Severe Aplastic Anemia (SAA)PB-Graft HaploNCT07629947
Peripheral Blood Versus Bone Marrow Plus Peripheral Blood Grafts for Haploidentical Transplantation in Severe Aplastic Anemia
A study testing G-CSF-Primed Peripheral Blood Stem Cell Graft and G-CSF-Primed Bone Marrow Plus Peripheral Blood Stem Cell Graft in people with Severe Aplastic Anemia (SAA), run by Peking University People's Hospital at 1 site in China.
Trial summary
Severe aplastic anemia is a life-threatening bone marrow failure disorder. Haploidentical hematopoietic stem cell transplantation has become an important curative treatment option for patients who do not have an HLA-matched sibling donor. Read more about this study
- Who is it for?
- Ages 18 to 40 years
- Conditions the study is for
- Severe Aplastic Anemia (SAA)
- What is being tested?
- G-CSF-Primed Peripheral Blood Stem Cell Graft and G-CSF-Primed Bone Marrow Plus Peripheral Blood Stem Cell Graft
- Type of treatment
- Surgery or procedure
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 180 participants
- Where
- 1 siteChina
- Study dates
- 15 Jun 2026to 31 Dec 2029
- Phase
- Interventional
- 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 18 to 40 years
- All sexes
- No healthy volunteers
- Adult
Who may be able to join
- Patients with newly diagnosed or relapsed severe aplastic anemia (SAA) or very severe aplastic anemia (vSAA) according to the Camitta criteria.
- SAA is defined as bone marrow cellularity <25% and at least two of the following peripheral blood criteria: absolute neutrophil count <0.5 × 10^9/L, platelet count <20 × 10^9/L, or reticulocyte count <20 × 10^9/L.
- vSAA is defined as SAA with an absolute neutrophil count <0.2 × 10^9/L.
- Age 18 to 40 years.
- Availability of a haploidentical related donor.
- Eastern Cooperative Oncology Group (ECOG) performance status of 0 to 2.
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- Adequate organ function, defined as follows:
- Cardiac function: left ventricular ejection fraction ≥50% and no severe arrhythmia.
- Hepatic function: total bilirubin ≤2 times the upper limit of normal and alanine aminotransferase/aspartate aminotransferase ≤3 times the upper limit of normal.
- Renal function: creatinine clearance ≥60 mL/min.
- No active infection.
- For women of childbearing potential, a negative pregnancy test is required.
- All patients must agree to use effective contraception during the study period.
- Written informed consent provided by the patient or legal representative.
Who cannot join
- Presence of hematologic malignancy or myelodysplastic syndrome-related chromosomal abnormalities, such as +8 or del(7q).
- Prior allogeneic hematopoietic stem cell transplantation or prior organ transplantation.
- Availability of an HLA-matched sibling donor.
- Active uncontrolled infection, including unresolved invasive fungal disease, active tuberculosis, sepsis, or other uncontrolled infections.
- Severe comorbidities, defined as a Hematopoietic Cell Transplantation-Comorbidity Index (HCT-CI) score ≥4, or uncontrolled autoimmune disease.
- Donor-related contraindications, including donor age <12 years or >65 years, donor body mass index <18 or >35 kg/m², contraindications to anesthesia, or contraindications to bone marrow harvest.
- Psychiatric illness that prevents cooperation with study procedures, or any condition considered by the investigator to make the patient unsuitable for study participation.
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
Peking university people's hospital
Beijing, Beijing Municipality, 100033
Zhengli Xu, 15652569255, xuzhengli0202@163.com
About this study
Official title: A Randomized Controlled Trial Comparing Different Graft Sources for Haploidentical Transplantation in Aplastic Anemia
Severe aplastic anemia is a life-threatening bone marrow failure disorder. Haploidentical hematopoietic stem cell transplantation has become an important curative treatment option for patients who do not have an HLA-matched sibling donor.
Traditionally, haploidentical transplantation for severe aplastic anemia uses a graft composed of granulocyte colony-stimulating factor-primed bone marrow plus peripheral blood stem cells. However, bone marrow collection is invasive and may increase donor burden. Peripheral blood stem cell collection is simpler and less invasive, but it remains unclear whether using peripheral blood stem cells alone provides similar clinical outcomes without increasing the risk of graft-versus-host disease.
This multicenter, randomized, open-label, non-inferiority trial will compare granulocyte colony-stimulating factor-primed peripheral blood stem cells alone with granulocyte colony-stimulating factor-primed bone marrow plus peripheral blood stem cells as graft sources for haploidentical transplantation in patients with severe or very severe aplastic anemia. Participants will be randomly assigned in a 1:1 ratio to either graft source group. The primary outcome is the cumulative incidence of grade II-IV acute graft-versus-host disease within 100 days after transplantation. Secondary outcomes include engraftment, chronic graft-versus-host disease, infections, immune reconstitution, survival, and donor safety.
