Combining standard immunosuppressive therapy with Romiplostim for severe aplastic anemia treatment

A Prospective, Single Center, Single-arm Study on the Efficacy and Safety of Standard Immunosuppressive Therapy Combined With Romiplostim N01 as First-line Treatment for Severe Aplastic Anemia

This study is testing if adding Romiplostim to standard treatments can help people with newly diagnosed severe aplastic anemia feel better and improve their health.

Quick facts

• Phase: PHASE2
• Study type: Interventional
• Enrollment: 48 (estimated)
• Ages: 12 Years and up
• Sex: All
• Sponsor: Institute of Hematology & Blood Diseases Hospital, China (other)
• Locations: 1 site (Tianjin, Tianjin Municipality)
• Trial ID: NCT06613880 on ClinicalTrials.gov

What this trial studies

This interventional study evaluates the efficacy and safety of combining anti-human T lymphocyte porcine immunoglobulin (p-ALG) with cyclosporine and Romiplostim in patients with newly diagnosed severe aplastic anemia (SAA). It is a single-center, single-arm study that aims to enroll 48 patients. The approach focuses on enhancing the treatment outcomes for SAA by utilizing a combination of established immunosuppressive therapies and a novel TPO receptor agonist. The study will assess both the effectiveness and safety of this combined treatment regimen.

Who should consider this trial

Good fit: Ideal candidates include individuals aged 12 years and older with a diagnosis of severe or very severe aplastic anemia who are not suitable for allogeneic hematopoietic stem cell transplantation.

Not a fit: Patients with a history of thromboembolic disease, uncontrolled infections, or significant comorbidities may not benefit from this study.

Why it matters

Potential benefit: If successful, this treatment could significantly improve the outcomes for patients suffering from severe aplastic anemia.

How similar studies have performed: While the combination of therapies is innovative, similar approaches in treating aplastic anemia have shown promise, suggesting potential for success.

Eligibility criteria

Inclusion Criteria:

* Age ≥ 12 years
* Diagnosis of severe or very severe aplastic anemia
* Patients are unconditionally or unacceptable undergoing allogeneic hematopoietic stem cell transplantation
* ECOG performance status ≤2
* Willing and able to comply with the requirements for this study and written informed consent.

Exclusion Criteria:

* Received \> 4 weeks of TPO-RA drug before treatment
* Received \> 4 weeks of immunosuppressive therapy before treatment
* History of thromboembolic disease
* Intolerance to Romiplostim N01 or cyclosporine
* Allergy to ALG
* Presence of uncontrolled active infection
* Presence of uncontrolled hypertension (≥140/90mmHg), diabetes mellitus (fasting blood glucose≥ 7.0 mmol/L or random blood glucose ≥ 11.1 mmol/L)
* Abnormal liver and kidney function: Aspartate Transaminase (AST) or Alanine Transaminase (ALT) \> 3 ULN, creatinine ≥ 2.5 ULN
* History of chemoradiotherapy for malignant solid tumors
* History of other systemic serious illnesses
* Females who are pregnant/lactating or need pregnancy
* Patients considered to be ineligible for the study by the investigator for reasons other than the above

Where this trial is running

Tianjin, Tianjin Municipality

• Regenerative Medicine Center and Red Blood Cell Disorders Center — Tianjin, Tianjin Municipality, China (RECRUITING)

Study contacts

• Principal investigator: Jun Shi, PhD — Institute of Hematology & Blood Diseases Hospital, China
• Study coordinator: Lele Zhang, PhD

Conditions: Aplastic Anemia, TPO Receptor Agonists, Severe aplastic anemia, Romiplostim N01