Poor Graft FunctionPhase 1NCT07907172
Expanded Donor Stem Cells for Poor Graft Function After Stem Cell Transplantation in Children
A Phase 1 study testing Ex vivo HEB-001-expanded donor-derived CD34+ hematopoietic stem cells in people with Poor Graft Function and Hematopoietic Stem Cell Transplantation Complications, run by wang xiaodong.
Trial summary
Poor graft function (PGF) is a serious complication after allogeneic hematopoietic stem cell transplantation (HSCT) and may cause persistent low blood cell counts despite donor-cell engraftment. Read more about this study
- Who is it for?
- Ages 6 to 18 years
- Conditions the study is for
- Poor Graft Function, Hematopoietic Stem Cell Transplantation Complications
- What is being tested?
- Ex vivo HEB-001-expanded donor-derived CD34+ hematopoietic stem cells
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 3 participants
- Where
- No sites listed yet
- Study dates
- 15 Nov 2026to 30 Dec 2029
- Phase
- Phase 1
- 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 6 to 18 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Age 6-18 years, regardless of sex.
- Availability of a related stem cell donor who was also the donor for the participant's original HSCT.
- Poor graft function following allogeneic HSCT, defined as ANC <0.5 × 10^9/L for >28 days with reduced bone marrow cellularity, after exclusion of acute GVHD, recurrence of the underlying disease, drug toxicity, and severe infection.
- Insufficient autologous hematopoietic stem cell reserve and failure of conventional hematopoietic supportive treatment according to the protocol, including persistent neutropenia despite appropriate G-CSF; persistent severe thrombocytopenia or transfusion dependence despite thrombopoietin-receptor agonist treatment where indicated; unsuitability for or refusal of donor lymphocyte infusion, or an unfavorable benefit-risk assessment; and an investigator assessment that other supportive approaches do not provide sustained hematopoietic recovery.
- No active infection, including normal body temperature for at least 72 hours and no evidence of infection on relevant cultures or imaging assessments.
- No acute or chronic GVHD, or GVHD no greater than Grade 1, as specified in the protocol (see harmonisation note).
- Written informed consent provided by the participant and/or legal guardian in accordance with applicable requirements.
Who cannot join
- Inherited bone marrow failure syndrome (e.g., Fanconi anemia) or hematologic malignancy.
- History of non-hematologic malignancy.
- Severe cardiac, hepatic, or renal dysfunction, including LVEF <50%, creatinine clearance <30 mL/min, or ALT/AST ≥3 × the upper limit of normal.
- Active bleeding or coagulation disorder, including INR >2 or APTT >2 × the upper limit of normal.
- Central nervous system involvement, including CNS infection or CNS involvement by malignancy.
- History of hypersensitivity to culture components, including HEB-001 or the cytokines used for ex vivo expansion.
Trial locations and contacts
No sites are listed yet. Contact the study team or a site near you to ask about taking part.
BMT Center, Shanghai Children's Medical Center contact
About this study
Official title: Safety and Efficacy of Ex Vivo Expanded Peripheral Hematopoietic Stem Cells (HEB-001) for the Treatment of Poor Graft Function in Pediatric Patients After Hematopoietic Stem Cell Transplantation: A Phase I, Single Center Pilot Study
Poor graft function (PGF) is a serious complication after allogeneic hematopoietic stem cell transplantation (HSCT) and may cause persistent low blood cell counts despite donor-cell engraftment. This single-center, open-label, Phase I pilot study will enroll three children aged 6-18 years with PGF after allogeneic HSCT who have not responded adequately to conventional hematopoietic supportive treatments. CD34-positive hematopoietic stem cells will be collected from each participant's original related transplant donor and expanded outside the body for ~10 days using the small-molecule compound HEB-001 together with the cytokines stem cell factor, thrombopoietin, and FLT3 ligand. The expanded cells will then be administered as a single intravenous infusion. The main objectives are to assess acute infusion-related adverse events during the first 30 days and to examine genetic stability through 1 year. Preliminary hematopoietic recovery will be assessed using neutrophil and platelet recovery and donor chimerism. All participants will receive the investigational cell infusion; there is no comparator group.
