GVHD - Graft-Versus-Host DiseasePhase 3NCT07568535
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
A Phase 3 study testing Remestemcel-L-rknd in people with GVHD - Graft-Versus-Host Disease, run by Mesoblast, Ltd..
Trial 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. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- GVHD - Graft-Versus-Host Disease
- What is being tested?
- Remestemcel-L-rknd
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 180 participants
- Where
- No sites listed yet
- Study dates
- 31 Aug 2026to Jul 2028
- Phase
- Phase 3
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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.
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- 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.
- If there was prior use of ruxolitinib for GVHD prophylaxis. If aGVHD developed while on ruxolitinib for GVHD prophylaxis or within 3 days of its discontinuation, the patient is not eligible.
- Evident myeloid and platelet engraftment. Absolute neutrophil count (ANC) > 1000/mm3 and platelets ≥ 20,000/ mm3. Use of growth factor supplementation and transfusion support is allowed.
- Minimum Karnofsky performance score of 30 at the time of study entry.
- Patient (or legal representative where appropriate) must be capable of providing written informed consent/assent.
- Female patients of childbearing potential must use a medically accepted method of contraception and must agree to continue use of this method for the entire duration of study participation. Acceptable methods of contraception include abstinence, barrier method with spermicide, intrauterine device, or steroid contraceptive (oral, transdermal, implanted, and injected) in conjunction with a barrier method.
- Male patients with partners of childbearing potential must agree to use adequate contraception (barrier method with spermicide or abstinence) for the entire duration of study participation.
- Willing and able to comply with study requirements, remain at the clinic, and return to the clinic for the follow-up evaluation, as specified in this protocol during the study period.
Who cannot join
- 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.
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- Prior treatment with any mesenchymal lineage cells, including remestemcel-L.
- Active or inadequately treated latent infection with Mycobacterium tuberculosis (i.e., tuberculosis).
- Female patients who are pregnant, lactating, or planning a pregnancy during the expected remestemcel-L treatment period.
- Concurrently receiving an investigational agent, device or procedure. An investigational agent, device or procedure is defined as having no known FDA-approved indications. Any prior and/or current participation in a clinical trial of an investigational medicinal product (IMP) that is registered and being used off label requires review by the study's Protocol Officer, Protocol Chairs, and Sponsor prior to enrollment.
- Known hypersensitivity to DMSO or to porcine or bovine proteins.
- Requiring vasopressor support.
- Uncontrolled infections. Infections are considered controlled if appropriate therapy has been instituted and, at the time of enrollment, no signs of progression are present. Persistent fever without other signs or symptoms will not be interpreted as progressing infection. Progression of infection is defined as: hemodynamic instability attributable to sepsis OR new symptoms attributable to infection OR worsening physical signs attributable to infection OR worsening radiographic findings attributable to infection. Patients with radiographic findings attributable to infection within 4 weeks prior to enrollment must have a repeat radiographic exam within one week of enrollment that documents stable or improved findings.
- Estimated creatinine clearance less than 15 mL/min or those requiring hemodialysis.
- Clinically significant liver disorders or bilirubin greater than 3 mg/dl not attributable to aGVHD or Gilbert's.
- Moderate to severe cGVHD that require systemic treatment. Chronic GVHD that is not requiring systemic therapy is allowed.
Trial locations and contacts
No sites are listed yet. Contact the study team or a site near you to ask about taking part.
About this study
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).
- Conditions
- GVHD - Graft-Versus-Host Disease
- Being tested
- Remestemcel-L-rknd, Placebo
- Medical subject headings
- Graft vs Host Disease
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Remestemcel-L-rkndDrug
Remestemcel-L is comprised of ex-vivo cultured adult human MSC
PlaceboOther
Placebo is infusion based to be given at the same points as remestemcel-L-rknd
| Study group | Role | What they receive |
|---|---|---|
| Remestemcel-L-rknd | Experimental | Remestemcel-L will be administered as intravenous infusions Receives: Drug: Remestemcel-L-rknd |
| Placebo | Placebo comparator | PTM will be administered as intravenous infusions Receives: Other: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Overall response
The primary clinical endpoint is overall response at Day 28 post-randomization. Overall response is defined as achieving a PR or CR on Day 28 post-randomization without requirement for new intervening systemic immunosuppressive therapies
Measured: 28 days post randomization
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted5 May 2026
- Study start31 Aug 2026 (estimated)
- Primary completion28 Feb 2028 (estimated)
- Study completionJul 2028 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for GVHD - Graft-Versus-Host DiseaseStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Model description
- Double Blinded Randomized
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 180 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Mesoblast, Ltd.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07568535
- Sponsor's protocol ID
- 2320
- Status last verified
- Apr 2026
- First submitted
- 28 Apr 2026
- Last updated
- 5 May 2026
- Results posted
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 5 May 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- 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
- ClinicalTrials.gov ID
- NCT07568535
- Acronym / sponsor ID
- 2320
- Conditions
- GVHD - Graft-Versus-Host Disease
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Not yet recruiting
- Enrolment
- 180 participants (estimated)
- Lead sponsor
- Mesoblast, Ltd.Industry (pharma, biotech, devices)
- Study start
- 31 Aug 2026 (estimated)
- Primary completion
- 28 Feb 2028 (estimated)
- Study completion
- Jul 2028 (estimated)
- First posted
- 5 May 2026 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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).
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
- Model description
- Double Blinded Randomized
| Arm | Type | Receives |
|---|---|---|
| Remestemcel-L-rknd | Experimental | Drug: Remestemcel-L-rkndRemestemcel-L will be administered as intravenous infusions |
| Placebo | Placebo comparator | Other: PlaceboPTM will be administered as intravenous infusions |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (15 listed)
- 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.
- Prior use of ruxolitinib is permissible in the following cases:
- Evident myeloid and platelet engraftment. Absolute neutrophil count (ANC) > 1000/mm3 and platelets ≥ 20,000/ mm3. Use of growth factor supplementation and transfusion support is allowed.
Key exclusion criteria (16 listed)
- 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].
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Remestemcel-L-rkndRemestemcel-L is comprised of ex-vivo cultured adult human MSC | Drug | Remestemcel-L-rknd |
| PlaceboPlacebo is infusion based to be given at the same points as remestemcel-L-rknd | Other | Placebo |
Primary outcome
Overall response
Time frame: 28 days post randomization
The primary clinical endpoint is overall response at Day 28 post-randomization. Overall response is defined as achieving a PR or CR on Day 28 post-randomization without requirement for new intervening systemic immunosuppressive therapies
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 5 May 2026.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Remestemcel-L-rknd in people with GVHD - Graft-Versus-Host Disease, run by Mesoblast, Ltd..
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 15 inclusion and 16 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: opening soon, as of the sponsor's last update on 5 May 2026. Status can change between updates, so confirm with the study contact before making plans.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Teresa Montagut, MD (19173294933, Teresa.Montagut@mesoblast.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Overall response (time frame: 28 days post randomization). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 5 May 2026.