Prevention of Acute Graft-versus-Host Disease (aGvHD) after allogeneic hematopoietic stem cell transplant MedDRA version: 20.0 Level: LLT Classification code 10068908 Term: AGVHD System Organ Class: 100000004870
Conditions
Sponsors
Jazz Pharmaceuticals Inc.
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 1. Patient must be =1 year of age at screening and undergoing allogeneic HSCT. 2. Patient must be diagnosed with acute leukemia in morphologic complete remission (CR1 or CR2) or with MDS with no circulating blasts and with less than 5% blasts in the bone marrow 3. Patient must have planned to receive either a myeloablative or reducedintensity conditioning regimen and have an unrelated donor who is HLA matched or single-allele mismatched 4. Graft must be a CD3+ T-cell replete PBSC graft or non-manipulated BM graft. 5. Adult patients must be able to understand and sign a written informed consent. For pediatric patients, the parent/legal guardian or representative must be able to understand and sign a written informed consent. Assent, when appropriate, will be obtained according to institutional guidelines. Are the trial subjects under 18? yes Number of subjects for this age range: 30 F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range 114 F.1.3 Elderly (>=65 years) yes F.1.3.1 Number of subjects for this age range 8
Exclusion criteria
Exclusion criteria: 1. Patient has had a prior autologous or allogeneic HSCT. 2. Patient is using or plans to use an investigational agent for the prevention of GvHD. 3. Patient is receiving or plans to receive other investigational therapy 4. Patient, in the opinion of the investigator, may not be able to comply with the safety monitoring requirements of the study. 5. Patient has a psychiatric illness that would prevent the patient or legal guardian or representative from giving informed consent and/or assent. 6. Patient has a serious active disease or co-morbid medical condition, as judged by the investigator, which would interfere with the conduct of this study. 7. Patient is pregnant or lactating and does not agree to stop breastfeeding. 8. Any other condition that would cause a risk to the patient if he/she participated in the trial. 9. Patient has a known history of hypersensitivity to defibrotide or any of its excipients. Patient has a known hypersensitivity to any agent(s) or excipients of agent(s) within patient’s planned immunoprophylaxis regimen.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: The primary objective of the study is to compare the efficacy of defibrotide added to standard of care immunoprophylaxis vs standard of care immunoprophylaxis alone for the prevention of acute graft-versus-host disease (aGvHD) as measured by the cumulative incidence of Grade B-D by Day +100 post-allogeneic hematopoietic stem cell transplant (HSCT) in adult and pediatric patients.;Secondary Objective: •To compare the efficacy of defibrotide added to standard of care immunoprophylaxis vs standard of care immunoprophylaxis alone on additional variables: -Grade B-D aGvHD-free survival by Days +100 and +180 post-HSCT -Cumulative incidence of Grade B-D aGvHD by Day +180 post-HSCT -Cumulative incidence of Grade C-D aGvHD by Days +100 and +180 post-HSCT -Cumulative incidence of relapse by Days +100 and +180 post-HSCT •To evaluate steroid use in the treatment of aGvHD by Day +180 post-HSCT •To compare the health-related quality of life (HRQoL) using the following questionnaires: -FACT-BMT-TOI (adults only) -EuroQoL-5D (EQ-5D; version dependent on age group) •To compare the safety of defibrotide added to standard of care immunoprophylaxis vs standard of care immunoprophylaxis alone, including AE profile, SAE profile, laboratory abnormalities, neutrophil and platelet engraftment, graft failure, and infection. ;Primary end point(s): The primary efficacy endpoint of this study is cumulative incidence of Grade B-D aGvHD by Day +100 post-HSCT.;Timepoint(s) of evaluation of this end point: Day +100 post-HSCT | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): Secondary efficacy endpoints include the following: • Grade B-D aGvHD-free survival by Days +100 and +180 post-HSCT • Cumulative incidence of Grade B-D aGvHD by Day +180 post-HSCT • Cumulative incidence of Grade C-D aGvHD by Days +100 and +180 post-HSCT • Cumulative incidence of relapse by Days +100 and +180 post-HSCT;Timepoint(s) of evaluation of this end point: Day +100 and +180 post-HSCT for endpoints except for Cumulative incidence of Grade B-D aGvHD: Day +180 post-HSCT only | — |
Countries
Austria, Belgium, Bulgaria, Canada, Croatia, France, Germany, Greece, Italy, Poland, Portugal, Spain, United Kingdom, United States
Contacts
Public ContactClinical Dev and Lifecycle Mgmt
Jazz Pharmaceuticals Inc.
Outcome results
None listed