Preventing severe acute graft-versus-host disease in children using a specific model
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
This study is testing if a medication called ruxolitinib can help prevent severe acute graft-versus-host disease in children who are at high risk after receiving a stem cell transplant.
Quick facts
| Phase | Phase 2 |
|---|---|
| Study type | Interventional |
| Enrollment | 40 (estimated) |
| Ages | N/A to 16 Years |
| Sex | All |
| Sponsor | Institute of Hematology & Blood Diseases Hospital, China Academic / other |
| Drugs / interventions | ruxolitinib |
| Locations | 1 site (Tianjin, Tianjin) |
| Trial ID | NCT05599256 on ClinicalTrials.gov |
What this trial studies
This study evaluates the efficacy and safety of ruxolitinib as a preventive treatment for pediatric patients at high risk of developing severe acute graft-versus-host disease (aGVHD) following hematopoietic stem cell transplantation. It utilizes the dynamic aGVHD Onset Anticipation Tianjin (daGOAT) model to identify patients who would benefit from this prophylactic therapy. The research is conducted at the Institute of Hematology, Chinese Academy of Medical Sciences, focusing on real-world clinical applications of the daGOAT model.
Who should consider this trial
Good fit: Ideal candidates for this study are children aged 16 years or younger who are undergoing high-risk allogeneic hematopoietic stem cell transplantation.
Not a fit: Patients who have undergone tandem or multiple transplantations or those who are allergic to ruxolitinib may not benefit from this study.
Why it matters
Potential benefit: If successful, this approach could significantly reduce the incidence of severe aGVHD in pediatric transplant patients.
How similar studies have performed: While the use of ruxolitinib in this context is innovative, similar studies have shown promise in the prevention of aGVHD, suggesting potential for success.
Eligibility criteria
Show full inclusion / exclusion 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.
Where this trial is running
Tianjin, Tianjin
- Institute of Hematology & Blood Diseases Hospital — Tianjin, Tianjin, China (Recruiting)
Study contacts
- Study coordinator: Xueou Liu, PhD
- Email: liuxueou@ihcams.ac.cn
- Phone: 022-23909051
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.