A Study of Palifermin for the Reduction of Oral Mucositis in Subjects With Multiple Myeloma
Condition: Multiple Myeloma
MeSH terms: Multiple Myeloma; Neoplasms, Plasma Cell; Mucositis; Stomatitis
Study type: Interventional
Phase: Phase 3

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).
    

Intervention name(s): Palifermin before only; Placebo; Palifermin before and after
Intervention type(s): Drug; Drug; Drug
Intervention description(s): 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.; 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.; 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.
Other intervention name(s): Kepivance; Kepivance
Allocation: Randomized
Intervention model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary purpose: Supportive Care
Keywords: Palifermin; KGF; Clinical Trial; Oncology; Oral Mucositis; Multiple Myeloma; Cataract