Aplastic AnaemiaaGVHDNCT06837987
Modified Transplantation Regimen and aGVHD Prophylaxis for Severe Aplastic Anemia in the Setting of Allogeneic HSCT.
An observational study of Aplastic Anaemia and Transplantation Conditioning, run by Hematology department of the 920th hospital at 1 site in China.
Trial summary
The investigators retrospectively evaluted the safety and efficacy of the modified transplantation conditioning and aGVHD prophylaxis in severe aplastic anemia in 4 transplantation centers. Read more about this study
- Who is it for?
- Ages 3 to 65 years
- Conditions the study is for
- Aplastic Anaemia, Transplantation Conditioning
- What is being tested?
- Modified transplantation system
- How many people
- 72 participants
- Where
- 1 siteChina
- Study dates
- 1 Jun 2020to 1 Aug 2025
- 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 3 to 65 years
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Study population: Patients who were diagnosed with severe aplastic anemia by NCCN guidelines, and has received HSCT with this transplantation preconditioning and aGVHD prophylaxis regimen;
Who may be able to join
- Patients who were diagnosed with severe aplastic anemia by NCCN guidelines, and has received HSCT with this transplantation preconditioning and aGVHD prophylaxis regimen;
- Age 3-65 years old;
- Weight 10Kg-100Kg;
- Eastern Cooperative Oncology Group (ECOG) score ≤3;
- No major organ injury (ECG ejection fraction >45%; bilirubin < 2 times the upper limit of normal value; AST and ALT < 3 times the upper limit of normal value; serum creatinine < 2 times the upper limit of normal value);
- No severe infection;
- Subjects voluntarily participated in this clinical trial and signed the informed consent.
Who cannot join
- Patients with other hematologic diseases who are not eligible for transplantation or who do not wish to receive transplantation;
- Patients with an expected survival of less than 1 month;
- Patients with previous autologous or allogeneic hematopoietic stem cell transplantation;
- pregnant patients;
- Patients with severe mental or neurological disorders that would affect the ability to provide informed consent and/or to report or observe adverse events;
- Other conditions that the investigator determines to be inappropriate for enrollment.
Trial locations and contacts
1 site in 1 country.
China1 site
Department of Hematology，920th Hospital of Joint Logistics Support Force
Kunming, Yunnan, 650000
About this study
Official title: An Observation of the Safety and Efficacy of a Modified Transplantation Conditioning and aGVHD Prophylaxis for Severe Aplastic Anemia--a Retrospective, Double-center, Single-arm Clinical Study.
The investigators retrospectively evaluted the safety and efficacy of the modified transplantation conditioning and aGVHD prophylaxis in severe aplastic anemia in 4 transplantation centers.
Read the detailed description
Aplastic anemia (AA) is a group of myelo-hemopoietic failure syndromes caused by a variety of etiologies. If not intervened, the average expected survival time is less than half a year.Allogeneic hematopoietic stem cell transplantation (allo-HSCT) is one of the possible cures for this disease. The success rate of treatment for this disease can be further improved under the previous regimen system. The survival rate reported in the literature is 60%-90%. From Jun. 2020 to Dec. 2023, 72 patients in 4 transplantation center received this modified transplantation system, The investigators designed this clinical trial to retrospectively evaluted the safety and efficacy of the modified transplantation conditioning and aGVHD prophylaxis in severe aplastic anemia in the 4 clinical center.
- Conditions
- Aplastic Anaemia, Transplantation Conditioning
- Being tested
- Modified transplantation system
- Keywords
- modified transplantation conditioning, aplastic anemia, aGVHD prophylaxis
- Medical subject headings
- Anemia, Aplastic
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Modified transplantation systemOther
Modified transplantation conditioning include: Fludarabine 30mg/m2*6 day, Melphalan 100mg/m2*1 day, cyclophosphamide 50mg/kg*2 day.
What is being measured
The primary outcome is the main question the study is designed to answer.
Primary graft falure rate
no apperance or complete loss of donor-derived neutrophils by +28 days
Measured: From the day of HSCs transfusion to +100 day after HSCT.
aGVHD incidence
proportion of patients who developed aGVHD within 100 days after HSCT
Measured: From the day of HSCT transfusion to 100 days after HSCT
Secondary outcomes (5)
Poor graft function rate
the presence of at least two cytopenic counts beyond +28 days with a transfusion requirement associated with hypoplastic-aplastic bone marrow
Measured: From the day of HSCs transfusion to 24 months after HSCT
cGVHD incidence
the proportion of patients who developed chronic GVHD within the observation duration
Measured: From +100 days after HSCT to the follow-up date
adverse events
the incidence of adverse events during the transplantation preconditioning periods.
Measured: From the first day of preconditioning to +100 days after hematopoietic stem cell Transfusion
2-year Overall survival rate
proportioin of patients who still be alive within 24 months after HSCT
Measured: 24 months
Disease-free survival
proportion of patients still be alive without GVHD or other diseases.
