Standard Immunosuppressive Therapy Combined With Romiplostim N01 as First-line Treatment for Severe Aplastic Anemia

Institute of Hematology & Blood Diseases Hospital, China

Status and phase: Enrolling; Phase 2

Conditions: TPO Receptor Agonists; Aplastic Anemia

Treatments: Drug: standard IST combined with Romiplostim N01

Study type: Interventional

Funder types: Other; Industry

Identifiers

NCT06613880

IIT2024070

Details and patient eligibility

About

This prospective, single center, single-arm study aims to evaluate the efficacy and safety of anti-human T lymphocyte porcine immunoglobulin (p-ALG) in combination with cyclosporine and Romiplostim in treating severe aplastic anemia (SAA). It will include 48 patients with newly diagnosed SAA.

Enrollment: 48 estimated patients

Sex: All

Ages: 12+ years old

Volunteers: No Healthy Volunteers

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

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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48 participants in 1 patient group

p-ALG+Romiplostim N01+CsA

Experimental group

Description:

The maximum screening period was 4 weeks, and after entering the treatment period, the patients were treated with p-ALG combined with cyclosporine and Romiplostim N01. Symptomatic leukocyte elevation, blood transfusion support, and anti-infective therapy are allowed during this period, according to the patient's blood routine. If a patient has a serious adverse event during treatment, it will be treated according to the criteria for suspension and recovery.

Treatment:

Drug: standard IST combined with Romiplostim N01