Aplastic AnemiaNCT05571332
Efficacy and Safety of Avatrombopag Combined With IST for the Treatment of HAAA and SAA With Abnormal Liver Function
A study testing Avatrombopag 20 MG Oral Tablet in people with Aplastic Anemia, run by Institute of Hematology & Blood Diseases Hospital, China at 1 site in China.
Trial summary
This is a multicenter, single-arm clinical study. The objective was to evaluate the efficacy and safety of Avatrombopag combined with IST in very/sever aplastic anemia patients with abnormal liver function or HAAA patients treated for the first time. Read more about this study
- Who is it for?
- Ages 18 to 70 years
- Conditions the study is for
- Aplastic Anemia
- What is being tested?
- Avatrombopag 20 MG Oral Tablet
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 39 participants
- Where
- 1 siteChina
- Study dates
- 28 Jun 2022to 28 Jun 2024
- Phase
- Interventional
- 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 70 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- patients with V/SAA with a definite diagnosis.
- age between 18-70 years, male or female.
- Subjects must complete all screening assessments as outlined in the trial protocol.
- Able to swallow or administer the drug orally.
- No prior application of TPO receptor agonists (including Thrombopoietin, Eltrombopag, Hetrombopag, etc.) or application of TPO receptor agonists for treatment with ≤ 5 total doses and ≤ 7 days of TPO receptor agonist drugs such as Eltrombopag, Hetrombopag, etc.
- Diagnosis as HAAA or abnormal liver function. ALT and AST more than 1.5 times of upper limit.
- Informed consent must be signed prior to the start of all specific study procedures, in consideration of the patient's condition, or by a member of the patient's immediate family if the patient's signature is not conducive to the treatment of the condition.
Who cannot join
- Known diagnosis of congenital hematopoietic failure disorders (e.g. Fanconi anemia) and other causes of allogeneic cytopenias and bone marrow hypoproliferative disorders (e.g. hemolytic PNH, hypoproliferative MDS/AML, autoantibody-mediated allogeneic cytopenias, etc.);
- Patients with uncontrolled bleeding and/or infection despite standard treatment.
- Patients with previous history of hematopoietic stem cell transplantation; previous history of thrombosis.
- Patients with concurrent malignancy or potential cancer on immunosuppressive therapy.
- Those who are considered unsuitable for enrollment by the investigator.
Trial locations and contacts
1 site in 1 country.
China1 site, 1 recruiting
- Recruiting
Institute of Hematology & Blood Diseases Hospital, Chinese Academy of Medical Sciences
Tianjin, Tianjin Municipality, 300020
Fengkui Zhang, Doctor, 8602223909229, fkzhang@ihcams.ac.cn
Liping Jing, Doctor, 8602223909223, jingliping@ihcams.ac.cn
About this study
This is a multicenter, single-arm clinical study. The objective was to evaluate the efficacy and safety of Avatrombopag combined with IST in very/sever aplastic anemia patients with abnormal liver function or HAAA patients treated for the first time. The design was: Patients received p-ATG for 5 consecutive days (day 1-5), at a dose of 20 mg/kg/day. Cyclosporine 3 mg/kg orally in two divided doses, with cyclosporine trough concentrations maintained at 200-250 ng/ml for 3 months to achieve maximum efficacy, and Avatrombopag, which was administered in the dose of 40 mg orally once daily for a total of 12 weeks. Thirty-nine patients are expected to be enrolled in this study. Evaluation endpoint: complete response rate at 12 weeks of treatment.
Read the detailed description
This is a multicenter, single-arm clinical study to evaluate the efficacy and safety of Avatrombopag combined with IST as the first-line regimen for aplastic anemia. The patients are diagnosed as hepatitis associated with very sever/sever aplastic anemia(V/SAA) or V/SAA with abnormal liver function before treatment.
Patients received p-ATG for 5 consecutive days (day 1-5), at a dose of 20 mg/kg/day. CSA is started at 3 mg/kg orally in two doses. Concentrations maintained at 200-250 ng/ml to achieve maximum efficacy and then tapered by 25 mg every 3 months; Avatrombopag: 40 mg orally once daily for a total of 12 weeks. A total of 39 patients were expected to be included.Complete response rate at 12 weeks of treatment and adverse events are the evaluation endpoint.Secondary study endpoints were: ORR at 12 , CRR and ORR at 24 weeks, survival, and clonal evolution in follow-up.
