Using Avatrombopag to Help Platelet Recovery After Cord Blood Transplantation

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

This study is testing if a medication called Avatrombopag can help people with bone marrow failure recover their platelet levels after receiving a cord blood transplant.

Quick facts

• Study type: Observational
• Enrollment: 40 (estimated)
• Ages: 12 Years and up
• Sex: All
• Sponsor: Anhui Provincial Hospital (Government)
• Locations: 1 site (Hefei, Anhui)
• Trial ID: NCT05823376 on ClinicalTrials.gov

What this trial studies

This observational study evaluates the efficacy and safety of Avatrombopag in promoting platelet recovery in patients with bone marrow failure disease following umbilical cord blood transplantation (UCBT). It is a single-center, single-arm phase II trial involving patients diagnosed with various forms of bone marrow failure who are undergoing UCBT. Participants will receive Avatrombopag treatment from the first day to the 28th day post-transplantation, with the primary outcome being the cumulative platelet implantation rate at 28 days. A total of 40 patients are planned for enrollment in this study.

Who should consider this trial

Good fit: Ideal candidates include individuals aged 12 and older with bone marrow failure diseases such as aplastic anemia, Fanconi anemia, or paroxysmal nocturnal hemoglobinuria undergoing UCBT.

Not a fit: Patients with a history of severe thrombotic events, active infections, or those who are pregnant or lactating 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 UCBT.

How similar studies have performed: While this approach is focused on a specific patient population, similar studies have shown promise in using Avatrombopag for platelet recovery, indicating potential for success.

Eligibility criteria

Inclusion Criteria:

* Age ≥12 years old, male or female;
* Patients diagnosed with bone marrow failure disease including aplastic anemia (AA), Fanconi anemia (FA), paroxysmal nocturnal hemoglobinuria (PNH) and undergoing UCBT;
* ECOG score ≤2;
* Voluntary participation in this clinical trial, patients fully understand the trial content and sign the informed consent；

Exclusion Criteria:

* Pregnant or lactating women；
* Known allergy to Avatrombopag;
* A history of severe thrombotic events or known risk factors for thrombosis or active thromboembolism requiring anticoagulation;
* A history of platelet dysfunction or bleeding prone disease or severe bleeding (requiring more than 2 units of red blood cell infusion or a hematocrit drop of ≥10%) within 7 days prior to screening;
* Chronic active hepatitis B and C;
* Repeat or multiple transplantation or multiple organ transplantation;
* HIV positive, EBV-DNA positive, CMV-DNA positive;
* Have severe infection or complicated with serious heart, liver, lung, kidney, neurological or metabolic diseases;
* A serious adverse event (Level 4 or 5) as defined by the Standard General Terminology for Adverse Events Version 4.0 occurred during the pre-treatment period;
* Participants participate in another clinical study with any exploratory drug or device within 30 days prior to baseline visit; Observational studies are allowed;
* Subjects with cognitive impairment or uncontrolled mental illness;
* Subjects and/or authorized family members to refuse treatment with Avatrombopag;
* Those who are judged by the researchers as not suitable for inclusion (such as accompanying medical history, which may affect the safety of the subjects or it is estimated that the treatment cannot be adhered to due to financial problems).

Where this trial is running

Hefei, Anhui

• The First Affiliated Hospital of University of Science and Technology of China (Anhui Provincial Hospital) — Hefei, Anhui, China (Recruiting)

Study contacts

• Principal investigator: Xiaoyu Zhu, ph.D — The First Affiliated Hospital of University of Science and Technology of China (Anhui Provincial Hospital)
• Study coordinator: Xiaoyu Zhu, ph.D

Conditions Platelet Recovery After Umbilical Cord Blood Transplantation; Avatrobopag