Severe Aplastic AnemiaNCT06039436
Conditioning Regimen Containing Low Dose ATG for The Treatment of Acquired SAA Receiving sUCBT
An observational study of Severe Aplastic Anemia, run by Anhui Provincial Hospital at 1 site in China.
Trial summary
To evaluate the safety and efficacy of Single Umbilical Cord blood transplantation (sUCBT) containing low dose ATG based conditioning regimen in the treatment of acquired Severe Aplastic Anemia (SAA). Read more about this study
- Who is it for?
- Ages 1 year to 50 years
- Conditions the study is for
- Severe Aplastic Anemia
- What is being tested?
- Anti-Thymocyte Globulin (Rabbit)
- How many people
- 72 participants
- Where
- 1 siteChina
- Study dates
- Oct 2023to 31 May 2025
- How long will I take part?
- 12 Months
- Phase
- Observational
- 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 1 year to 50 years
- All sexes
- No healthy volunteers
- Child, Adult
Study population: patients with indications for hematopoietic stem cell transplantation and initially diagnosed with severe aplastic anemia or have not responded to immunosuppressive therapy
Who may be able to join
- Patients diagnosed with severe aplastic anemia who do not respond to initial diagnosis or immunosuppressive therapy;
- Patients meet diagnostic criteria for severe aplastic anemia (Camitta criteria);
- Age from 1 to 50 years old, male or female;
- Patients without HLA-matched sibling or unrelated donors avaliable;
- Patients who have undergone unrelated cord blood transplantation need to have HLA high-resolution matching、TNC (before cord blood freezing)≥3.0×107/kg、CD34+(before cord blood freezing)≥1.5×107/kg、tubule recovery rate≥80%;
- patients and their family members or guardians agreed to the study and signed informed consent;
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- No severe organ failure;
- ECOG score≤2 and HCT-CI≤2;
- serum ferritin (SF)<1000ug/L;
- women of reproductive agemust have a negative pregnancy test and agree to use effective contraception during treatment and for one year thereafter.
Who cannot join
- patients with inherited bone marrow failure syndromes;
- Positive for HLA-A, B, Cw, DRB1, DQB1, DPB1 site-specific HLA antibodies for donor cord blood with solid-phase fluorescence assay;
- patients who have previously received allogeneic hematopoietic stem cell transplantation or solid organ transplantation;
- Uncontrolled bacterial, viral, or fungal infections;
- HIV infection or active viral hepatitis B or C infection;
- Pregnant or breastfeeding women;
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- patients with a history of primary malignancy within 3 years prior to transplant treatment;
- patients receiving ATG treatment within 2 weeks before transplantation;
- patients with drug dependent or uncontrolled mental illness or cognitive impairment;
- Participants in other similar clinical studies within 3 months;
- Patients allergic to ATG;
- patients considered unsuitable for inclusion by the researchers.
Trial locations and contacts
1 site in 1 country.
China1 site, 1 recruiting
- Recruiting
The First Affiliated Hospital of University of Science and Technology of China (Anhui Provincial Hospital)
Hefei, Anhui, 230036
Xiaoyu Zhu, ph.D, 15255456091, xiaoyuz@ustc.cn
Baolin Tang, M.D, 13856901277, zwcrystalbl@163.com
About this study
Official title: Conditioning Regimen Containing Low Dose ATG for The Treatment of Acquired Severe Aplastic Aplasia Receiving Single Cord Blood Transplant: A Multi-center, Single-arm Study
To evaluate the safety and efficacy of Single Umbilical Cord blood transplantation (sUCBT) containing low dose ATG based conditioning regimen in the treatment of acquired Severe Aplastic Anemia (SAA).
Read the detailed description
Hematopoietic stem cell transplantation (HSCT) from HLA-compatible sibling donors is the standard preferred therapy for Severe Aplastic Anemia (SAA). At present, HLA-compatible donors in China fail to meet the needs of patients for transplantation. Umbilical Cord blood transplantation (UCBT) is an alternative treatment for patients with emergency conditions who lack compatible sibling donors, have failed immunosuppressant therapies or cannot wait for unrelated fully compatible donors. However, implantation failure is one of the main problems in UCBT for SAA due to the low cord blood stem cells. Nowadays, there is no consensus on the optimal conditioning regimen for UCBT treatment of SAA, and whether ATG should be added to the conditioning regimen is still controversial. Early clinical studies in UCBT have shown that the sufficient ATG in the conditioning regimen will lead to a decrease rate of implantation rate and affect survival of UCBT ultimately. However, a range of recent studies have found that with the addition of low-dose ATG, T cell reconstitution seems to outperform bone marrow or peripheral blood stem cell transplantation. Therefore, how to properly use ATG to both retain its effective GVHD prevention effect and reduce its impact on immune reconstruction has important clinical significance for improving the transplantation efficacy. In this study, a multicenter clinical study will be conducted to observe the safety and efficacy of UCBT with low dose ATG in the treatment of SAA and the long-term quality of life of patients.
