Severe Aplastic AnemiaPhase 2NCT05720234
Avatrombopag Combined With IST as First-line Treatment for SAA
A Phase 2 study testing avatrombopag in people with Severe Aplastic Anemia, run by Institute of Hematology & Blood Diseases Hospital, China at 1 site in China.
Trial summary
This single-center study aims to evaluate the early efficacy and safety of avatrombopag combined with immunosuppressive therapy (IST) in the first-line treatment of severe aplastic anemia (SAA). Read more about this study
- Who is it for?
- Ages 12 to 60 years
- Conditions the study is for
- Severe Aplastic Anemia
- What is being tested?
- avatrombopag
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 53 participants
- Where
- 1 siteChina
- Study dates
- 10 Nov 2022to 30 Nov 2024
- 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 12 to 60 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Patients with newly diagnosed severe aplastic anemia.
- Men and women aged between 12 and 60.
- Subjects must complete all screening assessments as outlined in the test protocol.
- Able to swallow or administer orally.
- Before the start of the research procedure, the patient or guardian should fully understand the research procedure and purpose and sign the informed consent form. If the patient's signature is not conducive to the treatment of the disease, the patient's immediate family should sign the informed consent form.
Who cannot join
- Congenital bone marrow failure (eg. Fanconi anemia).
- Accompanied by cytogenetic cloning changes (chromosomal karyotype and FISH detection found somatic cloning abnormalities; Simple -Y abnormality can be included in this study;) .
- ATG or middle/high-dose cyclophosphamide was used in the past.
- Previous treatment with cyclosporine or tacrolimus > 6 months.
- The total course of treatment with TPO receptor agonists (including thrombopoietin, eltrombopag,hetrombopag and avatrombopag) was more than 1 month.
- Serious infectious diseases (tuberculosis without effective control, pulmonary aspergillosis, viral infections).
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- AIDS patients.
- Pregnant or breastfeeding, fertile but unwilling to take effective contraceptive measures.
- Patients with malignant tumors who are not suitable for ATG treatment.
- A newly diagnosed history of cardio/cerebral vascular thrombosis within 12 months.
- Those who are assessed as unsuitable for inclusion by the investigator.
Trial locations and contacts
1 site in 1 country.
China1 site, 1 recruiting
- Recruiting
Institute of Hematology & Blood Diseases Hospital
Tianjin, Tianjin Municipality, 300020
Xin Zhao, M.D, 13820961539, zhaoxin@ihcams.ac.cn
About this study
Official title: The Efficacy and Safety Study of Avatrombopag Combined With IST as First-line Treatment for Severe Aplastic Anemia, A Single-arm, Phase II Clinical Study
This single-center study aims to evaluate the early efficacy and safety of avatrombopag combined with immunosuppressive therapy (IST) in the first-line treatment of severe aplastic anemia (SAA).
Read the detailed description
This is a single center, single arm, phase II clinical study. Fifty-three patients will be enrolled.
Treatment protocol is as follows: 1) Anti-human thymocyte porcine immunoglobulin (P-ATG 20mg/kg/d) or rabbit anti human thymocyte globulin (R-ATG 3.0mg/kg/d) was administered intravenously for 5 days; 2) Cyclosporine (CSA) is given at 3-5 mg/kg.d in divided doses for at least 6 months. The trough concentration is maintained at 150-250 ng/ml. 3) Avatrombopag is given orally at 60 mg once a day for patients with body weight ≥ 50 kg, and 40 mg orally once a day for patients with body weight<50 kg, for a total of 12 weeks.
- Conditions
- Severe Aplastic Anemia
- Being tested
- avatrombopag
- Keywords
- avatrombopag
- Medical subject headings
- Anemia, Aplastic
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
avatrombopagDrug
Also called AVA, APAG
Patients with body weight ≥50kg were given 60mg/day and patients with body weight < 50kg were given 40mg/day for 12 weeks.
| Study group | Role | What they receive |
|---|---|---|
| Treatment group | Experimental | 53 subjects will be enrolled with the indicated treatment dose of avatrombopag. Receives: Drug: avatrombopag |
What is being measured
The primary outcome is the main question the study is designed to answer.
Treatment response
Percentage of patients who achieves complete response(CR) at 12 weeks.
Measured: From the start of study treatment (Day1) to end of week 12.
Secondary outcomes (6)
Treatment response
Percentage of patients achieving hematologic response (OR) at 12 weeks.
Measured: From the start of study treatment (Day1) to end of week 12.
Treatment response
Percentage of patients achieving hematologic response and complete response (OR and CR) at 24 weeks.
Measured: From the start of study treatment (Day1) to end of week 24.
Supportive treatment
The time of red blood cell or platelet recovery to transfusion independence.
Measured: From the start study treatment (Day1) up to transfusion independence.
Incidence of Treatment-Emergent Adverse Events by CTCAE
Number of participants with treatment-related adverse events as assessed by CTCAE v4.0
Measured: From the start study treatment (Day1) up to week 12.
Dose-effect relationship
Correlation between avatrombopag's serum concentration with total and complete hematological response rate.
