Clinical trial
Efficacy and Safety of Avatrombopag in the Treatment of Thrombocytopenia After Haploidentical Hematopoietic Stem Cell Transplantation: Prospective, Multi-center, Double-blinded, Randomized Placebo-controlled Study
Name
2023PHD009-001
Description
In this study, investigators aim to evaluate the efficacy of avatrombopag in thrombocytopenic patients after haploidentical hematopoietic stem cell transplantation (haplo-HSCT) through a prospective, multi-center, double-blinded, randomized placebo-controlled clinical trial.
Trial arms
Trial start
2024-01-15
Estimated PCD
2024-10-30
Trial end
2025-10-30
Status
Not yet recruiting
Phase
Early phase I
Treatment
avatrombopag
The avatrombopag 20mg/d will be orally taken from +D7 after haplo-HSCT until meeting the adjustment indication or to +D60 after haplo-HSCT; When PLT\<50×10\^9/L or PLT transfusion-dependent on the +D30 after haplo-HSCT, increase avatrombopag dosage to 40 mg/d; When PLT≥80×10\^9/L and without PLT transfusion within avatrombopag dosage at 40 mg/d, decrease avatrombopag dosage to 20 mg/d; When PLT≥80×10\^9/L for 7 consecutive days or PLT≥300×10\^9/L and without PLT transfusion, stop avatrombopag; When PLT\<50×10\^9/L or PLT transfusion-dependent after stopping avatrombopag , reuse avatrombopag at 40 mg/d.
Arms:
avatrombopag
Other names:
Doptelet
Placebo
The placebo 20mg/d will be orally taken from +D7 after haplo-HSCT until meeting the adjustment indication or to +D60 after haplo-HSCT; When PLT\<50×10\^9/L or PLT transfusion-dependent on the +D30 after haplo-HSCT, increase placebo dosage to 40 mg/d; When PLT≥80×10\^9/L and without PLT transfusion within placebo dosage at 40 mg/d, decrease placebo dosage to 20 mg/d; When PLT≥80×10\^9/L for 7 consecutive days or PLT≥300×10\^9/L and without PLT transfusion, stop placebo; When PLT\<50×10\^9/L or PLT transfusion-dependent after stopping placebo, reuse placebo at 40 mg/d.
Arms:
Placebo
Size
142
Primary endpoint
the proportion of complete response(CR) on day 60 after haplo-HSCT
from randomization to day 60 after haplo-HSCT
Eligibility criteria
Inclusion Criteria:
1. Male or female, aged between 18-65 years;
2. PLT\<20×10\^9/L or transfusion dependent on +D7 after haplo-HSCT;
3. Agree to receive the treatment of avatrombopag after Haplo-HSCT and sign the informed consent form.
Exclusion Criteria:
1. With active infection;
2. ALT or AST\>3ULN, or total Bil\>2ULN
3. Ccr\<50 mL/min;
4. With the history of arteriovenous thrombosis;
5. With history of cardiovascular disease (such as NYHA Class III/IV congestive heart failure, arrhythmia that increases the risk of thromboembolic events \[such as atrial fibrillation\] and angina), and subjects who have undergone coronary stent implantation, angioplasty, or coronary artery bypass grafting;
6. With treatment of drugs to promote platelet production two weekes before enrollment, including but not limited to rhTPO and TPO-RA;
7. HBsAg or anti-HCV or anti-HIV positive;
8. Known to be allergic to avatrombopag and any of its excipients;
9. With secondary or multiple HSCT;
10. Females who were pregnant or breastfeeding or who had fertile ability but refuse to take effective contraceptive measures during and one month after this trial;
11. With any other clinical trial of investigational product or device within 30 days prior to the baseline visit, except for observational study;
12. Deemed unsuitable for enrollment by the investigator for any history of or concomitant medical condition.
13. Concomitant medication:The rhIL-11, rhTPO or TPO-RA(such as eltrombopag, hetrombopag and romiplostim) and desitabine, etc. were not allowed for use during this trial.
Protocol
{'studyType': 'INTERVENTIONAL', 'phases': ['PHASE2', 'PHASE3'], 'designInfo': {'allocation': 'RANDOMIZED', 'interventionModel': 'PARALLEL', 'primaryPurpose': 'TREATMENT', 'maskingInfo': {'masking': 'QUADRUPLE', 'whoMasked': ['PARTICIPANT', 'CARE_PROVIDER', 'INVESTIGATOR', 'OUTCOMES_ASSESSOR']}}, 'enrollmentInfo': {'count': 142, 'type': 'ESTIMATED'}}
Updated at
2024-01-11
1 organization
2 products
2 indications
Organization
Peking University People's HospitalProduct
avatrombopagIndication
ThrombocytopeniaIndication
Stem Cell Transplant ComplicationsProduct
Placebo