Platelet Recovery After Umbilical Cord Blood TransplantationAVANCT05823376
Avatrombopag for Platelet Recovery Post-UCBT in Patients With Bone Marrow Failure Disease
An observational study of Platelet Recovery After Umbilical Cord Blood Transplantation, run by Anhui Provincial Hospital at 1 site in China.
Trial summary
To evaluate the efficacy and safety of Avatrombopag for platelet recovery after unrelated cord blood transplantation (UCBT) in patients with bone marrow failure disease Read more about this study
- Who is it for?
- Ages 12 years and older
- Conditions the study is for
- Platelet Recovery After Umbilical Cord Blood Transplantation
- What is being tested?
- Avatrombopag
- How many people
- 40 participants
- Where
- 1 siteChina
- Study dates
- 4 May 2023to 28 Feb 2025
- How long will I take part?
- 28 Days
- Phase
- Observational
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 12 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Study population: Patients diagnosed with bone marrow failure and receiving umbilical cord blood transplantation
Who may be able to join
- 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；
Who cannot join
- 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;
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- 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).
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
China1 site, 1 recruiting
- Recruiting
The First Affiliated Hospital of University of Science and Technology of China (Anhui Provincial Hospital)
Hefei, Anhui, 230036
Xiaoyu Zhu, ph.D, 15255456091, xiaoyuz@ustc.cn
Aijie Huang, M.D, 15221056490, huangaijie@mail.ustc.edu.cn
About this study
Official title: 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
To evaluate the efficacy and safety of Avatrombopag for platelet recovery after unrelated cord blood transplantation (UCBT) in patients with bone marrow failure disease
Read the detailed description
Persistent thrombocytopenia is a common complication after umbilical cord blood transplantation (UCBT), which necessitates platelet transfusion and leads to increased transplant-related mortality. This is a single-center, single-arm, phase II clinical trial aimed at evaluating the efficacy and safety of Avatrombopag for platelet recovery after UCBT in patients with bone marrow failure disease (BMFD). The study was conducted in patients diagnosed with BFMD and receiving UCBT. After the subjects signed and informed and qualified, they received Avatrombopag treatment from the first day to the 28th day after transplantation. In this study, the cumulative of platelet implantation rate at 28 days after transplantation was used as the main therapeutic index, and 40 patients were planned to be enrolled.
- Being tested
- Avatrombopag
- Keywords
- Avatrobopag
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
AvatrombopagDrug
Indications for drug reduction and withdrawal: excluding blood transfusion factors, the reduction will be 20mg/d when PLT≥50×10^9/L; Drug administration will be stopped when PLT≥100×10^9/L.
| Study group | Role | What they receive |
|---|---|---|
| Avatrombopag group | The duration of the study is 4 weeks, from +1 day to +28 days after UCBT, patients will be given Avatrombopag with a recommended dose of 40mg per day. After 28 days of administration, the patients may continue, change or discontinue medication based on the patients' platelet status. Receives: Drug: Avatrombopag |
What is being measured
The primary outcome is the main question the study is designed to answer.
Time of platelet engraftment
Platelet engraftment is defined as independence from platelet transfusion for at least 7 days with a platelet count of more than ≥20 × 10^9/L.
Measured: 28 days
The cumulative incidence of platelet engraftment at 28 days after transplantation
Platelet engraftment is defined as independence from platelet transfusion for at least 7 days with a platelet count of more than ≥20 × 10^9/L.
Measured: 28 days
Secondary outcomes (7)
The cumulative incidence of platelet recovery rate at 28 days after transplantation
Platelet recovery was defined as independence from platelet transfusion for at least 7 days with a platelet count of more than ≥50×10^9/L.
Measured: 28 days
Total platelet transfusion during 4 weeks of treatment
Total platelet transfusion during 4 weeks of treatment
Measured: 28 days
neutrophil reconstitution
neutrophil engraftment time was defined as the first of three consecutive days during which the neutrophil count was at least 0.5×10^9/L.
Measured: 28 days
erythroid reconstitution
RBC engraftment time was defined as the first day of achieving a reticulocyte count greater than 1% for 3 consecutive days.
Measured: 28 days
Megakaryocyte levels in bone marrow smears 4 weeks after treatment
Megakaryocyte levels in bone marrow smears 4 weeks after treatment
Measured: 4 weeks
Time of platelet recovery
Platelet count ≥50×10^9/L for 3 consecutive days without platelet transfusions for 7 consecutive days
Measured: 100 days
Time of platelet ≥100×10^9/L.
