Hematological MalignancyNCT05629260
The Optimization of Haploidentical Hematopoietic Stem Cell Transplantation
An observational study of Hematological Malignancy, run by Peking University People's Hospital at 1 site in China.
Trial summary
The goal of this observational study is to compare the incidence of relapse in G-CSF/ATG based and PT-Cy based haploidentical transplantation] in [patients aged 18 to 55 years with a diagnosis of hematological malignancies who unmanipulated haplo-HSCT with myeloablative conditioning]. Read more about this study
- Who is it for?
- Ages 18 to 55 years
- Conditions the study is for
- Hematological Malignancy
- What is being tested?
- ATG and Post-transplantation cyclophosphamide
- Type of treatment
- Pill or oral medicine, Injection or infusion
- How many people
- 300 participants
- Where
- 1 siteChina
- Study dates
- 1 Dec 2022to Dec 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 18 to 55 years
- All sexes
- No healthy volunteers
- Adult
Study population: Patients with hematologic malignancy who are indicated for allogeneic hematopoietic stem cell transplantation (allo-HSCT) and absence of HLA matched donors will be treated with haplo-HSCT with either G-CSF/ATG based or PT-Cy based protocols（4:1）
Who may be able to join
- Subjects diagnosed as acute leukemia with transplant indications in ≤ CR2;
- Lack of available, HLA-identical, related sibling or unrelated donor;
- Female or male, age: 18-55 years old;
- ECOG performance status 0-2;
- Adequate organ function as defined by the following criteria:
- Serum aspartate transaminase (AST) and serum alanine transaminase (ALT) ≤2.5× upper limit of normal (ULN), or AST and ALT ≤5× ULN if liver function abnormalities are due to underlying malignancy Total serum bilirubin≤1.5× ULN Serum creatinine≤2.5× ULN
- Signed and dated informed consent document indicating that the patient (or legally acceptable representative) has been informed of all the pertinent aspects of the trial prior to enrollment;
- Willingness and ability to comply with scheduled visits, treatment plans, laboratory tests, and other study procedures.
Who cannot join
- Uncontrollable active infection;
- Severe organic impairment: hepatic and renal impairment;
- Any of the following within 6 months prior to starting study treatment: myocardial infarction, severe/unstable angina, congestive heart failure, or cerebrovascular accident including transient ischemic attack;
- Pregnancy or breastfeeding;
- Psychiatric disorders;
- Don't sign the informed consent;
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- Prior/concurrent clinical study experience;
- Other conditions:
- Participant not suitable for participation, whatever the reason, as judged by the Investigator, including medical or clinical conditions, or participants potentially at risk of noncompliance to study procedures
- Participants are employees of the clinical study site or other individuals directly involved in the conduct of the study, or immediate family members of such individuals (in conjunction with section 1.61 of the ICH-GCP Ordinance E6)
- Any specific situation during study implementation/course that may rise ethics considerations
- Sensitivity to any of the study interventions, or components thereof, or drug or other allergy that, in the opinion of the Investigator, contraindicates participation in the study.
Trial locations and contacts
1 site in 1 country.
Xiao-Jun Huang, M.D contact
China1 site, 1 recruiting
- Recruiting
People's Hospital of Peking University
Beijing, Beijing Municipality, 100044
Zhengli Xu, M.D, xuzhengli0202@163.com
Xiao-Jun Huang, M.D
About this study
Official title: A Multi-center, Prospective Study of Clinical Outcome and Immune Reconstruction in G-CSF/ATG and PT-Cy Based Haploidentical Transplantation
The goal of this observational study is to compare the incidence of relapse in G-CSF/ATG based and PT-Cy based haploidentical transplantation] in [patients aged 18 to 55 years with a diagnosis of hematological malignancies who unmanipulated haplo-HSCT with myeloablative conditioning]. The main question it aims to answer are:
Primary objective: To compare the incidence of relapse in G-CSF/ATG based and PT-Cy based haploidentical transplantation and illustrate the possible immune mechanism.
Read more
Secondary objectives: To compare CMV infection, GVHD and survival outcomes, and to observe the dynamic immune reconstitution of G-CSF/ATG based or PT-Cy based model.
Exploratory objectives: To compare the long-term quality of life among recipients who receive G-CSF/ATG based or PT-Cy based protocol.
- Conditions
- Hematological Malignancy
- Being tested
- ATG, Post-transplantation cyclophosphamide
- Keywords
- Haploidentical Transplantation, G-CSF/ATG, PT-Cy
- Medical subject headings
- Hematologic Neoplasms
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
ATGDrug
Patients with intermediate-high risk hematologic malignancy who are indicated for allogeneic hematopoietic stem cell transplantation (allo-HSCT) and absence of HLA matched donors will be treated with haplo-HSCT, with G-CSF/ATG based protocol.
