Aplastic AnemiaPhase 2NCT05510505
GVHD Prophylaxis by Addition of CD20 Monoclonal Antibody to the Conditioning Regimen in SAA With Treatment of Allo-HSCT
A Phase 2 study testing CD20 monoclonal antibody and ATG in people with Aplastic Anemia, run by The First Affiliated Hospital of Soochow University at 1 site in China.
Trial summary
Objectives 2.1 Primary objectives 1) To observe and compare incidence and severity of aGVHD and cGVHD between the two arms within 2 years after transplantation. 2) To observe and compare the engraftment rate between the two arms. 3) To observe and compare the incidence of infections between the two arms. Read more about this study
- Who is it for?
- Ages Up to 50 years
- Conditions the study is for
- Aplastic Anemia
- What is being tested?
- CD20 monoclonal antibody and ATG
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 100 participants
- Where
- 1 siteChina
- Study dates
- 30 Dec 2021to 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 Up to 50 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Subjects eligible for inclusion in this study must meet all of the following criteria:
- SAA characterized Bone marrow cellularity< 25%, or 25-50% with <30% residual hematopoietic cells and pancytopenia, with at least two of the following parameters in peripheral blood Absolute neutrophil count < 0.5*10E9/L Platelet count < 20*10E9/L Absolute reticulocyte count < 20*10E9/L
- ALL patients will undergo allo-HSCT.
- Subjects aged <50 years old with KPS performance status ≥70 at the same time.
- Aspartate aminotransferase (AST) , alanine aminotransferase (ALT) and alkaline phosphatase≤2 times the upper limit of normal (ULN). Blood urea nitrogen and Creatinine ≤1.25 times ULN.
- Cardiac function of subjects must meet all of the following requirements: ECG examination do not reveal any acute myocardial infarction, arrhythmia, or first-degree or higher atrioventricular block. No signs of heart failure. No carrying of active rheumatoid heart disease. Chest radiograph or physical examination do not indicate an enlarged heart.
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- ALL subjects show none contraindication for allogeneic hematopoietic stem cell transplantation.
- Patients enrolled in the rituximab group have no contraindications for the use of rituximab.
- Patients and their clients are willing to perform hematopoietic stem cell transplantation.
- Potential donor is accessible.
- Patients have no anti-HLA antibodies.
Who cannot join
- Subject who is unable comprehend or is unwilling to sign an informed consent form or consent form due to severe physical or mental illness resulting in a survival of less than 2 years.
- Presence of clinically active uncontrolled significant chronic infections (including bacterial, fungal or viral infection), such as dental caries, otitis media, sinusitis, etc., need to be carried out after effective control.
- Past medical history of severe pulmonary dysfunction.
- Past medical history of diabetes with a propensity for ketoacidosis.
- Presence of severe coagulopathy, thrombophlebitis or pulmonary embolism.
- Presence of decompensated liver insufficiency or active hepatitis.
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- Presence of history of severe autoimmune disease.
- Past medical history of thyroid dysfunction with currently abnormal thyroid function.
- Any concomitant malignancies that have not been disease-free for 5 years.
- Past medical history of hypersensitivity to biological products (including antibiotics).
- Pregnant or nursing woman.
- Inherited bone marrow failure.
Trial locations and contacts
1 site in 1 country.
China1 site, 1 recruiting
- Recruiting
The First Affiliated Hospital of Soochow University
Suzhou, Jiangsu, 215215
depei wu, 67781856 ext. 0512, wudepei@suda.edu.cn
About this study
Official title: A Prospective, Randomized, Multi-center Study to Assess the Efficacy and Safety of GVHD Prophylaxis by Addition of CD20 Monoclonal Antibody to the Conditioning Regimen in Severe Aplastic Anemia Patients With Treatment of Allogeneic HSCT
Objectives 2.1 Primary objectives
- To observe and compare incidence and severity of aGVHD and cGVHD between the two arms within 2 years after transplantation.
