Using Ruxolitinib to Treat Chronic Graft-versus-Host Disease
Upfront Ruxolitinib Treatment for Chronic Graft-vs-host Disease in Children and Young Adults: A Corticosteroid-sparing Pilot Study
This study is testing if a new medication called ruxolitinib can help young people aged 12 to 30 with chronic graft-versus-host disease feel better without using steroids first.
Quick facts
| Phase | Phase 1 |
|---|---|
| Study type | Interventional |
| Enrollment | 30 (estimated) |
| Ages | 12 Years to 30 Years |
| Sex | All |
| Sponsor | Children's Hospital Medical Center, Cincinnati Academic / other |
| Drugs / interventions | ruxolitinib, prednisone |
| Locations | 1 site (Cincinnati, Ohio) |
| Trial ID | NCT06695507 on ClinicalTrials.gov |
What this trial studies
This study investigates the effectiveness of ruxolitinib as a first-line treatment for chronic graft-versus-host disease (cGVHD) in patients aged 12 to 30 years. Traditionally, corticosteroids have been the primary treatment for cGVHD, but this trial aims to evaluate ruxolitinib, a newer immunosuppressive agent, administered without prior steroid treatment. The goal is to determine if upfront ruxolitinib can provide better outcomes for patients newly diagnosed with moderate to severe cGVHD. Participants will be monitored for their response to the treatment and any potential side effects.
Who should consider this trial
Good fit: Ideal candidates are patients aged 12 to 30 years with newly diagnosed moderate to severe chronic graft-versus-host disease requiring systemic treatment.
Not a fit: Patients with active uncontrolled infections or those who have previously received systemic treatment for chronic GVHD may not benefit from this study.
Why it matters
Potential benefit: If successful, this treatment could offer a more effective and less toxic option for managing chronic graft-versus-host disease.
How similar studies have performed: Other studies have shown promising results with ruxolitinib for cGVHD, indicating a potential for success with this approach.
Eligibility criteria
Show full inclusion / exclusion criteria
Inclusion Criteria: * Newly diagnosed moderate to severe chronic graft versus host disease (as defined by NIH cGVHD consensus criteria), requiring systemic treatment * Patient aged ≥12 year-old and ≤30 year-old * Patient able to take oral or enteral medication * No active, clinically significant uncontrolled infections * ALT ≤ 5x upper limit of normal (ULN) and total bilirubin ≤ 5xULN (unless presumed liver GVHD) * Platelet ≥ 20k and ANC ≥ 500. The use of transfusions or growth factors is permitted to maintain counts at these thresholds * No prior systemic treatment for chronic GVHD. Patients previously treated for acute GVHD are eligible, including those who received ruxolitinib for treatment of their aGVHD * Patients must be on ≤ physiologic dosing (i.e. hydrocortisone 8-12mg/m2/day) at enrollment * Patients with prior acute GVHD on \< 1 mg/kg steroids with new onset moderate-severe chronic GVHD may be considered for enrollment if they can taper steroids to reach physiological hydrocortisone in 1 month. If unable to do so these patients will come off study and be replaced Exclusion Criteria: * Mild cGVHD (as defined by NIH cGVHD consensus criteria11), that does not require systemic therapy * Acute or late acute GVHD without any evidence of chronic GVHD features * Patients who have received corticosteroids for ≥ 24 hours at 1 mg/kg/day of methylprednisolone or prednisone with the intent to treat cGVHD at time of enrollment * Corticosteroid dosing above physiologic dose hydrocortisone (i.e. \> 8-12mg/m2/day) at time of enrollment * Clinical evidence suggesting active malignancy (including PTLD and primary/secondary malignancy) * Clinical evidence of clinically significant active infection * Ongoing cytopenias which cannot be supported with routine supportive care (keeping hemoglobin over 7 g/dL and platelets \>20,000 and absolute neutrophil count over 500/uL) * Active lower gastrointestinal bleeding * Thrombosis within 6 months (including myocardial infarction, stroke, deep venous thrombosis, pulmonary embolism). IV infiltration will not be included as an example of thrombosis * Pregnant or lactating females; patients of childbearing age who are not able to comply with contraceptive recommendations * Other condition that PI feels would preclude the patient from complying with study activities * ESRD \[CLcr \< 15 mL/min\] not on dialysis * Overlap syndrome
Where this trial is running
Cincinnati, Ohio
- Cincinnati Children's Hospital Medical Center — Cincinnati, Ohio, United States (Recruiting)
Study contacts
- Principal investigator: Pooja Khandelwal, MD — Children's Hospital Medical Center, Cincinnati
- Study coordinator: Claire Dusa
- Email: Claire.Dusa@cchmc.org
- Phone: (513) 803-4541
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.