Steroid refractory acute GVHD MedDRA version: 20.0 Level: LLT Classification code 10068908 Term: AGVHD System Organ Class: 100000004870
Conditions
Interventions
Product Name: Decidaua Stroma Cells
Product Code: DSC
Pharmaceutical Form: Solution for injection/infusion
Trade Name: Jakavi
Product Code: L01XE18
Pharmaceutical Form:
Trade Name: Entyvio
Product
Sponsors
Uppsala University Hospital
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 1. Completed study informed consent form. 2. Male or female patients aged 18 or older 3. Have undergone HSCT from any donor source 4. Clinically diagnosed Grades II to IV acute GvHD as per standard criteria occurring after HSCT requiring systemic immune suppressive therapy. 5. Confirmed diagnosis of steroid refractory aGvHD. Are the trial subjects under 18? no Number of subjects for this age range: F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range 46 F.1.3 Elderly (>=65 years) yes F.1.3.1 Number of subjects for this age range 10
Exclusion criteria
Exclusion criteria: 1. Has received systemic treatment for aGvHD apart from steroids. 2. Clinical presentation resembling de novo chronic GvHD or GvHD overlap syndrome 3. Presence of an active infection 4. Known human immunodeficiency virus infection (HIV). 5. Patients suffering on active tuberculosis or viral hepatitis. 6. Significant respiratory disease 7. Presence of severely impaired renal function 8. Presence of severely impaired liver function 9. Malignancy that has required treatment in the previous two years 10. Any corticosteroid therapy for indications other than aGvHD at doses > 1 mg/kg/day 11. History of progressive multifocal leuko-encephalopathy (PML). 12. Previous participation in a study of any investigational treatment agent within 30 days 13. Any condition that would, in the Investigator's judgment, interfere with full participation in the study 14. Known allergies, hypersensitivity, or intolerance to systemic immunosuppressive therapy. 15. Known hypersensitivity to dimethyl sulfoxide (DMSO) 16. Known hypersensitivity to Gentamycin 17. Patients with coagulopathy or history of past thrombo-embolic event 18. Uncontrolled hypertension 19. History of severe chronic history of heart disease 20. Pregnant or nursing (lactating) women
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: •Assess the Safety of DSC. •Durable Overall Response (DOR) at Day 56. ;Secondary Objective: •To assess Overall Response Rate at day 28 (ORR) •To assess 1-year Overall Survival (OS) •To assess 1-year Non-Relapse Mortality (NRM) •To assess incidence of infections Exploratory objectives •To assess the cumulative steroid dose until Day 56 and Day 90 •To assess Event-Free Survival (EFS) •To assess incidence of Malignancy Relapse/Progression (MR) •To assess the incidence of cGvHD ;Primary end point(s): Documenting AEs and SAEs and determining causality in relation to DSC. Causality of AEs and SAEs will be assessed by the investigator. Durable Overall response rate (DOR) at Day 56 after randomization, defined as the proportion of patients in each arm demonstrating a durable complete response (CR) or partial response (PR) between day 28 and day 56, without requirement for additional systemic therapies for an earlier progression, mixed response or nonresponse.;Timepoint(s) of evaluation of this end point: day 56 | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): 1. Proportion of patients in each arm who achieve a complete response (CR) or partial response (PR) at Day 28. 2. Overall survival (OS), defined as the time from the date of randomization to the date of death due to any cause. 3. Non-relapse mortality (NRM), defined as the time from date of randomization to death not preceded by hematologic disease relapse/progression. 4. Proportion of patients in each treatment arm who develop infections or other transplant related complications. 5. Safety and tolerability including myelosuppression, infections and bleeding will be assessed by monitoring the frequency, duration and severity of Adverse Events including occurrence of any secondary malignancies, infections, by performing physical exams and evaluating changes in vital signs from baseline, routine serum chemistry, hematology results and coagulation profile. 6. Weekly cumulative steroid dose for each patient up to Day 56 and Day 90 or end of treatment will be calculated. 7. Event-free survival, defined as the time from the date of randomization to the date of hematologic disease relapse/ progression, graft failure or death due to any cause. 8. Malignancy relapse/progression (MR), defined as the time from date of randomization to hematologic malignancy relapse or progression. Calculated for patients with underlying hematologic malignant disease. 9. Chronic GvHD, defined as the diagnosis of any cGvHD including mild, moderate and severe according to NIH criteria. (see Appendix 3) 10. Changes in immune reconstitution after aGvHD per randomization arm. ;Timepoint(s) of evaluation of this end point: 1 year | — |
Countries
Canada, Denmark, Norway, Sweden
Contacts
Public ContactKFUE
Uppsala University Hospital
Outcome results
None listed