Efficacy and Safety of CsA+AVA in the Treatment of NSAA in the Elderly

NCT06004752 | Unknown Phase 2

• 1 other identifier: Avatrombopag-2
• Study Type: interventional
• Target: 30
• Locations: 1 country
• Sites: 1

Brief Summary

For elderly patients who cannot tolerate anti-thymocyte globulin (ATG) treatment, the addition of avatrombopag (AVA), which has a slight adverse reaction, can theoretically improve the hematological response rate in elderly patients with non-severe aplastic anemia (NSAA) without significantly increasing adverse reactions. Based on this, this study treated NSAA patients older than 60 with AVA combined with CsA to evaluate the hematological response rate and safety of AVA in the elderly who could not tolerate ATG therapy.

Conditions

Aplastic Anemia

Outcome Measures

Primary Outcomes (1)

• ORR at 6 Months

  Overall Response Rate (ORR) Defined as the Number of Participants Who Met the Criteria of Either Complete Response (CR) or Partial Response (PR)

  6 months

Secondary Outcomes (5)

• Changes in Haemoglobin in the Absence of Red Blood Cells Transfusion

  6 months

• Changes in Platelet in the Absence of Platelet Transfusion

  6 months

• Percentage of patients with clonal evolution to myelodysplasia, PNH, acute leukemia

  12 months

• ORR at 1, 2, 3 Months and at the end of follow-up

  1, 2, 3 Months, last follow-up

• Relapse rate

  6 months, last follow-up

Study Arms (1)

treatment group

EXPERIMENTAL

Cyclosporine: 3mg/kg/d 3-5mg/kg bid was administered to maintain the trough concentration at 100-200 μg/ml; Avatrombopag: The dosage was 40\~60mg orally, once a day, and the dosage was adjusted according to the subject's platelet count. The drug was administered for 24 weeks (6 months).

Drug: Ciclosporin; Drug: Avatrombopag

Interventions

Ciclosporin DRUG

Cyclosporine was taken orally at 3-5mg/kg bid was administered to maintain the trough concentration at 100-200 μg/ml

treatment group

Avatrombopag DRUG

Avatrombopag was administrated at 40 mg/d and increased by 20 mg/d during the biweekly follow-up if platelet was not 20 × 109/L higher than baseline. AVA dose was gradually reduced if PLT ≥ 150 × 109/L. The maximum dose was 60 mg/d, while the minimum was 20 mg/week.

treatment group

Eligibility Criteria

• Age: 60 Years - 90 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Elderly patients with well-defined NSAA anemia who meet the diagnostic criteria for aplastic anemia (AA) but do not meet the diagnostic criteria for severe aplastic anemia (SAA).
• Age 60 years or older, male or female.
• Able to swallow or administer orally.
• Intolerant or refused anti-thymocyte globulin treatment
• \. No previous treatment with cyclosporine, tacrolimus or hormones or treatment for less than 1 month.
• \. No prior treatment with thrombopoietin (TPO) receptor agonists (including eltrombopag, herombopag, romiplostim, etc.) 8. Informed consent must be signed before the start of all specific research procedures. Considering the patient's condition, if the patient's signature is not conducive to the treatment of the disease, the informed consent shall be signed by the patient's immediate family.

You may not qualify if:

• Known congenital AA (such as Fanconi anemia) and other causes of pancytopenia and hematological bone marrow disorders;
• With paroxysmal nocturnal hemoglobinuria (PNH) clone ≥ 50%
• With a history of hematopoietic stem cell transplantation.
• History of thrombosis
• Patients with underlying cancer who also have malignant tumors or are receiving immunosuppressive therapy.
• Baseline creatine levels greater than twice of the upper limit of normal (ULN) and/or alanine aminotransferase (ALT) greater than 2.5 times of the ULN;
• Serious heart, liver and kidney disease.
• With uncontrolled bleeding and/or infection after standard treatment.

Sponsors & Collaborators

• Peking Union Medical College Hospital: lead

Study Sites (1)

Peking union medical college hospital

Beijing, China

RECRUITING

MeSH Terms

Conditions

Anemia, Aplastic

Interventions

Cyclosporine; avatrombopag

Condition Hierarchy (Ancestors)

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

Intervention Hierarchy (Ancestors)

Cyclosporins; Peptides, Cyclic; Macrocyclic Compounds; Polycyclic Compounds; Peptides; Amino Acids, Peptides, and Proteins

Central Study Contacts

Bing Han

CONTACT

+861069151235; hanbing_li@sina.com

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Professor

Study Record Dates

• First Submitted: August 16, 2023
• First Posted: August 22, 2023
• Study Start: August 17, 2023
• Primary Completion: August 1, 2024
• Study Completion: August 1, 2025
• Last Updated: December 8, 2023

Record last verified: 2023-12

Data Sharing

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

Locations

CN (1)