Severe Aplastic AnemiaPhase 2NCT05975996
Cyclophosphamide Added to Standard Immunosuppressive Therapy With Herombopag as Front-line Therapy in Patients With Severe Aplastic Anemia
A Phase 2 study testing Cyclophosphamide added to standard immunosuppressive therapy with herombopag in people with Severe Aplastic Anemia, run by Institute of Hematology & Blood Diseases Hospital, China at 1 site in China.
Trial summary
This is a prospective, single-center, single-arm, phase 2 study. This study aims to evaluate the efficacy and safety of Anti-lymphocyte globulin plus herombopag in combination with moderate-dose cyclophosphamide for severe aplastic anemia. Read more about this study
- Who is it for?
- Ages 12 years and older
- Conditions the study is for
- Severe Aplastic Anemia
- What is being tested?
- Cyclophosphamide added to standard immunosuppressive therapy with herombopag
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 43 participants
- Where
- 1 siteChina
- Study dates
- 10 Jul 2023to 31 Dec 2025
- 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 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Male or female age ≥ 12 years
- Subject has a diagnosis of naïve severe or very severe aplastic anemia
- ECOG performance status ≤2
- Unwilling or unable to receive allogeneic hematopoietic stem cell transplantation.
- Willing and able to comply with the requirements for this study and written informed consent.
Who cannot join
- Previously received immunosuppressive therapy > 4 weeks
- Previously treated with TPO-RA > 4 weeks
- Have an allergy or intolerance to either herombopag or cyclophosphamide.
- Have an allergy to ALG
- Uncontrolled systemic fungal, bacterial, or viral infection
- Poorly controlled hypertension (≥140/90mmHg) or diabetes (a fasting plasma glucose concentration ≥7.0mmol/L or a random venous plasma glucose concentration ≥11.1mmol/L)
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- Abnormal liver or kidney function: ALT or AST >3 ULN, or serum creatinine (sCr)≥ 2.5 ULN.
- History of radiotherapy and chemotherapy for malignant solid tumors
- Combined with other serious disorders
- Pregnant or breast-feeding patients
- Patients considered to be ineligible for the study by the investigator for reasons other than the above.
Trial locations and contacts
1 site in 1 country.
About this study
This is a prospective, single-center, single-arm, phase 2 study. This study aims to evaluate the efficacy and safety of Anti-lymphocyte globulin plus herombopag in combination with moderate-dose cyclophosphamide for severe aplastic anemia.
- Conditions
- Severe Aplastic Anemia
- Being tested
- Cyclophosphamide added to standard immunosuppressive therapy with herombopag
- Medical subject headings
- Anemia, Aplastic
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Cyclophosphamide added to standard immunosuppressive therapy with herombopagDrug
Severe aplastic anemia patients will receive a daily dose of Anti-lymphocyte globulin (25mg/kg) for the initial five days at the beginning of the treatment. Cyclosporine will be administered daily at a dosage of 3-5mg/kg. Herombopag will be administered daily at a dosage of 15mg starting from the first day of treatment and continuing for a duration of six months. The moderate-dose cyclophosphamide (20mg/kg) will be administered on days 29-30 and days 43-44.
| Study group | Role | What they receive |
|---|---|---|
| Experiemental | Experimental | ALG/CsA(Cyclosporine) and herombopag and moderate-dose cyclophosphamide Receives: Drug: Cyclophosphamide added to standard immunosuppressive therapy with herombopag |
What is being measured
The primary outcome is the main question the study is designed to answer.
Overall response rate
Percentage of patients with hematological response. Hematological response includes complete rate, near complete rate(CR), very good partial response(VGPR), good partial response(GPR) and partial response(PR).
Measured: Within 3 months
Secondary outcomes (4)
Robust response
Percentage of patients with robust response, including CR, near CR, VGPR and GPR.