- Conditions
- Severe Aplastic Anemia (SAA)
- Being tested
- G-CSF-Primed Peripheral Blood Stem Cell Graft, G-CSF-Primed Bone Marrow Plus Peripheral Blood Stem Cell Graft
- Medical subject headings
- Anemia, Aplastic
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
G-CSF-Primed Peripheral Blood Stem Cell GraftProcedure or surgery
Haploidentical hematopoietic stem cell transplantation using granulocyte colony-stimulating factor-primed peripheral blood stem cells alone as the graft source. Donors will receive granulocyte colony-stimulating factor mobilization, followed by peripheral blood stem cell collection by apheresis.
G-CSF-Primed Bone Marrow Plus Peripheral Blood Stem Cell GraftProcedure or surgery
Haploidentical hematopoietic stem cell transplantation using granulocyte colony-stimulating factor-primed bone marrow plus peripheral blood stem cells as the graft source. Donors will receive granulocyte colony-stimulating factor mobilization, followed by bone marrow harvest and peripheral blood stem cell collection.
| Study group | Role | What they receive |
|---|---|---|
| G-CSF-Primed Peripheral Blood Stem Cell Graft | Experimental | Participants assigned to this arm will undergo haploidentical hematopoietic stem cell transplantation using granulocyte colony-stimulating factor-primed peripheral blood stem cells alone as the graft source. Donors will receive granulocyte colony-stimulating factor mobilization, and peripheral blood stem cells will be collected by apheresis. Participants will receive the protocol-specified conditioning regimen and graft-versus-host disease prophylaxis. Receives: Procedure: G-CSF-Primed Peripheral Blood Stem Cell Graft |
| G-CSF-Primed Bone Marrow Plus Peripheral Blood Stem Cell Graft | Experimental | Participants assigned to this arm will undergo haploidentical hematopoietic stem cell transplantation using granulocyte colony-stimulating factor-primed bone marrow plus peripheral blood stem cells as the graft source. Donors will receive granulocyte colony-stimulating factor mobilization, followed by bone marrow harvest and peripheral blood stem cell collection. Participants will receive the protocol-specified conditioning regimen and graft-versus-host disease prophylaxis. Receives: Procedure: G-CSF-Primed Bone Marrow Plus Peripheral Blood Stem Cell Graft |
What is being measured
The primary outcome is the main question the study is designed to answer.
Cumulative Incidence of Grade II-IV Acute Graft-Versus-Host Disease Within 100 Days After Transplantation
The cumulative incidence of newly diagnosed or persistent grade II-IV acute graft-versus-host disease within 100 days after haploidentical hematopoietic stem cell transplantation. Acute graft-versus-host disease will be graded according to the MAGIC criteria and assessed based on skin, gastrointestinal, and liver manifestations. Endpoint adjudication will be performed by an independent blinded endpoint review committee using medical records, photographs, endoscopy reports, and/or biopsy reports, as applicable.
Measured: Up to 100 Days After Transplantation
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted5 Jun 2026
- Study start15 Jun 2026
- Primary completion15 Apr 2027 (estimated)
- Study completion31 Dec 2029 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Severe Aplastic Anemia (SAA)Studies 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 N/A
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 180 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Peking University People's Hospital
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Xiao-Jun Huang, Peking University People's Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07629947
- Sponsor's protocol ID
- 2026PHB428-001
- Acronym
- PB-Graft Haplo
- Status last verified
- May 2026
- First submitted
- 1 Jun 2026
- Last updated
- 16 Sep 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 16 Sep 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Randomized Controlled Trial Comparing Different Graft Sources for Haploidentical Transplantation in Aplastic Anemia
- ClinicalTrials.gov ID
- NCT07629947
- Acronym / sponsor ID
- PB-Graft Haplo / 2026PHB428-001
- Conditions
- Severe Aplastic Anemia (SAA)
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 180 participants (estimated)
- Lead sponsor
- Peking University People's HospitalUniversity, hospital or other
- Study start
- 15 Jun 2026 (actual)
- Primary completion
- 15 Apr 2027 (estimated)
- Study completion
- 31 Dec 2029 (estimated)
- First posted
- 5 Jun 2026 (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.
Severe aplastic anemia is a life-threatening bone marrow failure disorder. Haploidentical hematopoietic stem cell transplantation has become an important curative treatment option for patients who do not have an HLA-matched sibling donor.
Traditionally, haploidentical transplantation for severe aplastic anemia uses a graft composed of granulocyte colony-stimulating factor-primed bone marrow plus peripheral blood stem cells. However, bone marrow collection is invasive and may increase donor burden. Peripheral blood stem cell collection is simpler and less invasive, but it remains unclear whether using peripheral blood stem cells alone provides similar clinical outcomes without increasing the risk of graft-versus-host disease.
