Standard Immunosuppressive Therapy Combined With Romiplostim N01 as First-line Treatment for Severe Aplastic Anemia
For patients and families
In plain language
An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.
- What is being studied
- The protocol lists: standard IST combined with Romiplostim N01.
- Who it may be relevant to
- Registry conditions: Aplastic Anemia, TPO Receptor Agonists. Basic parameters: from 12 years · All.
- What needs checking
- Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
- Where it takes place
- China
- Next step
- Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →
Unsure about the terms? Read our patient guide →
Official title
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
Overview
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.
Interventions
- 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.
Primary outcome measures
- Overall response rate [Time frame: Within 27 weeks post treatment]
Secondary outcome measures (3)
- Overall response rate [Time frame: Within 3 months post treatment]
- Time to hematological response [Time frame: Within 27 weeks post treatment]
- Incidence of adverse events [Time frame: Within 27 weeks post treatment]
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
Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.
Healthy volunteers: No
Study design
- Allocation
- N/A
- Model
- Single group
- Masking
- Open label
- Primary purpose
- Treatment
Study locations
China · 1 center
- Regenerative Medicine Center and Red Blood Cell Disorders Center — Tianjin
Identifiers
NCT: NCT06613880 · IIT2024070