Aplastic AnemiaPhase 4NCT06426043
A Prospective Study on the Treatment of Recurrent/Refractory/Intolerable NSAA With Lusutrombopag
A Phase 4 study testing Lusutrombopag in people with Aplastic Anemia, run by Peking Union Medical College Hospital at 1 site in China.
Trial summary
In a prospective, single-arm study, the efficacy and safety of Lusutrombopag in the treatment of relapsed/refractory/intolerable non-severe aplastic anemia (NSAA) were explored. Read more about this study
- Who is it for?
- Ages 18 to 90 years
- Conditions the study is for
- Aplastic Anemia
- What is being tested?
- Lusutrombopag
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 40 participants
- Where
- 1 siteChina
- Study dates
- Jun 2024to Aug 2025
- Phase
- Phase 4
- 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 18 to 90 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Participants must be at least 18 years old, male or female.
- Participants must be diagnosed with NSAA and have a refractory/relapsed/intolerable response to standard-dose cyclosporine (CsA). The definition of refractory/relapsed is patients who have been treated with sufficient doses of cyclosporine (3-5mg/kg) for at least 6 months without response or relapse. The definition of intolerable is patients who cannot tolerate CsA and have stopped treatment due to significant side effects.
- Participants must meet the following criteria at enrollment: platelets <30×109/L.
- Baseline liver and kidney function must be within 2 times of normal range.
- No active infection; no pregnancy or breastfeeding.
- Participants must agree to sign the informed consent form.
- Eastern Cooperative Oncology Group (ECOG) performance status score of 0-2.
Who cannot join
- Other causes of pancytopenia, such as myelodysplastic syndrome (MDS).
- Evidence of clonal hematopoietic system bone marrow disease (MDS, AML) with cytogenetics.
- PNH clone ≥50%.
- Received hematopoietic stem cell transplant (HSCT) prior to enrollment.
- Received ATG treatment within 6 months prior to enrollment.
- Infection or bleeding that cannot be controlled with standard therapy.
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- Allergic to ruxolitinib.
- Active HIV, HCV, or HBV infection, cirrhosis, or portal hypertension.
- Any malignant tumor within 5 years, or local basal cell carcinoma of the skin.
- History of thromboembolic events, myocardial infarction, or stroke (including antiphospholipid syndrome) and current use of anticoagulants.
- Pregnant or breastfeeding (lactating) women.
- Participated in another clinical trial within 3 months.
Trial locations and contacts
1 site in 1 country.
China1 site
Peking Union Medical College Hospital
Beijing, Beijing Municipality, 100730
QLin Hu, PhD, 15810785167, HQLin2012@163.com
Bing Bing, PhD
About this study
Official title: An Exploratory Study on the Efficacy and Safety of Lusutrombopag in the Treatment of Recurrent/Refractory/Intolerable NSAA
In a prospective, single-arm study, the efficacy and safety of Lusutrombopag in the treatment of relapsed/refractory/intolerable non-severe aplastic anemia (NSAA) were explored.
Read the detailed description
The enrolled patients: were given Lusutrombopag at 3mg/qd orally for 12 weeks (the starting dose of lusutrombopag was 3mg, taken once daily. After 2 weeks of continuous administration, the dose was increased by 3mg every 2 weeks based on the platelet count and safety of the subjects. The dose was gradually increased to 9mg/d over a total of 12 weeks). The treatment duration was at least 3 months. When the platelet increase was <20×10^9/L, the daily dose was increased by 3mg, up to a maximum of 9mg/day. When the platelet increase was ≥50×109/L and ≤200×10^9/L, the dose was maintained at the previous level. When the platelet count was ≥200×10^9/L and ≤400×10^9/L, the daily dose was reduced by 3mg. When the platelet count was >400×10^9/L, the drug could be suspended, and the dose was reduced by 3mg when the platelet count decreased to <200×10^9/L. In this case, if the lowest dose of 3mg/day was used, the drug could be suspended. Responders continued treatment for 6 months. Other TPO-RA therapies were not allowed during the study period.
