Clinical trial NCT00434161
Conditions: Multiple Myeloma
Keywords: Palifermin KGF Clinical Trial Oncology Oral Mucositis Multiple Myeloma Cataract
Sponsor: Swedish Orphan Biovitrum
Start date: 2006-12-01; Completion date: 2012-05-01
Enrollment: 281 participants; Allocation: RANDOMIZED; Intervention model: PARALLEL

Brief summary:
The purpose of this study was to evaluate the efficacy and effect of palifermin on the incidence of oral mucositis in subjects with multiple myeloma receiving Melphalan followed by autologous peripheral blood stem cell transplantation. Amendment 01 (April 07) introduced three cataract assessments to be carried out at Screening, Month 6 and Month 12 in response to FDA and EMEA follow up measures.

Detailed description:
This was a double-blind, placebo-controlled, randomized, multicenter Phase IIIb study of palifermin given before and after dose chemotherapy (total 6 doses) or before dose chemotherapy only (total 3 doses), in subjects with Multiple Myeloma (MM)receiving high dose melphalan (chemotherapy), in a 1-day schedule, followed by autologous Peripheral Blood Stem Cell Transplantation (PBSCT).

All subjects were to be followed for disease progression, second primary tumors, additional malignancies and survival for up to 10 years.

Planned: 275 subjects, in fact, 281 subjects were randomized. Randomized: 115 subjects to palifermin pre/post-CT, 109 subjects to palifermin pre-CT and 57 subjects to placebo Analyzed: 281 subjects in the full analysis set, 277 subjects in the safety subset.

Efficacy Oral cavity assessment, patient reported outcome (PRO) questionnaires (Oral Mucositis Daily Questionnaire \[OMDQ\], Functional Assessment of Cancer Therapy Esophageal \[FACT-E\], European Quality of Life Utility Scale \[EQ 5D\], Mucositis Chronic Symptoms Questionnaire \[MCSQ\]).

Safety Physical examination (including body temperature), concomitant medications, transfusions, vital signs, laboratory assessments (hematology, chemistry), cataract assessments, adverse events (AEs).

Arms: arm 1: Subjects received palifermin before-high dose chemotherapy (total 3 doses) and matched placebo after-high dose chemotherapy (total 3 doses) arm 2: Subjects received matched placebo before- and after-high dose chemotherapy arm 3: Subjects received palifermin before- and after-high dose chemotherapy (total of 6 doses)
Interventions: intervention 1: Palifermin before only intervention 2: Placebo intervention 3: Palifermin before and after
Intervention descriptions: intervention 1: One bolus IV injection at 60 μg/kg/day, on Days 6, 5 \& 4 days before-high dose chemotherapy and one bolus IV injection at 60 μg/kg/day of matched placebo on days 0, 1 \& 2 days after-high dose chemotherapy. Minimum of 4 days between before-chemotherapy and after-transplantation dosing. intervention 2: One bolus IV injection at 60 μg/kg/day of matched placebo on Days 6, 5 \& 4 (before-high dose chemotherapy) and on Days 0, 1 \& 2 (after-high dose chemotherapy).

Minimum of 4 days between pre-chemotherapy and post-transplantation dosing. intervention 3: One bolus IV injection at 60 μg/kg/day, on Days 6, 5 \& 4 (before-high dose chemotherapy) and on Days 0, 1 \& 2 (after-high dose chemotherapy).

Minimum of 4 days between before-chemotherapy and after-transplantation dosing.

Protocol documents: