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

Severe Aplastic Anemia Clinical Trial

Official title:

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

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT05975996
• Other study ID #: ALG-HPAG-CTX2023
• Status: Recruiting
• Phase: Phase 2
• Start date: July 10, 2023
• Est. completion date: June 30, 2027

Study information

• Verified date: July 2023
• Source: Institute of Hematology & Blood Diseases Hospital
• Contact: Liwei Fang, MD
• Phone: 13752253515
• Email: fangliwei[@]ihcams.ac.cn
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

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

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 43
• Est. completion date: June 30, 2027
• Est. primary completion date: January 31, 2027
• Accepts healthy volunteers: No
• Gender: All
• Age group: 12 Years and older

Eligibility

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 eltrombopag 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.

Related Conditions & MeSH terms

• Anemia
• Anemia, Aplastic
• Severe Aplastic Anemia

Intervention

Drug:
• Cyclophosphamide added to standard immunosuppressive therapy with eltrombopag
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. Eltrombopag 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.

Locations

Country	Name	City	State
China	Regenerative Medicine Center	Tianjin	Tianjin

Sponsors (1)

Lead Sponsor	Collaborator
Institute of Hematology & Blood Diseases Hospital

Country where clinical trial is conducted

China, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Overall response rate	Percentage of patients with hematological response. Hematological response includes complete rate, near complete rate(CR), very good partial response(VGPR), Meaningful partial response(mPR) and partial response(PR).	Within 3 months
Secondary	Robust response	Percentage of patients with robust response, including CR, near CR, VGPR and mPR.	Within 3 months
Secondary	All-cause mortality		Within 3 months
Secondary	Time to achieve robust hematological response		Within 6 months
Secondary	Incidence of the adverse event	Use Common Terminology Criteria for Adverse Events (CTCAE) Version 5 to assess the adverse event	Within 6 months