Standard Immunosuppressive Therapy Combined With Romiplostim N01 as First-line Treatment for Severe Aplastic Anemia
NCT ID: NCT06613880
Last Updated: 2024-12-04
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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RECRUITING
PHASE2
48 participants
INTERVENTIONAL
2024-10-24
2026-09-30
Brief Summary
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Detailed Description
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Conditions
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Study Design
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NA
SINGLE_GROUP
TREATMENT
NONE
Study Groups
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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.
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.
Interventions
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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.
Eligibility Criteria
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Inclusion Criteria
* 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 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
12 Years
ALL
No
Sponsors
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Qilu Pharmaceutical Co., Ltd.
INDUSTRY
Institute of Hematology & Blood Diseases Hospital, China
OTHER
Responsible Party
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Principal Investigators
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Jun Shi, PhD
Role: PRINCIPAL_INVESTIGATOR
Institute of Hematology & Blood Diseases Hospital, China
Locations
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Regenerative Medicine Center and Red Blood Cell Disorders Center
Tianjin, Tianjin Municipality, China
Countries
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Central Contacts
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Meili Ge, PhD
Role: CONTACT
Facility Contacts
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Other Identifiers
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IIT2024070
Identifier Type: -
Identifier Source: org_study_id