A Randomized, Double-Blind, Placebo-Controlled, Multicenter Phase III Trial of Remestemcel-L-rknd… (NCT07568535) | Clinical Trial Compass
Not Yet RecruitingPhase 3
A Randomized, Double-Blind, Placebo-Controlled, Multicenter Phase III Trial of Remestemcel-L-rknd Added to Ruxolitinib for Grade III-IV Steroid-Refractory Acute Graft-Versus-Host Disease
180 participantsStarted 2026-08-31
Plain-language summary
This study is a randomized, double-blind, placebo-controlled, multicenter Phase III trial to compare the efficacy and safety of remestemcel-L-rknd (remestemcel-L), ex-vivo cultured adult human mesenchymal stromal cells (MSC), combined with ruxolitinib vs. ruxolitinib combined with placebo as second-line therapy in adult patients with Grade III-IV steroid-refractory acute graft-versus-host disease (SR-aGVHD).
Who can participate
Age range
18 Years
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Inclusion criteria
. Age 18 years or older at the time of enrollment.
. Able to take oral medications
. Have Grade III-IV SR-aGVHD at the time of enrollment, defined as aGVHD resistant to high dose corticosteroids at a dose of ≥ 1 mg/kg/day methylprednisolone (or equivalent), given alone or in combination with GVHD prophylaxis agents such as CNI, mTOR inhibitor, or MMF.
. The minimum time between the initiation of high-dose corticosteroids and enrollment is 3 days if aGVHD has progressed in at least one organ regardless of improvement in other organs, OR
. Minimum time of 5 days for aGVHD showing no improvement in GI or liver symptoms, OR
. Recurrence of Grade III-IV aGVHD after an initial response to steroid therapy.
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
. Prior use of ruxolitinib is permissible in the following cases:
. If no more than 2 doses of ruxolitinib were taken for aGVHD treatment prior to enrollment.
Exclusion criteria
. Underwent second allogeneic hematopoietic cell transplantation within six months from the first transplant.
. Received systemic treatment for aGVHD other than corticosteroids +/- agents used for aGVHD prophylaxis (e.g., CNIs, mTOR inhibitor, and/or MMF).
. Respiratory disease requiring continuous positive pressure ventilation or intubation. Patients who need intermittent continuous positive airway pressure (e.g., during sleep) are eligible.
. Any underlying or current medical or psychiatric condition that, in the opinion of the investigator, would interfere with the evaluation of the patient.
. Post-transplant morphologic relapsed malignancy \[defined as \> 5% blasts on bone marrow examination and/or extramedullary relapse\].
. Donor leukocyte infusion (DLI) for treatment of malignant disease relapse. Patients who received DLI for indications other than relapse, including for treatment of minimal residual disease (MRD) and mixed chimerism, are eligible.
. Prior treatment with any mesenchymal lineage cells, including remestemcel-L.
. Active or inadequately treated latent infection with Mycobacterium tuberculosis (i.e., tuberculosis).