Using PTCy and Ruxolitinib to prevent GVHD after stem cell transplants in children with immune disorders
Safety and Efficacy of Cyclophosphamide and Ruxolitinib for Graft-versus-host-disease Prophylaxis After Hematopoietic Stem Cell Transplantation With Thymoglobulin Serotherapy in Conditioning Regimen in Patients With Inborn Errors of Immunity
PHASE2 · Federal Research Institute of Pediatric Hematology, Oncology and Immunology · NCT06199427
This study is testing if a new combination of medications can help prevent graft-versus-host disease in children with immune disorders who are getting stem cell transplants.
Quick facts
| Phase | PHASE2 |
|---|---|
| Study type | Interventional |
| Enrollment | 100 (estimated) |
| Ages | 0 Months to 21 Years |
| Sex | All |
| Sponsor | Federal Research Institute of Pediatric Hematology, Oncology and Immunology (other) |
| Drugs / interventions | ruxolitinib, cyclophosphamide, fludarabine |
| Locations | 1 site (Moscow) |
| Trial ID | NCT06199427 on ClinicalTrials.gov |
What this trial studies
This study evaluates the effectiveness of a combined regimen for preventing graft-versus-host disease (GVHD) in patients with inborn errors of immunity undergoing hematopoietic stem cell transplantation (HSCT). It utilizes post-transplant cyclophosphamide (PTCY) and ruxolitinib alongside thymoglobulin in the conditioning regimen. The study aims to determine how well this combination works in reducing the incidence of GVHD, which is a significant risk in these patients. Participants will receive specific doses of cyclophosphamide and ruxolitinib at designated times post-transplant.
Who should consider this trial
Good fit: Ideal candidates are children and young adults aged 0 to 21 years diagnosed with inborn errors of immunity who are eligible for allogeneic HSCT.
Not a fit: Patients with severe concomitant somatic diseases that pose additional risks for complications may not benefit from this study.
Why it matters
Potential benefit: If successful, this approach could significantly reduce the risk of GVHD in pediatric patients with inborn errors of immunity undergoing HSCT.
How similar studies have performed: Previous studies have shown promising results with the use of PTCY and ruxolitinib for GVHD prophylaxis, indicating potential success for this combined approach.
Eligibility criteria
Show full inclusion / exclusion criteria
Inclusion Criteria: 1. Patients aged ≥ 0 months and \< 21 years 2. Patients diagnosed with NBS eligible for an allogeneic HSCT 3. Signed written informed consent signed by a parent or legal guardian Exclusion Criteria: Concomitant severe somatic disease associated with an additional risk of severe complications
Where this trial is running
Moscow
- HSCT department — Moscow, Russia (RECRUITING)
Study contacts
- Study coordinator: Dmitry Balashov, MD, PhD
- Email: bala8@yandex.ru
- Phone: +74956647091
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.
Conditions: Inborn Errors of Immunity