Combining ATRA and Eltrombopag for treating resistant ITP
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
This study is testing whether combining two medications, ATRA and eltrombopag, can help people with hard-to-treat immune thrombocytopenia (ITP) have better platelet counts and fewer bleeding issues compared to using eltrombopag alone.
Quick facts
| Phase | Phase 3 |
|---|---|
| Study type | Interventional |
| Enrollment | 96 (estimated) |
| Ages | 18 Years and up |
| Sex | All |
| Sponsor | Peking University People's Hospital Academic / other |
| Locations | 5 sites (Beijing, Beijing and 4 other locations) |
| Trial ID | NCT05438875 on ClinicalTrials.gov |
What this trial studies
This clinical trial is a prospective, randomized, open-label, multicenter study aimed at evaluating the efficacy and safety of combining all-trans retinoic acid (ATRA) with eltrombopag compared to eltrombopag alone in patients with steroid-resistant or relapsed immune thrombocytopenia (ITP). Patients will be tested for MSC-C5b-9 markers and divided into two groups based on their results, followed by randomization into treatment groups. The study will assess platelet counts, bleeding symptoms, and adverse events before and after treatment to determine the effectiveness of the combination therapy.
Who should consider this trial
Good fit: Ideal candidates are adults over 18 years with isolated thrombocytopenia and a history of ineffective or relapsed ITP after prior therapies.
Not a fit: Patients with secondary ITP or those with severe underlying health conditions may not benefit from this study.
Why it matters
Potential benefit: If successful, this treatment could provide a new effective option for patients with steroid-resistant or relapsed ITP.
How similar studies have performed: Other studies have explored similar combination therapies, but this specific approach is novel in the context of MSC-C5b-9 markers.
Eligibility criteria
Show full inclusion / exclusion criteria
Inclusion Criteria: * 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. Exclusion Criteria: * 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.
Where this trial is running
Beijing, Beijing and 4 other locations
- Peking University Insititute of Hematology, Peking University People's Hospital — Beijing, Beijing, China (Recruiting)
- Beijing Tongren hospital — Beijing, Beijing, China (Recruiting)
- Department of Hematology, Beijing Friendship Hospital, Capital Medical University — Beijing, China (Recruiting)
- Department of Hematology, Beijing Hospital — Beijing, China (Recruiting)
- Department of Hematology, Senior Department of Hematology, the Fifth Medical Center of PLA General Hospital — Beijing, China (Recruiting)
Study contacts
- Principal investigator: Xiaohui Zhang, MD — Study Principal Investigator Peking University People's Hospital
- Study coordinator: Xiaohui Zhang, MD
- Email: zhangxh100@sina.com
- Phone: +8613522338836
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.