Severe Aplastic AnemiaPhase 2NCT06378060
Clinical Study on Modified Allogeneic Hematopoietic Stem Cell Transplantation Regimen for Severe Aplastic Anemia
A Phase 2 study testing Modified transplantation system in people with Severe Aplastic Anemia, run by Hematology department of the 920th hospital at 1 site in China.
Trial summary
The aim of this study was to evaluate the safety and efficacy of a modified allogeneic hematopoietic stem cell transplantation regimen for aplastic anemia. Read more about this study
- Who is it for?
- Ages 3 to 65 yearsHealthy volunteers can take part
- Conditions the study is for
- Severe Aplastic Anemia
- What is being tested?
- Modified transplantation system
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 30 participants
- Where
- 1 siteChina
- Study dates
- 1 Mar 2024to 1 Apr 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 3 to 65 years
- All sexes
- Healthy volunteers accepted
- Child, Adult, Older adult
Who may be able to join
- Patients with benign or malignant hematological diseases such as leukemia, lymphoma, thalassemia, aplastic anemia, etc. diagnosed by NCCN guidelines, and requiring allogeneic hematopoietic stem cell transplantation as determined by the researchers;
- 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 nonhematologic 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. Contact the study team or a site near you to ask about taking part.
China1 site, 1 recruiting
- Recruiting
920th Hospital of Joint Logistics Support Force of People's Liberation Army of China
Kunming, Yunnan, 650000
Wang PI sanbin, Doctor, 0871-64774206, Wangsanbin2022@126.com
About this study
Official title: A Prospective, Multi-center, Single-arm Clinical Study to Evaluate the Safety and Efficacy of a Modified Allogeneic Hematopoietic Stem Cell Transplantation Regimen for Severe Aplastic Anemia
The aim of this study was to evaluate the safety and efficacy of a modified allogeneic hematopoietic stem cell transplantation regimen for aplastic anemia.
Read the detailed description
ObjectiveTo evaluate the safety and efficacy of a modified allogeneic hematopoietic stem cell transplantation regimen in aplastic anemia. 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%. We designed this study to improve the conditioning regimen and optimize the GHVD prevention measures to improve the transplant success rate and prolong patient survival, while minimizing the occurrence of GVHD and reducing the recurrence rate of the disease.30 patients with aplastic anemia were planned to be enrolled.
- Conditions
- Severe Aplastic Anemia
- Being tested
- Modified transplantation system
- Keywords
- Severe Aplastic Anemia, Modified transplantation system, Ruxolitinib, Melphalan
- 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 systemDrug
Conditioning regimen: fludarabine 30mg/m2×5~6d,cyclophosphamide 50mg/kg×2d, melphalan 100mg/m2×1d aGVHD prophylaxis: PTCY 25mg/kg×2d, ATG 4.5mg/kg total, MMF 15mg/kg +5d~+35d, Ruxolitinib 5mg bid -1d~+50d and 2.5mg bid +51d~+110d, CSA
| Study group | Role | What they receive |
|---|---|---|
| Experimental group | Experimental | Not described Receives: Drug: Modified transplantation system |
What is being measured
The primary outcome is the main question the study is designed to answer.
