Phase 0 Microdose Study to Evaluate the Effect of Melphalan, Bortezomib and Dexamethasone on Cellular Gene-expression in Patients With Multiple Myeloma
Trial Snapshot
- Phase
- Early Phase 1
- Sponsor
- Henrik Gregersen
- Enrollment
- 6
- Locations
- 1
- Primary Endpoint
- Change from baseline in gene expression at 15, 30, 60, 120 minutes upon microdose drug exposure.
Study Overview
Brief Summary
The purpose of the study is to identify specific genes that are up- or downregulated in multiple myeloma patients who receive a microdose of either Melphalan (Alkeran®), Bortezomib (Velcade®) or Dexamethasone (Dexaven®). The study treatment constitutes 1% of the planned standard myeloma treatment and will be given two hours prior to standard treatment. Blood samples are taken at baseline, 15, 30, 60 and 120 minutes for microarray analysis.
Study Design
- Study Type
- Interventional
- Allocation
- Non Randomized
- Intervention Model
- Single Group
- Primary Purpose
- Basic Science
- Masking
- None
Eligibility Criteria
- Ages
- 18 Years to — (Adult, Older Adult)
- Sex
- All
- Accepts Healthy Volunteers
- No
Inclusion Criteria
- •Planned treatment for Multiple Myeloma (newly diagnosed as well as relapse and refractory disease) with one of the following chemotherapy regimens: 1) Highdose melphalan, 2) Bortezomib or 3) Dexamethasone
- •18 years or older.
- •Understand and have the will to sign the informed consent.
Exclusion Criteria
- •Prior treatment with the study drug
- •Received treatment with biphosphonates in the week prior to study treatment
Arms & Interventions
Melphalan
A microdose of 2 mg/m2 iv Melphalan (1% of standard dose) is given two hours prior to planned standard dose Melphalan
Intervention: Melphalan (Drug)
Bortezomib
A microdose of 0.013 mg/m2 iv Bortezomib (1% of standard dose) is given two hours prior to planned standard dose Bortezomib
Intervention: Bortezomib (Drug)
Dexamethasone
A microdose of 0.4 mg iv Dexamethasone (1% of standard dose) is given two hours prior to planned standard dose of Dexamethasone
Intervention: Dexamethasone (Drug)
Outcomes
Primary Outcomes
Change from baseline in gene expression at 15, 30, 60, 120 minutes upon microdose drug exposure.
Time Frame: Prior to microdose and 15, 30, 60 and 120 minutes post-microdose
The primary outcome measure is determination of differential and significantly expressed genes across time successive samples from each individual patient. The analysis will be based on global gene expression profiling and differentially expressed genes will be identified using pairwise comparisons of samples means by two sample t-tests and corrections for multiple testing.
Secondary Outcomes
No secondary outcomes reported
Investigators
Henrik Gregersen
Consultant haematologist, MD, PhD
Aalborg University Hospital