Aplastic AnemiaPhase 4NCT06009965
Efficacy of IST Combined With TPO-RA in the Treatment of AA and Establishment of a Recurrence Prediction System
A Phase 4 study testing CsA+ATG+Herombopag and CsA+Herombopag in people with Aplastic Anemia and CSA, run by Peking Union Medical College Hospital at 1 site in China.
Trial summary
In this study, investigators intend to prospectively study treatment-naive AA patients (including SAA and NSAA) who are non-transplant candidates in northern China. Patients with SAA receive ATG+CsA+Herombopag, and patients with NSAA receive CsA+ Herombopag. Read more about this study
- Who is it for?
- Ages 18 to 75 years
- Conditions the study is for
- Aplastic Anemia, CSA, Drug Therapy
- What is being tested?
- CsA+ATG+Herombopag and CsA+Herombopag
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 210 participants
- Where
- 1 siteChina
- Study dates
- 20 Jan 2023to 31 Jan 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 75 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Age: 18-75 years old, gender is not limited
- Definite diagnosis of AA
- No HSCT indication or unconditional HSCT
- SAA/VSAA patients were willing to accept ATG+CsA+TPO-RA treatment, NSAA was willing to accept CsA+TPO-RA treatment, and were willing to follow up regularly
- Baseline serum transaminase, total bilirubin and serum creatinine were less than 1.5 times the normal value
- Baseline liver and kidney function was less than 1.5 times the normal value
- Eastern Cancer Cooperation Group (ECOG) score status 0-2
- Agree to sign the consent form
Who cannot join
- Congenital AA
- Cytogenetic evidence of clonal hematologic bone marrow disease
- PNH clone ≥50%
- Allergic to ATG, cyclosporine and hexapopal in the past
- Uncontrolled infection or bleeding at enrollment
- Received hematopoietic stem cell transplantation (HSCT) before enrollment
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- Any concomitant malignancy within 5 years, except for local skin basal cell carcinoma
- A history of thromboembolic events, myocardial infarction, or stroke (including antiphospholipid antibody syndrome); Currently using anticoagulants
- Pregnant or lactating women
- Situations considered unsuitable for clinical research by other researchers
Trial locations and contacts
1 site in 1 country.
China1 site, 1 recruiting
- Recruiting
Peking union medical college hospital
Beijing
Bing Han, Doctor, +86-010-69155760, hanbingtg123@163.com
About this study
Official title: Establishment of an Efficacy and Relapse Prediction System for Immunosuppressants Combined With Thrombopoietin Receptor Agonists in the Treatment of Aplastic Anemia
In this study, investigators intend to prospectively study treatment-naive AA patients (including SAA and NSAA) who are non-transplant candidates in northern China. Patients with SAA receive ATG+CsA+Herombopag, and patients with NSAA receive CsA+ Herombopag. Investigators explored possible indicators of participants' predictive efficacy and built predictive models. After the participants achieved response, they used a tapering regimen, observed relapse and clonal evolution, and developed a predictive model of relapse.
Read the detailed description
- Patients in the SAA group: CsA: 3-5mg/kg/day, monitor the trough concentration every month, and maintain the trough concentration at 100-200ng/ml. ATG: rabbit anti-thymocyte immunoglobulin (r-ATG) 3mg/kg/d×5 days, or porcine anti-lymphocyte immunoglobulin ((p-ATG) 25mg/kg/d×5 days; TPO-RA: Herombopag starts at 7.5 mg qd, monitor the blood picture every 2 weeks, if the effect is not good, add 1 tablet every 2 weeks, up to 6 tablets (15 mg) qd.
Patients in the NSAA group: CsA: 3-5mg/kg/day, monitor the trough concentration every month, and maintain the trough concentration at 100-200ng/ml. TPO-RA: start with 7.5mg qd of Herombopag, monitor the blood picture every 2 weeks, if the effect is not good, add 1 tablet every 2 weeks, up to 6 tablets (15mg) qd.
(2) Those who are effective after 6 months of treatment continue to receive sufficient CsA treatment for at least 1.5 years, and then slowly reduce the dose until the end of the reduction. Those who fail to withdraw from the clinical trial.
(3) Participants who achieve CR or PR after treatment, but when the curative effect does not increase after 3 months of maintenance treatment, start to reduce the dose of Herombopag, and reduce one tablet every 3 months until the reduction stops. If recurrence occurs, recover Up to the last dose or even increase the dose, after reaching the best curative effect, reduce one tablet every 3 months until the reduction stops, and those who cannot recover are defined as relapse.
