NCT07345000

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

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

• Status: Not Yet Recruiting
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 210 (estimated)
• Sponsor: Qilu Pharmaceutical Co., Ltd. · Industry
• Sex: All
• Age: 15 Years
• Healthy volunteers: Not accepted

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

Interventions

Type	Name	Description
DRUG	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.
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.
DRUG	pALG/ rATG	25 mg/kg/day on Days 1 through 5 of Week 1.
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.
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.
DRUG	pALG/ rATG	25 mg/kg/day on Days 1 through 5 of Week 1.

Timeline

• Start date: 2026-01-01
• Primary completion: 2029-10-01
• Completion: 2030-08-01
• First posted: 2026-01-15
• Last updated: 2026-01-15