Measured: Within 3 months
All-cause mortality
Measured: Within 3 months
Time to achieve robust hematological response
Measured: Within 6 months
Incidence of the adverse event
Use Common Terminology Criteria for Adverse Events (CTCAE) Version 5 to assess the adverse event
Measured: Within 6 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted4 Aug 2023
- Study start10 Jul 2023
- Primary completion31 Dec 2024 (estimated)
- Study completion31 Dec 2025 (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
- 43 participants (actual)
Sponsor and investigators
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Fang Liwei, Institute of Hematology & Blood Diseases Hospital, China
- Investigators
- Liwei Fang, MD, Principal investigator, Institute of Hematology & Blood Diseases Hospital, China
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05975996
- Sponsor's protocol ID
- IIT2023038
- Status last verified
- Sep 2024
- First submitted
- 27 Jul 2023
- Last updated
- 25 Sep 2024
- 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 25 Sep 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Cyclophosphamide Added to Standard Immunosuppressive Therapy With Herombopag as Front-line Therapy in Patients With Severe Aplastic Anemia
- ClinicalTrials.gov ID
- NCT05975996
- Acronym / sponsor ID
- IIT2023038
- Conditions
- Severe Aplastic Anemia
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 43 participants (actual)
- Lead sponsor
- Institute of Hematology & Blood Diseases Hospital, ChinaUniversity, hospital or other
- Study start
- 10 Jul 2023 (actual)
- Primary completion
- 31 Dec 2024 (estimated)
- Study completion
- 31 Dec 2025 (estimated)
- First posted
- 4 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.
This is a prospective, single-center, single-arm, phase 2 study. This study aims to evaluate the efficacy and safety of Anti-lymphocyte globulin plus herombopag in combination with moderate-dose cyclophosphamide for severe aplastic anemia.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Experiemental | Experimental | Drug: Cyclophosphamide added to standard immunosuppressive therapy with herombopagALG/CsA(Cyclosporine) and herombopag and moderate-dose cyclophosphamide |
Who may qualify
- Ages
- 12 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (5 listed)
- Male or female age ≥ 12 years
- Subject has a diagnosis of naïve severe or very severe aplastic anemia
- ECOG performance status ≤2
- Unwilling or unable to receive allogeneic hematopoietic stem cell transplantation.
- Willing and able to comply with the requirements for this study and written informed consent.
Key exclusion criteria (11 listed)
- Previously received immunosuppressive therapy > 4 weeks
- Previously treated with TPO-RA > 4 weeks
- Have an allergy or intolerance to either herombopag or cyclophosphamide.
- Have an allergy to ALG
- Uncontrolled systemic fungal, bacterial, or viral infection
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 |
|---|---|---|
| Cyclophosphamide added to standard immunosuppressive therapy with herombopagSevere aplastic anemia patients will receive a daily dose of Anti-lymphocyte globulin (25mg/kg) for the initial five days at the beginning of the treatment. Cyclosporine will be administered daily at a dosage of 3-5mg/kg. Herombopag will be administered daily at a dosage of 15mg starting from the first day of treatment and continuing for a duration of six months. The moderate-dose cyclophosphamide (20mg/kg) will be administered on days 29-30 and days 43-44. | Drug | Experiemental |
Primary outcome
Overall response rate
Time frame: Within 3 months
Percentage of patients with hematological response. Hematological response includes complete rate, near complete rate(CR), very good partial response(VGPR), good partial response(GPR) and partial response(PR).
4 secondary outcomes
Robust response
Time frame: Within 3 months
All-cause mortality
Time frame: Within 3 months
Time to achieve robust hematological response
Time frame: Within 6 months
Incidence of the adverse event
Time frame: Within 6 months
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 25 Sep 2024.
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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.
Liwei Fang, MD 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 Cyclophosphamide added to standard immunosuppressive therapy with herombopag 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 25 Sep 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 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?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Overall response rate (time frame: Within 3 months). 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 25 Sep 2024.