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

NCT ID: NCT07010237

Last Updated: 2025-06-08

Basic Information

• Recruitment Status: RECRUITING
• Clinical Phase: PHASE2
• Total Enrollment: 65 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2025-02-01
• Study Completion Date: 2029-07-01

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.

Conditions

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

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

A-IST

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.

Group Type: EXPERIMENTAL

Dose-attenuated IST and Hetrombopag

Intervention Type: DRUG

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.

Interventions

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.

Intervention Type: DRUG

Eligibility Criteria

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.

• Minimum Eligible Age: 65 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Institute of Hematology & Blood Diseases Hospital, China

OTHER

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Jun Shi, PhD

Role: PRINCIPAL_INVESTIGATOR

Institute of Hematology & Blood Diseases Hospital, CAMS

Locations

Red Blood Cell Diseases Center

Tianjin, Tianjin Municipality, China

Site Status: RECRUITING

Countries

China

Central Contacts

Jianping Li, MD

Role: CONTACT

lijianping@ihcams.ac.cn

+8613820961539

Jingyu Zhao, MPH

Role: CONTACT

zhaojingyu@ihcams.ac.cn

+8613752253515

Facility Contacts

Jianping Li, MD

Role: primary

lijianping@ihcams.ac.cn

13820961539

Other Identifiers

IIT2025013

Identifier Type: -

Identifier Source: org_study_id