Actively Recruiting
Avatrombopag for Platelet Recovery After Unrelated Cord Blood Transplantation in Patients With Bone Marrow Failure Disease: A Single-Arm Phase II Study
Led by Anhui Provincial Hospital · Updated on 2024-12-31
40
Participants Needed
1
Research Sites
95 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
Persistent thrombocytopenia is a common complication after umbilical cord blood transplantation (UCBT) that requires platelet transfusions and increases transplant-related mortality. Researchers are evaluating the efficacy and safety of Avatrombopag for improving platelet recovery in patients with bone marrow failure disease who have undergone UCBT. This is a single-center, single-arm, phase II clinical trial involving patients diagnosed with bone marrow failure diseases such as aplastic anemia, Fanconi anemia, and paroxysmal nocturnal hemoglobinuria. Participants will receive Avatrombopag treatment daily from the first day to the 28th day after transplantation. The drug dose will be reduced by 20 mg per day if platelet counts reach or exceed 50×10^9/L, and treatment will be stopped once platelet counts reach or exceed 100×10^9/L, excluding factors related to blood transfusion. The main goal is to assess the cumulative incidence of platelet engraftment 28 days after transplantation. During the study, participants will be closely monitored for platelet recovery and safety. Researchers will evaluate the time to platelet engraftment and the overall rate of platelet recovery at 28 days following UCBT. The study plans to enroll 40 patients, who will be followed throughout the treatment period to measure platelet counts and monitor for any adverse effects related to Avatrombopag treatment.
CONDITIONS
Official Title
Avatrombopag for Platelet Recovery Post-UCBT in Patients With Bone Marrow Failure Disease
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Age 6 years old, male or female
- Diagnosed with bone marrow failure disease including aplastic anemia, Fanconi anemia, paroxysmal nocturnal hemoglobinuria
- Undergoing unrelated cord blood transplantation (UCBT)
- ECOG score 6
- Voluntary participation with informed consent
You will not qualify if you...
- Pregnant or lactating women
- Known allergy to Avatrombopag
- History of severe thrombotic events or active thromboembolism requiring anticoagulation
- History of platelet dysfunction, bleeding prone disease, or severe bleeding within 7 days before screening
- Chronic active hepatitis B or C
- Repeat, multiple, or multiple organ transplantation
- HIV positive, EBV-DNA positive, or CMV-DNA positive
- Severe infection or serious heart, liver, lung, kidney, neurological, or metabolic diseases
- Serious adverse event (Level 4 or 5) during pre-treatment period
- Participation in another clinical trial with exploratory drug or device within 30 days before baseline
- Cognitive impairment or uncontrolled mental illness
- Refusal of treatment with Avatrombopag by participant or authorized family
- Judged unsuitable by researchers due to medical history or inability to adhere to treatment due to financial problems
AI-Screening
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Trial Site Locations
Total: 1 location
1
The First Affiliated Hospital of University of Science and Technology of China (Anhui Provincial Hospital)
Hefei, Anhui, China, 230036
Actively Recruiting
Research Team
X
Xiaoyu Zhu, ph.D
A
Aijie Huang, M.D
How is the study designed?
Study Type
OBSERVATIONAL
Masking
N/A
Allocation
N/A
Model
N/A
Primary Purpose
N/A
Number of Arms
1
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