Actively Recruiting
Randomized Pilot Study of Two Doses of Cord Blood Tissue-Derived Mesenchymal Stromal Cells With Ruxolitinib Versus Ruxolitinib Alone for Steroid-Refractory Acute Graft Versus Host Disease
Led by M.D. Anderson Cancer Center · Updated on 2026-05-05
24
Participants Needed
1
Research Sites
N/A
Total Duration
On this page
Sponsors
M
M.D. Anderson Cancer Center
Lead Sponsor
N
National Cancer Institute (NCI)
Collaborating Sponsor
AI-Summary
What this Trial Is About
Researchers are evaluating whether adding cord blood tissue-derived mesenchymal stromal cells (cb-MSCs) to ruxolitinib may help treat patients with steroid-refractory acute graft versus host disease (GVHD). This early phase I trial focuses on patients whose disease does not respond to steroid therapy. Ruxolitinib is being studied for its potential to stop cancer cell growth, and cb-MSCs are special cells from donated umbilical cord blood that may help treat GVHD and other diseases. Participants are randomly assigned to one of three groups. One group receives ruxolitinib orally twice daily for at least 3 days, with possible tapering after 6 months if the response is positive. The other two groups receive the same ruxolitinib treatment plus intravenous infusions of cb-MSCs twice weekly for 4 weeks, either at a lower or higher dose, totaling 8 doses. After treatment, participants are followed up on day 28 and monitored for up to 6 months. During the study, researchers will monitor participants for death from any cause, treatment response at day 28, and adverse events within 28 days of starting treatment. Additional assessments include GVHD status, response rates over time, organ-specific responses, survival, infections, and treatment-related toxicities up to 6 months. Participants will have regular evaluations and safety monitoring throughout the study period to track the effects and durability of the treatments.
CONDITIONS
Brief Title
Addition of Cord Blood Tissue-Derived Mesenchymal Stromal Cells to Ruxolitinib for the Treatment of Steroid-Refractory Acute Graft Versus Host Disease
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Participants aged between 12 and 80 years inclusive
- Diagnosed with steroid-refractory grades II-IV acute graft versus host disease of the lower gastrointestinal tract or liver
- No improvement after at least 7 days of methylprednisolone at 2.0 mg/kg/day or equivalent, or worsening symptoms after at least 3 days
- Karnofsky/Lansky Performance score of at least 30 at study entry
- Women of childbearing potential must be non-pregnant, not breastfeeding, and use adequate contraception
- Male participants must use adequate contraception
- Able to provide written informed consent and assent if applicable
You will not qualify if you...
- Diagnosis of de novo chronic graft versus host disease
- Isolated acute graft versus host disease of the skin only
- Treatment with ruxolitinib for acute graft versus host disease for more than 96 hours before starting study therapy
- Primary treatment with agents other than alpha-1 antitrypsin, glucocorticoids, or ruxolitinib
- Uncontrolled infections or signs of infection progression at enrollment
- Cognitive impairments or serious unstable medical or psychiatric conditions that interfere with safety or consent
- Need for supplemental oxygen above 6 liters per minute by nasal cannula
- Known allergy to bovine or porcine products
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Duration - At least 3 days for ruxolitinib; up to 4 weeks for cord blood tissue-derived mesenchymal stromal cells dosing
Participants receive ruxolitinib orally twice daily. Depending on the assigned arm, participants may also receive intravenous doses of cord blood tissue-derived mesenchymal stromal cells twice weekly for 4 weeks.
Twice weekly visits for up to 4 weeks for MSC infusions; daily oral medication (ruxolitinib)
Duration - Up to 6 months
After completing study treatment, participants are followed up to monitor response, adverse events, and survival outcomes.
Visits at Day 28 post treatment and additional follow-up visits up to 6 months
Trial Site Locations
Total: 1 location
1
M D Anderson Cancer Center
Houston, Texas, United States, 77030
Actively Recruiting
Research Team
P
Partow Kebriaei, MD
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
RANDOMIZED
Model
PARALLEL
Primary Purpose
TREATMENT
Number of Arms
3
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