Measured: From the start study treatment(Day1) up to week 24.
Change of CD34+ cell
Change of CD34+ cells' proportion in bone marrow before and after avatrombopag treatment at week 12 and 24.
Measured: From the start study treatment(Day1) up to end of week 12 and 24.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted9 Feb 2023
- Study start10 Nov 2022
- Primary completion30 Nov 2023 (estimated)
- Study completion30 Nov 2024 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Severe 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 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 53 participants (estimated)
Sponsor and investigators
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Xin Zhao, Principal investigator, Institute of Hematology & Blood Diseases Hospital, China
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05720234
- Sponsor's protocol ID
- AVA&IST-001
- Status last verified
- Nov 2022
- First submitted
- 11 Jan 2023
- Last updated
- 9 Feb 2023
- Results posted
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 9 Feb 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- The Efficacy and Safety Study of Avatrombopag Combined With IST as First-line Treatment for Severe Aplastic Anemia, A Single-arm, Phase II Clinical Study
- ClinicalTrials.gov ID
- NCT05720234
- Acronym / sponsor ID
- AVA&IST-001
- Conditions
- Severe Aplastic Anemia
- Keywords
- avatrombopag
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 53 participants (estimated)
- Lead sponsor
- Institute of Hematology & Blood Diseases Hospital, ChinaUniversity, hospital or other
- Study start
- 10 Nov 2022 (actual)
- Primary completion
- 30 Nov 2023 (estimated)
- Study completion
- 30 Nov 2024 (estimated)
- First posted
- 9 Feb 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.
This single-center study aims to evaluate the early efficacy and safety of avatrombopag combined with immunosuppressive therapy (IST) in the first-line treatment of severe aplastic anemia (SAA).
Detailed description
This is a single center, single arm, phase II clinical study. Fifty-three patients will be enrolled.
Treatment protocol is as follows: 1) Anti-human thymocyte porcine immunoglobulin (P-ATG 20mg/kg/d) or rabbit anti human thymocyte globulin (R-ATG 3.0mg/kg/d) was administered intravenously for 5 days; 2) Cyclosporine (CSA) is given at 3-5 mg/kg.d in divided doses for at least 6 months. The trough concentration is maintained at 150-250 ng/ml. 3) Avatrombopag is given orally at 60 mg once a day for patients with body weight ≥ 50 kg, and 40 mg orally once a day for patients with body weight<50 kg, for a total of 12 weeks.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Treatment group | Experimental | Drug: avatrombopag53 subjects will be enrolled with the indicated treatment dose of avatrombopag. |
Who may qualify
- Ages
- 12 to 60 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (5 listed)
- Patients with newly diagnosed severe aplastic anemia.
- Men and women aged between 12 and 60.
- Subjects must complete all screening assessments as outlined in the test protocol.
- Able to swallow or administer orally.
- Before the start of the research procedure, the patient or guardian should fully understand the research procedure and purpose and sign the informed consent form. If the patient's signature is not conducive to the treatment of the disease, the patient's immediate family should sign the informed consent form.
Key exclusion criteria (11 listed)
- Congenital bone marrow failure (eg. Fanconi anemia).
- Accompanied by cytogenetic cloning changes (chromosomal karyotype and FISH detection found somatic cloning abnormalities; Simple -Y abnormality can be included in this study;) .
- ATG or middle/high-dose cyclophosphamide was used in the past.
- Previous treatment with cyclosporine or tacrolimus > 6 months.
- The total course of treatment with TPO receptor agonists (including thrombopoietin, eltrombopag,hetrombopag and avatrombopag) was more than 1 month.
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 |
|---|---|---|
| avatrombopagAlso: AVA, APAGPatients with body weight ≥50kg were given 60mg/day and patients with body weight < 50kg were given 40mg/day for 12 weeks. | Drug | Treatment group |
Primary outcome
Treatment response
Time frame: From the start of study treatment (Day1) to end of week 12.
Percentage of patients who achieves complete response(CR) at 12 weeks.
6 secondary outcomes
Treatment response
Time frame: From the start of study treatment (Day1) to end of week 12.
Treatment response
Time frame: From the start of study treatment (Day1) to end of week 24.
Supportive treatment
Time frame: From the start study treatment (Day1) up to transfusion independence.
Incidence of Treatment-Emergent Adverse Events by CTCAE
Time frame: From the start study treatment (Day1) up to week 12.
Dose-effect relationship
Time frame: From the start study treatment(Day1) up to week 24.
Change of CD34+ cell
Time frame: From the start study treatment(Day1) up to end of week 12 and 24.
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 9 Feb 2023.
- 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.
Xin Zhao principal investigator
Institute of Hematology & Blood Diseases Hospital, China
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 avatrombopag in people with Severe 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 5 inclusion and 11 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 9 Feb 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. 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?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Xin Zhao, M.D (8613702041366, zhaoxin@ihcams.ac.cn), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Treatment response (time frame: From the start of study treatment (Day1) to end of week 12.). 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 9 Feb 2023.