A Study of Palifermin for the Reduction of Oral Mucositis in Subjects With Multiple Myeloma

NCT ID: NCT00434161

Last Updated: 2015-03-30

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE3
• Total Enrollment: 281 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2006-12-31
• Study Completion Date: 2012-05-31

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).

Conditions

Multiple Myeloma

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: PARALLEL
• Primary Study Purpose: SUPPORTIVE_CARE
• Blinding Strategy: QUADRUPLE

Study Groups

Palifermin before only

Subjects received palifermin before-high dose chemotherapy (total 3 doses) and matched placebo after-high dose chemotherapy (total 3 doses)

Group Type: ACTIVE_COMPARATOR

Palifermin before only

Intervention Type: DRUG

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.

Placebo (suger pill)

Subjects received matched placebo before- and after-high dose chemotherapy

Group Type: PLACEBO_COMPARATOR

Placebo

Intervention Type: DRUG

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.

Palifermin before and after

Subjects received palifermin before- and after-high dose chemotherapy (total of 6 doses)

Group Type: ACTIVE_COMPARATOR

Palifermin before and after

Intervention Type: DRUG

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.

Interventions

Palifermin before only

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 Type: DRUG

Placebo

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 Type: DRUG

Palifermin before and after

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.

Intervention Type: DRUG

Other Intervention Names

Kepivance; Kepivance

Eligibility Criteria

Inclusion Criteria

• Multiple myeloma (MM) subjects scheduled to receive high-dose Melphalan in a one day schedule followed by autologous peripheral blood progenitor cell (PBSCT)
• Body Mass Index (BMI) ≤ 35
• Eastern Cooperative Oncology Group (ECOG) performance status ≤ 2, or an ECOG status of 3 if the reason for a status of 3 is exclusively due to MM (e.g. pathological fracture)
• Functional hematopoietic, hepato-renal and pulmonary systems
• Subjects at minimum with a baseline best corrected visual acuity (BCVA) of 20/40, (6/12 or 0.5 on the decimal scale) or better using the ETDRS chart in one eye
• Subject at minimum with one eye with a natural, intact lens
• Subject who has a LOCS III score at baseline of P < 1.0, C < 2.0 and NO < 2.0 in at least one eye
• Women in child bearing potential must have a negative pregnancy test

Exclusion Criteria

• Presence or history of any other malignancy (other than curatively treated basal cell or squamous cell carcinoma of the skin, in situ cervical carcinoma, or other surgically cured malignancy, without evidence of disease for > 3 years
• Prior autologous or allogeneic transplants
• Prior treatment with palifermin, or other fibroblast or keratinocyte growth factors
• Receiving dialysis
• History of cataract surgery in both eyes
• Incapable of being responsive to mydriatic agents
• History of other ocular disease (e.g., macular degeneration, glaucoma, corneal disease) that would make assessment of visual status difficult
• Subject is scheduled to undergo cataract surgery
• Subject with any disease, that in the opinion of the ophthalmologist, could adversely effect the subject's vision during the course of the study
• Currently active oral mucositis infection
• Positive for HIV, hepatitis B or C
• Subject is unable or unwilling to follow with study procedures
• Subject is pregnant or is breast feeding
• Subject has not agreed to use adequate contraceptive precautions

• Minimum Eligible Age: 18 Years
• Maximum Eligible Age: 70 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Swedish Orphan Biovitrum

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Kristina Timdahl, MD

Role: STUDY_DIRECTOR

Swedish Orphan Biovitrum AB

Dietger Niederwieser, Professor

Role: PRINCIPAL_INVESTIGATOR

Universitatsklinikum Leipzig, Leipzig, Germany

Locations

Universitatsklinikum Leipzig

Leipzig, , Germany

Countries

Germany

Other Identifiers

20050219

Identifier Type: -

Identifier Source: org_study_id

NCT00964925

Identifier Type: -

Identifier Source: nct_alias