Sever Aplastic AnaemiaPhase 2NCT07010237
Dose-attenuated IST and Hetrombopag in Elderly (≥65 Years) Patients With Severe Aplastic Anemia
A Phase 2 study testing Dose-attenuated IST and Hetrombopag in people with Sever Aplastic Anaemia and Elderly (People Aged 65 or More), run by Institute of Hematology & Blood Diseases Hospital, China at 1 site in China.
Trial summary
This is a prospetive,Single-Center, Single-Arm, Phase IIB Clinical Study.This study aims to evaluate the efficacy and safety of dose-attenuated IST combined with Hetrombopag in elderly patients (≥65 years) with VSAA/SAA. Read more about this study
- Who is it for?
- Ages 65 years and older
- Conditions the study is for
- Sever Aplastic Anaemia, Elderly (People Aged 65 or More), Immunosuppressive Treatment
- What is being tested?
- Dose-attenuated IST and Hetrombopag
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 65 participants
- Where
- 1 siteChina
- Study dates
- 1 Feb 2025to 1 Jul 2029
- Phase
- Phase 2
- 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 65 years and older
- All sexes
- No healthy volunteers
- Older adult
Who may be able to join
- Clinical diagnosis of VSAA/SAA.
- Age ≥65 years.
- Completion of all screening assessments.
- Must be able to swallow tablets.
- Signed informed consent (by patient or legal guardian if patient is incapacitated).
Who cannot join
- Clonal cytogenetic abnormalities (excluding isolated -Y or +8).
- Prior treatment with ATG/high-dose cyclophosphamide.
- Prior cyclosporine/tacrolimus use >12 months.
- Prior TPO-RA therapy >3 months.
- Uncontrolled malignancies or conditions contraindicating ATG.
- Severe organ dysfunction (e.g., creatinine ≥177 μmol/L).
- Investigator judgment of unsuitability.
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
Red Blood Cell Diseases Center
Tianjin, Tianjin Municipality
Jianping Li, MD, 13820961539, lijianping@ihcams.ac.cn
About this study
Official title: Dose-attenuated IST and Hetrombopag in the Treatment of Elderly (≥65 Years) Patients With Very Severe/Severe Aplastic Anemia: A Single-Center, Single-Arm, Phase IIB Clinical Study on Efficacy and Safety
This is a prospetive,Single-Center, Single-Arm, Phase IIB Clinical Study.This study aims to evaluate the efficacy and safety of dose-attenuated IST combined with Hetrombopag in elderly patients (≥65 years) with VSAA/SAA.
Read the detailed description
Design : Prospective, single-center, single-arm, open-label Phase IIB trial.
Intervention :
P-ATG : 15 mg/kg/day IV ×5 days. Cyclosporine : 3 mg/kg/day (adjusted to trough 100-200 μg/L). Hetrombopag : 15 mg daily. Follow-Up : Weekly for 24 weeks, with bone marrow evaluation at 24 weeks.
Inclusion Criteria :
Confirmed diagnosis of VSAA/SAA. Age ≥65 years. Completion of all screening assessments. Ability to swallow oral medication. Signed informed consent (by patient or legal guardian if patient is incapacitated).
Exclusion Criteria :
Clonal cytogenetic abnormalities (excluding isolated -Y or +8). Prior treatment with ATG/high-dose cyclophosphamide. Prior cyclosporine/tacrolimus use >12 months. Prior TPO-RA therapy >3 months. Uncontrolled malignancies or conditions contraindicating ATG. Severe organ dysfunction (e.g., creatinine ≥177 μmol/L). Investigator judgment of unsuitability.
- Being tested
- Dose-attenuated IST and Hetrombopag
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Dose-attenuated IST and HetrombopagDrug
Porcine-ATG : 15 mg/kg/day IV ×5 days. Cyclosporine : 3-5 mg/kg/day (adjusted to trough 100-200 μg/L). Hetrombopag: 15 mg daily for 24weeks.
| Study group | Role | What they receive |
|---|---|---|
| A-IST | Experimental | Porcine-ATG : 15 mg/kg/day IV ×5 days. Cyclosporine : 3-5 mg/kg/day (adjusted to trough 100-200 μg/L). Hetrombopag: 15 mg daily. Receives: Drug: Dose-attenuated IST and Hetrombopag |
What is being measured
The primary outcome is the main question the study is designed to answer.
Hematologic response rate at 24 weeks post-ATG treatment.
PR+CR Partial Response (PR) : Independence from transfusions without meeting CR. No Response (NR) : Failure to achieve PR.
Measured: 24 weeks
Secondary outcomes (2)
Early mortality rate
Early mortality rate within 12weeks
Measured: 12 weeks
Complete Response
Complete Response (CR) : HGB ≥100 g/L, PLT ≥100×10⁹/L, ANC ≥1.0×10⁹/L.
