Clinical trial NCT05599256: Prevention of Severe Acute Graft-versus-host Disease in Pediatric Patients Using a daGOAT Model
Official title: A Prospective, Single-arm Clinical Trial of Prevention of Severe Acute Graft-versus-host Disease After Pediatric Patients Receiving Allogeneic Hematopoietic Stem Cell Transplantation Using a daGOAT Model
Study type: INTERVENTIONAL
Overall status: RECRUITING
Phase: PHASE2
Conditions: Transplant-Related Disorder
Lead sponsor: Institute of Hematology & Blood Diseases Hospital, China
Sponsor class: OTHER
First posted: 2022-10-31
Enrollment: 40
Sex: ALL
Maximum age (years): 16
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: PREVENTION

Brief summary: To evaluate the efficacy and safety of ruxolitinib for prophylactic therapy of child patients who are predicted to have a high risk for developing severe acute graftversus-host disease (aGVHD) by the dynamic aGVHD Onset Anticipation Tianjin (daGOAT) model.

Detailed description: This study aims to prospectively evaluate the use of the daGOAT model in real-world clinical settings at the Institute of Hematology, Chinese Academy of Medical Sciences (IHCAMS).

Eligibility criteria: Inclusion Criteria:

1. Patients must be ≤ 16 years of age;
2. Patients receiving human leukocyte antigen mismatched and non-cord blood allogeneic hematopoietic stem cell transplantation;
3. Patients who can take oral medication;
4. Patients or their guardians have to sign an informed consent form before the start of the research procedure.

Exclusion Criteria:

1. Tandem transplantation or multiple transplantations;
2. Patients who are allergic to or cannot tolerate ruxolitinib;
3. Mental or other medical conditions that make the patients unable to comply with the research treatment and monitoring requirements;
4. Patients who are pregnant or cannot take appropriate contraceptive measures during treatment;
5. Patients who are ineligible for the study due to other factors, or will bear great risk if participating in the study.

Primary outcomes:
measure: Severe aGVHD during 100 days after transplantation according to the MAGIC criteria
timeFrame: 100 days after transplantation
Secondary outcomes:
measure: aGVHD in various target organs during 100 days after transplantation according to the MAGIC criteria
timeFrame: 100 days after transplantation
measure: Overall survival during 1.5 year after transplantation
timeFrame: Days 14, 28, 42, 60, 90, 180, 270, 360 and 540 after transplantation
measure: Relapse-free survival rate and relapse rate during 1.5 year after transplantation
timeFrame: Days 14, 28, 42, 60, 90, 180, 270, 360 and 540 after transplantation
measure: Incidence of infections during 1.5 year after transplantation
timeFrame: 1.5 year after transplantation
measure: Safety of treatment during 100 days after transplantation according to the Common Terminology Criteria for Adverse Events version 5.0
timeFrame: 100 days after transplantation
measure: Total cost of treatment during 1.5 year after transplantation
timeFrame: 1.5 year after transplantation
Other outcomes:
Locations:
city: Tianjin
country: China
facility: Institute of Hematology & Blood Diseases Hospital
state: Tianjin
Study officials:
References: