP).

Read the detailed description

The investigators are undertaking a parallel group, multicenter, randomized controlled trial of patients with ITP in China. Patients were tested for MSCs, and they were divided into MSC-C5b-9+ group and MSC-C5b-9- group according to the test results, and the two groups were randomized to ATRA + eltrombopag and eltrombopag monotherapy group. Platelet count, bleeding and other symptoms were evaluated before and after treatment. Adverse events are also recorded throughout the study.

Being tested
all-trans retinoic acid, Eltrombopag
Keywords
Resistant/recurrent ITP, MSC-C5b-9 marker, all-trans retinoic acid, Eltrombopag, prospective clinical study
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 acidDrug

    Also called retinoic acid

    ATRA 10 mg, 2 times a day, orally.The treatment course is 12 weeks.

  • EltrombopagDrug

    Also called TPO-RA

    The initial dose of eltrombopag is 50 mg/time, once a day, and the dose is increased when the platelet count is lower than 5×109/L, the maximum is 75 mg/d, and the dose is higher than 200×109/L. When the drug is temporarily discontinued, the drug is re-administered according to the platelet count.The treatment course is 12 weeks.

Study groups in NCT05438875
Study groupRoleWhat they receive
EltrombopagActive comparatorThe initial dose of eltrombopag was 50 mg/time, once a day. According to the clinical standard of eltrombopag dose adjustment in the research protocol set by the research group, the dose was increased when the platelet count was lower than 5×109/L. The highest is 75mg/d, if the dose is higher than 200×109/L, the dose is reduced. When the dose is higher than 400×109/L, the drug is temporarily discontinued, and the drug is repeated according to the platelet count. The treatment course is 12 weeks.
Receives: Drug: Eltrombopag
ATRA and EltrombopagExperimentalATRA 10 mg, 2 times a day, orally; The initial dose of eltrombopag was 50 mg/time, once a day. According to the clinical standard of eltrombopag dose adjustment in the research protocol set by the research group, the dose was increased when the platelet count was lower than 5×109/L. The maximum dose is 75 mg/d, the dose is reduced if it is higher than 200×109/L, and the drug is temporarily discontinued when it is higher than 400×109/L, and the drug is repeated according to the platelet count. The treatment course is 12 weeks.
Receives: Drug: all-trans retinoic acid; Drug: Eltrombopag

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Sustained Response Rate (SR) at 18 months

    The maintenance of platelet count ≥ 30 x 10^9/L, at least 2-fold increase of the baseline count, the absence of bleeding, and no need for rescue medication at the 18-month follow-up

    Measured: 18 months

Secondary outcomes (8)

  1. Complete response rate (CR)

    The complete response (CR) was defined as platelet count more than 100×10^9/L and absence of bleeding.

    Measured: 18 months

  2. Response rate (R)

    The Response rate (R) was defined as platelet count more than 30×10^9/L and more than 2 times higher than baseline, without bleeding

    Measured: 18 months

  3. Inefficiency (NR)

    Platelet count <30×10^9/L, or less than 2-fold increase from baseline or associated with bleeding

    Measured: 18 months

  4. Recurrence rate (relapse)

    After the treatment is effective, the platelet count drops below 30×10^9/L or drops to less than 2 times the basal value, or bleeding symptoms occur

    Measured: 18 months

  5. Early response

    Platelet count ≥30×10^9/L and at least doubling baseline at 1 wk.

    Measured: 1 week

  6. Initial response

    Initial response as platelet count more than 30×10^9/L and at least 2-fold increase of the baseline count and absence of bleeding.

    Measured: 1 month

  7. Time to response (TTR)

    The time from starting treatment to time of achievement of CR or R

    Measured: 18 months

  8. Time to relapse (duration of efficacy)

    The time from achievement of CR or R to time of relapse

    Measured: 18 months

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted30 Jun 2022
  2. Study start12 Oct 2022
  3. Primary completion30 Nov 2024
  4. Study completion30 Nov 2024

Resources

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
None (open label)
Enrolment
96 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT05438875
Sponsor's protocol ID
2022-1-4082
Status last verified
Dec 2024
First submitted
25 Jun 2022
Last updated
9 Sep 2025
Results posted
No
Data monitoring committee
No
FDA-regulated drug
No
FDA-regulated device
No

Publications

  • Fu H, An Z, Li M, Liu Y, Liu H, Feng R, Wang Z, Zhang G, Wang J, Yang L, Huang Q, Zhao P, Zang M, Wang C, He Y, Zhu X, Liu H, Wang Y, Xu L, Kong Y, Chang Y, Zhao X, Wang Y, Feng G, Huang X, Zhang X. All-Trans Retinoic Acid plus Eltrombopag for Refractory/Relapsed Immune Thrombocytopenia. NEJM Evid. 2026 Aug;5(8):EVIDoa2500333. doi: 10.1056/EVIDoa2500333. Epub 2026 Jul 28.

    PubMed 42517709

Questions about this trial

What is this clinical trial studying?

A Phase 3 study testing all-trans retinoic acid and Eltrombopag in people with Purpura, Thrombocytopenic, Idiopathic, run by Peking University People's Hospital at 5 sites 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 1 inclusion and 1 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: finished, as of the sponsor's last update on 9 Sep 2025. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 5 sites 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. 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?

The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.

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?

Sustained Response Rate (SR) at 18 months (time frame: 18 months). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT05438875, which also lists 1 linked publication.

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 9 Sep 2025.

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