Read the detailed description
This investigator-initiated Phase I pilot study is a prospective, single-center, single-arm, open-label investigation of ex vivo expanded donor-derived peripheral blood hematopoietic stem cells for children with poor graft function after allogeneic HSCT. The sample size is three participants. Safety and feasibility are the main aims; the study is not designed to establish comparative efficacy.
Eligible participants are 6-18 years old, have an available related donor who provided the original HSCT graft, and have persistent post-transplant PGF despite standard hematopoietic treatment. Eligible PGF is characterized by an absolute neutrophil count below 0.5 × 10^9/L for more than 28 days, reduced bone marrow cellularity, and exclusion of relevant competing causes.
Donor peripheral blood CD34+ cells will be isolated using a magnetic selection system, then cultured in a GMP facility for ~10 days with HEB-001 (1 μM), SCF (100 ng/mL), TPO (100 ng/mL), and FLT3L (100 ng/mL) in serum-free medium. Expansion will be monitored during culture. A culture with less than 15-fold total-cell expansion by Day 7 will be considered an expansion failure according to the protocol. Before administration, the final cell product must satisfy defined quality criteria, including at least 70% CD34+ purity, at least 90% viability, and acceptable microbiological and other release testing. The planned treatment is a single intravenous infusion targeting at least 2 × 10^6 CD34+ cells/kg.
Infusion-related reactions, other adverse events, laboratory abnormalities, and serious adverse events will be monitored intensively during and after infusion and graded using CTCAE version 5.0. Assessment visits include the first 24 hours and approximately Days 7, 14, 30, and 100, with longer-term assessment through 1 year. Genetic stability will be examined through whole-exome sequencing and assessment of clonal hematopoiesis, supplemented by V(D)J clonality sequencing as described in the protocol. Recovery of neutrophils and platelets, donor chimerism, bone marrow findings, and selected biological markers will be reported descriptively. The protocol also lists survival outcomes at 6, 12, and 24 months; the final follow-up duration must be harmonized before the record is released.
Because of the very small sample size and absence of a control group, analyses will focus on individual safety profiles, descriptive statistics, and the feasibility of cell manufacture, infusion, and hematopoietic recovery.
- Being tested
- Ex vivo HEB-001-expanded donor-derived CD34+ hematopoietic stem cells
- Keywords
- Poor graft function, Pediatric allogeneic hematopoietic stem cell transplantation, Hematopoietic stem cells, CD34-positive cells, Ex vivo stem cell expansion, Donor-derived stem cell infusion, HEB-001, Hematopoietic recovery, Genetic stability
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Ex vivo HEB-001-expanded donor-derived CD34+ hematopoietic stem cellsBiological
Also called HEB-001-expanded hematopoietic stem cells
CD34+ cells are collected from the original related allogeneic HSCT donor and magnetically selected using a CliniMACS system. Cells are expanded ex vivo for ~10 days in serum-free culture medium with HEB-001 (1 μM), stem cell factor (100 ng/mL), thrombopoietin (100 ng/mL), and FLT3 ligand (100 ng/mL). The expanded product undergoes release testing, including CD34+ purity ≥70%, viability ≥90%, and microbiological testing. A single intravenous infusion is administered with a target dose of ≥2 × 10^6 CD34+ cells/kg, with infusion planned within approximately 30 minutes. HEB-001 is used in ex vivo cell culture; it is not described as a separately infused drug.
| Study group | Role | What they receive |
|---|---|---|
| Ex Vivo Expanded Donor CD34+ Stem Cells | Experimental | Participants with PGF after allogeneic HSCT will receive one intravenous infusion of CD34+ cells obtained from their original related HSCT donor and expanded ex vivo using HEB-001 and cytokines. Receives: Biological: Ex vivo HEB-001-expanded donor-derived CD34+ hematopoietic stem cells |
What is being measured
The primary outcome is the main question the study is designed to answer.
Participants With Treatment-Related Acute Adverse Events
Number and percentage of participants experiencing infusion-related treatment-associated adverse events within 30 days, including fever (≥38.5°C), allergic reactions, or microbiologically confirmed infections. Adverse event types, severity (CTCAE v5.0), and the number with Grade ≥3 events will be described for each participant.