- Conditions
- Severe Aplastic Anemia
- Being tested
- Anti-Thymocyte Globulin (Rabbit)
- Keywords
- Anti-Thymocyte Globulin (Rabbit), Umbilical Cord Blood Transplantation, Severe Aplastic Anemia
- Medical subject headings
- Anemia, Aplastic
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Anti-Thymocyte Globulin (Rabbit)Drug
Indications for trial stopping: subjects occur pregnancy events, severe organ function impairments, or emergency events related or unrelated to treatment; or subjects are unwilling to continue participating in the clinical trial.
| Study group | Role | What they receive |
|---|---|---|
| ATG group | Patients in the ATG group will be treated with conditioning regimen containing ATG 2mg/kg on day -7 prior to transplant. Receives: Drug: Anti-Thymocyte Globulin (Rabbit) |
What is being measured
The primary outcome is the main question the study is designed to answer.
The one-year probability of GRFS
GRFS is defined as survival without grade III-IV acute GVHD immunosuppression requiring chronic GVHD or relapse.
Measured: one year
Secondary outcomes (11)
The cumulative incidence of platelet engraftment at 28 days after transplantation
Platelet engraftment is defined as independent from platelet transfusion for at least 7 days with a platelet count of more than 20 × 10^9/L.
Measured: 28 days
The cumulative incidence of neutrophil engraftment at 28 days after transplantation
Neutrophil engraftment is defined as neutrophil count achieves 0.5 ×10^9/L for three consecutive days.
Measured: 28 days
Time of platelet engraftment
Platelet engraftment is defined as independent from platelet transfusion for at least 7 days with a platelet count of more than 20 × 10^9/L.
Measured: 28 days
Time of neutrophil engraftment
Neutrophil engraftment is defined as neutrophil count achieves 0.5 ×10^9/L for three consecutive days.
Measured: 28 days
The cumulative incidence of acute GVHD at 100 days after transplantation
Acute GVHD is defined that occurs within 100 days after transplantation
Measured: 100 days
The cumulative incidence of chronic GVHD at 100 days after transplantation
Chronic GVHD is defined that occurs 100 days after transplantation
Measured: one year
Immune reconstitution after transplantation
The level of immunoglobulin, T cells, B cells and NK cells reconstitution after transplantation
Measured: one year
The 180-day probability of TRM
TRM(transplant-related mortality) is defined that death due to any transplant-related cause within 180 days after transplantation
Measured: 180 days
The incidence of infection
Infection is defined as various post-transplant associated infections
Measured: one year
The one-year probability of OS
OS (overall survival) is defined as the time which begins at diagnosis and up to the time of death
Measured: one year
The one-year probability of DFS
DFS (disease free survival) is defined as the time from treatment until the recurrence of disease (or death) after transplantation
Measured: one year
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted15 Sep 2023
- Study startOct 2023 (estimated)
- Primary completion30 Apr 2025 (estimated)
- Study completion31 May 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 Anhui Provincial 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
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 72 participants (estimated)
- Target duration
- 12 Months
Sponsor and investigators
- Lead sponsor
- Anhui Provincial Hospital
- Sponsor type
- Other government
- Responsible party
- Principal investigator, Xiaoyu Zhu, Anhui Provincial Hospital
- Investigators
- Xiaoyu Zhu, ph.D, Principal investigator, The First Affiliated Hospital of University of Science and Technology of China (Anhui Provicial Hospital)
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06039436
- Sponsor's protocol ID
- CBSAA001
- Status last verified
- Oct 2023
- First submitted
- 9 Sep 2023
- Last updated
- 24 Oct 2023
- 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 24 Oct 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Conditioning Regimen Containing Low Dose ATG for The Treatment of Acquired Severe Aplastic Aplasia Receiving Single Cord Blood Transplant: A Multi-center, Single-arm Study
- ClinicalTrials.gov ID
- NCT06039436
- Acronym / sponsor ID
- CBSAA001
- Conditions
- Severe Aplastic Anemia
- Keywords
- Anti-Thymocyte Globulin (Rabbit), Umbilical Cord Blood Transplantation, Severe Aplastic Anemia
- Study type
- Observational
- Recruitment status
- Unknown status
- Enrolment
- 72 participants (estimated)
- Lead sponsor
- Anhui Provincial HospitalOther government
- Study start
- Oct 2023 (estimated)
- Primary completion
- 30 Apr 2025 (estimated)
- Study completion
- 31 May 2025 (estimated)
- First posted
- 15 Sep 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.