- Conditions
- Aplastic Anemia
- Being tested
- Avatrombopag 20 MG Oral Tablet
- Keywords
- Avatrombopag, aplastic anemia, abnormal liver function, hepatitis
- Medical subject headings
- Anemia, Aplastic, Liver Diseases, Hepatitis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Avatrombopag 20 MG Oral TabletDrug
Also called porcine ATG, Cyclosporine(CsA)
p-ATG and CsA in combination with Avatrombopag to treat
| Study group | Role | What they receive |
|---|---|---|
| Avatrombopag+CsA+ p-ATG | Experimental | Patients received p-ATG for 5 consecutive days (day 1-5), at a dose of 20 mg/kg/day. CSA is started at 3 mg/kg orally in two doses. Concentrations maintained at 200-250 ng/ml to achieve maximum efficacy and then tapered by 25 mg every 3 months; Avatrombopag: 40 mg orally once daily for a total of 12 weeks. A total of 39 patients were expected to be included. Receives: Drug: Avatrombopag 20 MG Oral Tablet |
What is being measured
The primary outcome is the main question the study is designed to answer.
CR rate at 12 weeks of treatment
Percentage of the total number of patients receiving treatment who received a complete response at 12 weeks of treatment
Measured: 12 weeks of treatment
ncidence of Treatment-Emergent Adverse Events as assessed by information on Common Toxicity Criteria (CTC) AE grading at 12 weeks of treatment
Incidence of Treatment-Emergent AE by CTCAE
Measured: 12 weeks of treatment
Secondary outcomes (1)
OR rate at 12 weeks of treatment
Percentage of the total number of patients receiving treatment who received a response at 12 weeks of treatment
Measured: 12 weeks of treatment
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted7 Oct 2022
- Study start28 Jun 2022
- Primary completion28 Dec 2023 (estimated)
- Study completion28 Jun 2024 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (2)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 Institute of Hematology & Blood Diseases Hospital, ChinaThe 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 N/A
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 39 participants (estimated)
Sponsor and investigators
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Wenrui Yang, Study director, Anemia Therapeutic center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05571332
- Sponsor's protocol ID
- IIT2021008-EC-1(2)
- Status last verified
- Aug 2022
- First submitted
- 28 Aug 2022
- Last updated
- 7 Oct 2022
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Yes: You can ask for the researcher after completing the experiment
Publications
Young NS, Kaufman DW. The epidemiology of acquired aplastic anemia. Haematologica. 2008 Apr;93(4):489-92. doi: 10.3324/haematol.12855. No abstract available.
PubMed 18379007Scheinberg P. Activity of eltrombopag in severe aplastic anemia. Hematology Am Soc Hematol Educ Program. 2018 Nov 30;2018(1):450-456. doi: 10.1182/asheducation-2018.1.450.
PubMed 30504345
Study at a glance
From the registry record, last updated by the sponsor on 7 Oct 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Efficacy and Safety of Avatrombopag Combined With IST for the Treatment of HAAA and SAA With Abnormal Liver Function
- ClinicalTrials.gov ID
- NCT05571332
- Acronym / sponsor ID
- IIT2021008-EC-1(2)
- Conditions
- Aplastic Anemia
- Keywords
- Avatrombopag, aplastic anemia, abnormal liver function, hepatitis
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 39 participants (estimated)
- Lead sponsor
- Institute of Hematology & Blood Diseases Hospital, ChinaUniversity, hospital or other
- Study start
- 28 Jun 2022 (actual)
- Primary completion
- 28 Dec 2023 (estimated)
- Study completion
- 28 Jun 2024 (estimated)
- First posted
- 7 Oct 2022 (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 multicenter, single-arm clinical study. The objective was to evaluate the efficacy and safety of Avatrombopag combined with IST in very/sever aplastic anemia patients with abnormal liver function or HAAA patients treated for the first time. The design was: Patients received p-ATG for 5 consecutive days (day 1-5), at a dose of 20 mg/kg/day. Cyclosporine 3 mg/kg orally in two divided doses, with cyclosporine trough concentrations maintained at 200-250 ng/ml for 3 months to achieve maximum efficacy, and Avatrombopag, which was administered in the dose of 40 mg orally once daily for a total of 12 weeks. Thirty-nine patients are expected to be enrolled in this study. Evaluation endpoint: complete response rate at 12 weeks of treatment.
Detailed description
This is a multicenter, single-arm clinical study to evaluate the efficacy and safety of Avatrombopag combined with IST as the first-line regimen for aplastic anemia. The patients are diagnosed as hepatitis associated with very sever/sever aplastic anemia(V/SAA) or V/SAA with abnormal liver function before treatment.
