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
Trial Snapshot
- Phase
- Phase 2
- Status
- Recruiting
- Enrollment
- 48
- Locations
- 1
- Primary Endpoint
- Overall response rate
Study Overview
Brief Summary
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.
Study Design
- Study Type
- Interventional
- Allocation
- Na
- Intervention Model
- Single Group
- Primary Purpose
- Treatment
- Masking
- None
Eligibility Criteria
- Ages
- 12 Years to — (Child, Adult, Older Adult)
- Sex
- All
- Accepts Healthy Volunteers
- No
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
Arms & Interventions
p-ALG+Romiplostim N01+CsA
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.
Intervention: standard IST combined with Romiplostim N01 (Drug)
Outcomes
Primary Outcomes
Overall response rate
Time Frame: Within 27 weeks post treatment
Percentage of patients with hematological response, including complete response (CR) or partial response (PR). Hematological response is evaluated by hemoglobin (Hb), platelet count (PLT) and absolute neutrophil count (ANC).
Secondary Outcomes
- Overall response rate(Within 3 months post treatment)
- Time to hematological response(Within 27 weeks post treatment)
- Incidence of adverse events(Within 27 weeks post treatment)