To evaluate the safety and efficacy of Single Umbilical Cord blood transplantation (sUCBT) containing low dose ATG based conditioning regimen in the treatment of acquired Severe Aplastic Anemia (SAA).
Detailed description
Hematopoietic stem cell transplantation (HSCT) from HLA-compatible sibling donors is the standard preferred therapy for Severe Aplastic Anemia (SAA). At present, HLA-compatible donors in China fail to meet the needs of patients for transplantation. Umbilical Cord blood transplantation (UCBT) is an alternative treatment for patients with emergency conditions who lack compatible sibling donors, have failed immunosuppressant therapies or cannot wait for unrelated fully compatible donors. However, implantation failure is one of the main problems in UCBT for SAA due to the low cord blood stem cells. Nowadays, there is no consensus on the optimal conditioning regimen for UCBT treatment of SAA, and whether ATG should be added to the conditioning regimen is still controversial. Early clinical studies in UCBT have shown that the sufficient ATG in the conditioning regimen will lead to a decrease rate of implantation rate and affect survival of UCBT ultimately. However, a range of recent studies have found that with the addition of low-dose ATG, T cell reconstitution seems to outperform bone marrow or peripheral blood stem cell transplantation. Therefore, how to properly use ATG to both retain its effective GVHD prevention effect and reduce its impact on immune reconstruction has important clinical significance for improving the transplantation efficacy. In this study, a multicenter clinical study will be conducted to observe the safety and efficacy of UCBT with low dose ATG in the treatment of SAA and the long-term quality of life of patients.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Target follow-up
- 12 Months
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| ATG group | – | Drug: Anti-Thymocyte Globulin (Rabbit)Patients in the ATG group will be treated with conditioning regimen containing ATG 2mg/kg on day -7 prior to transplant. |
Who may qualify
- Ages
- 1 year to 50 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
- Study population
- patients with indications for hematopoietic stem cell transplantation and initially diagnosed with severe aplastic anemia or have not responded to immunosuppressive therapy
Key inclusion criteria (10 listed)
- Patients diagnosed with severe aplastic anemia who do not respond to initial diagnosis or immunosuppressive therapy;
- Patients meet diagnostic criteria for severe aplastic anemia (Camitta criteria);
- Age from 1 to 50 years old, male or female;
- Patients without HLA-matched sibling or unrelated donors avaliable;
- Patients who have undergone unrelated cord blood transplantation need to have HLA high-resolution matching、TNC (before cord blood freezing)≥3.0×107/kg、CD34+(before cord blood freezing)≥1.5×107/kg、tubule recovery rate≥80%;
Key exclusion criteria (12 listed)
- patients with inherited bone marrow failure syndromes;
- Positive for HLA-A, B, Cw, DRB1, DQB1, DPB1 site-specific HLA antibodies for donor cord blood with solid-phase fluorescence assay;
- patients who have previously received allogeneic hematopoietic stem cell transplantation or solid organ transplantation;
- Uncontrolled bacterial, viral, or fungal infections;
- HIV infection or active viral hepatitis B or C 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 |
|---|---|---|
| Anti-Thymocyte Globulin (Rabbit)Indications for trial stopping: subjects occur pregnancy events, severe organ function impairments, or emergency events related or unrelated to treatment; or subjects are unwilling to continue participating in the clinical trial. | Drug | ATG group |
Primary outcome
The one-year probability of GRFS
Time frame: one year
GRFS is defined as survival without grade III-IV acute GVHD immunosuppression requiring chronic GVHD or relapse.
11 secondary outcomes
The cumulative incidence of platelet engraftment at 28 days after transplantation
Time frame: 28 days
The cumulative incidence of neutrophil engraftment at 28 days after transplantation
Time frame: 28 days
Time of platelet engraftment
Time frame: 28 days
Time of neutrophil engraftment
Time frame: 28 days
The cumulative incidence of acute GVHD at 100 days after transplantation
Time frame: 100 days
The cumulative incidence of chronic GVHD at 100 days after transplantation
Time frame: one year
Immune reconstitution after transplantation
Time frame: one year
The 180-day probability of TRM
Time frame: 180 days
The incidence of infection
Time frame: one year
The one-year probability of OS
Time frame: one year
The one-year probability of DFS
Time frame: one year
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 24 Oct 2023.
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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.
Xiaoyu Zhu, ph.D principal investigator
The First Affiliated Hospital of University of Science and Technology of China (Anhui Provicial Hospital)
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Severe Aplastic Anemia, run by Anhui Provincial 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 10 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 Oct 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.
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 Xiaoyu Zhu, ph.D (15255456091, xiaoyuz@ustc.edu.cn), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
The one-year probability of GRFS (time frame: one year). 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 Oct 2023.