Patients received p-ATG for 5 consecutive days (day 1-5), at a dose of 20 mg/kg/day. CSA is started at 3 mg/kg orally in two doses. Concentrations maintained at 200-250 ng/ml to achieve maximum efficacy and then tapered by 25 mg every 3 months; Avatrombopag: 40 mg orally once daily for a total of 12 weeks. A total of 39 patients were expected to be included.Complete response rate at 12 weeks of treatment and adverse events are the evaluation endpoint.Secondary study endpoints were: ORR at 12 , CRR and ORR at 24 weeks, survival, and clonal evolution in follow-up.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Avatrombopag+CsA+ p-ATG | Experimental | Drug: Avatrombopag 20 MG Oral TabletPatients received p-ATG for 5 consecutive days (day 1-5), at a dose of 20 mg/kg/day. CSA is started at 3 mg/kg orally in two doses. Concentrations maintained at 200-250 ng/ml to achieve maximum efficacy and then tapered by 25 mg every 3 months; Avatrombopag: 40 mg orally once daily for a total of 12 weeks. A total of 39 patients were expected to be included. |
Who may qualify
- Ages
- 18 to 70 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (7 listed)
- patients with V/SAA with a definite diagnosis.
- age between 18-70 years, male or female.
- Subjects must complete all screening assessments as outlined in the trial protocol.
- Able to swallow or administer the drug orally.
- No prior application of TPO receptor agonists (including Thrombopoietin, Eltrombopag, Hetrombopag, etc.) or application of TPO receptor agonists for treatment with ≤ 5 total doses and ≤ 7 days of TPO receptor agonist drugs such as Eltrombopag, Hetrombopag, etc.
Key exclusion criteria (5 listed)
- Known diagnosis of congenital hematopoietic failure disorders (e.g. Fanconi anemia) and other causes of allogeneic cytopenias and bone marrow hypoproliferative disorders (e.g. hemolytic PNH, hypoproliferative MDS/AML, autoantibody-mediated allogeneic cytopenias, etc.);
- Patients with uncontrolled bleeding and/or infection despite standard treatment.
- Patients with previous history of hematopoietic stem cell transplantation; previous history of thrombosis.
- Patients with concurrent malignancy or potential cancer on immunosuppressive therapy.
- Those who are considered unsuitable for enrollment by the investigator.
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 |
|---|---|---|
| Avatrombopag 20 MG Oral TabletAlso: porcine ATG, Cyclosporine(CsA)p-ATG and CsA in combination with Avatrombopag to treat | Drug | Avatrombopag+CsA+ p-ATG |
Primary outcome
CR rate at 12 weeks of treatment
Time frame: 12 weeks of treatment
Percentage of the total number of patients receiving treatment who received a complete response at 12 weeks of treatment
ncidence of Treatment-Emergent Adverse Events as assessed by information on Common Toxicity Criteria (CTC) AE grading at 12 weeks of treatment
Time frame: 12 weeks of treatment
Incidence of Treatment-Emergent AE by CTCAE
1 secondary outcome
OR rate at 12 weeks of treatment
Time frame: 12 weeks of treatment
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 7 Oct 2022.
- Results publication: PubMed 18379007Young NS, Kaufman DW. The epidemiology of acquired aplastic anemia. Haematologica. 2008 Apr;93(4):489-92. doi: 10.3324/haematol.12855. No abstract available.
- Results publication: PubMed 30504345Scheinberg P. Activity of eltrombopag in severe aplastic anemia. Hematology Am Soc Hematol Educ Program. 2018 Nov 30;2018(1):450-456. doi: 10.1182/asheducation-2018.1.450.
- Individual participant dataSharing planned: Yes. You can ask for the researcher after completing the experiment
Related trials for Aplastic Anemia
RecruitingPhase 1/2
The Safety and Efficacy of Daratumumab in Patients With Refractory Aplastic Anemia
Aplastic Anemia, Refractory
RecruitingPhase 1/2
Pacritinib, a Kinase Inhibitor of CSF1R, IRAK1, JAK2, and FLT3, in Adults and Pediatric Participants 12 Years of Age or Older With Myelodysplastic Syndromes or Myelodysplastic/Myeloproliferative Neoplasms
Myelodysplastic Syndromes
RecruitingObservational study
Defining the Natural History of Squamous Cell Carcinoma in Fanconi Anemia
Fanconi Anemia, Inherited Bone Marrow Failure Syndrome
Not yet recruitingObservational study
Clinical Genetics Branch Eligibility Screening Survey
Melanoma, Li-Fraumeni Syndrome
RecruitingObservational study
HPC Offered for PRESERVE Expansion
Bone Marrow Transplant (BMT)
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.
Wenrui Yang study director
Anemia Therapeutic center
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing Avatrombopag 20 MG Oral Tablet in people with Aplastic Anemia, run by Institute of Hematology & Blood Diseases Hospital, China 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 5 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 7 Oct 2022. 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?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Fengkui Zhang, Dr. (+8602223909229, zhangfenkui@ihcams.ac.cn), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
CR rate at 12 weeks of treatment (time frame: 12 weeks of treatment). 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 NCT05571332, which also lists 2 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 7 Oct 2022.