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

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 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 Overview

• Status: Recruiting
• Conditions: Aplastic Anemia; TPO Receptor Agonists
• Intervention / Treatment: Drug: standard IST combined with Romiplostim N01

• Study Type: Interventional
• Enrollment (Estimated): 48
• Phase: Phase 2

Contacts and Locations

• Study Contact: Name: Lele Zhang, PhD; Phone Number: ‭15811139278‬; Email: zhanglele@ihcams.ac.cn
• Study Contact Backup: Name: Meili Ge, PhD

Study Locations

• China
  • Tianjin
    • Tianjin, Tianjin, China
      • Recruiting
      • Regenerative Medicine Center and Red Blood Cell Disorders Center
      • Contact: Lele Zhang, PhD; Phone Number: ‭15811139278‬; Email: zhanglele@ihcams.ac.cn

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Child; Adult; Older Adult
• Accepts Healthy Volunteers: No

Description

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

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm

Intervention / Treatment

Experimental: 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.

Drug: standard IST combined with Romiplostim N01; Romiplostim N01 is a class of TPO receptor agonists. Its TPO peptide can bind to TPO receptors on megakaryocyte precursors in the bone marrow and activate them, triggering a series of intracellular signalling pathways, including JAK2/STAT5, PI3K/Akt, MEK/ERK, and p38, which ultimately lead to gene transcription and increased megakaryocyte proliferation and differentiation.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Overall response rate	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).	Within 27 weeks post treatment

Secondary Outcome Measures

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

Collaborators and Investigators

• Sponsor: Institute of Hematology & Blood Diseases Hospital, China
• Collaborators: Qilu Pharmaceutical Co., Ltd.
• Investigators: Principal Investigator: Jun Shi, PhD, Institute of Hematology & Blood Diseases Hospital, China

Study record dates

Study Major Dates

• Study Start (Actual): October 24, 2024
• Primary Completion (Estimated): January 31, 2026
• Study Completion (Estimated): September 30, 2026

Study Registration Dates

• First Submitted: September 23, 2024
• First Submitted That Met QC Criteria: September 23, 2024
• First Posted (Actual): September 26, 2024

Study Record Updates

• Last Update Posted (Estimated): December 4, 2024
• Last Update Submitted That Met QC Criteria: November 29, 2024
• Last Verified: September 1, 2024

More Information

Terms related to this study

• Keywords: Severe aplastic anemia; Romiplostim N01
• Additional Relevant MeSH Terms: Bone Marrow Failure Disorders; Hematologic Diseases; Bone Marrow Diseases; Anemia; Anemia, Aplastic
• Other Study ID Numbers: IIT2024070

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No