Efficacy and Safety of Sirolimus With or Without Cyclosporin A in Chinese Patients With Aplastic Anemia Refractory/Intolerant to Cyclosporin A

PHASE2RecruitingINTERVENTIONAL
Enrollment

40

Participants

Timeline

Start Date

January 25, 2025

Primary Completion Date

January 31, 2026

Study Completion Date

January 31, 2027

Conditions
Aplastic Anaemia
Interventions
DRUG

Sirolimus (Rapamune®)

Sirolimus 1-3 mg once daily orally, with a target trough blood concentration of 4-12 ng/ml.

DRUG

Cyclosporin A (CsA)

Cyclosporine A 25-150 mg orally every 12 hours, with the dose adjusted based on renal function and trough concentration. For patients with normal renal function, the target trough concentration is approximately 150 ng/ml. For patients with impaired renal function, the cyclosporine A dose is reduced to 25-50 mg every 12 hours, aiming for recovery or stabilization of renal function.

Trial Locations (1)

100730

RECRUITING

Peking Union Medical College Hospital, Beijing

All Listed Sponsors
lead

Peking Union Medical College Hospital

OTHER