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

NCT06009965 | Unknown Phase 4

• 1 other identifier: SN-01
• Study Type: interventional
• Target: 210
• Locations: 1 country
• Sites: 1

Brief Summary

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.

Conditions

Aplastic Anemia; CSA; Drug Therapy

Keywords

Aplastic Anemia; CsA; ATG; TPO-RA

Outcome Measures

Primary Outcomes (2)

• Overall response rate

  Overall response rate

  24 weeks after treatment

• 24-month recurrence rate

  .24-month recurrence rate

  96 weeks after treatment

Secondary Outcomes (2)

• Overall response rate

  56 weeks after treatment

• 36-month recurrence rate

  144 weeks after treatment

Study Arms (2)

Severe aplastic anemia group

EXPERIMENTAL

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

Non-severe aplastic anemia group

EXPERIMENTAL

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

Interventions

CsA+ATG+Herombopag DRUG

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.

Also known as: SAA group intervention treatment

Severe aplastic anemia group

CsA+Herombopag DRUG

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.

Also known as: NSAA group intervention treatment

Non-severe aplastic anemia group

Eligibility Criteria

• Age: 18 Years - 75 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Age: 18-75 years old, gender is not limited
• Definite diagnosis of AA
• No HSCT indication or unconditional HSCT
• 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
• Baseline serum transaminase, total bilirubin and serum creatinine were less than 1.5 times the normal value
• Baseline liver and kidney function was less than 1.5 times the normal value
• Eastern Cancer Cooperation Group (ECOG) score status 0-2
• Agree to sign the consent form

You may not qualify if:

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

Sponsors & Collaborators

• Peking Union Medical College Hospital: lead

Study Sites (1)

Peking union medical college hospital

Beijing, China

RECRUITING

MeSH Terms

Conditions

Anemia, Aplastic

Condition Hierarchy (Ancestors)

Anemia; Hematologic Diseases; Hemic and Lymphatic Diseases; Bone Marrow Failure Disorders; Bone Marrow Diseases

Study Officials

• Bing Bing, PhD

  Peking Union Medical College Hospital

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Yali Du, Master

CONTACT

+8615845992396; yali_crazy@126.com

Study Design

• Study Type: interventional
• Phase: phase 4
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Professor

Study Record Dates

• First Submitted: August 6, 2023
• First Posted: August 24, 2023
• Study Start: January 20, 2023
• Primary Completion: January 30, 2025
• Study Completion: January 31, 2025
• Last Updated: August 24, 2023

Record last verified: 2023-08

Data Sharing

• IPD Sharing: Will share
• Shared Documents: STUDY PROTOCOL, SAP, ICF, CSR
• Time Frame: 10 years
• Access Criteria: email request

Locations

CN (1)