Combination treatment for high-risk acute graft-versus-host disease
Ruxolitinib, Human Chorionic Gonadotropin (uhCG/EGF), and Dose De-escalated Corticosteroids for Treatment of Minnesota High-Risk Acute GVHD (aGVHD): A Phase I/II Study
PHASE1; PHASE2 · Masonic Cancer Center, University of Minnesota · NCT05123040
This study is testing the lowest safe dose of corticosteroids combined with ruxolitinib and uhCG/EGF to help patients with high-risk acute graft-versus-host disease feel better without more side effects.
Quick facts
| Phase | PHASE1; PHASE2 |
|---|---|
| Study type | Interventional |
| Enrollment | 55 (estimated) |
| Ages | 12 Years and up |
| Sex | All |
| Sponsor | Masonic Cancer Center, University of Minnesota (other) |
| Drugs / interventions | ruxolitinib |
| Locations | 1 site (Minneapolis, Minnesota) |
| Trial ID | NCT05123040 on ClinicalTrials.gov |
What this trial studies
This multi-center phase I/II study aims to determine the lowest recommended dose of corticosteroids when used alongside ruxolitinib and uhCG/EGF for patients with high-risk acute graft-versus-host disease (aGVHD). It is a single-arm study focused on finding the minimum corticosteroid dose that maintains efficacy without increasing toxicity. The study will assess the complete or partial response rates at day 28 and will carry forward the determined dose into a phase II extension trial to further evaluate safety and preliminary efficacy.
Who should consider this trial
Good fit: Ideal candidates include hematopoietic cell transplant recipients over 12 years old who are within the first week of treatment for high-risk aGVHD.
Not a fit: Patients with progressive malignancy or uncontrolled infections may not benefit from this study.
Why it matters
Potential benefit: If successful, this study could provide a safer and more effective treatment option for patients with high-risk aGVHD.
How similar studies have performed: Other studies have explored similar combinations for aGVHD, but this specific approach is novel and has not been extensively tested.
Eligibility criteria
Show full inclusion / exclusion criteria
Inclusion Criteria: HCT recipients over 12 years of age within the first 7 days of initial treatment of high-risk aGVHD, defined as: * Newly diagnosed Minnesota high-risk aGVHD -OR- * Newly diagnosed Minnesota standard risk aGVHD with plasma amphiregulin ≥ 33 pg/ml tested at the UMN Cytokine Reference Lab. For amphiregulin lab ordering information, see Fairview Lab Guide: http://labguide.fairview.org/showtest.asp?testid=6766\&format=long -OR- * Newly diagnosed Minnesota standard risk aGVHD Ann Arbor 3 biomarkers tested by Viracor. For ordering information, see: https://www.viracor-eurofins.com/test-menu/403572p-agvhd-symptomatic- onset-algorithm/ * Renal: Serum creatinine ≤2.5x upper limit of normal (ULN) * Cardiac: Left ventricular ejection fraction (LVEF) ≥ 35% * Voluntary written consent (adult or parent/guardian with minor assent for 12 through 17-year-olds). Exclusion Criteria: * Progressive malignancy * Uncontrolled bacterial, fungal, parasitic, or viral infection at initiation of protocol treatment * Unwilling or unable to stop supplemental sex hormone therapy (estrogen, progesterone, and/or testosterone preparations) * Unwilling or unable to stop GnRH antagonists, aromatase inhibitors, or anti-androgens * History of a hormone responsive malignancy * Current thromboembolic disease requiring full-dose anticoagulation - patients receiving pharmacologic prophylaxis for thromboembolic disease will be eligible * Active or recent (within prior 3 months) thrombus, irrespective of anticoagulation status * Pregnancy * Women or men of childbearing potential unwilling to take adequate precautions to avoid unintended pregnancy from the start of protocol treatment through 30 days after the last treatment
Where this trial is running
Minneapolis, Minnesota
- Masonic Cancer Center at University of Minnesota — Minneapolis, Minnesota, United States (RECRUITING)
Study contacts
- Principal investigator: Sherman Holtan, MD — Masonic Cancer Center, University of Minnesota
- Study coordinator: Cancer Center Clinical Trials Office
- Email: ccinfo@umn.edu
- Phone: 612 624 2620
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: Acute-graft-versus-host Disease, aGVHD