- Conditions
- Aplastic Anemia, CSA, Drug Therapy
- Being tested
- CsA+ATG+Herombopag, CsA+Herombopag
- Keywords
- Aplastic Anemia, CsA, ATG, TPO-RA
- Medical subject headings
- Anemia, Aplastic
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
CsA+ATG+HerombopagDrug
Also called SAA group intervention treatment
CsA: 3-5 mg/kg/day, monitor trough concentration monthly, maintain trough concentration at 100-200 ng/ml.ATG: rabbit anti-thymocyte immunoglobulin (r-ATG) 3 mg/kg/d x 5 days or porcine anti-lymphocyte immunoglobulin ((p-ATG) 25 mg/kg/d x 5 days.TPO-RA: Heptapepto-Papa 7.5 mg qd to start. Monitor blood every 2 weeks and if ineffective, increase by 1 tablet every 2 weeks up to a maximum of 6 tablets (15mg) qd.
CsA+HerombopagDrug
Also called NSAA group intervention treatment
CsA: 3-5 mg/kg/day, monitor trough concentrations monthly, maintain trough concentrations at 100-200 ng/ml.TPO-RA: Start with Hetropoxyphene 7.5 mg qd, monitor blood every 2 weeks, and if ineffective, increase by 1 tablet every 2 weeks up to a maximum of 6 tablets (15 mg) qd.
| Study group | Role | What they receive |
|---|---|---|
| Severe aplastic anemia group | Experimental | CsA: 3-5 mg/kg/day, monitor trough concentration monthly, maintain trough concentration at 100-200 ng/ml.ATG: rabbit anti-thymocyte immunoglobulin (r-ATG) 3 mg/kg/d x 5 days or porcine anti-lymphocyte immunoglobulin ((p-ATG) 25 mg/kg/d x 5 days.TPO-RA: Heptapepto-Papa 7.5 mg qd to start. Monitor blood every 2 weeks and if ineffective, increase by 1 tablet every 2 weeks up to a maximum of 6 tablets (15mg) qd. Receives: Drug: CsA+ATG+Herombopag |
| Non-severe aplastic anemia group | Experimental | CsA: 3-5 mg/kg/day, monitor trough concentrations monthly, maintain trough concentrations at 100-200 ng/ml.TPO-RA: Start with Hetropoxyphene 7.5 mg qd, monitor blood every 2 weeks, and if ineffective, increase by 1 tablet every 2 weeks up to a maximum of 6 tablets (15 mg) qd. Receives: Drug: CsA+Herombopag |
What is being measured
The primary outcome is the main question the study is designed to answer.
Overall response rate
Overall response rate
Measured: 24 weeks after treatment
24-month recurrence rate
.24-month recurrence rate
Measured: 96 weeks after treatment
Secondary outcomes (2)
Overall response rate
Overall response rate
Measured: 56 weeks after treatment
36-month recurrence rate
36-month recurrence rate
Measured: 144 weeks after treatment
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted24 Aug 2023
- Study start20 Jan 2023
- Primary completion30 Jan 2025 (estimated)
- Study completion31 Jan 2025 (estimated)
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 4
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 210 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
- NCT06009965
- Sponsor's protocol ID
- SN-01
- Status last verified
- Aug 2023
- First submitted
- 6 Aug 2023
- Last updated
- 24 Aug 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 24 Aug 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Establishment of an Efficacy and Relapse Prediction System for Immunosuppressants Combined With Thrombopoietin Receptor Agonists in the Treatment of Aplastic Anemia
- ClinicalTrials.gov ID
- NCT06009965
- Acronym / sponsor ID
- SN-01
- Conditions
- Aplastic Anemia, CSA, Drug Therapy
- Keywords
- Aplastic Anemia, CsA, ATG, TPO-RA
- Phase
- Phase 4
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 210 participants (estimated)
- Lead sponsor
- Peking Union Medical College HospitalUniversity, hospital or other
- Study start
- 20 Jan 2023 (actual)
- Primary completion
- 30 Jan 2025 (estimated)
- Study completion
- 31 Jan 2025 (estimated)
- First posted
- 24 Aug 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.
In this study, investigators intend to prospectively study treatment-naive AA patients (including SAA and NSAA) who are non-transplant candidates in northern China. Patients with SAA receive ATG+CsA+Herombopag, and patients with NSAA receive CsA+ Herombopag. Investigators explored possible indicators of participants' predictive efficacy and built predictive models. After the participants achieved response, they used a tapering regimen, observed relapse and clonal evolution, and developed a predictive model of relapse.
Detailed description
- Patients in the SAA group: CsA: 3-5mg/kg/day, monitor the trough concentration every month, and maintain the trough concentration at 100-200ng/ml. ATG: rabbit anti-thymocyte immunoglobulin (r-ATG) 3mg/kg/d×5 days, or porcine anti-lymphocyte immunoglobulin ((p-ATG) 25mg/kg/d×5 days; TPO-RA: Herombopag starts at 7.5 mg qd, monitor the blood picture every 2 weeks, if the effect is not good, add 1 tablet every 2 weeks, up to 6 tablets (15 mg) qd.
