A Two-part Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Efficacy of KRP203 in Patients Undergoing Stem Cell Transplant for Hematological Malignancies

Novartis

Status and phase: Completed; Phase 1

Conditions: Hematological Malignancies

Treatments: Drug: Study Part 2: KRP203 higher dose; Drug: Study Part 2: KRP203 lower dose; Drug: Study Part 1: KRP203

Study type: Interventional

Funder types: Industry

Identifiers

NCT01830010

CKRP203A2105

Details and patient eligibility

About

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

Enrollment: 23 patients

Sex: All

Ages: 18 to 65 years old

Volunteers: No Healthy Volunteers

Inclusion and exclusion 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)

Trial design

Primary purpose: Prevention

Allocation: Non-Randomized

Interventional model: Parallel Assignment

Masking: None (Open label)

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23 participants in 3 patient groups

Study Part 1: KRP203

Experimental group

Description:

All patients to receive KRP203 for 111days

Treatment:

Drug: Study Part 1: KRP203

Study Part 2: lower KRP203 dose

Experimental group

Description:

in this treatment arm patients will receive the lower KRP203 dose for 111 days on top of the standard treatment with cyclosporine A and methotrexte for GVHD prophylaxis

Treatment:

Drug: Study Part 2: KRP203 lower dose

Study Part 2: higher KRP203 dose

Experimental group

Description:

in this treatment arm patients will recieve the higher KRP203 dose for 111 days on top of standard treatment with tacrolimus and methotrexate for GVHD prophylaxis

Treatment:

Drug: Study Part 2: KRP203 higher dose