Post-transplantation cyclophosphamideDrug
Patients with intermediate-high risk hematologic malignancy who are indicated for allogeneic hematopoietic stem cell transplantation (allo-HSCT) and absence of HLA matched donors will be treated with haplo-HSCT with PT-Cy based protocol.
| Study group | Role | What they receive |
|---|---|---|
| G-CSF/ATG group | Patients in the G-CSF/ATG group received a modified Bu/Cy plus ATG conditioning regimen as follows: cytarabine (4/g m2 per day IV.) on days -10 to -9; Bu (3.2 mg/kg per day IV.) on days -8 to -6; Cy (1.8 g/m2 per day IV.) on days -5 to -4; methyl chloride hexamethylene urea nitrate (Me-CCNU) (250 mg/m2 per day orally) once on day -3; and ATG (2.5 mg/kg per day IV; rabbit, Sang Stat, Lyon, France) on days -5 to -2. Receives: Drug: ATG | |
| PT-Cy group | Patients in PT-Cy group received a Bu/Flu/Cy-based conditioning regimen as follows: Flu (40 mg/m2 or 1 mg/kg IV) on days -8 to -4; Bu (3.2 mg kg/ d-1 IV) on days -7 to -4; Cy (14.5-40 mg/kg IV) on days -3 to -2;±cytarabine (3 g/m2/d IV) on days -9 to -8; and ± IDA (15 mg/m2/d IV) on days -9 to -8. The addition of IDA and/or cytarabine depended on the performance status of the patients and whether the patients were in relapse status. High-dose PTCy (50 mg/kg/d IV) was administered on days +3 and +4. Receives: Drug: Post-transplantation cyclophosphamide |
What is being measured
The primary outcome is the main question the study is designed to answer.
Percent of participants with disease relapse
The cumulative incidence of relapse of the primary disease.
Measured: 1 year
Secondary outcomes (8)
Incidence of CMV disease
The cumulative incidences of CMV disease in participants after transplantation
Measured: 6 months
Cumulative incidences of aGVHD
The diagnosis and grading of aGVHD are based on the modified Glucksberg grading standard.
Measured: 100 days
Cumulative incidences of cGVHD
Chronic GVHD can be classified as "limited" or "extensive" according to the Seattle criteria, and also be classified as "mild" or "moderate" or "severe" according to the National Institutes of Health (NIH) criteria.
Measured: 1 year
Percent of participants with overall survival
Overall survival (OS) is defined as the time from randomization to death resulting from any cause.
Measured: 1 year
Dynamic immune reconstitution
The main immune cell subsets include: T cell, B cell, NK cell, and Monocytes
Measured: 1 year
Neutrophil engraftment
Neutrophil engraftment is defined as the first of 3 consecutive days with an absolute neutrophil count > 0.5 × 10^9/L.
Measured: 1 month
Platelet engraftment
Platelet engraftment is defined as the first of 7 consecutive days with an absolute platelet count > 20 × 10^9/L independent from transfusion
Measured: 1 month
Transplantation-related mortality
Death due to causes unrelated to the underlying disease
Measured: 1 year
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted29 Nov 2022
- Study start1 Dec 2022
- Primary completionJul 2025 (estimated)
- Study completionDec 2025 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Hematological MalignancyStudies of the same condition, open and under way.
- More trials run by Peking University People's HospitalThe sponsor's other registered studies.
- 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
- Cohort
- Time perspective
- Prospective
- Enrolment
- 300 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Peking University People's Hospital
- Sponsor type
- University, hospital or other
- Collaborators
- Sanofi
- Responsible party
- Principal investigator, Xiao-Jun Huang, Peking University People's Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05629260
- Sponsor's protocol ID
- GM-2021-13560
- Status last verified
- Sep 2023
- First submitted
- 17 Nov 2022
- Last updated
- 11 Sep 2023
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Undecided
Publications
Lyu ZS, Shen MZ, Zhang YY, Gao H, Liang M, Chen YH, Wang ZK, Zhang XY, Chen DD, Zhang YY, Lv M, Mo XD, Xu LP, Zhang XH, Wang Y, Wang YC, Kong Y, Huang XJ. TGF-beta1-triggered maladaptive bone marrow endothelium impedes hematopoietic recovery. Signal Transduct Target Ther. 2025 Oct 7;10(1):332. doi: 10.1038/s41392-025-02429-y.
PubMed 41053000
Study at a glance
From the registry record, last updated by the sponsor on 11 Sep 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Multi-center, Prospective Study of Clinical Outcome and Immune Reconstruction in G-CSF/ATG and PT-Cy Based Haploidentical Transplantation
- ClinicalTrials.gov ID
- NCT05629260
- Acronym / sponsor ID
- GM-2021-13560
- Conditions
- Hematological Malignancy
- Keywords
- Haploidentical Transplantation, G-CSF/ATG, PT-Cy
- Study type
- Observational
- Recruitment status
- Unknown status
- Enrolment
- 300 participants (estimated)
- Lead sponsor
- Peking University People's HospitalUniversity, hospital or other
- Collaborators
- Sanofi
- Study start
- 1 Dec 2022 (actual)
- Primary completion
- Jul 2025 (estimated)
- Study completion
- Dec 2025 (estimated)
- First posted
- 29 Nov 2022 (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 goal of this observational study is to compare the incidence of relapse in G-CSF/ATG based and PT-Cy based haploidentical transplantation] in [patients aged 18 to 55 years with a diagnosis of hematological malignancies who unmanipulated haplo-HSCT with myeloablative conditioning]. The main question it aims to answer are:
Primary objective: To compare the incidence of relapse in G-CSF/ATG based and PT-Cy based haploidentical transplantation and illustrate the possible immune mechanism.
