Clinical trial NCT05438875: The Combination of ATRA and Eltrombopag as the Treatment of Steroid-resistant/Relapse ITP Based on MSC-C5b-9
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
Study type: INTERVENTIONAL
Overall status: RECRUITING
Phase: PHASE3
Conditions: Purpura, Thrombocytopenic, Idiopathic
Keywords: Resistant/recurrent ITP
MSC-C5b-9 marker
all-trans retinoic acid
Eltrombopag
prospective clinical study
Lead sponsor: Peking University People's Hospital
Sponsor class: OTHER
Collaborators: name: Beijing Friendship Hospital
name: Beijing Tongren Hospital
name: Beijing Hospital
name: Navy General Hospital, Beijing
First posted: 2022-06-30
Enrollment: 96
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief 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).

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.

Eligibility 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.

Primary outcomes:
measure: Sustained Response Rate (SR) at 18 months
timeFrame: 18 months
Secondary outcomes:
measure: Complete response rate (CR)
timeFrame: 18 months
measure: Response rate (R)
timeFrame: 18 months
measure: Inefficiency (NR)
timeFrame: 18 months
measure: Recurrence rate (relapse)
timeFrame: 18 months
measure: Early response
timeFrame: 1 week
measure: Initial response
timeFrame: 1 month
measure: Time to response (TTR)
timeFrame: 18 months
measure: Time to relapse (duration of efficacy)
timeFrame: 18 months
Other outcomes:
Locations:
city: Beijing
country: China
facility: Peking University Insititute of Hematology, Peking University People's Hospital
state: Beijing
city: Beijing
country: China
facility: Beijing Tongren hospital
state: Beijing
city: Beijing
country: China
facility: Department of Hematology, Beijing Friendship Hospital, Capital Medical University
city: Beijing
country: China
facility: Department of Hematology, Beijing Hospital
city: Beijing
country: China
facility: Department of Hematology, Senior Department of Hematology, the Fifth Medical Center of PLA General Hospital
Study officials:
affiliation: Study Principal Investigator Peking University People's Hospital
name: Xiaohui Zhang, MD
role: PRINCIPAL_INVESTIGATOR
References: