Severe Aplastic AnemiaPhase 2hypo-CASHNCT06695741
Reduced Dose of Cyclophosphamide Combined With Standard Immunosuppressive Therapy to Treat Severe Aplastic Anemia
A Phase 2 study testing Reduced dose of cyclophosphamide combined with standard immunosuppressive therapy in people with Severe Aplastic Anemia and Cyclophosphamide Reduced-dose, 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 the reduced dose of cyclophosphamide (hypo-CASH) 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, Cyclophosphamide Reduced-dose, Immunosuppressive Therapy
- What is being tested?
- Reduced dose of cyclophosphamide combined with standard immunosuppressive therapy
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 75 participants
- Where
- 1 siteChina
- Study dates
- 1 Nov 2024to 30 Jun 2027
- 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 12 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Subject has a diagnosis of naïve severe or very severe aplastic anemia
- Male or female age ≥ 12 years
- Unwilling or unable to receive allogeneic hematopoietic stem cell transplantation.
- ECOG performance status ≤2
- 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 anti-lymphocyte globulin, cyclosporine, herombopag or cyclophosphamide.
- Uncontrolled fungal, bacterial, or viral infection. Hepatitis-associated aplastic anemia is allowed; hepatitis B or C infection is permitted unless it causes severe liver failure.
- Tested positive for HIV or syphilis
- Presence of severe liver, kidney, or heart failure, or other life-threatening comorbidities.
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- History of radiotherapy and chemotherapy for malignant solid tumors in recent 5 years
- 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. Contact the study team or a site near you to ask about taking part.
China1 site, 1 recruiting
- Recruiting
Red Blood Cell Disorders Center and Regenerative Medicine Center
Tianjin, Tianjin Municipality, 301617
Hong Pan, MD, 15822458611, panhong@ihcams.ac.cn
About this study
Official title: Reduced Dose of Cyclophosphamide Combined With Standard Immunosuppressive Therapy as Front-line Therapy in Patients With Severe Aplastic Anemia
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 the reduced dose of cyclophosphamide (hypo-CASH) for severe aplastic anemia.
- Being tested
- Reduced dose of cyclophosphamide combined with standard immunosuppressive therapy
- Medical subject headings
- Anemia, Aplastic
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Reduced dose of cyclophosphamide combined with standard immunosuppressive therapyDrug
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. Cyclophosphamide (20mg/kg) will be administered on days 15-16.
| Study group | Role | What they receive |
|---|---|---|
| hypo-CASH | Experimental | ALG/CsA and herombopag and reduced dose of cyclophosphamide Receives: Drug: Reduced dose of cyclophosphamide combined with standard immunosuppressive therapy |
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 (5)
Superior response rate
Percentage of patients with superior response, including CR, VGPR and GPR.
Measured: With in 6 months
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: With in 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
Time to achieve robust superior response
Measured: Within 6 months
First time to response
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 posted19 Nov 2024
- Study start1 Nov 2024
- Primary completion1 Feb 2027 (estimated)
- Study completion30 Jun 2027 (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.
- 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
- 75 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Institute of Hematology & Blood Diseases Hospital, China
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Jun Shi, Institute of Hematology & Blood Diseases Hospital, China
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06695741
- Sponsor's protocol ID
- Hypo-CASH-2024
- Acronym
- hypo-CASH
- Status last verified
- Nov 2024
- First submitted
- 16 Nov 2024
- Last updated
- 19 Nov 2024
- Results posted
- No
- Data monitoring committee
- 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 19 Nov 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Reduced Dose of Cyclophosphamide Combined With Standard Immunosuppressive Therapy as Front-line Therapy in Patients With Severe Aplastic Anemia
- ClinicalTrials.gov ID
- NCT06695741
- Acronym / sponsor ID
- hypo-CASH / Hypo-CASH-2024
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 75 participants (estimated)
- Lead sponsor
- Institute of Hematology & Blood Diseases Hospital, ChinaUniversity, hospital or other
- Study start
- 1 Nov 2024 (actual)
- Primary completion
- 1 Feb 2027 (estimated)
- Study completion
- 30 Jun 2027 (estimated)
- First posted
- 19 Nov 2024 (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 the reduced dose of cyclophosphamide (hypo-CASH) 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 |
|---|---|---|
| hypo-CASH | Experimental | Drug: Reduced dose of cyclophosphamide combined with standard immunosuppressive therapyALG/CsA and herombopag and reduced dose of 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)
- Subject has a diagnosis of naïve severe or very severe aplastic anemia
- Male or female age ≥ 12 years
- Unwilling or unable to receive allogeneic hematopoietic stem cell transplantation.
- ECOG performance status ≤2
- Willing and able to comply with the requirements for this study and written informed consent.
Key exclusion criteria (10 listed)
- Previously received immunosuppressive therapy > 4 weeks
- Previously treated with TPO-RA > 4 weeks
- Have an allergy or intolerance to anti-lymphocyte globulin, cyclosporine, herombopag or cyclophosphamide.
- Uncontrolled fungal, bacterial, or viral infection. Hepatitis-associated aplastic anemia is allowed; hepatitis B or C infection is permitted unless it causes severe liver failure.
- Tested positive for HIV or syphilis
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 |
|---|---|---|
| Reduced dose of cyclophosphamide combined with standard immunosuppressive therapySevere 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. Cyclophosphamide (20mg/kg) will be administered on days 15-16. | Drug | hypo-CASH |
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).
5 secondary outcomes
Superior response rate
Time frame: With in 6 months
Overall response rate
Time frame: With in 6 months
Incidence of the adverse event
Time frame: Within 6 months
Time to achieve robust superior response
Time frame: Within 6 months
First time to response
Time frame: Within 6 months
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 19 Nov 2024.
- 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.
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 Reduced dose of cyclophosphamide combined with standard immunosuppressive therapy in people with Severe Aplastic Anemia and Cyclophosphamide Reduced-dose, 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 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: looking for participants now, as of the sponsor's last update on 19 Nov 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?
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 Hong Pan, MD (15822458611, panhong@ihcams.ac.cn), 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: 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 19 Nov 2024.