- Conditions
- Aplastic Anemia
- Being tested
- Lusutrombopag
- Keywords
- Non-severe aplastic anemia, Lusutrombopag, Refractory, Recurrent
- Medical subject headings
- Anemia, Aplastic, Recurrence
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
LusutrombopagDrug
Administer lusutrombopag at 3mg/qd orally for 12 weeks (lusutrombopag starting dose is 3mg, once daily. After 2 weeks of continuous administration, the dose can be increased by 3mg every 2 weeks based on the platelet count and safety of the subject. The dose can be gradually increased to 9mg/d over a total of 12 weeks). The course should be at least 3 months. When the platelet increase is <20×109/L, the daily dose can be increased by 3mg up to a maximum of 9mg/day; when the platelet increase is ≥50×10^9/L and ≤200×10^9/L, the dose can be maintained; when the platelet count is ≥200×10^9/L and ≤400×10^9/L, the daily dose can be reduced by 3mg; when the platelet count is >400×10^9/L, the drug can be suspended and resumed when the platelet count decreases to <200×10^9/L, with the daily dose reduced by 3mg. In this case, if the lowest dose of 3mg/day is used, the drug can be suspended. Responders continue treatment until 6 months.
| Study group | Role | What they receive |
|---|---|---|
| Lusutrombopag | Experimental | Administer lusutrombopag at 3mg/qd orally for 12 weeks (lusutrombopag starting dose is 3mg, once daily. After 2 weeks of continuous administration, the dose can be increased by 3mg every 2 weeks based on the platelet count and safety of the subject. The dose can be gradually increased to 9mg/d over a total of 12 weeks). The course should be at least 3 months. When the platelet increase is <20×10^9/L, the daily dose can be increased by 3mg up to a maximum of 9mg/day; when the platelet increase is ≥50×10^9/L and ≤200×10^9/L, the dose can be maintained; when the platelet count is ≥200×10^9/L and ≤400×10^9/L, the daily dose can be reduced by 3mg; when the platelet count is >400×10^9/L, the drug can be suspended and resumed when the platelet count decreases to <200×10^9/L, with the daily dose reduced by 3mg. In this case, if the lowest dose of 3mg/day is used, the drug can be suspended. Responders continue treatment until 6 months. Receives: Drug: Lusutrombopag |
What is being measured
The primary outcome is the main question the study is designed to answer.
Overall response rate at 3 months
Proportion of patients who achieved complete response, partial response and hematological response
Measured: 3 month
Overall response rate at 6 months
Proportion of patients who achieved complete response, partial response and hematological response
Measured: 6 month
Secondary outcomes (2)
adverse event rate at 3 months
Proportion of patients with adverse eventsProportion of patients with adverse events
Measured: 3 month
adverse event rate at 6 months
Proportion of patients with adverse events
Measured: 6 month
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted23 May 2024
- Study startJun 2024 (estimated)
- Primary completionJun 2025 (estimated)
- Study completionAug 2025 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (5)Papers the sponsor has linked to this study.
- 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 4
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 40 participants (estimated)
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
- Investigators
- Bing Bing, PhD, Principal investigator, Peking Union Medical College Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06426043
- Sponsor's protocol ID
- LNA-2024
- Status last verified
- May 2024
- First submitted
- 17 May 2024
- Last updated
- 23 May 2024
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
Young NS. Aplastic Anemia. N Engl J Med. 2018 Oct 25;379(17):1643-1656. doi: 10.1056/NEJMra1413485. No abstract available.
PubMed 30354958Ruan J, Zuo W, Chen M, Yang C, Han B. Eltrombopag is effective in patients with relapse/refractory aplastic anemia-report from a single center in China. Ann Hematol. 2020 Dec;99(12):2755-2761. doi: 10.1007/s00277-020-04266-1. Epub 2020 Sep 17.
PubMed 32944791Katsube T, Wajima T, Fukuhara T, Kano T. Effects of Food and Calcium Carbonate on the Pharmacokinetics of Lusutrombopag, a Novel Thrombopoietin Receptor Agonist. Clin Ther. 2019 Sep;41(9):1747-1754.e2. doi: 10.1016/j.clinthera.2019.06.004. Epub 2019 Jul 11.
PubMed 31303281Hidaka H, Kurosaki M, Tanaka H, Kudo M, Abiru S, Igura T, Ishikawa T, Seike M, Katsube T, Ochiai T, Kimura K, Fukuhara T, Kano T, Nagata T, Tanaka K, Kurokawa M, Yamamoto K, Osaki Y, Izumi N, Imawari M. Lusutrombopag Reduces Need for Platelet Transfusion in Patients With Thrombocytopenia Undergoing Invasive Procedures. Clin Gastroenterol Hepatol. 2019 May;17(6):1192-1200. doi: 10.1016/j.cgh.2018.11.047. Epub 2018 Nov 28.