- To observe and compare the engraftment rate between the two arms. 3) To observe and compare the incidence of infections between the two arms. 2.2 Secondary objectives
- To conduct pharmacogenomic assay in CD20 arm(treatment arm) before conditioning and monitor plasma concentration of CD20 dynamically（7d、14d、28d、56d、91d）.
- To monitor levels of B cells in peripheral blood dynamically (+90d、+180d、+270d、+360d、+450d、+540d、+630d、+720d) in all patients.
- To observe and compare the incidence of PTLD between the two arms.
- To observe and compare immunoglobulin levels after transplantation in all patients.
- To evaluate transplant-related mortality.
- To evaluate the effect on hematopoietic reconstruction.
Read the detailed description
- Study design 3.1 Principle of design: prospective, randomized, control, open label 3.2 Subjects: patients with SAA undergoing allogeneic HSCT 3.3 Grouping: In this study, central randomization was used for random enrolment (1:1). After signing the informed consent, patients were randomized into rituximab conditioning group (test group) or non- rituximab conditioning group (control group). Treatment was assigned on a randomized basis according to a 1:1 ratio. The test group and the control group each will include 100 cases.
3.4 Study schedule: This clinical research is to be completed from September 2020 to September 2023.
- Subject enrollment 36months
- Transplantation to the end of follow-up 24months
- Data collection and report writing 3months In total 63months
- Conditions
- Aplastic Anemia
- Being tested
- CD20 monoclonal antibody, ATG
- Medical subject headings
- Anemia, Aplastic
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
CD20 monoclonal antibodyDrug
Also called allogeneic hematopoietic stem cell transplantation
4.2 Conditioning Regimen
4.2.1 Matched sibling donor
- ATG arm (control group) Fludarabine 30mg/m2/d×6d（-7d ~ -2d）+ Cyclophosphamide 50mg/kg/d×2d （-4d ~ -3d）+ ATG 2.5mg/kg/d×5d（-8d ~ -4d）
- ATG + CD20 monoclonal antibody (test arm) Fludarabine 30mg/m2/d×6d（-7d ~ -2d） + Cyclophosphamide 50mg/kg/d×2d （-4d ~ -3d）+ ATG 2.5mg/kg/d×5d（-8d ~ -4d）+ CD20 monoclonal antibody 375mg/m2, -1d
4.2.2 Unrelated donor and haploidentical donor
- ATG arm (control group) Busulfan 3.2 mg/kg/d（0.8 mg/kg，q6h）×2d（-7d ~ -6d） + Cyclophosphamide 50mg/kg/d×4d （-5d ~ -2d）+ ATG 2.5mg/kg/d×4d（-5d ~ -2d）
- ATG + CD20 monoclonal antibody (test arm) Busulfan 3.2 mg/kg/d（0.8 mg/kg，q6h）×2d（-7d ~ -6d） + Cyclophosphamide 50mg/kg/d×4d （-5d ~ -2d）+ ATG 2.5mg/kg/d×4d（-5d ~ -2d）+ CD20 monoclonal antibody 375mg/m2, -1d
ATGDrug
4.2 Conditioning Regimen
4.2.1 Matched sibling donor
- ATG arm (control group) Fludarabine 30mg/m2/d×6d（-7d ~ -2d）+ Cyclophosphamide 50mg/kg/d×2d （-4d ~ -3d）+ ATG 2.5mg/kg/d×5d（-8d ~ -4d）
- ATG + CD20 monoclonal antibody (test arm) Fludarabine 30mg/m2/d×6d（-7d ~ -2d） + Cyclophosphamide 50mg/kg/d×2d （-4d ~ -3d）+ ATG 2.5mg/kg/d×5d（-8d ~ -4d）+ CD20 monoclonal antibody 375mg/m2, -1d
4.2.2 Unrelated donor and haploidentical donor
- ATG arm (control group) Busulfan 3.2 mg/kg/d（0.8 mg/kg，q6h）×2d（-7d ~ -6d） + Cyclophosphamide 50mg/kg/d×4d （-5d ~ -2d）+ ATG 2.5mg/kg/d×4d（-5d ~ -2d）
- ATG + CD20 monoclonal antibody (test arm) Busulfan 3.2 mg/kg/d（0.8 mg/kg，q6h）×2d（-7d ~ -6d） + Cyclophosphamide 50mg/kg/d×4d （-5d ~ -2d）+ ATG 2.5mg/kg/d×4d（-5d ~ -2d）+ CD20 monoclonal antibody 375mg/m2, -1d
| Study group | Role | What they receive |
|---|---|---|
