NCT06426043

A Prospective Study on the Treatment of Recurrent/Refractory/Intolerable NSAA With Lusutrombopag

An Exploratory Study on the Efficacy and Safety of Lusutrombopag in the Treatment of Recurrent/Refractory/Intolerable NSAA

• Status: Not Yet Recruiting
• Phase: Phase 4
• Study type: Interventional
• Enrollment: 40 (estimated)
• Sponsor: Peking Union Medical College Hospital · Academic / Other
• Sex: All
• Age: 18 Years – 90 Years
• Healthy volunteers: Not accepted

Summary

In a prospective, single-arm study, the efficacy and safety of Lusutrombopag in the treatment of relapsed/refractory/intolerable non-severe aplastic anemia (NSAA) were explored.

Detailed description

The enrolled patients: were given Lusutrombopag at 3mg/qd orally for 12 weeks (the starting dose of lusutrombopag was 3mg, taken once daily. After 2 weeks of continuous administration, the dose was increased by 3mg every 2 weeks based on the platelet count and safety of the subjects. The dose was gradually increased to 9mg/d over a total of 12 weeks). The treatment duration was at least 3 months. When the platelet increase was \<20×10\^9/L, the daily dose was increased by 3mg, up to a maximum of 9mg/day. When the platelet increase was ≥50×109/L and ≤200×10\^9/L, the dose was maintained at the previous level. When the platelet count was ≥200×10\^9/L and ≤400×10\^9/L, the daily dose was reduced by 3mg. When the platelet count was \>400×10\^9/L, the drug could be suspended, and the dose was reduced by 3mg when the platelet count decreased to \<200×10\^9/L. In this case, if the lowest dose of 3mg/day was used, the drug could be suspended. Responders continued treatment for 6 months. Other TPO-RA therapies were not allowed during the study period.

Conditions

• Aplastic Anemia

Interventions

Type	Name	Description
DRUG	Lusutrombopag	Administer lusutrombopag at 3mg/qd orally for 12 weeks (lusutrombopag starting dose is 3mg, once daily. After 2 weeks of continuous administration, the dose can be increased by 3mg every 2 weeks based on the platelet count and safety of the subject. The dose can be gradually increased to 9mg/d over a total of 12 weeks). The course should be at least 3 months. When the platelet increase is \<20×109/L, the daily dose can be increased by 3mg up to a maximum of 9mg/day; when the platelet increase is ≥50×10\^9/L and ≤200×10\^9/L, the dose can be maintained; when the platelet count is ≥200×10\^9/L and ≤400×10\^9/L, the daily dose can be reduced by 3mg; when the platelet count is \>400×10\^9/L, the drug can be suspended and resumed when the platelet count decreases to \<200×10\^9/L, with the daily dose reduced by 3mg. In this case, if the lowest dose of 3mg/day is used, the drug can be suspended. Responders continue treatment until 6 months.

Timeline

• Start date: 2024-06-01
• Primary completion: 2025-06-01
• Completion: 2025-08-01
• First posted: 2024-05-23
• Last updated: 2024-05-23

Locations

1 site across 1 country: China