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

NCT07345000 | Not Yet Recruiting Phase 3

• 1 other identifier: QL0911-304
• Study Type: interventional
• Target: 210
• Locations: 0 countries
• Sites: N/A

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

Outcome Measures

Primary Outcomes (1)

• Complete Response (CR) rate at the 6-month time point of treatment.

  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.

  During 6 months of therapy

Study Arms (2)

Romiplostim N01+ IST

EXPERIMENTAL

Drug: Romiplostim N01; Drug: Cyclosporine A (CsA); Drug: pALG/ rATG

Placebo+ IST

ACTIVE COMPARATOR

Drug: Placebo; Drug: Cyclosporine A (CsA); Drug: pALG/ rATG

Interventions

Romiplostim N01 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.

Romiplostim N01+ IST

Cyclosporine A (CsA) 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.

Romiplostim N01+ IST

Placebo 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.

Placebo+ IST

pALG/ rATG DRUG

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

Romiplostim N01+ IST

Eligibility Criteria

• Age: 15 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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.
• 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:
• ATG/ALG
• 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.

Sponsors & Collaborators

• Qilu Pharmaceutical Co., Ltd.: lead

MeSH Terms

Interventions

Cyclosporine

Intervention Hierarchy (Ancestors)

Cyclosporins; Peptides, Cyclic; Macrocyclic Compounds; Polycyclic Compounds; Peptides; Amino Acids, Peptides, and Proteins

Central Study Contacts

jun shi, Professor

CONTACT

022-23608326; shijun@ihcams.ac.cn

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: RANDOMIZED
• Masking: DOUBLE
• Who Masked: PARTICIPANT, INVESTIGATOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: December 23, 2025
• First Posted: January 15, 2026
• Study Start: January 1, 2026
• Primary Completion (Estimated): October 1, 2029
• Study Completion (Estimated): August 1, 2030
• Last Updated: January 15, 2026

Record last verified: 2026-01