The Optimization of Haploidentical Hematopoietic Stem Cell Transplantation

NCT05629260 | Unknown FDA Drug

• 1 other identifier: GM-2021-13560
• Study Type: observational
• Target: 300
• Locations: 1 country
• Sites: 1

Brief 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.

Conditions

Hematological Malignancy

Keywords

Haploidentical Transplantation; G-CSF/ATG; PT-Cy

Outcome Measures

Primary Outcomes (1)

• Percent of participants with disease relapse

  The cumulative incidence of relapse of the primary disease.

  1 year

Secondary Outcomes (8)

• Incidence of CMV disease

  6 months

• Cumulative incidences of aGVHD

  100 days

• Cumulative incidences of cGVHD

  1 year

• Percent of participants with overall survival

  1 year

• Dynamic immune reconstitution

  1 year

Study Arms (2)

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.

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.

Drug: Post-transplantation cyclophosphamide

Interventions

ATG DRUG

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.

G-CSF/ATG group

Post-transplantation cyclophosphamide DRUG

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.

PT-Cy group

Eligibility Criteria

• Age: 18 Years - 55 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64)
• Sampling Method: Non-Probability Sample

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）

You may qualify if:

• 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.

You may not qualify if:

• 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;
• 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.

Sponsors & Collaborators

• Peking University People's Hospital: lead
• Sanofi: collaborator

Study Sites (1)

People's Hospital of Peking University

Beijing, Beijing Municipality, 100044, China

RECRUITING

MeSH Terms

Conditions

Hematologic Neoplasms

Condition Hierarchy (Ancestors)

Neoplasms by Site; Neoplasms; Hematologic Diseases; Hemic and Lymphatic Diseases

Central Study Contacts

Xu Zhengli, M.D

CONTACT

86-10-88326904; xuzhengli0202@163.com

Xiao-Jun Huang, M.D

CONTACT

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Prof.

Study Record Dates

• First Submitted: November 17, 2022
• First Posted: November 29, 2022
• Study Start: December 1, 2022
• Primary Completion: July 1, 2025
• Study Completion: December 1, 2025
• Last Updated: September 11, 2023

Record last verified: 2023-09

Locations

CN (1)