Clinical trial NCT05660785: Herombopag Added to Cyclosporine in Non Severe Aplastic Anemia
Official title: The Efficacy and Safety of Herombopag Combined With Cyclosporine for Patients With Non Severe Aplastic Anemia
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Non Severe Aplastic Anemia
Untreated
Lead sponsor: Institute of Hematology & Blood Diseases Hospital, China
Sponsor class: OTHER
Collaborators: name: Jiangsu Hengrui Pharmaceutical Co., Ltd.
First posted: 2022-12-21
Enrollment: 54
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: 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).

Detailed 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.

Eligibility criteria: 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

Primary outcomes:
measure: Overall response rate
timeFrame: 24 weeks
Secondary outcomes:
measure: Robust response rate
timeFrame: 24 weeks
measure: Proportion of patients with abnormal karyotype changes
timeFrame: Baseline and 24 weeks
measure: Time duration for patients achieving hematological response
timeFrame: A minimum of 2 years of planned follow-up
measure: Change of the health-related quality of life
timeFrame: Baseline and 24 weeks
measure: Incidence of the adverse event
timeFrame: 24 weeks
measure: Severity of the adverse event
timeFrame: 24 weeks
Other outcomes:
Locations:
city: Tangshan
country: China
facility: Tangshan Central Hospital
state: Hebei
city: Zhoukou
country: China
facility: Zhoukou Central Hospital
state: Henan
city: Tai'an
country: China
facility: The Second Affilated Hospital of Shandong First Medical University
state: Shandong
city: Tianjin
country: China
facility: Regenerative Medicine Center
state: Tianjin
Study officials:
References: