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 range18 Years
SexALL
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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.
✓. 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).