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

Phase 2

Recruiting

• Conditions: Aplastic Anemia; TPO Receptor Agonists
• Interventions: Drug: standard IST combined with Romiplostim N01

• Registration Number: NCT06613880
• Lead Sponsor: Institute of Hematology & Blood Diseases Hospital, China

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.

Detailed Description

Not available

Basic Information
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Recruitment & Eligibility
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Study & Design
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Trial Locations

Study Timeline

• Status Verified: 2024-09
• Study First Submitted: 2024-09-23
• Study First Submitted QC: 2024-09-23
• Study First Posted: 2024-09-26
• Study Start: 2024-10-24
• Last Update Submitted: 2024-11-29
• Last Update Posted: 2024-12-04
• Primary Completion: 2026-01-31
• Study Completion: 2026-09-30

Recruitment & Eligibility

• Status: RECRUITING
• Sex: All
• Target Recruitment: 48

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.

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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

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Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
p-ALG+Romiplostim N01+CsA	standard IST combined with Romiplostim N01	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.

Primary Outcome Measures

Name	Time	Method
Overall response rate	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 Outcome Measures

Name	Time	Method
Overall response rate	Within 3 months 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).
Time to hematological response	Within 27 weeks post treatment	Duration time was calculated from enrollment to achieve hematological response.
Incidence of adverse events	Within 27 weeks post treatment	Use Common Terminology Criteria for Adverse Events (CTCAE) Version 5 to assess the adverse event

Trial Locations

Locations (1)

Regenerative Medicine Center and Red Blood Cell Disorders Center; 🇨🇳 Tianjin, Tianjin, China