Measured: From infusion (Day 0) through Day 30
Participants With Evidence of Clonal Hematopoiesis or Driver Mutations
Whole-exome sequencing will be used to describe detected hematopoietic driver mutations (including DNMT3A, TP53, and ASXL1) and variant allele frequencies; V(D)J sequencing will assess clonality. The number of participants with evidence of clonal hematopoiesis or relevant driver mutations will be reported at follow-up relative to baseline.
Measured: Before infusion (baseline), Day 100, and 1 year after infusion
Secondary outcomes (5)
Time to Neutrophil Recovery
Elapsed days from cell infusion to the first of three consecutive days with an absolute neutrophil count (ANC) ≥0.5 × 10^9/L. Individual recovery times and the median/range will be reported.
Measured: From infusion (Day 0) through Day 100
Time to Platelet Recovery
Elapsed days from cell infusion to the first day of a sustained platelet count ≥20 × 10^9/L without platelet transfusion for at least 7 days. Individual recovery times and the median/range will be reported.
Measured: From infusion (Day 0) through Day 100
Donor Chimerism Following Expanded Stem Cell Infusion
Donor-derived hematopoietic chimerism measured by short tandem repeat polymerase chain reaction (STR-PCR), expressed as percentage donor-derived cells; achievement of ≥90% donor chimerism will also be described.
Measured: Days 30 and 100 after infusion
Overall Survival
Percentage of participants alive at the specified post-infusion follow-up times. As an uncomparative three-participant study, individual survival status and descriptive estimates will be reported.
Measured: 6, 12, and 24 months after infusion
Event-Free Survival
Time from infusion to the first protocol-defined event or death.
Measured: 6, 12, and 24 months after infusion
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted30 Oct 2026
- Study start15 Nov 2026 (estimated)
- Primary completion30 Jun 2028 (estimated)
- Study completion30 Dec 2029 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Poor Graft FunctionStudies 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 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Model description
- Three eligible children will each receive a single infusion of ex vivo HEB-001-expanded donor-derived CD34+ hematopoietic stem cells. No control group or randomized allocation is planned.
- Masking
- None (open label)
- Enrolment
- 3 participants (estimated)
Sponsor and investigators
- Lead sponsor
- wang xiaodong
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor-investigator, wang xiaodong, Shanghai Jiao Tong University School of Medicine
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07907172
- Sponsor's protocol ID
- HEB-001
- Status last verified
- Oct 2026
- First submitted
- 26 Oct 2026
- Last updated
- 30 Oct 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Undecided: A decision regarding external sharing of de-identified individual participant data has not yet been finalized. Any proposed sharing would require institutional and ethics review and compliance with applicable privacy, consent, and human genetic data requirements. Particular care is required because the study enrolls only three children and includes genomic sequencing data.
Study at a glance
From the registry record, last updated by the sponsor on 30 Oct 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Safety and Efficacy of Ex Vivo Expanded Peripheral Hematopoietic Stem Cells (HEB-001) for the Treatment of Poor Graft Function in Pediatric Patients After Hematopoietic Stem Cell Transplantation: A Phase I, Single Center Pilot Study
- ClinicalTrials.gov ID
- NCT07907172
- Acronym / sponsor ID
- HEB-001
- Keywords
- Poor graft function, Pediatric allogeneic hematopoietic stem cell transplantation, Hematopoietic stem cells, CD34-positive cells, Ex vivo stem cell expansion, Donor-derived stem cell infusion, HEB-001, Hematopoietic recovery
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Not yet recruiting
- Enrolment
- 3 participants (estimated)
- Lead sponsor
- wang xiaodongUniversity, hospital or other
- Study start
- 15 Nov 2026 (estimated)
- Primary completion
- 30 Jun 2028 (estimated)
- Study completion
- 30 Dec 2029 (estimated)
- First posted
- 30 Oct 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.
