Combined Steroid and Cyclosporin as First-line Treatment in Adults With Primary Immune Thrombocytopenia

Immune Thrombocytopenia Clinical Trial

Official title:

A Multicenter Randomized Trial of First Line Treatment for Newly Diagnosed Immune Thrombocytopenia: Standard Steroid Treatment Versus Combined Steroid and Cyclosporin

Clinical Trial Details — Status: Not yet recruiting

Administrative data

• NCT number: NCT05459649
• Other study ID #: CSA-ITP
• Status: Not yet recruiting
• Phase: Phase 2
• Start date: July 20, 2022
• Est. completion date: July 20, 2025

Study information

• Verified date: July 2022
• Source: Peking University People's Hospital
• Contact: Xiao-Hui Zhang, MD
• Phone: +8613522338836
• Email: zhangxh100[@]sina.com
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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).

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.

Recruitment information / eligibility

• Status: Not yet recruiting
• Enrollment: 253
• Est. completion date: July 20, 2025
• Est. primary completion date: July 20, 2024
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years to 80 Years

Eligibility

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.

Related Conditions & MeSH terms

• Immune Thrombocytopenia
• Purpura, Thrombocytopenic, Idiopathic
• Thrombocytopenia

Intervention

Drug:
• Cyclosporine Oral Product
A combination of cyclosporin with standard steroid in newly diagnosed ITP patients: cyclosporin was started orally 1 mg/kg/d in two divided doses for 1 week, increased to 1.5 mg/kg/d for 1week, further increased to 2.5 mg/kg/d and then continued for 24 weeks. The therapeutic serum level of cyclosporin was 75 to 150 ug/L. After 26 weeks of cyclosporin treatment, the dose for patients who achieved complete response was reduced by 25 mg/d every 2 weeks to ensure continuing the lowest dose that achieved the targeted serum level of cyclosporin and a safe platelet count; standard steroid regimen included orally prednisone for a total of 10 weeks: 1 mg per kilogram of body weight for 2 weeks followed by 40 mg daily for 2 weeks, 20 mg daily for 2 weeks, 10 mg daily for 2 weeks, 5 mg daily for 1 week and 5 mg every other day for the final week.
• Prednisone
Standard steroid in newly diagnosed ITP patients: orally prednisone for a total of 10 weeks: 1 mg per kilogram of body weight for 2 weeks followed by 40 mg daily for 2 weeks, 20 mg daily for 2 weeks, 10 mg daily for 2 weeks, 5 mg daily for 1 week and 5 mg every other day for the final week.

Locations

Country	Name	City	State
China	Peking University Insititute of Hematology, Peking University People's Hospital	Beijing	Beijing

Sponsors (1)

Lead Sponsor	Collaborator
Peking University People's Hospital

Country where clinical trial is conducted

China, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Treatment failure	Nonresponse or loss of response for those who had achieved overall response (assessed in a time-to-event analysis). Overall response was defined as platelet count = 30,000 per cubic millimeter and at least 2-fold increase of the baseline count and absence of bleeding.	From date of randomization until 2 years or the end of follow-up
Secondary	Number of patients with side effects	Number of patients with Medication adverse events.	From date of randomization until 2 years or the end of follow-up
Secondary	Number of patients with bleeding	Number of patients with bleeding complication (WHO bleeding score)	From date of randomization until 2 years or the end of follow-up
Secondary	Sustained response	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 6-month follow-up.	From date of randomization until 2 years or the end of follow-up
Secondary	Overall response (OR)	Platelet count = 30,000 per cubic millimeter and at least 2-fold increase of the baseline count and absence of bleeding.	From date of randomization until 2 years or the end of follow-up
Secondary	Complete response (CR)	Platelet count > 100,000 per cubic millimeter and absence of bleeding.	From date of randomization until 2 years or the end of follow-up
Secondary	Time to response	The time from starting treatment to time of achievement of CR or OR	From date of randomization until 2 years or the end of follow-up
Secondary	Duration of response	time from OR until loss of response or until the last follow-up visit	From date of randomization until 2 years or the end of follow-up
Secondary	Remission	a durable platelet count =30×10^9/L without bleeding up to 12 months from randomization.	at 12-month follow-up
Secondary	Rescue therapy	any new medical intervention taken to increase the platelet count or prevent bleeding events or an increase in the dose of concomitant treatments	From date of randomization until 2 years or the end of follow-up
Secondary	Associated factors of treatment failure, OR, SR and remission	Factors that are associated with treatment failure, OR, SR and remission	From date of randomization until 2 years or the end of follow-up