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

Phase 2

Recruiting

• Conditions: Sever Aplastic Anaemia; Elderly (People Aged 65 or More); Immunosuppressive Treatment
• Interventions: Drug: Dose-attenuated IST and Hetrombopag

• Registration Number: NCT07010237
• Lead Sponsor: Institute of Hematology & Blood Diseases Hospital, China

Brief 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.

Study Timeline

• Status Verified: 2025-02
• Study Start: 2025-02-01
• Study First Submitted: 2025-04-30
• Study First Submitted QC: 2025-06-05
• Last Update Submitted: 2025-06-05
• Study First Posted: 2025-06-08
• Last Update Posted: 2025-06-08
• Primary Completion: 2028-12-31
• Study Completion: 2029-07-01

Recruitment & Eligibility

• Status: RECRUITING
• Sex: All
• Target Recruitment: 65

Inclusion Criteria

• Clinical diagnosis of VSAA/SAA.
• Age ≥65 years.
• Completion of all screening assessments.
• Must be able to swallow tablets.
• 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.

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
A-IST	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.

Primary Outcome Measures

Name	Time	Method
Hematologic response rate at 24 weeks post-ATG treatment.	24 weeks	PR+CR Partial Response (PR) : Independence from transfusions without meeting CR. No Response (NR) : Failure to achieve PR.

Secondary Outcome Measures

Name	Time	Method
Early mortality rate	12 weeks	Early mortality rate within 12weeks
Complete Response	24 weeks	Complete Response (CR) : HGB ≥100 g/L, PLT ≥100×10⁹/L, ANC ≥1.0×10⁹/L.

Trial Locations

Locations (1)

Red Blood Cell Diseases Center; 🇨🇳 Tianjin, Tianjin, China