Secondary objectives: To compare CMV infection, GVHD and survival outcomes, and to observe the dynamic immune reconstitution of G-CSF/ATG based or PT-Cy based model.
Exploratory objectives: To compare the long-term quality of life among recipients who receive G-CSF/ATG based or PT-Cy based protocol.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| G-CSF/ATG group | – | Drug: ATGPatients in the G-CSF/ATG group received a modified Bu/Cy plus ATG conditioning regimen as follows: cytarabine (4/g m2 per day IV.) on days -10 to -9; Bu (3.2 mg/kg per day IV.) on days -8 to -6; Cy (1.8 g/m2 per day IV.) on days -5 to -4; methyl chloride hexamethylene urea nitrate (Me-CCNU) (250 mg/m2 per day orally) once on day -3; and ATG (2.5 mg/kg per day IV; rabbit, Sang Stat, Lyon, France) on days -5 to -2. |
| PT-Cy group | – | Drug: Post-transplantation cyclophosphamidePatients in PT-Cy group received a Bu/Flu/Cy-based conditioning regimen as follows: Flu (40 mg/m2 or 1 mg/kg IV) on days -8 to -4; Bu (3.2 mg kg/ d-1 IV) on days -7 to -4; Cy (14.5-40 mg/kg IV) on days -3 to -2;±cytarabine (3 g/m2/d IV) on days -9 to -8; and ± IDA (15 mg/m2/d IV) on days -9 to -8. The addition of IDA and/or cytarabine depended on the performance status of the patients and whether the patients were in relapse status. High-dose PTCy (50 mg/kg/d IV) was administered on days +3 and +4. |
Who may qualify
- Ages
- 18 to 55 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult
- Study population
- Patients with hematologic malignancy who are indicated for allogeneic hematopoietic stem cell transplantation (allo-HSCT) and absence of HLA matched donors will be treated with haplo-HSCT with either G-CSF/ATG based or PT-Cy based protocols（4:1）
Key inclusion criteria (8 listed)
- Subjects diagnosed as acute leukemia with transplant indications in ≤ CR2;
- Lack of available, HLA-identical, related sibling or unrelated donor;
- Female or male, age: 18-55 years old;
- ECOG performance status 0-2;
- Adequate organ function as defined by the following criteria:
Key exclusion criteria (12 listed)
- Uncontrollable active infection;
- Severe organic impairment: hepatic and renal impairment;
- Any of the following within 6 months prior to starting study treatment: myocardial infarction, severe/unstable angina, congestive heart failure, or cerebrovascular accident including transient ischemic attack;
- Pregnancy or breastfeeding;
- Psychiatric disorders;
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 |
|---|---|---|
| ATGPatients with intermediate-high risk hematologic malignancy who are indicated for allogeneic hematopoietic stem cell transplantation (allo-HSCT) and absence of HLA matched donors will be treated with haplo-HSCT, with G-CSF/ATG based protocol. | Drug | G-CSF/ATG group |
| Post-transplantation cyclophosphamidePatients with intermediate-high risk hematologic malignancy who are indicated for allogeneic hematopoietic stem cell transplantation (allo-HSCT) and absence of HLA matched donors will be treated with haplo-HSCT with PT-Cy based protocol. | Drug | PT-Cy group |
Primary outcome
Percent of participants with disease relapse
Time frame: 1 year
The cumulative incidence of relapse of the primary disease.
8 secondary outcomes
Incidence of CMV disease
Time frame: 6 months
Cumulative incidences of aGVHD
Time frame: 100 days
Cumulative incidences of cGVHD
Time frame: 1 year
Percent of participants with overall survival
Time frame: 1 year
Dynamic immune reconstitution
Time frame: 1 year
Neutrophil engraftment
Time frame: 1 month
Platelet engraftment
Time frame: 1 month
Transplantation-related mortality
Time frame: 1 year
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 11 Sep 2023.
- Derived from this study: PubMed 41053000Lyu ZS, Shen MZ, Zhang YY, Gao H, Liang M, Chen YH, Wang ZK, Zhang XY, Chen DD, Zhang YY, Lv M, Mo XD, Xu LP, Zhang XH, Wang Y, Wang YC, Kong Y, Huang XJ. TGF-beta1-triggered maladaptive bone marrow endothelium impedes hematopoietic recovery. Signal Transduct Target Ther. 2025 Oct 7;10(1):332. doi: 10.1038/s41392-025-02429-y.
- 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.
Xiao-Jun Huang, M.D contact
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 Hematological Malignancy, 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 8 inclusion and 12 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: status not confirmed, as of the sponsor's last update on 11 Sep 2023. 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?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Xu Zhengli, M.D (86-10-88326904, xuzhengli0202@163.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Percent of participants with disease relapse (time frame: 1 year). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT05629260, which also lists 1 linked publication.
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 11 Sep 2023.