Treatment of non-severe aplastic anemia in elderly patients using CsA and AVA

A Single-center, Single-arm, Prospective Clinical Study on the Efficacy and Safety of Cyclosporine Combined With Avatrombopag in the Treatment of Non-severe Aplastic Anemia in the Elderly

This study is testing if a combination of two medications, ciclosporin and avatrombopag, can help older adults with non-severe aplastic anemia who can’t use standard treatment feel better and stay safe.

Quick facts

• Phase: Phase 2
• Study type: Interventional
• Enrollment: 30 (estimated)
• Ages: 60 Years to 90 Years
• Sex: All
• Sponsor: Peking Union Medical College Hospital (Academic / other)
• Locations: 1 site (Beijing)
• Trial ID: NCT06004752 on ClinicalTrials.gov

What this trial studies

This study evaluates the efficacy and safety of combining ciclosporin (CsA) with avatrombopag (AVA) in elderly patients over 60 years old who have non-severe aplastic anemia (NSAA) and cannot tolerate anti-thymocyte globulin (ATG) treatment. The aim is to improve hematological response rates while minimizing adverse reactions. Participants will receive the treatment and be monitored for their response and safety outcomes. This phase 2 interventional study focuses on a specific demographic that has limited treatment options.

Who should consider this trial

Good fit: Ideal candidates are elderly patients aged 60 and older with well-defined non-severe aplastic anemia who are intolerant to ATG treatment.

Not a fit: Patients with severe aplastic anemia or those with known congenital aplastic anemia will not benefit from this study.

Why it matters

Potential benefit: If successful, this treatment could provide a safer and effective option for elderly patients with NSAA who cannot tolerate standard therapies.

How similar studies have performed: While the combination of CsA and AVA is a novel approach for this specific patient population, similar studies have shown promise in treating aplastic anemia with alternative therapies.

Eligibility criteria

Inclusion Criteria:

1. 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).
2. Age 60 years or older, male or female.
3. Able to swallow or administer orally.
4. Intolerant or refused anti-thymocyte globulin treatment

6. No previous treatment with cyclosporine, tacrolimus or hormones or treatment for less than 1 month.

7. 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.

Exclusion Criteria:

1. Known congenital AA (such as Fanconi anemia) and other causes of pancytopenia and hematological bone marrow disorders;
2. With paroxysmal nocturnal hemoglobinuria (PNH) clone ≥ 50%
3. With a history of hematopoietic stem cell transplantation.
4. History of thrombosis
5. Patients with underlying cancer who also have malignant tumors or are receiving immunosuppressive therapy.
6. 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;
7. Serious heart, liver and kidney disease.
8. With uncontrolled bleeding and/or infection after standard treatment.
9. Participants considered unsuitable for inclusion by the researchers.

Where this trial is running

Beijing

• Peking union medical college hospital — Beijing, China (Recruiting)

Study contacts

• Study coordinator: Bing Han

Conditions Aplastic Anemia