Measured: 24 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted8 Jun 2025
- Study start1 Feb 2025
- Primary completion31 Dec 2028 (estimated)
- Study completion1 Jul 2029 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Sever Aplastic AnaemiaStudies 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
- 65 participants (estimated)
Sponsor and investigators
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Jun Shi, PhD, Principal investigator, Institute of Hematology & Blood Diseases Hospital, CAMS
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07010237
- Sponsor's protocol ID
- IIT2025013
- Status last verified
- Feb 2025
- First submitted
- 30 Apr 2025
- Last updated
- 8 Jun 2025
- 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 8 Jun 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Dose-attenuated IST and Hetrombopag in the Treatment of Elderly (≥65 Years) Patients With Very Severe/Severe Aplastic Anemia: A Single-Center, Single-Arm, Phase IIB Clinical Study on Efficacy and Safety
- ClinicalTrials.gov ID
- NCT07010237
- Acronym / sponsor ID
- IIT2025013
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 65 participants (estimated)
- Lead sponsor
- Institute of Hematology & Blood Diseases Hospital, ChinaUniversity, hospital or other
- Study start
- 1 Feb 2025 (actual)
- Primary completion
- 31 Dec 2028 (estimated)
- Study completion
- 1 Jul 2029 (estimated)
- First posted
- 8 Jun 2025 (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 prospetive,Single-Center, Single-Arm, Phase IIB Clinical Study.This study aims to evaluate the efficacy and safety of dose-attenuated IST combined with Hetrombopag in elderly patients (≥65 years) with VSAA/SAA.
Detailed description
Design : Prospective, single-center, single-arm, open-label Phase IIB trial.
Intervention :
P-ATG : 15 mg/kg/day IV ×5 days. Cyclosporine : 3 mg/kg/day (adjusted to trough 100-200 μg/L). Hetrombopag : 15 mg daily. Follow-Up : Weekly for 24 weeks, with bone marrow evaluation at 24 weeks.
Inclusion Criteria :
Confirmed diagnosis of VSAA/SAA. Age ≥65 years. Completion of all screening assessments. Ability to swallow oral medication. Signed informed consent (by patient or legal guardian if patient is incapacitated).
Exclusion Criteria :
Clonal cytogenetic abnormalities (excluding isolated -Y or +8). Prior treatment with ATG/high-dose cyclophosphamide. Prior cyclosporine/tacrolimus use >12 months. Prior TPO-RA therapy >3 months. Uncontrolled malignancies or conditions contraindicating ATG. Severe organ dysfunction (e.g., creatinine ≥177 μmol/L). Investigator judgment of unsuitability.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| A-IST | Experimental | Drug: Dose-attenuated IST and HetrombopagPorcine-ATG : 15 mg/kg/day IV ×5 days. Cyclosporine : 3-5 mg/kg/day (adjusted to trough 100-200 μg/L). Hetrombopag: 15 mg daily. |
Who may qualify
- Ages
- 65 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Older adult
Key inclusion criteria (5 listed)
- Clinical diagnosis of VSAA/SAA.
- Age ≥65 years.
- Completion of all screening assessments.
- Must be able to swallow tablets.
- Signed informed consent (by patient or legal guardian if patient is incapacitated).
Key exclusion criteria (7 listed)
- Clonal cytogenetic abnormalities (excluding isolated -Y or +8).
- Prior treatment with ATG/high-dose cyclophosphamide.
- Prior cyclosporine/tacrolimus use >12 months.
- Prior TPO-RA therapy >3 months.
- Uncontrolled malignancies or conditions contraindicating ATG.
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 |
|---|---|---|
| Dose-attenuated IST and HetrombopagPorcine-ATG : 15 mg/kg/day IV ×5 days. Cyclosporine : 3-5 mg/kg/day (adjusted to trough 100-200 μg/L). Hetrombopag: 15 mg daily for 24weeks. | Drug | A-IST |
Primary outcome
Hematologic response rate at 24 weeks post-ATG treatment.
Time frame: 24 weeks
PR+CR Partial Response (PR) : Independence from transfusions without meeting CR. No Response (NR) : Failure to achieve PR.
2 secondary outcomes
Early mortality rate
Time frame: 12 weeks
Complete Response
Time frame: 24 weeks
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 8 Jun 2025.
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.
Jun Shi, PhD principal investigator
Institute of Hematology & Blood Diseases Hospital, CAMS
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 Dose-attenuated IST and Hetrombopag in people with Sever Aplastic Anaemia and Elderly (People Aged 65 or More), 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 7 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 8 Jun 2025. 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?
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 Jianping Li, MD (+8613820961539, lijianping@ihcams.ac.cn), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Hematologic response rate at 24 weeks post-ATG treatment. (time frame: 24 weeks). 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 8 Jun 2025; AzTrials last refreshed its copy on 5 Nov 2026.