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

Phases

Phase 2

Study type

Interventional

Source

ClinicalTrials.gov

Registry ID

NCT07010237

Enrollment

Registered

2025-06-08

Start date

2025-02-01

Completion date

2029-07-01

Last updated

2025-06-08

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

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

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.

Interventions

DRUGDose-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.

Study design

Allocation

Intervention model

SINGLE_GROUP

Primary purpose

TREATMENT

Masking

NONE

Eligibility

Sex/Gender

ALL

Age

65 Years to No maximum

Healthy volunteers

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.

Design outcomes

Primary

Measure	Time frame	Description
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

Measure	Time frame	Description
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.

Countries

China

Contacts

Primary ContactJianping Li, MD

lijianping@ihcams.ac.cn+8613820961539

Backup ContactJingyu Zhao, MPH

zhaojingyu@ihcams.ac.cn+8613752253515

Outcome results

None listed

Source: ClinicalTrials.gov · Data processed: Feb 4, 2026