Measured: 24 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted20 Feb 2025
- Study start1 Jun 2020
- Primary completion30 Jun 2023
- Study completion1 Aug 2025
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Aplastic AnaemiaStudies 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
- Observational
- Observational model
- Case only
- Time perspective
- Retrospective
- Enrolment
- 72 participants (actual)
Sponsor and investigators
- Lead sponsor
- Hematology department of the 920th hospital
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06837987
- Sponsor's protocol ID
- KM-08
- Other IDs
- 202301AY070001-226
- Acronym
- aGVHD
- Status last verified
- Sep 2025
- First submitted
- 16 Feb 2025
- Last updated
- 22 Sep 2025
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Undecided
Study at a glance
From the registry record, last updated by the sponsor on 22 Sep 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Observation of the Safety and Efficacy of a Modified Transplantation Conditioning and aGVHD Prophylaxis for Severe Aplastic Anemia--a Retrospective, Double-center, Single-arm Clinical Study.
- ClinicalTrials.gov ID
- NCT06837987
- Acronym / sponsor ID
- aGVHD / KM-08
- Conditions
- Aplastic Anaemia, Transplantation Conditioning
- Keywords
- modified transplantation conditioning, aplastic anemia, aGVHD prophylaxis
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 72 participants (actual)
- Lead sponsor
- Hematology department of the 920th hospitalUniversity, hospital or other
- Study start
- 1 Jun 2020 (actual)
- Primary completion
- 30 Jun 2023 (actual)
- Study completion
- 1 Aug 2025 (actual)
- First posted
- 20 Feb 2025 (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.
The investigators retrospectively evaluted the safety and efficacy of the modified transplantation conditioning and aGVHD prophylaxis in severe aplastic anemia in 4 transplantation centers.
Detailed description
Aplastic anemia (AA) is a group of myelo-hemopoietic failure syndromes caused by a variety of etiologies. If not intervened, the average expected survival time is less than half a year.Allogeneic hematopoietic stem cell transplantation (allo-HSCT) is one of the possible cures for this disease. The success rate of treatment for this disease can be further improved under the previous regimen system. The survival rate reported in the literature is 60%-90%. From Jun. 2020 to Dec. 2023, 72 patients in 4 transplantation center received this modified transplantation system, The investigators designed this clinical trial to retrospectively evaluted the safety and efficacy of the modified transplantation conditioning and aGVHD prophylaxis in severe aplastic anemia in the 4 clinical center.
Study design
- Observational model
- Case only
- Time perspective
- Retrospective
Who may qualify
- Ages
- 3 to 65 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- Study population
- Patients who were diagnosed with severe aplastic anemia by NCCN guidelines, and has received HSCT with this transplantation preconditioning and aGVHD prophylaxis regimen;
Key inclusion criteria (7 listed)
- Patients who were diagnosed with severe aplastic anemia by NCCN guidelines, and has received HSCT with this transplantation preconditioning and aGVHD prophylaxis regimen;
- Age 3-65 years old;
- Weight 10Kg-100Kg;
- Eastern Cooperative Oncology Group (ECOG) score ≤3;
- No major organ injury (ECG ejection fraction >45%; bilirubin < 2 times the upper limit of normal value; AST and ALT < 3 times the upper limit of normal value; serum creatinine < 2 times the upper limit of normal value);
Key exclusion criteria (6 listed)
- Patients with other hematologic diseases who are not eligible for transplantation or who do not wish to receive transplantation;
- Patients with an expected survival of less than 1 month;
- Patients with previous autologous or allogeneic hematopoietic stem cell transplantation;
- pregnant patients;
- Patients with severe mental or neurological disorders that would affect the ability to provide informed consent and/or to report or observe adverse events;
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 |
|---|---|---|
| Modified transplantation systemModified transplantation conditioning include: Fludarabine 30mg/m2*6 day, Melphalan 100mg/m2*1 day, cyclophosphamide 50mg/kg*2 day. | Other | – |
Primary outcome
Primary graft falure rate
Time frame: From the day of HSCs transfusion to +100 day after HSCT.
no apperance or complete loss of donor-derived neutrophils by +28 days
aGVHD incidence
Time frame: From the day of HSCT transfusion to 100 days after HSCT
proportion of patients who developed aGVHD within 100 days after HSCT
5 secondary outcomes
Poor graft function rate
Time frame: From the day of HSCs transfusion to 24 months after HSCT
cGVHD incidence
Time frame: From +100 days after HSCT to the follow-up date
adverse events
Time frame: From the first day of preconditioning to +100 days after hematopoietic stem cell Transfusion
2-year Overall survival rate
Time frame: 24 months
Disease-free survival
Time frame: 24 months
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 22 Sep 2025.
- Individual participant dataSharing planned: Undecided
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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.
The record lists no contact. Use the official record to reach the sponsor.
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 Aplastic Anaemia and Transplantation Conditioning, run by Hematology department of the 920th 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 7 inclusion and 6 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: finished, as of the sponsor's last update on 22 Sep 2025. 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?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
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?
Primary graft falure rate (time frame: From the day of HSCs transfusion to +100 day after HSCT.). 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 22 Sep 2025.