Poor graft function (PGF) is a serious complication after allogeneic hematopoietic stem cell transplantation (HSCT) and may cause persistent low blood cell counts despite donor-cell engraftment. This single-center, open-label, Phase I pilot study will enroll three children aged 6-18 years with PGF after allogeneic HSCT who have not responded adequately to conventional hematopoietic supportive treatments. CD34-positive hematopoietic stem cells will be collected from each participant's original related transplant donor and expanded outside the body for ~10 days using the small-molecule compound HEB-001 together with the cytokines stem cell factor, thrombopoietin, and FLT3 ligand. The expanded cells will then be administered as a single intravenous infusion. The main objectives are to assess acute infusion-related adverse events during the first 30 days and to examine genetic stability through 1 year. Preliminary hematopoietic recovery will be assessed using neutrophil and platelet recovery and donor chimerism. All participants will receive the investigational cell infusion; there is no comparator group.
Detailed description
This investigator-initiated Phase I pilot study is a prospective, single-center, single-arm, open-label investigation of ex vivo expanded donor-derived peripheral blood hematopoietic stem cells for children with poor graft function after allogeneic HSCT. The sample size is three participants. Safety and feasibility are the main aims; the study is not designed to establish comparative efficacy.
Eligible participants are 6-18 years old, have an available related donor who provided the original HSCT graft, and have persistent post-transplant PGF despite standard hematopoietic treatment. Eligible PGF is characterized by an absolute neutrophil count below 0.5 × 10^9/L for more than 28 days, reduced bone marrow cellularity, and exclusion of relevant competing causes.
Donor peripheral blood CD34+ cells will be isolated using a magnetic selection system, then cultured in a GMP facility for ~10 days with HEB-001 (1 μM), SCF (100 ng/mL), TPO (100 ng/mL), and FLT3L (100 ng/mL) in serum-free medium. Expansion will be monitored during culture. A culture with less than 15-fold total-cell expansion by Day 7 will be considered an expansion failure according to the protocol. Before administration, the final cell product must satisfy defined quality criteria, including at least 70% CD34+ purity, at least 90% viability, and acceptable microbiological and other release testing. The planned treatment is a single intravenous infusion targeting at least 2 × 10^6 CD34+ cells/kg.
Infusion-related reactions, other adverse events, laboratory abnormalities, and serious adverse events will be monitored intensively during and after infusion and graded using CTCAE version 5.0. Assessment visits include the first 24 hours and approximately Days 7, 14, 30, and 100, with longer-term assessment through 1 year. Genetic stability will be examined through whole-exome sequencing and assessment of clonal hematopoiesis, supplemented by V(D)J clonality sequencing as described in the protocol. Recovery of neutrophils and platelets, donor chimerism, bone marrow findings, and selected biological markers will be reported descriptively. The protocol also lists survival outcomes at 6, 12, and 24 months; the final follow-up duration must be harmonized before the record is released.
Because of the very small sample size and absence of a control group, analyses will focus on individual safety profiles, descriptive statistics, and the feasibility of cell manufacture, infusion, and hematopoietic recovery.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
- Model description
- Three eligible children will each receive a single infusion of ex vivo HEB-001-expanded donor-derived CD34+ hematopoietic stem cells. No control group or randomized allocation is planned.
| Arm | Type | Receives |
|---|---|---|
| Ex Vivo Expanded Donor CD34+ Stem Cells | Experimental | Biological: Ex vivo HEB-001-expanded donor-derived CD34+ hematopoietic stem cellsParticipants with PGF after allogeneic HSCT will receive one intravenous infusion of CD34+ cells obtained from their original related HSCT donor and expanded ex vivo using HEB-001 and cytokines. |
Who may qualify
- Ages
- 6 to 18 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (7 listed)
- Age 6-18 years, regardless of sex.
- Availability of a related stem cell donor who was also the donor for the participant's original HSCT.
- Poor graft function following allogeneic HSCT, defined as ANC <0.5 × 10^9/L for >28 days with reduced bone marrow cellularity, after exclusion of acute GVHD, recurrence of the underlying disease, drug toxicity, and severe infection.
