NCT07345000Not yet recruitingPhase 3
A Randomized, Controlled, Multicenter, Double-blind Phase III Study Evaluating the Efficacy and Safety of Romiplostim N01 Combined With Standard Immunosuppressive Therapy (IST) Versus Placebo Combined With IST in Treatment-naïve Subjects With Severe Aplastic Anemia
Trial Snapshot
- Phase
- Phase 3
- Status
- Not yet recruiting
- Enrollment
- 210
- Primary Endpoint
- Complete Response (CR) rate at the 6-month time point of treatment.
Study Overview
Brief Summary
This is a randomized, double-blind, multicenter trial designed to evaluate treatment with romiplostim N01+ IST compared with placebo + IST in the participants with treatment-naïve severe aplastic anemia.
Study Design
- Study Type
- Interventional
- Allocation
- Randomized
- Intervention Model
- Parallel
- Primary Purpose
- Treatment
- Masking
- Double (Participant, Investigator)
Eligibility Criteria
- Ages
- 15 Years to — (Child, Adult, Older Adult)
- Sex
- All
- Accepts Healthy Volunteers
- No
Inclusion Criteria
- •Age ≥15 years, regardless of sex (subjects ≥18 years old will be enrolled first; enrollment of subjects aged 15-18 years will commence after sufficient PK/PD data are obtained).
- •Diagnosis of SAA or VSAA according to the British Journal of Haematology (BJH) guidelines. The diagnostic criteria for SAA are as follows:
- •①Bone marrow cellularity <25% of normal; or between 25% and <50%, with residual hematopoietic cells comprising <30%.
- •②Peripheral blood counts must meet at least two of the following three criteria (based on the lowest values from tests within 28 days prior to the first dose):
- •Absolute neutrophil count (ANC) <0.5×10⁹/L
- •Platelet count (PLT) <20×10⁹/L
- •Absolute reticulocyte count (RET) <60×10⁹/L The diagnostic criterion for VSAA is: meeting the SAA criteria + ANC <0.2×10⁹/L.
- •Written informed consent
Exclusion Criteria
- •History and/or concomitant presence of other primary or secondary bone marrow failure (BMF) syndromes, such as:
- •①Primary: Fanconi anemia, dyskeratosis congenita, congenital amegakaryocytic thrombocytopenia or Shwachman-Diamond syndrome, symptomatic paroxysmal nocturnal hemoglobinuria (PNH), myelodysplastic syndromes (MDS), clonal cytopenia of undetermined significance (CCUS), antibody-mediated BMF, idiopathic cytopenia of undetermined significance (ICUS), etc.
- •Secondary: large granular lymphocyte (LGL) leukemia, infiltration of the bone marrow by other systemic malignancies, myelofibrosis, and acute hematopoietic arrest, etc.
- •Note: Asymptomatic PNH and hepatitis-associated SAA may be included if they meet all other inclusion criteria.
- •Evidence of clonal cytogenetic abnormalities at screening.
- •Participation in another clinical trial with investigational drugs or medical devices within 30 days prior to the first dose or within 5 half-lives of the investigational product (whichever is longer).
- •Previous use of any of the following agents prior to the first dose:
- •Alemtuzumab
- •Mycophenolate mofetil ④Sirolimus
- •Tacrolimus ⑥High-dose cyclophosphamide (≥45 mg/kg/day)
- •Cumulative cyclosporine A (CsA) therapy exceeding 4 weeks prior to the first dose. If cumulative use is ≤4 weeks, a washout period of >14 days prior to the first dose is required.
- •Cumulative use of thrombopoietin receptor agonists (TPO-RAs) for >14 days prior to the first dose, or cumulative use ≤14 days with a washout period of <14 days, including:
- •Romiplostim / Nplate® (romiplostim)
- •Eltrombopag ③Hetrombopag ④Recombinant human thrombopoietin, etc.
- •Previous history of hematopoietic stem cell transplantation.
- •Uncontrolled bleeding and/or infection after standard treatment prior to the first dose [defined as persistent signs/symptoms related to infection without improvement despite appropriate antibiotic and/or other therapy], or requiring intravenous (IV) antibiotic administration.
- •Concomitant active CMV and EBV infection (positive test).
Outcomes
Primary Outcomes
Complete Response (CR) rate at the 6-month time point of treatment.
Time Frame: During 6 months of therapy
Proportion of subjects achieving hematopoietic complete response at the 6-month. Hematopoietic complete response is defined as: hemoglobin ≥10 g/dL, absolute neutrophil count ≥1×10⁹/L, and platelet count ≥100×10⁹/L.
Secondary Outcomes
No secondary outcomes reported
Investigators
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