| ATG arm (control group) | Active comparator | 4.2.1 Matched sibling donor 1) ATG arm (control group) Fludarabine 30mg/m2/d×6d（-7d ~ -2d）+ Cyclophosphamide 50mg/kg/d×2d （-4d ~ -3d）+ ATG 2.5mg/kg/d×5d（-8d ~ -4d）
4.2.2 Unrelated donor and haploidentical donor
1) ATG arm (control group) Busulfan 3.2 mg/kg/d（0.8 mg/kg，q6h）×2d（-7d ~ -6d） + Cyclophosphamide 50mg/kg/d×4d （-5d ~ -2d）+ ATG 2.5mg/kg/d×4d（-5d ~ -2d） Receives: Drug: ATG |
| ATG + CD20 monoclonal antibody (test arm) | Experimental | 4.2.1 Matched sibling donor 2) ATG + CD20 monoclonal antibody (test arm) Fludarabine 30mg/m2/d×6d（-7d ~ -2d） + Cyclophosphamide 50mg/kg/d×2d （-4d ~ -3d）+ ATG 2.5mg/kg/d×5d（-8d ~ -4d）+ CD20 monoclonal antibody 375mg/m2, -1d
4.2.2 Unrelated donor and haploidentical donor 2) ATG + CD20 monoclonal antibody (test arm) Busulfan 3.2 mg/kg/d（0.8 mg/kg，q6h）×2d（-7d ~ -6d） + Cyclophosphamide 50mg/kg/d×4d （-5d ~ -2d）+ ATG 2.5mg/kg/d×4d（-5d ~ -2d）+ CD20 monoclonal antibody 375mg/m2, -1d Receives: Drug: CD20 monoclonal antibody |
What is being measured
The primary outcome is the main question the study is designed to answer.
GVHD incidence
GVHD incidence, location and grade. Infection incidence and recurrence rate.
Measured: 2 years
Secondary outcomes (4)
Infection incidence
Cumulative incidence of infection post-transplant
Measured: 2 years
GVHD-free survival rate
defined by the percentage of patients who are alive without evidence of moderate or severe chronic GVHD at 2 year
Measured: 2 years
transplant related mortality
Defined as the number of days from the date of transplant to the date of death related to transplant
Measured: 2 years
overall survival rate
OS is defined as the number of days from the date of transplant to the date of death
Measured: 2 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted22 Aug 2022
- Study start30 Dec 2021
- Primary completionSep 2023 (estimated)
- Study completionDec 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 The First Affiliated Hospital of Soochow UniversityThe 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
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind
- Who is masked
- Participant, Investigator
- Enrolment
- 100 participants (estimated)
Sponsor and investigators
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Wu Depei, The First Affiliated Hospital of Soochow University
- Investigators
- depei wu, Principal investigator, The First Affiliated Hospital of Soochow University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05510505
- Sponsor's protocol ID
- SAA-HSCT-2021
- Status last verified
- Aug 2022
- First submitted
- 8 Aug 2022
- Last updated
- 24 Aug 2022
- 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 24 Aug 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Prospective, Randomized, Multi-center Study to Assess the Efficacy and Safety of GVHD Prophylaxis by Addition of CD20 Monoclonal Antibody to the Conditioning Regimen in Severe Aplastic Anemia Patients With Treatment of Allogeneic HSCT
- ClinicalTrials.gov ID
- NCT05510505
- Acronym / sponsor ID
- SAA-HSCT-2021
- Conditions
- Aplastic Anemia
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 100 participants (estimated)
- Lead sponsor
- The First Affiliated Hospital of Soochow UniversityUniversity, hospital or other
- Study start
- 30 Dec 2021 (actual)
- Primary completion
- Sep 2023 (estimated)
- Study completion
- Dec 2025 (estimated)
- First posted
- 22 Aug 2022 (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.
