Herombopag Added to Cyclosporine in Non Severe Aplastic Anemia

Institute of Hematology & Blood Diseases Hospital, China

Status and phase

Completed

Phase 2

Conditions

Untreated

Non Severe Aplastic Anemia

Treatments

Drug: Herombopag

Study type

Interventional

Funder types

Other

Industry

Identifiers

NCT05660785

IIT2022049

Details and patient eligibility

About

This is a prospective, multicenter, single-arm, phase 2 trial. The aim of this study is to evaluate the efficacy and safety of herombopag combined with cyclosporine for patients with non severe aplastic anemia (NSAA).

Full description

This study aims to improve the 24 weeks response rate. The sample size is calculated based on Simon's two-stage design. The first stage of the study enrolled a cohort of 15 patients. If after 24 weeks at least 9 patients achieved a response, then enrollment was expanded to a total of 43 patients. The null hypothesis was unaccepted if more than 26 of 43 patients achieved the response. Accounting for a 20% dropout rate, the estimated final sample size was 54 patients.

Enrollment

54 patients

Sex

All

Ages

18+ years old

Volunteers

No Healthy Volunteers

Inclusion criteria

Willing and able to comply with the requirements for this study and written informed consent.
Male or female age ≥ 18 years
Diagnosis of untreated non severe aplastic anemia.
Platelet counts < 50 x 10^9/L at least 2 times consecutively (time interval ≥ 1 week)

Exclusion criteria

Receive immunosuppressive therapy more than 4 weeks before enrollment
Treatment with TPO-RA within 1 week before enrollment
Inherited bone marrow failure syndromes
Bone marrow fibrosis grade ≥ 2
The presence of hemolytic PNH clone
The presence of clonal karyotypic abnormalities (del(20q), +8 and -Y are not included in this category)
Previously treated with TPO-RA ≥ 4 weeks
Previously received immunosuppressive therapy ≥ 12 weeks
Ferritin > 1000 ng/ml (The increased level of Ferritin led by infection is not included in this category)
Have an allergy to eltrombopag or any other part of this medicine.
History of radiotherapy and chemotherapy for malignant solid tumors
Cytopenia caused by other non-hematologic diseases, including liver cirrhosis, active rheumatic connective tissue disease, and persistence of infectious diseases, etc
Abnormal liver function: ALT or AST > 3 ULN, or TBil > 1.5 ULN after treatment.
Abnormal kidney function: Creatinine clearance < 30 ml/min, or serum creatinine (sCr) >1.5 ULN
Patients with diabetic nephropathy, neuropathy, or eye disease
Patients with poorly controlled hypertension or cardiac arrhythmia
Patients with congestive heart failure and the NYHA grade ≥ 3 historically or currently, and LVEF < 45% within 4 weeks before enrollment
History of arteriovenous thrombosis within 1 year before enrollment
Participation in another clinical trial within 4 months before the start of this trial
Pregnant or breast-feeding patients
Patients considered to be ineligible for the study by the investigator for reasons other than the above