PubMed 30502505Wan Z, Chen M, Han B. Avatrombopag, a promising novel thrombopoietin receptor agonist for refractory/relapsed/intolerant non-severe aplastic anemia: a phase 2 single-arm clinical trial. Ann Med. 2023 Dec;55(1):2224044. doi: 10.1080/07853890.2023.2224044.
PubMed 37318085
Study at a glance
From the registry record, last updated by the sponsor on 23 May 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Exploratory Study on the Efficacy and Safety of Lusutrombopag in the Treatment of Recurrent/Refractory/Intolerable NSAA
- ClinicalTrials.gov ID
- NCT06426043
- Acronym / sponsor ID
- LNA-2024
- Conditions
- Aplastic Anemia
- Keywords
- Non-severe aplastic anemia, Lusutrombopag, Refractory, Recurrent
- Phase
- Phase 4
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 40 participants (estimated)
- Lead sponsor
- Peking Union Medical College HospitalUniversity, hospital or other
- Study start
- Jun 2024 (estimated)
- Primary completion
- Jun 2025 (estimated)
- Study completion
- Aug 2025 (estimated)
- First posted
- 23 May 2024 (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.
In a prospective, single-arm study, the efficacy and safety of Lusutrombopag in the treatment of relapsed/refractory/intolerable non-severe aplastic anemia (NSAA) were explored.
Detailed description
The enrolled patients: were given Lusutrombopag at 3mg/qd orally for 12 weeks (the starting dose of lusutrombopag was 3mg, taken once daily. After 2 weeks of continuous administration, the dose was increased by 3mg every 2 weeks based on the platelet count and safety of the subjects. The dose was gradually increased to 9mg/d over a total of 12 weeks). The treatment duration was at least 3 months. When the platelet increase was <20×10^9/L, the daily dose was increased by 3mg, up to a maximum of 9mg/day. When the platelet increase was ≥50×109/L and ≤200×10^9/L, the dose was maintained at the previous level. When the platelet count was ≥200×10^9/L and ≤400×10^9/L, the daily dose was reduced by 3mg. When the platelet count was >400×10^9/L, the drug could be suspended, and the dose was reduced by 3mg when the platelet count decreased to <200×10^9/L. In this case, if the lowest dose of 3mg/day was used, the drug could be suspended. Responders continued treatment for 6 months. Other TPO-RA therapies were not allowed during the study period.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Lusutrombopag | Experimental | Drug: LusutrombopagAdminister lusutrombopag at 3mg/qd orally for 12 weeks (lusutrombopag starting dose is 3mg, once daily. After 2 weeks of continuous administration, the dose can be increased by 3mg every 2 weeks based on the platelet count and safety of the subject. The dose can be gradually increased to 9mg/d over a total of 12 weeks). The course should be at least 3 months. When the platelet increase is <20×10^9/L, the daily dose can be increased by 3mg up to a maximum of 9mg/day; when the platelet increase is ≥50×10^9/L and ≤200×10^9/L, the dose can be maintained; when the platelet count is ≥200×10^9/L and ≤400×10^9/L, the daily dose can be reduced by 3mg; when the platelet count is >400×10^9/L, the drug can be suspended and resumed when the platelet count decreases to <200×10^9/L, with the daily dose reduced by 3mg. In this case, if the lowest dose of 3mg/day is used, the drug can be suspended. Responders continue treatment until 6 months. |
Who may qualify
- Ages
- 18 to 90 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (7 listed)
- Participants must be at least 18 years old, male or female.
- Participants must be diagnosed with NSAA and have a refractory/relapsed/intolerable response to standard-dose cyclosporine (CsA). The definition of refractory/relapsed is patients who have been treated with sufficient doses of cyclosporine (3-5mg/kg) for at least 6 months without response or relapse. The definition of intolerable is patients who cannot tolerate CsA and have stopped treatment due to significant side effects.
- Participants must meet the following criteria at enrollment: platelets <30×109/L.
- Baseline liver and kidney function must be within 2 times of normal range.
- No active infection; no pregnancy or breastfeeding.
Key exclusion criteria (12 listed)
- Other causes of pancytopenia, such as myelodysplastic syndrome (MDS).