Patients in the NSAA group: CsA: 3-5mg/kg/day, monitor the trough concentration every month, and maintain the trough concentration at 100-200ng/ml. TPO-RA: start with 7.5mg qd of Herombopag, monitor the blood picture every 2 weeks, if the effect is not good, add 1 tablet every 2 weeks, up to 6 tablets (15mg) qd.
(2) Those who are effective after 6 months of treatment continue to receive sufficient CsA treatment for at least 1.5 years, and then slowly reduce the dose until the end of the reduction. Those who fail to withdraw from the clinical trial.
(3) Participants who achieve CR or PR after treatment, but when the curative effect does not increase after 3 months of maintenance treatment, start to reduce the dose of Herombopag, and reduce one tablet every 3 months until the reduction stops. If recurrence occurs, recover Up to the last dose or even increase the dose, after reaching the best curative effect, reduce one tablet every 3 months until the reduction stops, and those who cannot recover are defined as relapse.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Severe aplastic anemia group | Experimental | Drug: CsA+ATG+HerombopagCsA: 3-5 mg/kg/day, monitor trough concentration monthly, maintain trough concentration at 100-200 ng/ml.ATG: rabbit anti-thymocyte immunoglobulin (r-ATG) 3 mg/kg/d x 5 days or porcine anti-lymphocyte immunoglobulin ((p-ATG) 25 mg/kg/d x 5 days.TPO-RA: Heptapepto-Papa 7.5 mg qd to start. Monitor blood every 2 weeks and if ineffective, increase by 1 tablet every 2 weeks up to a maximum of 6 tablets (15mg) qd. |
| Non-severe aplastic anemia group | Experimental | Drug: CsA+HerombopagCsA: 3-5 mg/kg/day, monitor trough concentrations monthly, maintain trough concentrations at 100-200 ng/ml.TPO-RA: Start with Hetropoxyphene 7.5 mg qd, monitor blood every 2 weeks, and if ineffective, increase by 1 tablet every 2 weeks up to a maximum of 6 tablets (15 mg) qd. |
Who may qualify
- Ages
- 18 to 75 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (8 listed)
- Age: 18-75 years old, gender is not limited
- Definite diagnosis of AA
- No HSCT indication or unconditional HSCT
- SAA/VSAA patients were willing to accept ATG+CsA+TPO-RA treatment, NSAA was willing to accept CsA+TPO-RA treatment, and were willing to follow up regularly
- Baseline serum transaminase, total bilirubin and serum creatinine were less than 1.5 times the normal value
Key exclusion criteria (10 listed)
- Congenital AA
- Cytogenetic evidence of clonal hematologic bone marrow disease
- PNH clone ≥50%
- Allergic to ATG, cyclosporine and hexapopal in the past
- Uncontrolled infection or bleeding at 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 |
|---|---|---|
| CsA+ATG+HerombopagAlso: SAA group intervention treatmentCsA: 3-5 mg/kg/day, monitor trough concentration monthly, maintain trough concentration at 100-200 ng/ml.ATG: rabbit anti-thymocyte immunoglobulin (r-ATG) 3 mg/kg/d x 5 days or porcine anti-lymphocyte immunoglobulin ((p-ATG) 25 mg/kg/d x 5 days.TPO-RA: Heptapepto-Papa 7.5 mg qd to start. Monitor blood every 2 weeks and if ineffective, increase by 1 tablet every 2 weeks up to a maximum of 6 tablets (15mg) qd. | Drug | Severe aplastic anemia group |
| CsA+HerombopagAlso: NSAA group intervention treatmentCsA: 3-5 mg/kg/day, monitor trough concentrations monthly, maintain trough concentrations at 100-200 ng/ml.TPO-RA: Start with Hetropoxyphene 7.5 mg qd, monitor blood every 2 weeks, and if ineffective, increase by 1 tablet every 2 weeks up to a maximum of 6 tablets (15 mg) qd. | Drug | Non-severe aplastic anemia group |
Primary outcome
Overall response rate
Time frame: 24 weeks after treatment
Overall response rate
24-month recurrence rate
Time frame: 96 weeks after treatment
.24-month recurrence rate
2 secondary outcomes
Overall response rate
Time frame: 56 weeks after treatment
36-month recurrence rate
Time frame: 144 weeks after treatment
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 24 Aug 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.
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 CsA+ATG+Herombopag and CsA+Herombopag in people with Aplastic Anemia and CSA, 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 8 inclusion and 10 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 24 Aug 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 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 Yali Du, Master (+8615845992396, yali_crazy@126.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Overall response rate (time frame: 24 weeks after 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?
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 24 Aug 2023.