Hematological Malignancies
Conditions
Brief summary
Two part study to evaluate the safety, tolerability, pharmacokinetics, and efficacy (in Part 2 only) of KRP203 in patients undergoing allogeneic hemopoietic stem cell transplant for hematological malignancies
Interventions
DRUGStudy Part 1: KRP203
All subjects will receive KRP203 for 111 days
DRUGStudy Part 2: KRP203 lower dose
DRUGStudy Part 2: KRP203 higher dose
Sponsors
Novartis Pharmaceuticals
Study design
Allocation
NON_RANDOMIZED
Intervention model
PARALLEL
Primary purpose
PREVENTION
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
18 Years to 65 Years
Healthy volunteers
No
Inclusion criteria
* Patients aged 18 to 65 years, inclusive * Patients must have a hematological malignancy that as per standard medical practice requires myeloablative conditioning (including short term myeloablative reduced intensity conditioning) followed by allogeneic hematopoetic stem cell transplant * Karnofsky Performance status ≥60%. * Suitable stem cell source available according to the graft selection algorithm using T-cell replete peripheral stem cells as a graft source
Exclusion criteria
* Resting heart rate below 55 * Significant cardiac disease (such as arrhytmia, heart failure) or any significant condition which in the investigators opinion would make the patient ineligible * Previous allogeneic HSCT * Any drug required that is not compatible with KRP203 (e.g. beta-blockers or anti-thymocyte globulin)
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Number of participants with Adverse Events as a Measure of safety | 111 days | Safety and tolerability of KRP203 in patients undergoing allogeneic hematopoetic stem cell transplant for hematological malignancies |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Plasma Pharmacokinetics of KRP203: Area under the Plasma Concentration-time Curve (AUC) | 111 days | The main PK parameters will be determined in whole blood using non-compartmental methods. Pk parameters being measured are: AUCtau AUC during a dosing interval (tau) of 24 hours \[h.ng/mL\] , AUCtauR Molar ratios between KRP203-P and KRP203 based on Cmax or AUCtau |
| Plasma Pharmacokinetics (PK) of KRP203: Observed Maximum Plasma Concentration Following Drug Administration (Cmax) | 111 days | Cmax Maximum (peak) blood drug concentration after drug administration \[ng/mL\] |
| Plasma Pharmacokinetics (PK) of KRP203: Time to reach the maximum concentration after drug administration | 111 days | Tmax Time to reach maximum (peak) concentration \[ng/mL\] |
| GVHD-free, relapse free survival | 1 years post-transplant | occurence of GVHD, disease relaps and death will be assessed |
Countries
France, Germany, Switzerland
Outcome results
None listed