Reduced Dose of Cyclophosphamide Combined With Standard Immunosuppressive Therapy to Treat Severe Aplastic Anemia
For patients and families
In plain language
An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.
- What is being studied
- The protocol lists: Reduced dose of cyclophosphamide combined with standard immunosuppressive therapy.
- Who it may be relevant to
- Registry conditions: Severe Aplastic Anemia, Cyclophosphamide Reduced-dose, Immunosuppressive Therapy. Basic parameters: from 12 years · All.
- What needs checking
- Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
- Where it takes place
- China
- Next step
- Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →
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Official title
Reduced Dose of Cyclophosphamide Combined With Standard Immunosuppressive Therapy as Front-line Therapy in Patients With Severe Aplastic Anemia
Overview
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.
Interventions
- Drug Reduced dose of cyclophosphamide combined with standard immunosuppressive therapy
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. Cyclophosphamide (20mg/kg) will be administered on days 15-16.
Primary outcome measures
- Overall response rate [Time frame: Within 3 months]
Secondary outcome measures (5)
- Superior response rate [Time frame: With in 6 months]
- Overall response rate [Time frame: With in 6 months]
- Incidence of the adverse event [Time frame: Within 6 months]
- Time to achieve robust superior response [Time frame: Within 6 months]
- First time to response [Time frame: Within 6 months]
Eligibility criteria
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.
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.
Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.
Healthy volunteers: No
Study design
- Allocation
- N/A
- Model
- Single group
- Masking
- Open label
- Primary purpose
- Treatment
Study locations
China · 1 center
- Red Blood Cell Disorders Center and Regenerative Medicine Center — Tianjin
Identifiers
NCT: NCT06695741 · Hypo-CASH-2024