A Prospective Study of Remestemcel-L, Ex-vivo Cultured Adult Human Mesenchymal Stromal Cells, for the Treatment of Pediatric Participants Who Have Failed to Respond to Steroid Treatment for Acute Graft-Versus-Host Disease (aGVHD)
CompletedPhase 3Results postedLast updated 17 March 2022
What this trial tests
Phase 3 trial testing remestemcel-L in Grade B aGVHD in 55 participants. Completed in 9 April 2018.
Adults 2 Months to 17, any sex, with Grade B aGVHD or Grade C aGVHD. Patients with the condition only — healthy volunteers not accepted.
Results — posted to ClinicalTrials.gov
Per-arm endpoint measurements with 95% confidence intervals where reported. Source: trial results section.
Overall Response Rate (ORR) at Day 28 Post Initiation of TherapyPrimary· Day 28
ORR was defined as the percentage of participants who had achieved overall response. Overall response was defined as complete response (CR) plus partial response (PR) as per aGVHD response criteria. CR was defined as resolution of aGVHD in all involved organs. PR was defined as organ improvement of at least 1 stage without worsening of any other organ.
Group
Value
95% CI
Remestemcel-L 2×10^6 MSCs/kg
69.1
Overall Survival (OS) Rate at Day 100 Post Initiation of TherapySecondary· Day 100
Overall survival rate was defined as percentage of participants who survived. OS was defined as the time to death from the start of drug therapy.
Group
Value
95% CI
Remestemcel-L 2×10^6 MSCs/kg
74.5
OS Rate at Day 100 Post Initiation of Therapy, Stratified by Responder Status at Day 28Secondary· Day 100
Overall survival rate was defined as percentage of participants who survived. OS was defined as the time to death from the start of drug therapy.
Responders
Group
Value
95% CI
Remestemcel-L 2×10^6 MSCs/kg
86.8
Non- Responders
Group
Value
95% CI
Remestemcel-L 2×10^6 MSCs/kg
47.1
OS Rate at Day 100 Post Initiation of Therapy, Stratified by Baseline aGVHD GradeSecondary· Day 100
OS rate was defined as percentage of participants who survived. OS was defined as the time to death from the start of drug therapy. Maximum severity of acute GVHD was assessed by using International Bone Marrow Transplant Registry (IBMTR) index. The severity index was defined as: Grade A (skin Stage 1: extent of rash \<25%); Grade B (skin Stage 2: extent of rash 25 to 50% or liver Stage 1 to 2: total bilirubin 34 to 102 micromoles per liter \[mcmol/L\] or intestinal tract Stage 1 to 2: volume of diarrhea 550 to 1500 milliliters per day \[mL/day\]); Grade C (skin Stage 3: extent of rash \> 50%
Grade B
Group
Value
95% CI
Remestemcel-L 2×10^6 MSCs/kg
50.0
11.81 – 88.19
Grade C
Group
Value
95% CI
Remestemcel-L 2×10^6 MSCs/kg
82.6
61.22 – 95.05
Grade D
Group
Value
95% CI
Remestemcel-L 2×10^6 MSCs/kg
73.1
52.21 – 88.43
OS Rate at Day 100 Post Initiation of Therapy, Stratified by Organ InvolvementSecondary· Day 100
OS rate was defined as percentage of participants who survived. OS was defined as the time to death from the start of drug therapy. The data was summarized for organ involvement: skin only, lower GI only, and multi-organ.
Skin Only
Group
Value
95% CI
Remestemcel-L 2×10^6 MSCs/kg
78.6
Lower GI Only
Group
Value
95% CI
Remestemcel-L 2×10^6 MSCs/kg
76.2
Multi-organ (Any Combination)
Group
Value
95% CI
Remestemcel-L 2×10^6 MSCs/kg
70.0
OR Rate at Day 56 and 100 Post Initiation of TherapySecondary· Day 56 and Day 100
OR rate was defined as the percentage of participants who had achieved overall response. Overall response was defined as CR plus PR as per aGVHD response criteria. CR was defined as resolution of aGVHD in all involved organs. PR was defined as organ improvement of at least 1 stage without worsening of any other organ.
Day 56
Group
Value
95% CI
Remestemcel-L 2×10^6 MSCs/kg
58.2
Day 100
Group
Value
95% CI
Remestemcel-L 2×10^6 MSCs/kg
69.1
Adverse events — posted to ClinicalTrials.gov
Time frame: From Baseline through 100 days of follow up.
Reporting threshold: 5%.
Adverse-event reports describe events observed during the trial — not all are caused by the drug.
Remestemcel-L 2×10^6 MSCs/kg
Serious: 35/54 (65%)
Deaths: 13/55
Serious adverse events (64 terms)
Reaction
System
Remestemcel-L 2×10^6 MSCs/kg
Pyrexia
General disorders
—
Multiple organ dysfunction syndrome
General disorders
—
Respiratory failure
Respiratory, thoracic and mediastinal disorders
—
Pneumatosis intestinalis
Gastrointestinal disorders
—
Acute graft versus host disease
Immune system disorders
—
Staphylococcal infection
Infections and infestations
—
Haemolytic uraemic syndrome
Blood and lymphatic system disorders
—
Graft versus host disease in skin
Immune system disorders
—
BK virus infection
Infections and infestations
—
Escherichia urinary tract infection
Infections and infestations
—
Human herpesvirus 6 infection
Infections and infestations
—
Pneumonia
Infections and infestations
—
Sepsis
Infections and infestations
—
Staphylococcal bacteraemia
Infections and infestations
—
Dehydration
Metabolism and nutrition disorders
—
Acute myeloid leukaemia recurrent
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
The study plans to treat at least 60 pediatric participants, male and female, between the ages of 2 months and 17 years inclusive with aGVHD following allogeneic hematopoietic stem cell transplant (HSCT) that has failed to respond to treatment with systemic corticosteroid therapy. Participants may have Grades C and D aGVHD involving the skin, liver and/or gastrointestinal (GI) tract or Grade B aGVHD involving the liver and/or GI tract, with or without concomitant skin disease.
Publications & conference data
8 peer-reviewed publications reference this trial (live from Europe PMC):
NCT00759018 — Prochymal Expanded Access Treatment for Pediatric Patients Who Have Failed Steroids for Acute GVHD
· no longer available
Other Mesoblast, Inc. trials
Trials by the same sponsor.
NCT02652130 — Safety Follow-up of Treatment With Remestemcel-L in Pediatric Participants Who Have Failed to Respond to Steroid Treatme
· Phase 3
· completed
NCT02032004 — Efficacy and Safety of Allogeneic Mesenchymal Precursor Cells (Rexlemestrocel-L) for the Treatment of Heart Failure
· Phase 3
· completed
NCT01781390 — Safety Study of Allogeneic Mesenchymal Precursor Cell Infusion in Myocardial Infarction
· Phase 2
· completed
NCT01233960 — Evaluation of PROCHYMAL® for Treatment-refractory Moderate-to-severe Crohn's Disease
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· completed
Publications: Europe PMC API search by NCT ID, retrieved 10 June 2026
Drug + disease cross-links: matched in real time against Drug Landscape's normalised drug + company + condition tables
Sponsor: as reported to ClinicalTrials.gov by Mesoblast, Inc.
Last refreshed: 17 March 2022
Drug Landscape aggregates and links these public records for informational use only. Always verify against the primary source before clinical or regulatory decisions. Canonical URL: https://druglandscape.com/trial/NCT02336230.