Modified Transplantation Regimen and aGVHD Prophylaxis for Severe Aplastic Anemia in the Setting of Allogeneic HSCT. (aGVHD)

September 16, 2025 updated by: Hematology department of the 920th hospital

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.

Study Overview

Status

Status

Indicates the current recruitment status or the expanded access status.
Completed

Conditions

Conditions

The disease, disorder, syndrome, illness, or injury that is being studied. On ClinicalTrials.gov, conditions may also include other health-related issues, such as lifespan, quality of life, and health risks.

Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.

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 Type

Study Type

Describes the nature of a clinical study. Study types include interventional studies (also called clinical trials), observational studies (including patient registries), and expanded access.
Observational

Enrollment (Actual)

Enrollment

The number of participants in a clinical study. The "anticipated" enrollment is the target number of participants that the researchers need for the study.
72

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • China
    • Yunnan
      • Kunming, Yunnan, China, 650000
        • Department of Hematology，920th Hospital of Joint Logistics Support Force

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Eligibility Criteria

The key requirements that people who want to participate in a clinical study must meet or the characteristics they must have. Eligibility criteria consist of both inclusion criteria (which are required for a person to participate in the study) and exclusion criteria (which prevent a person from participating). Types of eligibility criteria include whether a study accepts healthy volunteers, has age or age group requirements, or is limited by sex.

Ages Eligible for Study

  • Child
  • Adult
  • Older Adult

Accepts Healthy Volunteers

No

Sampling Method

Non-Probability Sample

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;

Description

Inclusion Criteria:

  1. Patients who were diagnosed with severe aplastic anemia by NCCN guidelines, and has received HSCT with this transplantation preconditioning and aGVHD prophylaxis regimen;
  2. Age 3-65 years old;
  3. Weight 10Kg-100Kg;
  4. Eastern Cooperative Oncology Group (ECOG) score ≤3;
  5. 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);
  6. No severe infection;
  7. Subjects voluntarily participated in this clinical trial and signed the informed consent.

Exclusion Criteria:

  1. Patients with other hematologic diseases who are not eligible for transplantation or who do not wish to receive transplantation;
  2. Patients with an expected survival of less than 1 month;
  3. Patients with previous autologous or allogeneic hematopoietic stem cell transplantation;
  4. pregnant patients;
  5. Patients with severe mental or neurological disorders that would affect the ability to provide informed consent and/or to report or observe adverse events;
  6. Other conditions that the investigator determines to be inappropriate for enrollment.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

What is the study measuring?

Primary Outcome Measures

Primary Outcome Measures

In a clinical study's protocol, the planned outcome measure that is the most important for evaluating the effect of an intervention/treatment. Most clinical studies have one primary outcome measure, but some have more than one.
Outcome Measure
Measure Description
Time Frame
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
From the day of HSCs transfusion to +100 day after HSCT.
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
From the day of HSCT transfusion to 100 days after HSCT

Secondary Outcome Measures

Secondary Outcome Measures

In a clinical study's protocol, a planned outcome measure that is not as important as the primary outcome measure for evaluating the effect of an intervention but is still of interest. Most clinical studies have more than one secondary outcome measure.
Outcome Measure
Measure Description
Time Frame
2-year Overall survival rate
Time Frame: 24 months
proportioin of patients who still be alive within 24 months after HSCT
24 months
Disease-free survival
Time Frame: 24 months
proportion of patients still be alive without GVHD or other diseases.
24 months
Poor graft function rate
Time Frame: From the day of HSCs transfusion to 24 months after HSCT
the presence of at least two cytopenic counts beyond +28 days with a transfusion requirement associated with hypoplastic-aplastic bone marrow
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
the proportion of patients who developed chronic GVHD within the observation duration
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
the incidence of adverse events during the transplantation preconditioning periods.
From the first day of preconditioning to +100 days after hematopoietic stem cell Transfusion

