Efficacy of IST Combined With TPO-RA in the Treatment of AA and Establishment of a Recurrence Prediction System

August 20, 2023 updated by: Bing Han, Peking Union Medical College Hospital

Establishment of an Efficacy and Relapse Prediction System for Immunosuppressants Combined With Thrombopoietin Receptor Agonists in the Treatment of Aplastic Anemia

In this study, investigators intend to prospectively study treatment-naive AA patients (including SAA and NSAA) who are non-transplant candidates in northern China. Patients with SAA receive ATG+CsA+Herombopag, and patients with NSAA receive CsA+ Herombopag. Investigators explored possible indicators of participants' predictive efficacy and built predictive models. After the participants achieved response, they used a tapering regimen, observed relapse and clonal evolution, and developed a predictive model of relapse.

Study Overview

Status

Recruiting

Conditions

Intervention / Treatment

Detailed Description

1) Patients in the SAA group: CsA: 3-5mg/kg/day, monitor the trough concentration every month, and maintain the trough concentration at 100-200ng/ml. ATG: rabbit anti-thymocyte immunoglobulin (r-ATG) 3mg/kg/d×5 days, or porcine anti-lymphocyte immunoglobulin ((p-ATG) 25mg/kg/d×5 days; TPO-RA: Herombopag starts at 7.5 mg qd, monitor the blood picture every 2 weeks, if the effect is not good, add 1 tablet every 2 weeks, up to 6 tablets (15 mg) qd.

Patients in the NSAA group: CsA: 3-5mg/kg/day, monitor the trough concentration every month, and maintain the trough concentration at 100-200ng/ml. TPO-RA: start with 7.5mg qd of Herombopag, monitor the blood picture every 2 weeks, if the effect is not good, add 1 tablet every 2 weeks, up to 6 tablets (15mg) qd.

(2) Those who are effective after 6 months of treatment continue to receive sufficient CsA treatment for at least 1.5 years, and then slowly reduce the dose until the end of the reduction. Those who fail to withdraw from the clinical trial.

(3) Participants who achieve CR or PR after treatment, but when the curative effect does not increase after 3 months of maintenance treatment, start to reduce the dose of Herombopag, and reduce one tablet every 3 months until the reduction stops. If recurrence occurs, recover Up to the last dose or even increase the dose, after reaching the best curative effect, reduce one tablet every 3 months until the reduction stops, and those who cannot recover are defined as relapse.

Study Type

Interventional

Enrollment (Estimated)

210

Phase

  • Phase 4

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Locations

  • China
      • Beijing, China
        • Recruiting
        • Peking Union Medical College Hospital
        • Contact:

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Adult
  • Older Adult

Accepts Healthy Volunteers

No

Description

Inclusion Criteria:

  1. Age: 18-75 years old, gender is not limited
  2. Definite diagnosis of AA
  3. No HSCT indication or unconditional HSCT
  4. SAA/VSAA patients were willing to accept ATG+CsA+TPO-RA treatment, NSAA was willing to accept CsA+TPO-RA treatment, and were willing to follow up regularly
  5. Baseline serum transaminase, total bilirubin and serum creatinine were less than 1.5 times the normal value
  6. Baseline liver and kidney function was less than 1.5 times the normal value
  7. Eastern Cancer Cooperation Group (ECOG) score status 0-2
  8. Agree to sign the consent form

Exclusion Criteria:

  1. Congenital AA
  2. Cytogenetic evidence of clonal hematologic bone marrow disease
  3. PNH clone ≥50%
  4. Allergic to ATG, cyclosporine and hexapopal in the past
  5. Uncontrolled infection or bleeding at enrollment
  6. Received hematopoietic stem cell transplantation (HSCT) before enrollment
  7. Any concomitant malignancy within 5 years, except for local skin basal cell carcinoma
  8. A history of thromboembolic events, myocardial infarction, or stroke (including antiphospholipid antibody syndrome); Currently using anticoagulants
  9. Pregnant or lactating women
  10. Situations considered unsuitable for clinical research by other researchers

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Non-Randomized
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: Severe aplastic anemia group
CsA: 3-5 mg/kg/day, monitor trough concentration monthly, maintain trough concentration at 100-200 ng/ml.ATG: rabbit anti-thymocyte immunoglobulin (r-ATG) 3 mg/kg/d x 5 days or porcine anti-lymphocyte immunoglobulin ((p-ATG) 25 mg/kg/d x 5 days.TPO-RA: Heptapepto-Papa 7.5 mg qd to start. Monitor blood every 2 weeks and if ineffective, increase by 1 tablet every 2 weeks up to a maximum of 6 tablets (15mg) qd.
Drug: CsA+ATG+Herombopag
CsA: 3-5 mg/kg/day, monitor trough concentration monthly, maintain trough concentration at 100-200 ng/ml.ATG: rabbit anti-thymocyte immunoglobulin (r-ATG) 3 mg/kg/d x 5 days or porcine anti-lymphocyte immunoglobulin ((p-ATG) 25 mg/kg/d x 5 days.TPO-RA: Heptapepto-Papa 7.5 mg qd to start. Monitor blood every 2 weeks and if ineffective, increase by 1 tablet every 2 weeks up to a maximum of 6 tablets (15mg) qd.
Other Names:
  • SAA group intervention treatment
Experimental: Non-severe aplastic anemia group
CsA: 3-5 mg/kg/day, monitor trough concentrations monthly, maintain trough concentrations at 100-200 ng/ml.TPO-RA: Start with Hetropoxyphene 7.5 mg qd, monitor blood every 2 weeks, and if ineffective, increase by 1 tablet every 2 weeks up to a maximum of 6 tablets (15 mg) qd.
Drug: CsA+Herombopag
CsA: 3-5 mg/kg/day, monitor trough concentrations monthly, maintain trough concentrations at 100-200 ng/ml.TPO-RA: Start with Hetropoxyphene 7.5 mg qd, monitor blood every 2 weeks, and if ineffective, increase by 1 tablet every 2 weeks up to a maximum of 6 tablets (15 mg) qd.
Other Names:
  • NSAA group intervention treatment

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Overall response rate
Time Frame: 24 weeks after treatment
Overall response rate
24 weeks after treatment
24-month recurrence rate
Time Frame: 96 weeks after treatment
.24-month recurrence rate
96 weeks after treatment

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Overall response rate
Time Frame: 56 weeks after treatment
Overall response rate
56 weeks after treatment
36-month recurrence rate
Time Frame: 144 weeks after treatment
36-month recurrence rate
144 weeks after treatment

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Peking Union Medical College Hospital

Investigators

  • Principal Investigator: Bing Bing, PhD, Peking Union Medical College Hospital

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

January 20, 2023

Primary Completion (Estimated)

January 30, 2025

Study Completion (Estimated)

January 31, 2025

Study Registration Dates

First Submitted

August 6, 2023

First Submitted That Met QC Criteria

August 20, 2023

First Posted (Actual)

August 24, 2023

Study Record Updates

Last Update Posted (Actual)

August 24, 2023

Last Update Submitted That Met QC Criteria

August 20, 2023

Last Verified

August 1, 2023

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • SN-01

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

YES

IPD Plan Description

individual participant data would be accepted upon request

IPD Sharing Time Frame

10 years

IPD Sharing Access Criteria

email request

IPD Sharing Supporting Information Type

  • STUDY_PROTOCOL
  • SAP
  • ICF
  • CSR

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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