Actively Recruiting
Efficacy and Safety of Avatrombopag for Platelet Recovery After Unrelated Cord Blood Transplantation in Patients With Bone Marrow Failure Disease: a Single-center, Single-arm, Phase II Trial
Led by Anhui Provincial Hospital · Updated on 2024-12-31
40
Participants Needed
1
Research Sites
4 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
Researchers are evaluating the effectiveness and safety of Avatrombopag for helping platelet recovery after unrelated cord blood transplantation (UCBT) in patients with bone marrow failure disease (BMFD). This single-center, single-arm, phase II trial focuses on patients diagnosed with BMFD who undergo UCBT. Persistent low platelet counts after UCBT often require platelet transfusions and increase transplant-related risks, prompting this study to find better recovery options. Participants will receive Avatrombopag at a recommended dose of 40 mg daily, starting from the first day after UCBT and continuing for 28 days. After this treatment period, based on individual platelet levels, patients may continue, adjust, or stop the medication. The main goal is to measure the rate of platelet engraftment within 28 days following transplantation. During the study, patients will be closely monitored for platelet recovery and other blood cell reconstitution. Researchers will assess platelet engraftment time, cumulative platelet recovery rates, total platelet transfusions needed, and bone marrow megakaryocyte levels. Safety and treatment effects will be observed throughout the 28-day treatment, with follow-up assessments up to 100 days to evaluate sustained platelet recovery.
CONDITIONS
Brief 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 12 years or older, male or female
- Diagnosis of bone marrow failure disease including aplastic anemia, Fanconi anemia, or paroxysmal nocturnal hemoglobinuria
- Undergoing unrelated cord blood transplantation
- ECOG performance status score of 2 or less
- Voluntary participation with informed consent signed
You will not qualify if you...
- Pregnant or breastfeeding women
- Known allergy to Avatrombopag
- History of severe blood clotting events or active thromboembolism requiring anticoagulation
- History of platelet dysfunction, bleeding prone diseases, or severe bleeding requiring significant blood transfusion within 7 days before screening
- Chronic active hepatitis B or C
- Repeat or multiple organ transplantation
- Positive for HIV, EBV-DNA, or CMV-DNA
- Severe infection or serious heart, liver, lung, kidney, neurological, or metabolic diseases
- Serious adverse events during pre-treatment period
- Participation in another clinical study with exploratory drugs or devices within 30 days prior to baseline, except observational studies
- Cognitive impairment or uncontrolled mental illness
- Refusal of treatment with Avatrombopag by patient or authorized family
- Judged unsuitable by researchers due to medical history or inability to adhere to treatment due to financial or other issues
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
Duration - 28 days
Participants receive Avatrombopag treatment daily for platelet recovery starting from the first day after transplantation up to 28 days.
Daily treatment from day 1 to day 28 after transplantation
Duration - Up to 100 days
Participants may continue, change, or discontinue medication based on platelet status after the initial 28-day treatment period.
Visits as needed based on platelet status after treatment
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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