This multicenter, randomized, open-label, non-inferiority trial will compare granulocyte colony-stimulating factor-primed peripheral blood stem cells alone with granulocyte colony-stimulating factor-primed bone marrow plus peripheral blood stem cells as graft sources for haploidentical transplantation in patients with severe or very severe aplastic anemia. Participants will be randomly assigned in a 1:1 ratio to either graft source group. The primary outcome is the cumulative incidence of grade II-IV acute graft-versus-host disease within 100 days after transplantation. Secondary outcomes include engraftment, chronic graft-versus-host disease, infections, immune reconstitution, survival, and donor safety.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| G-CSF-Primed Peripheral Blood Stem Cell Graft | Experimental | Procedure: G-CSF-Primed Peripheral Blood Stem Cell GraftParticipants assigned to this arm will undergo haploidentical hematopoietic stem cell transplantation using granulocyte colony-stimulating factor-primed peripheral blood stem cells alone as the graft source. Donors will receive granulocyte colony-stimulating factor mobilization, and peripheral blood stem cells will be collected by apheresis. Participants will receive the protocol-specified conditioning regimen and graft-versus-host disease prophylaxis. |
| G-CSF-Primed Bone Marrow Plus Peripheral Blood Stem Cell Graft | Experimental | Procedure: G-CSF-Primed Bone Marrow Plus Peripheral Blood Stem Cell GraftParticipants assigned to this arm will undergo haploidentical hematopoietic stem cell transplantation using granulocyte colony-stimulating factor-primed bone marrow plus peripheral blood stem cells as the graft source. Donors will receive granulocyte colony-stimulating factor mobilization, followed by bone marrow harvest and peripheral blood stem cell collection. Participants will receive the protocol-specified conditioning regimen and graft-versus-host disease prophylaxis. |
Who may qualify
- Ages
- 18 to 40 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult
Key inclusion criteria (14 listed)
- Patients with newly diagnosed or relapsed severe aplastic anemia (SAA) or very severe aplastic anemia (vSAA) according to the Camitta criteria.
- SAA is defined as bone marrow cellularity <25% and at least two of the following peripheral blood criteria: absolute neutrophil count <0.5 × 10^9/L, platelet count <20 × 10^9/L, or reticulocyte count <20 × 10^9/L.
- vSAA is defined as SAA with an absolute neutrophil count <0.2 × 10^9/L.
- Age 18 to 40 years.
- Availability of a haploidentical related donor.
Key exclusion criteria (7 listed)
- Presence of hematologic malignancy or myelodysplastic syndrome-related chromosomal abnormalities, such as +8 or del(7q).
- Prior allogeneic hematopoietic stem cell transplantation or prior organ transplantation.
- Availability of an HLA-matched sibling donor.
- Active uncontrolled infection, including unresolved invasive fungal disease, active tuberculosis, sepsis, or other uncontrolled infections.
- Severe comorbidities, defined as a Hematopoietic Cell Transplantation-Comorbidity Index (HCT-CI) score ≥4, or uncontrolled autoimmune disease.
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 |
|---|---|---|
| G-CSF-Primed Peripheral Blood Stem Cell GraftHaploidentical hematopoietic stem cell transplantation using granulocyte colony-stimulating factor-primed peripheral blood stem cells alone as the graft source. Donors will receive granulocyte colony-stimulating factor mobilization, followed by peripheral blood stem cell collection by apheresis. | Procedure or surgery | G-CSF-Primed Peripheral Blood Stem Cell Graft |
| G-CSF-Primed Bone Marrow Plus Peripheral Blood Stem Cell GraftHaploidentical hematopoietic stem cell transplantation using granulocyte colony-stimulating factor-primed bone marrow plus peripheral blood stem cells as the graft source. Donors will receive granulocyte colony-stimulating factor mobilization, followed by bone marrow harvest and peripheral blood stem cell collection. | Procedure or surgery | G-CSF-Primed Bone Marrow Plus Peripheral Blood Stem Cell Graft |
Primary outcome
Cumulative Incidence of Grade II-IV Acute Graft-Versus-Host Disease Within 100 Days After Transplantation
Time frame: Up to 100 Days After Transplantation
The cumulative incidence of newly diagnosed or persistent grade II-IV acute graft-versus-host disease within 100 days after haploidentical hematopoietic stem cell transplantation. Acute graft-versus-host disease will be graded according to the MAGIC criteria and assessed based on skin, gastrointestinal, and liver manifestations. Endpoint adjudication will be performed by an independent blinded endpoint review committee using medical records, photographs, endoscopy reports, and/or biopsy reports, as applicable.
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 16 Sep 2026.
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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 study testing G-CSF-Primed Peripheral Blood Stem Cell Graft and G-CSF-Primed Bone Marrow Plus Peripheral Blood Stem Cell Graft in people with Severe Aplastic Anemia (SAA), run by Peking University People's 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 14 inclusion and 7 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 16 Sep 2026. 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?
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?
Each site lists its own contact: choose the nearest one under Trial locations. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Cumulative Incidence of Grade II-IV Acute Graft-Versus-Host Disease Within 100 Days After Transplantation (time frame: Up to 100 Days After Transplantation). 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 16 Sep 2026.