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

NCT07010237 | Recruiting Phase 2

• 1 other identifier: IIT2025013
• Study Type: interventional
• Target: 65
• Locations: 1 country
• Sites: 1

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.

Conditions

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

Outcome Measures

Primary Outcomes (1)

• Hematologic response rate at 24 weeks post-ATG treatment.

  PR+CR Partial Response (PR) : Independence from transfusions without meeting CR. No Response (NR) : Failure to achieve PR.

  24 weeks

Secondary Outcomes (2)

• Early mortality rate

  12 weeks

• Complete Response

  24 weeks

Study Arms (1)

A-IST

EXPERIMENTAL

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.

Drug: Dose-attenuated IST and Hetrombopag

Interventions

Dose-attenuated IST and Hetrombopag 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.

A-IST

Eligibility Criteria

• Age: 65 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Older Adult (65+)

You may qualify if:

• 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).

You may not qualify if:

• 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.

Sponsors & Collaborators

• Institute of Hematology & Blood Diseases Hospital, China: lead

Study Sites (1)

Red Blood Cell Diseases Center

Tianjin, Tianjin Municipality, China

RECRUITING

MeSH Terms

Interventions

hetrombopag

Study Officials

• Jun Shi, PhD

  Institute of Hematology & Blood Diseases Hospital, CAMS

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Jianping Li, MD

CONTACT

+8613820961539; lijianping@ihcams.ac.cn

Jingyu Zhao, MPH

CONTACT

+8613752253515; zhaojingyu@ihcams.ac.cn

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: April 30, 2025
• First Posted: June 8, 2025
• Study Start: February 1, 2025
• Primary Completion (Estimated): December 31, 2028
• Study Completion (Estimated): July 1, 2029
• Last Updated: June 8, 2025

Record last verified: 2025-02

Locations

CN (1)