Using avatrombopag to treat low platelet counts after stem cell transplant

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

This study is testing if a new drug called avatrombopag can help people with low platelet counts after a stem cell transplant to raise their platelet levels without needing blood transfusions.

Quick facts

• Phase: Phase2; Phase3
• Study type: Interventional
• Enrollment: 142 (estimated)
• Ages: 18 Years to 65 Years
• Sex: All
• Sponsor: Peking University People's Hospital (Academic / other)
• Locations: 14 sites (Beijing, Beijing Municipality and 13 other locations)
• Trial ID: NCT06202625 on ClinicalTrials.gov

What this trial studies

This clinical trial evaluates the efficacy and safety of avatrombopag in patients experiencing thrombocytopenia following haploidentical hematopoietic stem cell transplantation (haplo-HSCT). It is a prospective, multi-center, double-blinded, randomized placebo-controlled trial involving patients with platelet counts below 20×10^9/L or those who are transfusion dependent. Participants will be randomly assigned to receive either avatrombopag or a placebo, with the primary endpoint being the proportion of patients achieving a platelet count of at least 50×10^9/L without transfusions by day 60 post-transplant. The study aims to provide evidence for the use of avatrombopag in this specific patient population.

Who should consider this trial

Good fit: Ideal candidates are adults aged 18-65 with thrombocytopenia (PLT<20×10^9/L) or those dependent on transfusions within 7 days after haplo-HSCT.

Not a fit: Patients with active infections, significant liver dysfunction, or a history of cardiovascular disease may not benefit from this study.

Why it matters

Potential benefit: If successful, this treatment could significantly improve platelet recovery and reduce the need for transfusions in patients after haplo-HSCT.

How similar studies have performed: Previous studies have shown positive outcomes with avatrombopag in other thrombocytopenic conditions, suggesting potential success in this novel application.

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.

Where this trial is running

Beijing, Beijing Municipality and 13 other locations

• Peking University People's Hospital — Beijing, Beijing Municipality, China (Recruiting)
• Guangzhou First People's Hospital, School of Medicine, South China University of Technology — Guangzhou, Guangdong, China (Not_yet_recruiting)
• Peking University Shenzhen Hospital — Shenzhen, Guangdong, China (Recruiting)
• The First Affiliated Hospital, Harbin Medical University — Harbin, Heilongjiang, China (Not_yet_recruiting)
• The First Affiliated Hospital of Zhengzhou University — Zhengzhou, Henan, China (Not_yet_recruiting)
• Tongji Hospital, Tongji Medical College, Huazhong University of Science and Technology — Wuhan, Hubei, China (Not_yet_recruiting)
• Union Hospital, Tongji Medical College, Huazhong University of Science and Technology — Wuhan, Hubei, China (Not_yet_recruiting)
• Xiangya Hospital, Central South University — Changsha, Hunan, China (Not_yet_recruiting)
• The First Affiliated Hospital of Nanchang University — Nanchang, Jiangxi, China (Not_yet_recruiting)
• Shanxi Tumor Hospital Affiliated to Shanxi Medical University — Taiyuan, Shanxi, China (Not_yet_recruiting)
• Tangdu Hospital, PLA Air Force Military Medical University — Xi’an, Shanxi, China (Not_yet_recruiting)
• Xinqiao Hospital, Army Military Medical University — Chongqing, Sichuan, China (Not_yet_recruiting)
• 920th Hospital of Joint Logistics Support Force of People's Liberation Army of China — Kunming, Yunnan, China (Not_yet_recruiting)
• Chinese Academy of Medical Sciences & Peking Union Medical College — Tianjin, China (Not_yet_recruiting)

Study contacts

• Principal investigator: Xiaohui Zhang — Peking University People's Hospital
• Study coordinator: Haixia Fu

Conditions Thrombocytopenia; Stem Cell Transplant Complications; hematopoietic stem cell transplantation; haploidentical