Platelet count ≥100×10^9/L for 3 consecutive days without platelet transfusions for 7 consecutive days
Measured: 100 days
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted21 Apr 2023
- Study start4 May 2023
- Primary completion31 Jan 2025 (estimated)
- Study completion28 Feb 2025 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Platelet Recovery After Umbilical Cord Blood TransplantationStudies of the same condition, open and under way.
- More trials run by Anhui Provincial HospitalThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 40 participants (estimated)
- Target duration
- 28 Days
Sponsor and investigators
- Lead sponsor
- Anhui Provincial Hospital
- Sponsor type
- Other government
- Responsible party
- Principal investigator, Xiaoyu Zhu, Anhui Provincial Hospital
- Investigators
- Xiaoyu Zhu, ph.D, Principal investigator, The First Affiliated Hospital of University of Science and Technology of China (Anhui Provincial Hospital)
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05823376
- Sponsor's protocol ID
- AVA&MDS-001
- Acronym
- AVA
- Status last verified
- Dec 2024
- First submitted
- 24 Mar 2023
- Last updated
- 31 Dec 2024
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 31 Dec 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- 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
- ClinicalTrials.gov ID
- NCT05823376
- Acronym / sponsor ID
- AVA / AVA&MDS-001
- Keywords
- Avatrobopag
- Study type
- Observational
- Recruitment status
- Recruiting
- Enrolment
- 40 participants (estimated)
- Lead sponsor
- Anhui Provincial HospitalOther government
- Study start
- 4 May 2023 (actual)
- Primary completion
- 31 Jan 2025 (estimated)
- Study completion
- 28 Feb 2025 (estimated)
- First posted
- 21 Apr 2023 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
To evaluate the efficacy and safety of Avatrombopag for platelet recovery after unrelated cord blood transplantation (UCBT) in patients with bone marrow failure disease
Detailed description
Persistent thrombocytopenia is a common complication after umbilical cord blood transplantation (UCBT), which necessitates platelet transfusion and leads to increased transplant-related mortality. This is a single-center, single-arm, phase II clinical trial aimed at evaluating the efficacy and safety of Avatrombopag for platelet recovery after UCBT in patients with bone marrow failure disease (BMFD). The study was conducted in patients diagnosed with BFMD and receiving UCBT. After the subjects signed and informed and qualified, they received Avatrombopag treatment from the first day to the 28th day after transplantation. In this study, the cumulative of platelet implantation rate at 28 days after transplantation was used as the main therapeutic index, and 40 patients were planned to be enrolled.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Target follow-up
- 28 Days
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Avatrombopag group | – | Drug: AvatrombopagThe duration of the study is 4 weeks, from +1 day to +28 days after UCBT, patients will be given Avatrombopag with a recommended dose of 40mg per day. After 28 days of administration, the patients may continue, change or discontinue medication based on the patients' platelet status. |
Who may qualify
- Ages
- 12 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- Study population
- Patients diagnosed with bone marrow failure and receiving umbilical cord blood transplantation
Key inclusion criteria (4 listed)
- 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；
Key exclusion criteria (13 listed)
- 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;
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| AvatrombopagIndications for drug reduction and withdrawal: excluding blood transfusion factors, the reduction will be 20mg/d when PLT≥50×10^9/L; Drug administration will be stopped when PLT≥100×10^9/L. | Drug | Avatrombopag group |
Primary outcome
Time of platelet engraftment
Time frame: 28 days
Platelet engraftment is defined as independence from platelet transfusion for at least 7 days with a platelet count of more than ≥20 × 10^9/L.
The cumulative incidence of platelet engraftment at 28 days after transplantation
Time frame: 28 days
Platelet engraftment is defined as independence from platelet transfusion for at least 7 days with a platelet count of more than ≥20 × 10^9/L.
7 secondary outcomes
The cumulative incidence of platelet recovery rate at 28 days after transplantation
Time frame: 28 days
Total platelet transfusion during 4 weeks of treatment
Time frame: 28 days
neutrophil reconstitution
Time frame: 28 days
erythroid reconstitution
Time frame: 28 days
Megakaryocyte levels in bone marrow smears 4 weeks after treatment
Time frame: 4 weeks
Time of platelet recovery
Time frame: 100 days
Time of platelet ≥100×10^9/L.
Time frame: 100 days
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 31 Dec 2024.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Xiaoyu Zhu, ph.D principal investigator
The First Affiliated Hospital of University of Science and Technology of China (Anhui Provincial Hospital)
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Platelet Recovery After Umbilical Cord Blood Transplantation, run by Anhui Provincial Hospital at 1 site in China.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 4 inclusion and 13 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 31 Dec 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in China. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Xiaoyu Zhu, ph.D (15255456091, xiaoyuz@ustc.edu.cn), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Time of platelet engraftment (time frame: 28 days). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 31 Dec 2024.