Other Outcome Measures

Other Outcome Measures

For clinical trials, a planned measurement described in the protocol that is used to determine the effect of an intervention/treatment on participants. For observational studies, a measurement or observation that is used to describe patterns of diseases or traits, or associations with exposures, risk factors, or treatment. Types of outcome measures include primary outcome measure and secondary outcome measure.
Outcome Measure
Measure Description
Time Frame
Fungal infections rate
Time Frame: 2 years
Proportion of patients who developed fungal infection after HSCT
2 years
EB virus reactivation rate
Time Frame: within 100 days after HSCT
Proportion of patient who experience EB virus reactivation
within 100 days after HSCT
cytomegalovirus reactivatioin
Time Frame: within 100 days after HSCT
proportion of patient who experience cytomegalovirus reactivatioin
within 100 days after HSCT

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Sponsor

The organization or person who initiates the study and who has authority and control over the study.
Hematology department of the 920th hospital

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

Study Start

The actual date on which the first participant was enrolled in a clinical study. The "anticipated" study start date is the date that the researchers think will be the study start date.
June 1, 2020

Primary Completion (Actual)

Primary Completion

The date on which the last participant in a clinical study was examined or received an intervention to collect final data for the primary outcome measure. Whether the clinical study ended according to the protocol or was terminated does not affect this date. For clinical studies with more than one primary outcome measure with different completion dates, this term refers to the date on which data collection is completed for all the primary outcome measures. The "anticipated" primary completion date is the date that the researchers think will be the primary completion date for the study.
June 30, 2023

Study Completion (Actual)

Study Completion

The date on which the last participant in a clinical study was examined or received an intervention/treatment to collect final data for the primary outcome measures, secondary outcome measures, and adverse events (that is, the last participant's last visit). The "anticipated" study completion date is the date that the researchers think will be the study completion date.
August 1, 2025

Study Registration Dates

First Submitted

First Submitted

The date on which the study sponsor or investigator first submitted a study record to ClinicalTrials.gov. There is typically a delay of a few days between the first submitted date and the record's availability on ClinicalTrials.gov (the first posted date).
February 16, 2025

First Submitted That Met QC Criteria

First Submitted That Met QC Criteria

The date on which the study sponsor or investigator first submits a study record that is consistent with National Library of Medicine (NLM) quality control (QC) review criteria. The sponsor or investigator may need to revise and submit a study record one or more times before NLM's QC review criteria are met. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
February 16, 2025

First Posted (Actual)

First Posted

The date on which the study record was first available on ClinicalTrials.gov after National Library of Medicine (NLM) quality control (QC) review has concluded. There is typically a delay of a few days between the date the study sponsor or investigator submitted the study record and the first posted date.
February 20, 2025

Study Record Updates

Last Update Posted (Actual)

Last Update Posted

The most recent date on which changes to a study record were made available on ClinicalTrials.gov. There may be a delay between when the changes were submitted to ClinicalTrials.gov by the study's sponsor or investigator (the last update submitted date) and the last update posted date.
September 22, 2025

Last Update Submitted That Met QC Criteria

Last Update Submitted That Met QC Criteria

The most recent date on which the study sponsor or investigator submitted changes to a study record that are consistent with National Library of Medicine (NLM) quality control (QC) review criteria. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
September 16, 2025

Last Verified

Last Verified

The most recent date on which the study sponsor or investigator confirmed the information about a clinical study on ClinicalTrials.gov as accurate and current. If a study with a recruitment status of recruiting; not yet recruiting; or active, not recruiting has not been confirmed within the past 2 years, the study's recruitment status is shown as unknown.
September 1, 2025

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

Other Study ID Numbers

Identifiers or ID numbers other than the NCT number that are assigned to a clinical study by the study's sponsor, funders, or others. These numbers may include unique identifiers from other trial registries and National Institutes of Health grant numbers.
  • KM-08
  • 202301AY070001-226 (Other Grant/Funding Number: the Joint and Special Project of Kunming medical university)

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

UNDECIDED

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

product manufactured in and exported from the U.S.

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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