NCT07010237

Dose-attenuated IST and Hetrombopag in Elderly (≥65 Years) Patients With Severe Aplastic Anemia

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

• Status: Recruiting
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 65 (estimated)
• Sponsor: Institute of Hematology & Blood Diseases Hospital, China · Academic / Other
• Sex: All
• Age: 65 Years
• Healthy volunteers: Not accepted

Summary

This is a prospetive,Single-Center, Single-Arm, Phase IIB Clinical Study.This study aims to evaluate the efficacy and safety of dose-attenuated IST combined with Hetrombopag in elderly patients (≥65 years) with VSAA/SAA.

Detailed description

Design : Prospective, single-center, single-arm, open-label Phase IIB trial. Intervention : P-ATG : 15 mg/kg/day IV ×5 days. Cyclosporine : 3 mg/kg/day (adjusted to trough 100-200 μg/L). Hetrombopag : 15 mg daily. Follow-Up : Weekly for 24 weeks, with bone marrow evaluation at 24 weeks. Inclusion Criteria : Confirmed diagnosis of VSAA/SAA. Age ≥65 years. Completion of all screening assessments. Ability to swallow oral medication. Signed informed consent (by patient or legal guardian if patient is incapacitated). Exclusion Criteria : Clonal cytogenetic abnormalities (excluding isolated -Y or +8). Prior treatment with ATG/high-dose cyclophosphamide. Prior cyclosporine/tacrolimus use \>12 months. Prior TPO-RA therapy \>3 months. Uncontrolled malignancies or conditions contraindicating ATG. Severe organ dysfunction (e.g., creatinine ≥177 μmol/L). Investigator judgment of unsuitability.

Conditions

• Sever Aplastic Anaemia
• Elderly (People Aged 65 or More)
• Immunosuppressive Treatment

Interventions

Type	Name	Description
DRUG	Dose-attenuated IST and Hetrombopag	Porcine-ATG : 15 mg/kg/day IV ×5 days. Cyclosporine : 3-5 mg/kg/day (adjusted to trough 100-200 μg/L). Hetrombopag: 15 mg daily for 24weeks.

Timeline

• Start date: 2025-02-01
• Primary completion: 2028-12-31
• Completion: 2029-07-01
• First posted: 2025-06-08
• Last updated: 2025-06-08

Locations

1 site across 1 country: China