The Optimization of Haploidentical Hematopoietic Stem Cell Transplantation

Hematological Malignancy Clinical Trial

Official title:

A Multi-center, Prospective Study of Clinical Outcome and Immune Reconstruction in G-CSF/ATG and PT-Cy Based Haploidentical Transplantation

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT05629260
• Other study ID #: GM-2021-13560
• Status: Recruiting
• Start date: December 1, 2022
• Est. completion date: December 2025

Study information

• Verified date: September 2023
• Source: Peking University People's Hospital
• Contact: Xu Zhengli, M.D
• Phone: 86-10-88326904
• Email: xuzhengli0202[@]163.com
• Is FDA regulated: No
• Study type: Observational

Clinical 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]. 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.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 300
• Est. completion date: December 2025
• Est. primary completion date: July 2025
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years to 55 Years

Eligibility

Inclusion Criteria:

1. Subjects diagnosed as acute leukemia with transplant indications in = CR2;

2. Lack of available, HLA-identical, related sibling or unrelated donor;

3. Female or male, age: 18-55 years old;

4. ECOG performance status 0-2;

5. 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

6. 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;

7. Willingness and ability to comply with scheduled visits, treatment plans, laboratory tests, and other study procedures.

Exclusion Criteria:

1. Uncontrollable active infection;

2. Severe organic impairment: hepatic and renal impairment;

3. 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;

4. Pregnancy or breastfeeding;

5. Psychiatric disorders;

6. Don't sign the informed consent;

7. Prior/concurrent clinical study experience;

8. 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.

Related Conditions & MeSH terms

• Hematologic Neoplasms
• Hematological Malignancy

Intervention

Drug:
• ATG
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 cyclophosphamide
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.

Locations

Country	Name	City	State
China	People's Hospital of Peking University	Beijing	Beijing

Sponsors (2)

Lead Sponsor	Collaborator
Peking University People's Hospital	Sanofi

Country where clinical trial is conducted

China, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Percent of participants with disease relapse	The cumulative incidence of relapse of the primary disease.	1 year
Secondary	Incidence of CMV disease	The cumulative incidences of CMV disease in participants after transplantation	6 months
Secondary	Cumulative incidences of aGVHD	The diagnosis and grading of aGVHD are based on the modified Glucksberg grading standard.	100 days
Secondary	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.	1 year
Secondary	Percent of participants with overall survival	Overall survival (OS) is defined as the time from randomization to death resulting from any cause.	1 year
Secondary	Dynamic immune reconstitution	The main immune cell subsets include: T cell, B cell, NK cell, and Monocytes	1 year
Secondary	Neutrophil engraftment	Neutrophil engraftment is defined as the first of 3 consecutive days with an absolute neutrophil count > 0.5 × 10^9/L.	1 month
Secondary	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	1 month
Secondary	Transplantation-related mortality	Death due to causes unrelated to the underlying disease	1 year