Overview

This trial is active, not recruiting.

Condition multiple myeloma
Treatments palifermin pre only, placebo, palifermin pre and post
Phase phase 3
Sponsor Swedish Orphan Biovitrum
Start date December 2006
End date February 2009
Trial size 281 participants
Trial identifier NCT00434161, 20050219, NCT00964925

Summary

The purpose of this study is 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.

United States No locations recruiting
Other Countries No locations recruiting

Study Design

Allocation randomized
Endpoint classification safety/efficacy study
Intervention model parallel assignment
Masking double blind (subject, caregiver, investigator, outcomes assessor)
Primary purpose supportive care
Arm
(Experimental)
110 subjects to receive palifermin pre-high dose chemotherapy (total 3 doses) and matched placebo post-high dose chemotherapy (total 3 doses)
palifermin pre only
one bolus IV injection at 60 μg/kg/day, on Days -6, -5 & -4 (pre-high dose chemotherapy) and one bolus IV injection at 60 μg/kg/day of matched placebo on days 0, 1 & 2 (post-high dose chemotherapy). Minimum of 4 days between pre-chemotherapy and post-transplantation dosing.
(Placebo Comparator)
55 subjects to receive matched placebo pre- and post-high dose chemotherapy
placebo
one bolus IV injection at 60 μg/kg/day of matched placebo
(Experimental)
110 subjects to receive palifermin pre- and post-high dose chemotherapy (total of 6 doses)
palifermin pre and post
one bolus IV injection at 60 μg/kg/day, on Days -6, -5 & -4 (pre-high dose chemotherapy) and on Days 0, 1 & 2 (post-high dose chemotherapy). Minimum of 4 days between pre-chemotherapy and post-transplantation dosing.

Primary Outcomes

Measure
Maximum severity of oral mucositis (WHO grades 0/1, 2, 3, or 4)
time frame: at Day 32
Incidence of cataract development or progression (change of ≥0.3 in LOCS III score)
time frame: at month 12

Secondary Outcomes

Measure
Incidence and duration of ulcerative mucositis (WHO grades 2, 3, and 4)
time frame: at Day 32
Incidence and duration of severe oral mucositis (WHO grades 3, and 4)
time frame: at Day 32
Patient-reported mouth and throat soreness score (area under the curve)
time frame: at Day 32
Incidence of cataract development or progression (change of ≥0.3 in LOCS III score)
time frame: at Month 6
Incidence of an increase from baseline in cataract progression (increase of ≥0.3 in LOCS III score)
time frame: at months 6 and 12
Change from baseline in cataract progression (based on LOCS III score)
time frame: at months 6 and 12
Incidence of a decreased from baseline in best corrected visual acuity (BCVA) as measured by a change of 10 letters on the ETDRS (Early Termination Diabetic Retinopathy Study) at 4 meters
time frame: at months 6 and 12
Incidence of all adverse events and laboratory abnormalities
time frame: at Day 32
Overall Survival
time frame: during long-term follow up phase (maximum of 10 years)
Progression Free Survival
time frame: during long-term follow up phase (maximum of 10 years)
Time to disease progression
time frame: during long-term follow up phase (maximum of 10 years)
Incidence of second primary malignancies or other malignancies
time frame: during long-term follow up phase (maximum of 10 years)

Eligibility Criteria

Male or female participants from 18 years up to 70 years old.

Inclusion Criteria: - Multiple myeloma (MM) subjects scheduled to receive high-dose Melphalan in a one day schedule followed by autologous PBSCT - BMI ≤ 25 - 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 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

Additional Information

Official title A Double-Blind, Randomized, Placebo-Controlled Study of Two Different Schedules of Palifermin for Reduction in Severity of Oral Mucositis in Subjects With Multiple Myeloma (MM) Receiving High Dose Melphalan Followed by Autologous Peripheral Blood Stem Cell Transplantation (PBSCT)
Trial information was received from ClinicalTrials.gov and was last updated in February 2009.
Information provided to ClinicalTrials.gov by Swedish Orphan Biovitrum.