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

NCT05459649 | Unknown Phase 2

• 1 other identifier: CSA-ITP
• Study Type: interventional
• Target: 253
• Locations: 1 country
• Sites: 1

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).

Conditions

Immune Thrombocytopenia

Outcome Measures

Primary Outcomes (1)

• 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 Outcomes (10)

• Number of patients with side effects

  From date of randomization until 2 years or the end of follow-up

• Number of patients with bleeding

  From date of randomization until 2 years or the end of follow-up

• Sustained response

  From date of randomization until 2 years or the end of follow-up

• Overall response (OR)

  From date of randomization until 2 years or the end of follow-up

• Complete response (CR)

  From date of randomization until 2 years or the end of follow-up

Study Arms (2)

Cyclosporin plus Steroid

EXPERIMENTAL

Cyclosporin: 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. 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 regimen of steroid 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.

Drug: Cyclosporine Oral Product; Drug: Prednisone

Standard steroid

ACTIVE COMPARATOR

Standard regimen 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.

Drug: Prednisone

Interventions

Cyclosporine Oral Product DRUG

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.

Also known as: Prednisone

Cyclosporin plus Steroid

Prednisone DRUG

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.

Cyclosporin plus Steroid; Standard steroid

Eligibility Criteria

• Age: 18 Years - 80 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Newly-diagnosed, treatment-naive primary ITP;
• Platelet counts \<30×10\^9/L;
• Platelet counts \< 50×10\^9/L and significant bleeding symptoms (WHO bleeding scale 2 or above);
• Willing and able to sign written informed consent.

You may not qualify if:

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

Sponsors & Collaborators

• Peking University People's Hospital: lead

Study Sites (1)

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

Beijing, Beijing Municipality, 100010, China

Location

MeSH Terms

Conditions

Purpura, Thrombocytopenic, Idiopathic

Interventions

Prednisone

Condition Hierarchy (Ancestors)

Purpura, Thrombocytopenic; Purpura; Blood Coagulation Disorders; Hematologic Diseases; Hemic and Lymphatic Diseases; Thrombotic Microangiopathies; Thrombocytopenia; Blood Platelet Disorders; Cytopenia; Hemorrhagic Disorders; Autoimmune Diseases; Immune System Diseases; Hemorrhage; Pathologic Processes; Pathological Conditions, Signs and Symptoms; Skin Manifestations; Signs and Symptoms

Intervention Hierarchy (Ancestors)

Pregnadienediols; Pregnadienes; Pregnanes; Steroids; Fused-Ring Compounds; Polycyclic Compounds

Study Officials

• Xiao-Hui Zhang, MD

  Peking University People's Hospital

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Xiao-Hui Zhang, MD

CONTACT

+8613522338836; zhangxh100@sina.com

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: SINGLE
• Who Masked: OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Vice president of Peking Univeristy Institute of Hematology

Study Record Dates

• First Submitted: July 10, 2022
• First Posted: July 15, 2022
• Study Start: July 20, 2022
• Primary Completion: July 20, 2024
• Study Completion: July 20, 2025
• Last Updated: July 15, 2022

Record last verified: 2022-07

Locations

CN (1)