Reduced Dose of Cyclophosphamide Combined With Standard Immunosuppressive Therapy to Treat Severe Aplastic Anemia

Phase 2

Recruiting

• Conditions: Severe Aplastic Anemia; Cyclophosphamide Reduced-dose; Immunosuppressive Therapy
• Interventions: Drug: Reduced dose of cyclophosphamide combined with standard immunosuppressive therapy

• Registration Number: NCT06695741
• Lead Sponsor: Institute of Hematology & Blood Diseases Hospital, China

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 the reduced dose of cyclophosphamide (hypo-CASH) for severe aplastic anemia.

Detailed Description

Not available

Study Timeline

• Status Verified: 2024-11
• Study Start: 2024-11-01
• Study First Submitted: 2024-11-16
• Study First Submitted QC: 2024-11-16
• Last Update Submitted: 2024-11-16
• Study First Posted: 2024-11-19
• Last Update Posted: 2024-11-19
• Primary Completion: 2027-02-01
• Study Completion: 2027-06-30

Recruitment & Eligibility

• Status: RECRUITING
• Sex: All
• Target Recruitment: 75

Inclusion Criteria

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

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Exclusion Criteria

• Previously received immunosuppressive therapy > 4 weeks
• Previously treated with TPO-RA > 4 weeks
• Have an allergy or intolerance to anti-lymphocyte globulin, cyclosporine, herombopag or cyclophosphamide.
• Uncontrolled fungal, bacterial, or viral infection. Hepatitis-associated aplastic anemia is allowed; hepatitis B or C infection is permitted unless it causes severe liver failure.
• Tested positive for HIV or syphilis
• Presence of severe liver, kidney, or heart failure, or other life-threatening comorbidities.
• History of radiotherapy and chemotherapy for malignant solid tumors in recent 5 years
• 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.

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Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
hypo-CASH	Reduced dose of cyclophosphamide combined with standard immunosuppressive therapy	ALG/CsA and herombopag and reduced dose of cyclophosphamide

Primary Outcome Measures

Name	Time	Method
Overall response rate	Within 3 months	Percentage of patients with hematological response. Hematological response includes complete rate, near complete rate(CR), very good partial response(VGPR), good partial response(GPR) and partial response(PR).

Secondary Outcome Measures

Name	Time	Method
Superior response rate	With in 6 months	Percentage of patients with superior response, including CR, VGPR and GPR.
Overall response rate	With in 6 months	Percentage of patients with hematological response. Hematological response includes complete rate, near complete rate(CR), very good partial response(VGPR), good partial response(GPR) and partial response(PR).
Incidence of the adverse event	Within 6 months	Use Common Terminology Criteria for Adverse Events (CTCAE) Version 5 to assess the adverse event
Time to achieve robust superior response	Within 6 months
First time to response	Within 6 months

Trial Locations

Locations (1)

Red Blood Cell Disorders Center and Regenerative Medicine Center; 🇨🇳 Tianjin, Tianjin, China