- Insufficient autologous hematopoietic stem cell reserve and failure of conventional hematopoietic supportive treatment according to the protocol, including persistent neutropenia despite appropriate G-CSF; persistent severe thrombocytopenia or transfusion dependence despite thrombopoietin-receptor agonist treatment where indicated; unsuitability for or refusal of donor lymphocyte infusion, or an unfavorable benefit-risk assessment; and an investigator assessment that other supportive approaches do not provide sustained hematopoietic recovery.
- No active infection, including normal body temperature for at least 72 hours and no evidence of infection on relevant cultures or imaging assessments.
Key exclusion criteria (6 listed)
- Inherited bone marrow failure syndrome (e.g., Fanconi anemia) or hematologic malignancy.
- History of non-hematologic malignancy.
- Severe cardiac, hepatic, or renal dysfunction, including LVEF <50%, creatinine clearance <30 mL/min, or ALT/AST ≥3 × the upper limit of normal.
- Active bleeding or coagulation disorder, including INR >2 or APTT >2 × the upper limit of normal.
- Central nervous system involvement, including CNS infection or CNS involvement by malignancy.
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 |
|---|---|---|
| Ex vivo HEB-001-expanded donor-derived CD34+ hematopoietic stem cellsAlso: HEB-001-expanded hematopoietic stem cellsCD34+ cells are collected from the original related allogeneic HSCT donor and magnetically selected using a CliniMACS system. Cells are expanded ex vivo for ~10 days in serum-free culture medium with HEB-001 (1 μM), stem cell factor (100 ng/mL), thrombopoietin (100 ng/mL), and FLT3 ligand (100 ng/mL). The expanded product undergoes release testing, including CD34+ purity ≥70%, viability ≥90%, and microbiological testing. A single intravenous infusion is administered with a target dose of ≥2 × 10^6 CD34+ cells/kg, with infusion planned within approximately 30 minutes. HEB-001 is used in ex vivo cell culture; it is not described as a separately infused drug. | Biological | Ex Vivo Expanded Donor CD34+ Stem Cells |
Primary outcome
Participants With Treatment-Related Acute Adverse Events
Time frame: From infusion (Day 0) through Day 30
Number and percentage of participants experiencing infusion-related treatment-associated adverse events within 30 days, including fever (≥38.5°C), allergic reactions, or microbiologically confirmed infections. Adverse event types, severity (CTCAE v5.0), and the number with Grade ≥3 events will be described for each participant.
Participants With Evidence of Clonal Hematopoiesis or Driver Mutations
Time frame: Before infusion (baseline), Day 100, and 1 year after infusion
Whole-exome sequencing will be used to describe detected hematopoietic driver mutations (including DNMT3A, TP53, and ASXL1) and variant allele frequencies; V(D)J sequencing will assess clonality. The number of participants with evidence of clonal hematopoiesis or relevant driver mutations will be reported at follow-up relative to baseline.
5 secondary outcomes
Time to Neutrophil Recovery
Time frame: From infusion (Day 0) through Day 100
Time to Platelet Recovery
Time frame: From infusion (Day 0) through Day 100
Donor Chimerism Following Expanded Stem Cell Infusion
Time frame: Days 30 and 100 after infusion
Overall Survival
Time frame: 6, 12, and 24 months after infusion
Event-Free Survival
Time frame: 6, 12, and 24 months after infusion
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 30 Oct 2026.
- Individual participant dataSharing planned: Undecided. A decision regarding external sharing of de-identified individual participant data has not yet been finalized. Any proposed sharing would require institutional and ethics review and compliance with applicable privacy, consent, and human genetic data requirements. Particular care is required because the study enrolls only three children and includes genomic sequencing data.
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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.
BMT Center, Shanghai Children's Medical Center contact
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing Ex vivo HEB-001-expanded donor-derived CD34+ hematopoietic stem cells in people with Poor Graft Function and Hematopoietic Stem Cell Transplantation Complications, run by wang xiaodong.
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: opening soon, as of the sponsor's last update on 30 Oct 2026. Status can change between updates, so confirm with the study contact before making plans.
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 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
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 BMT Center, Shanghai Children's Medical Center (86-021-38626161 ext. 51224, BMT-SCMC@outlook.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Participants With Treatment-Related Acute Adverse Events (time frame: From infusion (Day 0) through Day 30). 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 30 Oct 2026.