Objectives 2.1 Primary objectives
- To observe and compare incidence and severity of aGVHD and cGVHD between the two arms within 2 years after transplantation.
- To observe and compare the engraftment rate between the two arms. 3) To observe and compare the incidence of infections between the two arms. 2.2 Secondary objectives
- To conduct pharmacogenomic assay in CD20 arm(treatment arm) before conditioning and monitor plasma concentration of CD20 dynamically（7d、14d、28d、56d、91d）.
- To monitor levels of B cells in peripheral blood dynamically (+90d、+180d、+270d、+360d、+450d、+540d、+630d、+720d) in all patients.
- To observe and compare the incidence of PTLD between the two arms.
- To observe and compare immunoglobulin levels after transplantation in all patients.
- To evaluate transplant-related mortality.
- To evaluate the effect on hematopoietic reconstruction.
Detailed description
- Study design 3.1 Principle of design: prospective, randomized, control, open label 3.2 Subjects: patients with SAA undergoing allogeneic HSCT 3.3 Grouping: In this study, central randomization was used for random enrolment (1:1). After signing the informed consent, patients were randomized into rituximab conditioning group (test group) or non- rituximab conditioning group (control group). Treatment was assigned on a randomized basis according to a 1:1 ratio. The test group and the control group each will include 100 cases.
3.4 Study schedule: This clinical research is to be completed from September 2020 to September 2023.
- Subject enrollment 36months
- Transplantation to the end of follow-up 24months
- Data collection and report writing 3months In total 63months
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind (participant, investigator)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| ATG arm (control group) | Active comparator | Drug: ATG4.2.1 Matched sibling donor 1) ATG arm (control group) Fludarabine 30mg/m2/d×6d（-7d ~ -2d）+ Cyclophosphamide 50mg/kg/d×2d （-4d ~ -3d）+ ATG 2.5mg/kg/d×5d（-8d ~ -4d） 4.2.2 Unrelated donor and haploidentical donor 1) ATG arm (control group) Busulfan 3.2 mg/kg/d（0.8 mg/kg，q6h）×2d（-7d ~ -6d） + Cyclophosphamide 50mg/kg/d×4d （-5d ~ -2d）+ ATG 2.5mg/kg/d×4d（-5d ~ -2d） |
| ATG + CD20 monoclonal antibody (test arm) | Experimental | Drug: CD20 monoclonal antibody4.2.1 Matched sibling donor 2) ATG + CD20 monoclonal antibody (test arm) Fludarabine 30mg/m2/d×6d（-7d ~ -2d） + Cyclophosphamide 50mg/kg/d×2d （-4d ~ -3d）+ ATG 2.5mg/kg/d×5d（-8d ~ -4d）+ CD20 monoclonal antibody 375mg/m2, -1d 4.2.2 Unrelated donor and haploidentical donor 2) ATG + CD20 monoclonal antibody (test arm) Busulfan 3.2 mg/kg/d（0.8 mg/kg，q6h）×2d（-7d ~ -6d） + Cyclophosphamide 50mg/kg/d×4d （-5d ~ -2d）+ ATG 2.5mg/kg/d×4d（-5d ~ -2d）+ CD20 monoclonal antibody 375mg/m2, -1d |
Who may qualify
- Ages
- Up to 50 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (11 listed)
- Subjects eligible for inclusion in this study must meet all of the following criteria:
Key exclusion criteria (12 listed)
- Subject who is unable comprehend or is unwilling to sign an informed consent form or consent form due to severe physical or mental illness resulting in a survival of less than 2 years.
- Presence of clinically active uncontrolled significant chronic infections (including bacterial, fungal or viral infection), such as dental caries, otitis media, sinusitis, etc., need to be carried out after effective control.
- Past medical history of severe pulmonary dysfunction.
