Aplastic AnemiaPhase 2NCT04403321
Efficacy and Safety of Eltrombopag + Tacrolimus in Chinese Refractory or Relapsed Aplastic Anemia Patients
A Phase 2 study testing Tacrolimus in people with Aplastic Anemia and Drug Effect, run by Peking Union Medical College Hospital at 1 site in China.
Trial summary
This is a randomized, open-label, phase II study to compare the efficacy of eltrombopag combined with tacrolimus to eltrombopag alone in Chinese subjects with refractory or relapsed aplastic anemia. The safety would also be evaluated. Read more about this study
- Who is it for?
- Ages 14 to 85 years
- Conditions the study is for
- Aplastic Anemia, Drug Effect
- What is being tested?
- Tacrolimus
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 114 participants
- Where
- 1 siteChina
- Study dates
- 1 Jul 2020to 31 Jul 2022
- Phase
- Phase 2
- Are there results?
- None posted in the registry
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 14 to 85 years
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Patient with a previous diagnosis of aplastic anemia and had no response or relapsed following at least one treatment course in a period time of ≥ 6 months of immunosuppression containing CsA or CsA+anti-thymocyte globulin (ATG);
- Current diagnosis of aplastic anemia by bone marrow biopsy;
- did not receive HSCT nor were HSCT candidates;
- Patient has an Eastern Cooperative Oncology Group (ECOG) performance status 0-2;
- Patient with QTcF (Fridericia's QT correction formula) at screening <450 msec, or <480 msec with bundle branch block, as determined via the mean of a triplicate ECG and assessed at site.
- Subjects are able to understand and comply with protocol requirements and instructions and have signed and dated informed consent.
Who cannot join
- Congenital aplastic anemia;
- Presence of chromosomal aberration;
- Evidence of a clonal hematologic bone marrow disorder on cytogenetics;
- Have any concomitant malignancies and must be fully recovered from treatment for any other malignancy and have been disease-free for 5 years;
- AST or ALT ≥3 times the upper limit of normal;
- Serum creatinine, total bilirubin, or alkaline phosphatase >1.5 x ULN;
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- Cardiac disorder (NYHA) functional classification Grade II/III/IV;
- Past history of thromboembolic event (including anti-phospholipid antibody syndrome) and current use of anticoagulants;
- Infection not adequately responding to appropriate therapy;
- Other known or suspected underlying primary immunodeficiency;
- Prior treatment with eltrombopag, romiplostim, or any other TPO (thrombopoietin) receptor agonist;
- Pregnant or nursing (lactating) woman;
Trial locations and contacts
1 site in 1 country.
About this study
This is a randomized, open-label, phase II study to compare the efficacy of eltrombopag combined with tacrolimus to eltrombopag alone in Chinese subjects with refractory or relapsed aplastic anemia. The safety would also be evaluated. Patients would be randomized to receive eltrombopag alone or eltrombopag combined with tacrolimus. Treatment with eltrombopag will be started at 25 mg/day and increased by 25 mg/day every 2 weeks according to the platelet count up to 150 mg/day, or the best response was achieved. Tacrolimus will be given at 1mg bid with the target trough concentration of 4-10 ng/mL throughout the study. The hematological response rate and safety will be recorded and compared at 3, 6 months and 1 year after starting the study treatment (Week 13, 26 and 52).
- Conditions
- Aplastic Anemia, Drug Effect
- Being tested
- Tacrolimus, Placebo (for Tacrolimus)
- Medical subject headings
- Anemia, Aplastic
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
TacrolimusDrug
Also called experimental
Tacrolimus will be given at 1mg bid with the target trough concentration to be 4-10 ng/mL.
Placebo (for Tacrolimus)Drug
Also called control
placebo will be given at 1mg bid.
| Study group | Role | What they receive |
|---|---|---|
| Eltrombopag | Placebo comparator | Eltrombopag and the placebo would be applied. Eltrombopag will be started at 25 mg/day and increased by 25 mg/day every 2 weeks according to the platelet count up to 150 mg/day, or the best response was achieved. Receives: Drug: Placebo (for Tacrolimus) |
| Eltrombopag + Tacrolimus | Experimental | Eltrombopag and tacrolimus would be applied. Eltrombopag will be started at 25 mg/day and increased by 25 mg/day every 2 weeks according to the platelet count up to 150 mg/day, or the best response was achieved. Tacrolimus will be given at 1mg bid with the target trough concentration of 4-10 ng/mL throughout the study. Receives: Drug: Tacrolimus |
What is being measured
The primary outcome is the main question the study is designed to answer.
ORR at 6 Months
Overall Response Rate (ORR) Defined as the Number of Participants Who Met the Criteria of Either Complete Response (CR) or Partial Response (PR) at Week 26
Measured: Week 26
Secondary outcomes (5)
ORR at 3 Months
ORR will be calculated after 3 months of treatment by measuring platelet, reticulocyte, neutrophil and transfusion independence.
Measured: Week 14
Changes in Haemoglobin in the Absence of Red Blood Cells Transfusion
The change in hematology values ( haemoglobin) were evaluated
Measured: Week 26
Changes in Platelet in the Absence of Platelet Transfusion
The change in hematology values (platelet) were evaluated
Measured: Week 26
Duration of hematologic response
Time from the date of the start of the first response to the date of first relapse defined as again meeting criteria for aplastic anemia
Measured: by 6 months (all patients), at 24 months (responders only)
Percentage of patients with clonal evolution to myelodysplasia, PNH, acute leukemia
Clonal evolution to myelodysplasia is defined as a new marrow cytogenic abnormality with or without characteristic dysplastic marrow findings. Evolution to leukemia is defined as greater than 20% peripheral blood and/or marrow blasts. Evolution to paroxysmal nocturnal hemoglobinuria (PNH) is defined as a clone at baseline < 10% that rose to greater than 50% on study.
