A Study of Romiplostim N01 Plus IST vs. Placebo Plus IST for Treatment-Naive Severe Aplastic Anemia

NCT ID: NCT07345000

Last Updated: 2026-01-15

Basic Information

• Recruitment Status: NOT_YET_RECRUITING
• Clinical Phase: PHASE3
• Total Enrollment: 210 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2026-01-31
• Study Completion Date: 2030-08-31

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.

Conditions

Treatment-naïve Severe Aplastic Anemia

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: PARALLEL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: DOUBLE

Study Groups

Romiplostim N01+ IST

Group Type: EXPERIMENTAL

Romiplostim N01

Intervention Type: DRUG

Starting from Day 1 of Week 1, administer the initial dose of romiplostim N01: 10 μg/kg. Platelet count (PLT) must be monitored weekly. The dose should be adjusted based on platelet values (adjusted by 5 μg/kg per change, see the table below for specific rules), administered once weekly, with a maximum dose of 20 μg/kg.

Cyclosporine A (CsA)

Intervention Type: DRUG

Initiate at a dose of 5 mg/kg/day, administered orally in two divided doses (recommended at 12-hour intervals). The dose may be adjusted between 3 and 5 mg/kg/day based on the subject's tolerance.

pALG/ rATG

Intervention Type: DRUG

25 mg/kg/day on Days 1 through 5 of Week 1.

Placebo+ IST

Group Type: ACTIVE_COMPARATOR

Placebo

Intervention Type: DRUG

Starting from Day 1 of Week 1, administer the initial dose of placebo: 10 μg/kg. Platelet count (PLT) must be monitored weekly. The dose should be adjusted based on platelet values (adjusted by 5 μg/kg per change, see the table below for specific rules), administered once weekly, with a maximum dose of 20 μg/kg.

Cyclosporine A (CsA)

Intervention Type: DRUG

Initiate at a dose of 5 mg/kg/day, administered orally in two divided doses (recommended at 12-hour intervals). The dose may be adjusted between 3 and 5 mg/kg/day based on the subject's tolerance.

pALG/ rATG

Intervention Type: DRUG

25 mg/kg/day on Days 1 through 5 of Week 1.

Interventions

Romiplostim N01

Starting from Day 1 of Week 1, administer the initial dose of romiplostim N01: 10 μg/kg. Platelet count (PLT) must be monitored weekly. The dose should be adjusted based on platelet values (adjusted by 5 μg/kg per change, see the table below for specific rules), administered once weekly, with a maximum dose of 20 μg/kg.

Intervention Type: DRUG

Cyclosporine A (CsA)

Initiate at a dose of 5 mg/kg/day, administered orally in two divided doses (recommended at 12-hour intervals). The dose may be adjusted between 3 and 5 mg/kg/day based on the subject's tolerance.

Intervention Type: DRUG

pALG/ rATG

25 mg/kg/day on Days 1 through 5 of Week 1.

Intervention Type: DRUG

Placebo

Starting from Day 1 of Week 1, administer the initial dose of placebo: 10 μg/kg. Platelet count (PLT) must be monitored weekly. The dose should be adjusted based on platelet values (adjusted by 5 μg/kg per change, see the table below for specific rules), administered once weekly, with a maximum dose of 20 μg/kg.

Intervention Type: DRUG

Cyclosporine A (CsA)

Initiate at a dose of 5 mg/kg/day, administered orally in two divided doses (recommended at 12-hour intervals). The dose may be adjusted between 3 and 5 mg/kg/day based on the subject's tolerance.

Intervention Type: DRUG

pALG/ rATG

25 mg/kg/day on Days 1 through 5 of Week 1.

Intervention Type: DRUG

Eligibility Criteria

Inclusion Criteria

1. 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).

2. 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):

1. Absolute neutrophil count (ANC) <0.5×10⁹/L

2. Platelet count (PLT) <20×10⁹/L

3. Absolute reticulocyte count (RET) <60×10⁹/L The diagnostic criterion for VSAA is: meeting the SAA criteria + ANC <0.2×10⁹/L.

3. Written informed consent

2. Evidence of clonal cytogenetic abnormalities at screening.

3. 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).

4. Previous use of any of the following agents prior to the first dose:

• ATG/ALG

• Alemtuzumab

• Mycophenolate mofetil ④Sirolimus

• Tacrolimus ⑥High-dose cyclophosphamide (≥45 mg/kg/day)

5. 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.

6. 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.

7. Previous history of hematopoietic stem cell transplantation.

8. 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.

9. Concomitant active CMV and EBV infection (positive test).

Exclusion Criteria

1. 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.

• Minimum Eligible Age: 15 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Qilu Pharmaceutical Co., Ltd.

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Central Contacts

jun shi, Professor

Role: CONTACT

shijun@ihcams.ac.cn

022-23608326

Other Identifiers

QL0911-304

Identifier Type: -

Identifier Source: org_study_id