- Past medical history of diabetes with a propensity for ketoacidosis.
- Presence of severe coagulopathy, thrombophlebitis or pulmonary embolism.
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 |
|---|---|---|
| CD20 monoclonal antibodyAlso: allogeneic hematopoietic stem cell transplantation4.2 Conditioning Regimen 4.2.1 Matched sibling donor 1. ATG arm (control group) Fludarabine 30mg/m2/d×6d（-7d ~ -2d）+ Cyclophosphamide 50mg/kg/d×2d （-4d ~ -3d）+ ATG 2.5mg/kg/d×5d（-8d ~ -4d） 2. ATG + CD20 monoclonal antibody (test arm) Fludarabine 30mg/m2/d×6d（-7d ~ -2d） + Cyclophosphamide 50mg/kg/d×2d （-4d ~ -3d）+ ATG 2.5mg/kg/d×5d（-8d ~ -4d）+ CD20 monoclonal antibody 375mg/m2, -1d 4.2.2 Unrelated donor and haploidentical donor 1. ATG arm (control group) Busulfan 3.2 mg/kg/d（0.8 mg/kg，q6h）×2d（-7d ~ -6d） + Cyclophosphamide 50mg/kg/d×4d （-5d ~ -2d）+ ATG 2.5mg/kg/d×4d（-5d ~ -2d） 2. ATG + CD20 monoclonal antibody (test arm) Busulfan 3.2 mg/kg/d（0.8 mg/kg，q6h）×2d（-7d ~ -6d） + Cyclophosphamide 50mg/kg/d×4d （-5d ~ -2d）+ ATG 2.5mg/kg/d×4d（-5d ~ -2d）+ CD20 monoclonal antibody 375mg/m2, -1d | Drug | ATG + CD20 monoclonal antibody (test arm) |
| ATG4.2 Conditioning Regimen 4.2.1 Matched sibling donor 1. ATG arm (control group) Fludarabine 30mg/m2/d×6d（-7d ~ -2d）+ Cyclophosphamide 50mg/kg/d×2d （-4d ~ -3d）+ ATG 2.5mg/kg/d×5d（-8d ~ -4d） 2. ATG + CD20 monoclonal antibody (test arm) Fludarabine 30mg/m2/d×6d（-7d ~ -2d） + Cyclophosphamide 50mg/kg/d×2d （-4d ~ -3d）+ ATG 2.5mg/kg/d×5d（-8d ~ -4d）+ CD20 monoclonal antibody 375mg/m2, -1d 4.2.2 Unrelated donor and haploidentical donor 1. ATG arm (control group) Busulfan 3.2 mg/kg/d（0.8 mg/kg，q6h）×2d（-7d ~ -6d） + Cyclophosphamide 50mg/kg/d×4d （-5d ~ -2d）+ ATG 2.5mg/kg/d×4d（-5d ~ -2d） 2. ATG + CD20 monoclonal antibody (test arm) Busulfan 3.2 mg/kg/d（0.8 mg/kg，q6h）×2d（-7d ~ -6d） + Cyclophosphamide 50mg/kg/d×4d （-5d ~ -2d）+ ATG 2.5mg/kg/d×4d（-5d ~ -2d）+ CD20 monoclonal antibody 375mg/m2, -1d | Drug | ATG arm (control group) |
Primary outcome
GVHD incidence
Time frame: 2 years
GVHD incidence, location and grade. Infection incidence and recurrence rate.
4 secondary outcomes
Infection incidence
Time frame: 2 years
GVHD-free survival rate
Time frame: 2 years
transplant related mortality
Time frame: 2 years
overall survival rate
Time frame: 2 years
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 24 Aug 2022.
- 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.
depei wu principal investigator
The First Affiliated Hospital of Soochow University
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 CD20 monoclonal antibody and ATG in people with Aplastic Anemia, run by The First Affiliated Hospital of Soochow University 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 11 inclusion and 12 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 2022. 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 depei wu (67781856 ext. 0512, wudepei@suda.edu.cn), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
GVHD incidence (time frame: 2 years). 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 2022.