Actively Recruiting
Dose-attenuated IST and Hetrombopag in the Treatment of Elderly (≥65 Years) Patients With Very Severe/Severe Aplastic Anemia: A Single-Center, Single-Arm, Phase IIB Clinical Study on Efficacy and Safety
Led by Institute of Hematology & Blood Diseases Hospital, China · Updated on 2025-06-08
65
Participants Needed
1
Research Sites
26 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
Researchers are conducting a prospective, single-center, single-arm, open-label Phase IIB clinical trial to evaluate the safety and effectiveness of dose-attenuated immunosuppressive therapy (IST) combined with Hetrombopag in elderly patients aged 65 years or older diagnosed with very severe aplastic anemia (VSAA) or severe aplastic anemia (SAA). This study focuses on this older population to better understand treatment options for these serious blood disorders. The treatment involves receiving Porcine antithymocyte globulin (P-ATG) intravenously at 15 mg/kg per day for 5 days, along with oral cyclosporine at 3 to 5 mg/kg per day adjusted to maintain specific blood levels, and Hetrombopag at 15 mg daily for 24 weeks. Participants will be monitored weekly for 24 weeks, including a bone marrow evaluation at the end of this period to assess response. During the study, participants will undergo regular follow-up visits each week for six months to track their blood health and treatment effects. Researchers will measure the hematologic response rate at 24 weeks after treatment and monitor early mortality at 12 weeks. The study also observes complete response rates at 24 weeks. Safety and efficacy will be carefully assessed throughout the study to understand how well this treatment approach works in elderly patients.
CONDITIONS
Brief Title
Dose-attenuated IST and Hetrombopag in Elderly (≥65 Years) Patients With Severe Aplastic Anemia
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Clinical diagnosis of very severe or severe aplastic anemia (VSAA/SAA)
- Age 65 years or older
- Completion of all screening assessments
- Ability to swallow oral medication
- Signed informed consent by patient or legal guardian if patient is incapacitated
You will not qualify if you...
- Clonal cytogenetic abnormalities except isolated -Y or +8
- Prior treatment with antithymocyte globulin (ATG) or high-dose cyclophosphamide
- Prior use of cyclosporine or tacrolimus for more than 12 months
- Prior thrombopoietin receptor agonist (TPO-RA) therapy for more than 3 months
- Uncontrolled malignancies or conditions that prevent use of ATG
- Severe organ dysfunction such as creatinine level ≥177 µmol/L
- Investigator judgment that the patient is unsuitable for the study
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Duration - 24 weeks
Participants receive dose-attenuated immunosuppressive therapy including Porcine-ATG for 5 days, cyclosporine daily with dose adjustments, and daily Hetrombopag for up to 24 weeks.
Weekly visits for up to 24 weeks
Duration - Up to 24 weeks during treatment
Participants are monitored weekly for safety and efficacy, including a bone marrow evaluation at 24 weeks post-treatment.
Weekly visits for up to 24 weeks
Trial Site Locations
Total: 1 location
1
Red Blood Cell Diseases Center
Tianjin, Tianjin Municipality, China
Actively Recruiting
Research Team
J
Jianping Li, MD
J
Jingyu Zhao, MPH
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SINGLE_GROUP
Primary Purpose
TREATMENT
Number of Arms
1
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