Aplastic Anemia, TPO Receptor Agonists
Conditions
Keywords
Severe aplastic anemia, Romiplostim N01
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.
Interventions
DRUGstandard 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.
Sponsors
Qilu Pharmaceutical Co., Ltd.
Institute of Hematology & Blood Diseases Hospital, China
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
12 Years to No maximum
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
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| 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
| Measure | Time frame | Description |
|---|---|---|
| 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 |
Countries
China
Contacts
Primary ContactLele Zhang, PhD
Backup ContactMeili Ge, PhD
Outcome results
None listed