- Evidence of clonal hematopoietic system bone marrow disease (MDS, AML) with cytogenetics.
- PNH clone ≥50%.
- Received hematopoietic stem cell transplant (HSCT) prior to enrollment.
- Received ATG treatment within 6 months prior to enrollment.
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 |
|---|---|---|
| LusutrombopagAdminister lusutrombopag at 3mg/qd orally for 12 weeks (lusutrombopag starting dose is 3mg, once daily. After 2 weeks of continuous administration, the dose can be increased by 3mg every 2 weeks based on the platelet count and safety of the subject. The dose can be gradually increased to 9mg/d over a total of 12 weeks). The course should be at least 3 months. When the platelet increase is <20×109/L, the daily dose can be increased by 3mg up to a maximum of 9mg/day; when the platelet increase is ≥50×10^9/L and ≤200×10^9/L, the dose can be maintained; when the platelet count is ≥200×10^9/L and ≤400×10^9/L, the daily dose can be reduced by 3mg; when the platelet count is >400×10^9/L, the drug can be suspended and resumed when the platelet count decreases to <200×10^9/L, with the daily dose reduced by 3mg. In this case, if the lowest dose of 3mg/day is used, the drug can be suspended. Responders continue treatment until 6 months. | Drug | Lusutrombopag |
Primary outcome
Overall response rate at 3 months
Time frame: 3 month
Proportion of patients who achieved complete response, partial response and hematological response
Overall response rate at 6 months
Time frame: 6 month
Proportion of patients who achieved complete response, partial response and hematological response
2 secondary outcomes
adverse event rate at 3 months
Time frame: 3 month
adverse event rate at 6 months
Time frame: 6 month
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 23 May 2024.
- Background: PubMed 30354958Young NS. Aplastic Anemia. N Engl J Med. 2018 Oct 25;379(17):1643-1656. doi: 10.1056/NEJMra1413485. No abstract available.
- Background: PubMed 32944791Ruan J, Zuo W, Chen M, Yang C, Han B. Eltrombopag is effective in patients with relapse/refractory aplastic anemia-report from a single center in China. Ann Hematol. 2020 Dec;99(12):2755-2761. doi: 10.1007/s00277-020-04266-1. Epub 2020 Sep 17.
- Background: PubMed 31303281Katsube T, Wajima T, Fukuhara T, Kano T. Effects of Food and Calcium Carbonate on the Pharmacokinetics of Lusutrombopag, a Novel Thrombopoietin Receptor Agonist. Clin Ther. 2019 Sep;41(9):1747-1754.e2. doi: 10.1016/j.clinthera.2019.06.004. Epub 2019 Jul 11.
- Background: PubMed 30502505Hidaka H, Kurosaki M, Tanaka H, Kudo M, Abiru S, Igura T, Ishikawa T, Seike M, Katsube T, Ochiai T, Kimura K, Fukuhara T, Kano T, Nagata T, Tanaka K, Kurokawa M, Yamamoto K, Osaki Y, Izumi N, Imawari M. Lusutrombopag Reduces Need for Platelet Transfusion in Patients With Thrombocytopenia Undergoing Invasive Procedures. Clin Gastroenterol Hepatol. 2019 May;17(6):1192-1200. doi: 10.1016/j.cgh.2018.11.047. Epub 2018 Nov 28.
- Background: PubMed 37318085Wan Z, Chen M, Han B. Avatrombopag, a promising novel thrombopoietin receptor agonist for refractory/relapsed/intolerant non-severe aplastic anemia: a phase 2 single-arm clinical trial. Ann Med. 2023 Dec;55(1):2224044. doi: 10.1080/07853890.2023.2224044.
- Individual participant dataSharing planned: No
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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.
Bing Bing, PhD principal investigator
Peking Union Medical College Hospital
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 4 study testing Lusutrombopag in people with Aplastic Anemia, 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 7 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: status not confirmed, as of the sponsor's last update on 23 May 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.
What does this phase mean?
Phase 4 studies take place after a treatment is approved, to learn more about its long-term benefits and risks in everyday use.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Bing Bing, PhD (13601059938, Hanbing_li@sina.com.cn), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Overall response rate at 3 months (time frame: 3 month). 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?
On the ClinicalTrials.gov record for NCT06426043, which also lists 5 linked publications.
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 23 May 2024.