Clinical trial NCT01830010: A Two-part Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Efficacy of KRP203 in Patients Undergoing Stem Cell Transplant for Hematological Malignancies
Official title: A Two-part, Single- and Two Arm Randomized, Open-label Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Efficacy (in Part 2 Only) of KRP203 in Patients Undergoing Stem Cell Transplant for Hematological Malignancies
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE1
Conditions: Hematological Malignancies
Lead sponsor: Novartis
Sponsor class: INDUSTRY
First posted: 2013-04-11
Enrollment: 23
Sex: ALL
Minimum age (years): 18
Maximum age (years): 65
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NON_RANDOMIZED
Intervention model: PARALLEL
Primary purpose: PREVENTION

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


Eligibility criteria: 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)

Primary outcomes:
measure: Number of participants with Adverse Events as a Measure of safety
timeFrame: 111 days
Secondary outcomes:
measure: Plasma Pharmacokinetics of KRP203: Area under the Plasma Concentration-time Curve (AUC)
timeFrame: 111 days
measure: Plasma Pharmacokinetics (PK) of KRP203: Observed Maximum Plasma Concentration Following Drug Administration (Cmax)
timeFrame: 111 days
measure: Plasma Pharmacokinetics (PK) of KRP203: Time to reach the maximum concentration after drug administration
timeFrame: 111 days
measure: GVHD-free, relapse free survival
timeFrame: 1 years post-transplant
measure: GVHD-free, relapse free survival
timeFrame: 2 years post transplant
Other outcomes:
Locations:
city: Paris
country: France
facility: Novartis Investigative Site
city: Regensburg
country: Germany
facility: Novartis Investigative Site
state: Bavaria
city: Freiburg
country: Germany
facility: Novartis Investigative Site
city: Hamburg
country: Germany
facility: Novartis Investigative Site
city: Jena
country: Germany
facility: Novartis Investigative Site
city: Koeln
country: Germany
facility: Novartis Investigative Site
city: Basel
country: Switzerland
facility: Novartis Investigative Site
city: Zürich
country: Switzerland
facility: Novartis Investigative Site
Study officials:
affiliation: Novartis Pharmaceuticals
name: Novartis Pharmaceuticals
role: STUDY_DIRECTOR
References:
citation: Dertschnig S, Gergely P, Finke J, Schanz U, Holler E, Holtick U, Socie G, Medinger M, Passweg J, Teshima T, Stylianou C, Oehen S, Heim D, Bucher C. Mocravimod, a Selective Sphingosine-1-Phosphate Receptor Modulator, in Allogeneic Hematopoietic Stem Cell Transplantation for Malignancy. Transplant Cell Ther. 2023 Jan;29(1):41.e1-41.e9. doi: 10.1016/j.jtct.2022.10.029. Epub 2022 Nov 4.