NCT05660785

Brief Summary

This is a prospective, multicenter, single-arm, phase 2 trial. The aim of this study is to evaluate the efficacy and safety of herombopag combined with cyclosporine for patients with non severe aplastic anemia (NSAA).

Trial Health

87
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
54

participants targeted

Target at P25-P50 for phase_2

Timeline
Completed

Started Dec 2022

Geographic Reach
1 country

4 active sites

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

November 23, 2022

Completed
8 days until next milestone

Study Start

First participant enrolled

December 1, 2022

Completed
20 days until next milestone

First Posted

Study publicly available on registry

December 21, 2022

Completed
2.1 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

January 20, 2025

Completed
8 days until next milestone

Study Completion

Last participant's last visit for all outcomes

January 28, 2025

Completed
Last Updated

August 5, 2025

Status Verified

January 1, 2025

Enrollment Period

2.1 years

First QC Date

November 23, 2022

Last Update Submit

July 31, 2025

Conditions

Non Severe Aplastic AnemiaUntreated

Outcome Measures

Primary Outcomes (1)

  • Overall response rate

    Percentage of patients with hematological response. Hematological response is evaluated by hemoglobin, platelet and neutrophil count in the routine blood test.

    24 weeks

Secondary Outcomes (6)

  • Robust response rate

    24 weeks

  • Proportion of patients with abnormal karyotype changes

    Baseline and 24 weeks

  • Time duration for patients achieving hematological response

    A minimum of 2 years of planned follow-up

  • Change of the health-related quality of life

    Baseline and 24 weeks

  • Incidence of the adverse event

    24 weeks

  • +1 more secondary outcomes

Study Arms (1)

CsA + Herombopag

EXPERIMENTAL

Herombopag combined with cyclosporine

Drug: Herombopag

Interventions

HerombopagDRUG

Hetrombopag is a TPO receptor agonist approved in China in 2021 for idiopathic thrombocytopenic purpura (ITP) and second-line severe aplastic anemia (SAA). Indications of chemotherapy-induced thrombocytopenia (CIT), pediatric/juvenile ITP and naive severe aplastic anemia are under development. Hetrombopag was granted Orphan Drug Designation by FDA for the treatment of CIT. Cyclosporine A is a calcineurin inhibitor, which has an effect on reducing T-cell proliferation and activation, can reverse pancytopenia and alleviate transfusion requirements in NSAA.

Also known as: Cyclosporine A
CsA + Herombopag

Eligibility Criteria

Age18 Years+
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

  • Willing and able to comply with the requirements for this study and written informed consent.
  • Male or female age ≥ 18 years
  • Diagnosis of untreated non severe aplastic anemia.
  • Platelet counts \< 50 x 10\^9/L at least 2 times consecutively (time interval ≥ 1 week)

You may not qualify if:

  • Receive immunosuppressive therapy more than 4 weeks before enrollment
  • Treatment with TPO-RA within 1 week before enrollment
  • Inherited bone marrow failure syndromes
  • Bone marrow fibrosis grade ≥ 2
  • The presence of hemolytic PNH clone
  • The presence of clonal karyotypic abnormalities (del(20q), +8 and -Y are not included in this category)
  • Previously treated with TPO-RA ≥ 4 weeks
  • Previously received immunosuppressive therapy ≥ 12 weeks
  • Ferritin \> 1000 ng/ml (The increased level of Ferritin led by infection is not included in this category)
  • Have an allergy to eltrombopag or any other part of this medicine.
  • History of radiotherapy and chemotherapy for malignant solid tumors
  • Cytopenia caused by other non-hematologic diseases, including liver cirrhosis, active rheumatic connective tissue disease, and persistence of infectious diseases, etc
  • Abnormal liver function: ALT or AST \> 3 ULN, or TBil \> 1.5 ULN after treatment.
  • Abnormal kidney function: Creatinine clearance \< 30 ml/min, or serum creatinine (sCr) \>1.5 ULN
  • Patients with diabetic nephropathy, neuropathy, or eye disease
  • +6 more criteria

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (4)

Tangshan Central Hospital

Tangshan, Hebei, China

Location

Zhoukou Central Hospital

Zhoukou, Henan, China

Location

The Second Affilated Hospital of Shandong First Medical University

Tai’an, Shandong, China

Location

Regenerative Medicine Center

Tianjin, Tianjin Municipality, China

Location

MeSH Terms

Interventions

Cyclosporine

Intervention Hierarchy (Ancestors)

CyclosporinsPeptides, CyclicMacrocyclic CompoundsPolycyclic CompoundsPeptidesAmino Acids, Peptides, and Proteins

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

November 23, 2022

First Posted

December 21, 2022

Study Start

December 1, 2022

Primary Completion

January 20, 2025

Study Completion

January 28, 2025

Last Updated

August 5, 2025

Record last verified: 2025-01

Locations

CN(4)