Cyclophosphamide Added to Standard Immunosuppressive Therapy With Herombopag as Front-line Therapy in Patients With Severe Aplastic Anemia

NCT ID: NCT05975996

Last Updated: 2024-09-25

Basic Information

• Recruitment Status: ACTIVE_NOT_RECRUITING
• Clinical Phase: PHASE2
• Total Enrollment: 43 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2023-07-10
• Study Completion Date: 2025-12-31

Brief Summary

This is a prospective, single-center, single-arm, phase 2 study. This study aims to evaluate the efficacy and safety of Anti-lymphocyte globulin plus herombopag in combination with moderate-dose cyclophosphamide for severe aplastic anemia.

Conditions

Severe Aplastic Anemia

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

Experiemental

ALG/CsA(Cyclosporine) and herombopag and moderate-dose cyclophosphamide

Group Type: EXPERIMENTAL

Cyclophosphamide added to standard immunosuppressive therapy with herombopag

Intervention Type: DRUG

Severe aplastic anemia patients will receive a daily dose of Anti-lymphocyte globulin (25mg/kg) for the initial five days at the beginning of the treatment. Cyclosporine will be administered daily at a dosage of 3-5mg/kg. Herombopag will be administered daily at a dosage of 15mg starting from the first day of treatment and continuing for a duration of six months. The moderate-dose cyclophosphamide (20mg/kg) will be administered on days 29-30 and days 43-44.

Interventions

Cyclophosphamide added to standard immunosuppressive therapy with herombopag

Severe aplastic anemia patients will receive a daily dose of Anti-lymphocyte globulin (25mg/kg) for the initial five days at the beginning of the treatment. Cyclosporine will be administered daily at a dosage of 3-5mg/kg. Herombopag will be administered daily at a dosage of 15mg starting from the first day of treatment and continuing for a duration of six months. The moderate-dose cyclophosphamide (20mg/kg) will be administered on days 29-30 and days 43-44.

Intervention Type: DRUG

Eligibility Criteria

Inclusion Criteria

• Male or female age ≥ 12 years
• Subject has a diagnosis of naïve severe or very severe aplastic anemia
• ECOG performance status ≤2
• Unwilling or unable to receive allogeneic hematopoietic stem cell transplantation.
• Willing and able to comply with the requirements for this study and written informed consent.

Exclusion Criteria

• Previously received immunosuppressive therapy > 4 weeks
• Previously treated with TPO-RA > 4 weeks
• Have an allergy or intolerance to either herombopag or cyclophosphamide.
• Have an allergy to ALG
• Uncontrolled systemic fungal, bacterial, or viral infection
• Poorly controlled hypertension (≥140/90mmHg) or diabetes (a fasting plasma glucose concentration ≥7.0mmol/L or a random venous plasma glucose concentration ≥11.1mmol/L)
• Abnormal liver or kidney function: ALT or AST >3 ULN, or serum creatinine (sCr)≥ 2.5 ULN.
• History of radiotherapy and chemotherapy for malignant solid tumors
• Combined with other serious disorders
• Pregnant or breast-feeding patients
• Patients considered to be ineligible for the study by the investigator for reasons other than the above.

• Minimum Eligible Age: 12 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Institute of Hematology & Blood Diseases Hospital, China

OTHER

Sponsor Role: lead

Responsible Party

Fang Liwei

Associate Chief Physician of Regenerative Medical Center

Responsibility Role: PRINCIPAL_INVESTIGATOR

Principal Investigators

Liwei Fang, MD

Role: PRINCIPAL_INVESTIGATOR

Institute of Hematology & Blood Diseases Hospital, China

Locations

Regenerative Medicine Center

Tianjin, Tianjin Municipality, China

Countries

China

Other Identifiers

IIT2023038

Identifier Type: -

Identifier Source: org_study_id