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Completed

NCT Number: NCT05996393

CsA+ATG+AVA vs. CsA+AVA for the Treatment of Newly-diagnosed SAA in the Elderly

This is a multicenter, prospective, randonmized study. Our previous retrospective study showed that for SAA patients who were intolerant to ATG, CsA+ eltrombopag (EPAG) had similar efficacy to CsA+ATG+EPAG. Since the action mechanism of AVA and EPAG is not exactly the same, and the metabolic level of the elderly is not the same as that of younger patients, it is unknown whether there are predictive factors of efficacy in the treatment of AVA. We wondered whether CsA+AVA could achieve an efficacy similar to CsA+ATG+AVA in the Elderly. Meanwhile, to explore the predictive factors of efficacy, to find out a safe and effective treatment strategy for the Elderly.

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Key information

Conditions

Aplastic Anemia Anemia Anemia, Aplastic Bone Marrow Diseases Bone Marrow Failure Disorders Hematologic Diseases Hemic and Lymphatic Diseases

Age range

60 year and older

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 4

Primary location

Peking union medical college hospital

Beijing, China

About this study

Aplastic anemia (AA) can be divided into severe AA (SAA) and non-severe AA (NSAA), according to the severity of the disease. For SAA patients with o condition of hematopoietic stem cell transplantation, guideline recommended ciclosporin A (CsA) + anti-thymocyte immunoglobulin (ATG) + thrombopoietin receptor agonists (TPO-RA) as the first-line therapy. Our previous retrospective study showed that for SAA patients who were intolerant to ATG, CsA+ eltrombopag (EPAG) had similar efficacy to CsA+ATG+EPAG. However, the adverse effects such as hepatotoxicity and gasrointestinal disorder limit its application in elderly SAA.Compared with EPAG, AVA has no hepatotoxicity and less other adverse effects, but there was few studies for AA. Our previous study demonstrated that AVA was effective in 55% of refractory AA. Since the action mechanism of AVA and EPAG is not exactly the same, and the metabolic level of the elderly is not the same as that of younger patients, it is unknown whether there are predictive factors of efficacy in the treatment of AVA. Thus, we wondered whether CsA+AVA could achieve an efficacy similar to CsA+ATG+AVA in the Elderly.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

  • Patients with newly diagnosed severe aplastic anemia, aged greater than 60 years.
  • Patients met the diagnostic criteria of severe aplastic anemia (SAA).
  • Complete all screening assessments as outlined in the test protocol.
  • Without or with no more than 1 month treatment of ciclosporine, tacrolimus, glucocortocoid, or TPO-RAs.
  • Agree to sign the informed consent form.

Exclusion criteria

  • Known diagnosis of congenital hematopoietic failure disorders (e.g. Fanconi anemia) and other causes of cytopenia.
  • Patients with uncontrolled bleeding and/or infection despite standard treatment.
  • Patients with previous history of hematopoietic stem cell transplantation.
  • Patients with previous history of thrombosis in 1 year.
  • Patients with concurrent malignancy.
  • Those who are considered unsuitable for enrollment by the investigator.
  • Abnormal renal function: creatinine > 1.2 normal upper limit, albumin < 0.9 normal lower limit, or CLcr < 30 ml/min.
  • Abnormal liver function: transaminase > 2.5 normal upper limit, or tota bilirubin > 2.5 normal upper limit.
  • Patients with severe heart, liver or renal disease.

Treatment and study plan

Ciclosporin

Drug

3-5 mg/kg/d orally, trough concentrations: 100-200 ng/ml

Avatrombopag

Drug

60 mg/d orally, 24 weeks

Anti-Human Thymocyte Immunoglobulin, Rabbit

Drug

rabbit anti-human thymocyte immunoglobulin (r-ATG 3mg/kg/d), intravenously, 5 days;

Primary outcomes

  1. Overall response rate

    Time frame: 6 month

    Proportion of patient who achieved partial response or complete response

Secondary outcomes

  1. Complete response rate

    Time frame: 6 month

    Proportion of patient who achieved complete response (HGB>100g/L, PLT>100×109/L, ANC>1.0×109/L)

  2. Relapse rate

    Time frame: 12 month

    Proportion of patient who relapse

  3. Adverse event rate

    Time frame: 3, 6, 12 month

    Proportion of patient who adverse events

Sponsors and collaborators

Lead sponsor

Peking Union Medical College Hospital

Other

Registry information

Official study title

Ciclosporin + Antithymocyte Immunoglobulin + Avatrombpag Versus Ciclosporin + Avatrombpag for the Treatment of Severe Aplastic Anemia in the Elderly

Important dates

Study start
2023
Primary completion
2025
Study completion
2025
First posted
Aug 18, 2023
Registry last updated
Apr 30, 2025

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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