Phase I Dose Escalation Trial of High Dose Melphalan Conditioning Regimen With Palifermin for Cytoprotection Followed by Autologous Peripheral Blood Stem Cell Transplantation for Multiple Myeloma
- Determine the maximum tolerated dose of high-dose melphalan when administered with
palifermin in patients undergoing autologous peripheral blood stem cell transplantation
for stage II or III multiple myeloma.
- Assess overall response (complete and partial response and stable disease) in these
patients at 28 and 100 days post-transplantation.
- Assess the efficacy of palifermin as a cytoprotective agent in reducing incidence and
duration of mucositis in patients treated with this regimen.
- Assess patient-reported outcomes and impact of palifermin on quality of life of these
- Assess the qualitative and quantitative toxicities of this regimen in these patients.
OUTLINE: This is a dose-escalation study of melphalan. Patients are stratified according to
creatinine clearance (normal vs < 60 mL/min).
Patients receive high-dose melphalan IV on day -2 and palifermin IV on days -5 to -3 and
1-3. Patients undergo autologous peripheral blood stem cell transplantation on day 0.
In each stratum, cohorts of 3-6 patients receive escalating doses of melphalan until the
maximum tolerated dose (MTD) is determined. The MTD is defined as the dose preceding that at
which 2 of 3 or 2 of 6 patients experience dose-limiting toxicity.
Patients complete questionnaires about overall health, mouth and throat soreness (MTS), and
activity limitations due to MTS once daily on days -5 to 28.
After completion of study treatment, patients are followed at days 28 and 100 and then
Endpoint Classification: Safety Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Supportive Care
Maximum tolerated dose of melphalan when treated with palifermin to prevent mucositis
Days -5, -4, -3, 2, +1, +2 and +3
Muneer H. Abidi, MD
Barbara Ann Karmanos Cancer Institute
United States: Federal Government
|Barbara Ann Karmanos Cancer Institute||Detroit, Michigan 48201|