Measured: 12 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted27 May 2020
- Study start1 Jul 2020
- Primary completion30 Jul 2022
- Study completion31 Jul 2022
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Aplastic AnemiaStudies of the same condition, open and under way.
- More trials run by Peking Union Medical College 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
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 114 participants (actual)
Sponsor and investigators
- Lead sponsor
- Peking Union Medical College Hospital
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Bing Han, Peking Union Medical College Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04403321
- Sponsor's protocol ID
- ELT-1
- Status last verified
- Jan 2023
- First submitted
- 21 May 2020
- Last updated
- 19 Jan 2023
- Results posted
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Yes: individual participant data would be accepted upon request
Study at a glance
From the registry record, last updated by the sponsor on 19 Jan 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Efficacy and Safety of Eltrombopag + Tacrolimus in Chinese Refractory or Relapsed Aplastic Anemia Patients
- ClinicalTrials.gov ID
- NCT04403321
- Acronym / sponsor ID
- ELT-1
- Conditions
- Aplastic Anemia, Drug Effect
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 114 participants (actual)
- Lead sponsor
- Peking Union Medical College HospitalUniversity, hospital or other
- Study start
- 1 Jul 2020 (actual)
- Primary completion
- 30 Jul 2022 (actual)
- Study completion
- 31 Jul 2022 (actual)
- First posted
- 27 May 2020 (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.
This is a randomized, open-label, phase II study to compare the efficacy of eltrombopag combined with tacrolimus to eltrombopag alone in Chinese subjects with refractory or relapsed aplastic anemia. The safety would also be evaluated. Patients would be randomized to receive eltrombopag alone or eltrombopag combined with tacrolimus. Treatment with eltrombopag will be started at 25 mg/day and increased by 25 mg/day every 2 weeks according to the platelet count up to 150 mg/day, or the best response was achieved. Tacrolimus will be given at 1mg bid with the target trough concentration of 4-10 ng/mL throughout the study. The hematological response rate and safety will be recorded and compared at 3, 6 months and 1 year after starting the study treatment (Week 13, 26 and 52).
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Eltrombopag | Placebo comparator | Drug: Placebo (for Tacrolimus)Eltrombopag and the placebo would be applied. Eltrombopag will be started at 25 mg/day and increased by 25 mg/day every 2 weeks according to the platelet count up to 150 mg/day, or the best response was achieved. |
| Eltrombopag + Tacrolimus | Experimental | Drug: TacrolimusEltrombopag and tacrolimus would be applied. Eltrombopag will be started at 25 mg/day and increased by 25 mg/day every 2 weeks according to the platelet count up to 150 mg/day, or the best response was achieved. Tacrolimus will be given at 1mg bid with the target trough concentration of 4-10 ng/mL throughout the study. |
Who may qualify
- Ages
- 14 to 85 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (6 listed)
- Patient with a previous diagnosis of aplastic anemia and had no response or relapsed following at least one treatment course in a period time of ≥ 6 months of immunosuppression containing CsA or CsA+anti-thymocyte globulin (ATG);
- Current diagnosis of aplastic anemia by bone marrow biopsy;
- did not receive HSCT nor were HSCT candidates;
- Patient has an Eastern Cooperative Oncology Group (ECOG) performance status 0-2;
- Patient with QTcF (Fridericia's QT correction formula) at screening <450 msec, or <480 msec with bundle branch block, as determined via the mean of a triplicate ECG and assessed at site.
Key exclusion criteria (12 listed)
- Congenital aplastic anemia;
- Presence of chromosomal aberration;
- Evidence of a clonal hematologic bone marrow disorder on cytogenetics;
- Have any concomitant malignancies and must be fully recovered from treatment for any other malignancy and have been disease-free for 5 years;
- AST or ALT ≥3 times the upper limit of normal;
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 |
|---|---|---|
| TacrolimusAlso: experimentalTacrolimus will be given at 1mg bid with the target trough concentration to be 4-10 ng/mL. | Drug | Eltrombopag + Tacrolimus |
| Placebo (for Tacrolimus)Also: controlplacebo will be given at 1mg bid. | Drug | Eltrombopag |
Primary outcome
ORR at 6 Months
Time frame: Week 26
Overall Response Rate (ORR) Defined as the Number of Participants Who Met the Criteria of Either Complete Response (CR) or Partial Response (PR) at Week 26
5 secondary outcomes
ORR at 3 Months
Time frame: Week 14
Changes in Haemoglobin in the Absence of Red Blood Cells Transfusion
Time frame: Week 26
Changes in Platelet in the Absence of Platelet Transfusion
Time frame: Week 26
Duration of hematologic response
Time frame: by 6 months (all patients), at 24 months (responders only)
Percentage of patients with clonal evolution to myelodysplasia, PNH, acute leukemia
Time frame: 12 months
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 19 Jan 2023.
- Individual participant dataSharing planned: Yes. individual participant data would be accepted upon request
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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. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Tacrolimus in people with Aplastic Anemia and Drug Effect, run by Peking Union Medical College 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 6 inclusion and 12 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: finished, as of the sponsor's last update on 19 Jan 2023. 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. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
ORR at 6 Months (time frame: Week 26). 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 19 Jan 2023; AzTrials last refreshed its copy on 8 Nov 2026.