overall survival (OS)
2-year overall survival (OS) and graft-versus-host disease and relapse-free survival (GRFS) after transplantation
Measured: [Time Frame: 2 years after allogeneic transplantation with improved conditioning regimen]
Acute graft-versus-host disease incidence
Measured: [Time Frame: 2 years after allogeneic transplantation with improved conditioning regimen]
Secondary outcomes (5)
Transplantation-related motality
Measured: [Time Frame: 2 years after allogeneic transplantation with improved conditioning regimen]
Chronic graft-versus-host disease incidence
Measured: [Time Frame: 2 years after allogeneic transplantation with improved conditioning regimen]
incidence of Intensive fungal disease
Measured: [Time Frame: 2 years after allogeneic transplantation with improved conditioning regimen]
EB virus reactivation rate
Measured: [Time Frame: 2 years after allogeneic transplantation with improved conditioning regimen]
CMV reactivation rate
Measured: [Time Frame: 2 years after allogeneic transplantation with improved conditioning regimen]
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted22 Apr 2024
- Study start1 Mar 2024
- Primary completion1 Apr 2026 (estimated)
- Study completion1 Apr 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
- 30 participants (estimated)
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
- NCT06378060
- Sponsor's protocol ID
- KM04
- Status last verified
- Mar 2024
- First submitted
- 17 Apr 2024
- Last updated
- 22 Apr 2024
- Results posted
- No
- Data monitoring committee
- Yes
- 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 22 Apr 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Prospective, Multi-center, Single-arm Clinical Study to Evaluate the Safety and Efficacy of a Modified Allogeneic Hematopoietic Stem Cell Transplantation Regimen for Severe Aplastic Anemia
- ClinicalTrials.gov ID
- NCT06378060
- Acronym / sponsor ID
- KM04
- Conditions
- Severe Aplastic Anemia
- Keywords
- Severe Aplastic Anemia, Modified transplantation system, Ruxolitinib, Melphalan
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 30 participants (estimated)
- Lead sponsor
- Hematology department of the 920th hospitalUniversity, hospital or other
- Study start
- 1 Mar 2024 (actual)
- Primary completion
- 1 Apr 2026 (estimated)
- Study completion
- 1 Apr 2027 (estimated)
- First posted
- 22 Apr 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.
The aim of this study was to evaluate the safety and efficacy of a modified allogeneic hematopoietic stem cell transplantation regimen for aplastic anemia.
Detailed description
ObjectiveTo evaluate the safety and efficacy of a modified allogeneic hematopoietic stem cell transplantation regimen in aplastic anemia. 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%. We designed this study to improve the conditioning regimen and optimize the GHVD prevention measures to improve the transplant success rate and prolong patient survival, while minimizing the occurrence of GVHD and reducing the recurrence rate of the disease.30 patients with aplastic anemia were planned to be enrolled.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Experimental group | Experimental | Drug: Modified transplantation system |
Who may qualify
- Ages
- 3 to 65 years
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (7 listed)
- Patients with benign or malignant hematological diseases such as leukemia, lymphoma, thalassemia, aplastic anemia, etc. diagnosed by NCCN guidelines, and requiring allogeneic hematopoietic stem cell transplantation as determined by the researchers;
- 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 nonhematologic 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 systemConditioning regimen: fludarabine 30mg/m2×5~6d,cyclophosphamide 50mg/kg×2d, melphalan 100mg/m2×1d aGVHD prophylaxis: PTCY 25mg/kg×2d, ATG 4.5mg/kg total, MMF 15mg/kg +5d~+35d, Ruxolitinib 5mg bid -1d~+50d and 2.5mg bid +51d~+110d, CSA | Drug | Experimental group |
Primary outcome
overall survival (OS)
Time frame: [Time Frame: 2 years after allogeneic transplantation with improved conditioning regimen]
2-year overall survival (OS) and graft-versus-host disease and relapse-free survival (GRFS) after transplantation
Acute graft-versus-host disease incidence
Time frame: [Time Frame: 2 years after allogeneic transplantation with improved conditioning regimen]
5 secondary outcomes
Transplantation-related motality
Time frame: [Time Frame: 2 years after allogeneic transplantation with improved conditioning regimen]
Chronic graft-versus-host disease incidence
Time frame: [Time Frame: 2 years after allogeneic transplantation with improved conditioning regimen]
incidence of Intensive fungal disease
Time frame: [Time Frame: 2 years after allogeneic transplantation with improved conditioning regimen]
EB virus reactivation rate
Time frame: [Time Frame: 2 years after allogeneic transplantation with improved conditioning regimen]
CMV reactivation rate
Time frame: [Time Frame: 2 years after allogeneic transplantation with improved conditioning regimen]
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 22 Apr 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 Modified transplantation system in people with Severe Aplastic Anemia, 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: looking for participants now, as of the sponsor's last update on 22 Apr 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 wang PI sanbin, Doctor (0871-64774206, Wangsanbin2022@126.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
overall survival (OS) (time frame: [Time Frame: 2 years after allogeneic transplantation with improved conditioning regimen]). 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 Apr 2024.