Clinical trial NCT05459649: Combined Steroid and Cyclosporin as First-line Treatment in Adults With Primary Immune Thrombocytopenia
Official title: A Multicenter Randomized Trial of First Line Treatment for Newly Diagnosed Immune Thrombocytopenia: Standard Steroid Treatment Versus Combined Steroid and Cyclosporin
Study type: INTERVENTIONAL
Overall status: NOT_YET_RECRUITING
Phase: PHASE2
Conditions: Immune Thrombocytopenia
Lead sponsor: Peking University People's Hospital
Sponsor class: OTHER
First posted: 2022-07-15
Enrollment: 253
Sex: ALL
Minimum age (years): 18
Maximum age (years): 80
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: Randomized, open-label, multicenter study to compare the efficacy and safety of cyclosporin plus standard steroid compared to standard steroid monotherapy for the first-line treatment of adults with primary immune thrombocytopenia (ITP).

Detailed description: The investigators are undertaking a parallel group, multicenter, randomized controlled trial of 253 adults with ITP in China. Patients were randomized to cyclosporin plus standard steroid compared to standard steroid 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. Newly-diagnosed, treatment-naive primary ITP;
2. Platelet counts \<30×10\^9/L;
3. Platelet counts \< 50×10\^9/L and significant bleeding symptoms (WHO bleeding scale 2 or above);
4. Willing and able to sign written informed consent.

Exclusion Criteria:

1. Pregnant or lactating women;
2. Secondary ITP (have a known diagnosis of connective tissue diseases, malignancy, active infection, HIV infections or hepatitis B virus or hepatitis C virus infections);
3. Received first-line and second-line ITP specific treatments (e.g., steroids, intravenous immunoglobulin, TPO-RAs, rhTPO, rituximab, etc);
4. Received drugs affecting the platelet counts within 6 months before the screening visit (e.g., chemotherapy, anticoagulants, etc);
5. Severe medical condition (lung, heart, hepatic or renal disorder);
6. Patients who are deemed unsuitable for the study by the investigator.

Primary outcomes:
measure: Treatment failure
timeFrame: From date of randomization until 2 years or the end of follow-up
Secondary outcomes:
measure: Number of patients with side effects
timeFrame: From date of randomization until 2 years or the end of follow-up
measure: Number of patients with bleeding
timeFrame: From date of randomization until 2 years or the end of follow-up
measure: Sustained response
timeFrame: From date of randomization until 2 years or the end of follow-up
measure: Overall response (OR)
timeFrame: From date of randomization until 2 years or the end of follow-up
measure: Complete response (CR)
timeFrame: From date of randomization until 2 years or the end of follow-up
measure: Time to response
timeFrame: From date of randomization until 2 years or the end of follow-up
measure: Duration of response
timeFrame: From date of randomization until 2 years or the end of follow-up
measure: Remission
timeFrame: at 12-month follow-up
measure: Rescue therapy
timeFrame: From date of randomization until 2 years or the end of follow-up
measure: Associated factors of treatment failure, OR, SR and remission
timeFrame: From date of randomization until 2 years or the end of follow-up
Other outcomes:
Locations:
city: Beijing
country: China
facility: Peking University Insititute of Hematology, Peking University People's Hospital
state: Beijing
Study officials:
affiliation: Peking University People's Hospital
name: Xiao-Hui Zhang, MD
role: PRINCIPAL_INVESTIGATOR
References: