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NCT00434161 · REGISTRY CONDITION SEARCH

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

Found through the registry condition index, not selected as a treatment recommendation. This discovery collection was restricted to studies with posted results.

Phase
PHASE3
Status at capture
COMPLETED
Registry last update
2015-03-30

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.

Open the original ClinicalTrials.gov record → · Download preserved record

What did the study report?

Outcomes, safety and baseline populations are separate source sections. Quality-of-life measures appear under their original outcome titles.

flow

Participant flow

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baseline

Baseline characteristics

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outcome · POSTED

Maximum Severity of Oral Mucositis (World Health Organization (WHO) Grades 0/1, 2, 3, or 4)

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outcome · POSTED

Incidence Ulcerative Mucositis (WHO Grades 2, 3, and 4)

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outcome · POSTED

Duration of Ulcerative Mucositis (WHO Grades 2, 3, and 4)

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outcome · POSTED

The Area Under the Curve (AUC) Was Calculated From the Patient-reported Outcome Mouth and Throat Soreness (MTS) Score.

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outcome · POSTED

Incidence of Cataract Development or Progression (Change of ≥0.3 in Lens Opacities Classification System III (LOCS III Score)) at Month 6.

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outcome · POSTED

Incidence of an Increase Posterior Subcapsular Cataract (P), Cortical Cataract (C) and Nuclear Opalescence (NO) at Month 6 and 12

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outcome · POSTED

Change From Baseline in Posterior Subcapsular (P), Cortical (C) Cataract and Nuclear Opalescence (NO) on the Lens Opacities Classification System III (LOCS III) Scale at Months 6.

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outcome · POSTED

Change From Baseline in Posterior Subcapsular (P), Cortical (C) Cataract and Nuclear Opalescence (NO) on the Lens Opacities Classification System III (LOCS III) Scale at Months 12.

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outcome · POSTED

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 at Months 6

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outcome · POSTED

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 at Months 12.

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outcome · POSTED

Incidence of Adverse Events and Laboratory Abnormalities

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outcome · POSTED

Overall Survival

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outcome · POSTED

Incidence of Cataract Development or Progression at Month 12.

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outcome · POSTED

Progression Free Survival

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outcome · POSTED

Time Death or Disease Progression

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outcome · POSTED

Incidence of Second Primary Malignancies or Other Malignancies

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adverse events

Adverse events

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more info

Limitations, agreements, and source contact

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Who could take part
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
healthy Volunteers
false
maximum Age
70 Years
minimum Age
18 Years
sex
ALL
std Ages
  1. ADULT
  2. OLDER_ADULT
Treatment arms and interventions
arm Groups
  1. description
    Subjects received palifermin before-high dose chemotherapy (total 3 doses) and matched placebo after-high dose chemotherapy (total 3 doses)
    intervention Names
    1. Drug: Palifermin before only
    label
    Palifermin before only
    type
    ACTIVE_COMPARATOR
  2. description
    Subjects received matched placebo before- and after-high dose chemotherapy
    intervention Names
    1. Drug: Placebo
    label
    Placebo (suger pill)
    type
    PLACEBO_COMPARATOR
  3. description
    Subjects received palifermin before- and after-high dose chemotherapy (total of 6 doses)
    intervention Names
    1. Drug: Palifermin before and after
    label
    Palifermin before and after
    type
    ACTIVE_COMPARATOR
interventions
  1. arm Group Labels
    1. Palifermin before only
    description
    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.
    name
    Palifermin before only
    other Names
    1. Kepivance
    type
    DRUG
  2. arm Group Labels
    1. Placebo (suger pill)
    description
    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.
    name
    Placebo
    type
    DRUG
  3. arm Group Labels
    1. Palifermin before and after
    description
    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.
    name
    Palifermin before and after
    other Names
    1. Kepivance
    type
    DRUG
Study design
allocation
RANDOMIZED
intervention Model
PARALLEL
masking Info
masking
QUADRUPLE
who Masked
  1. PARTICIPANT
  2. CARE_PROVIDER
  3. INVESTIGATOR
  4. OUTCOMES_ASSESSOR
primary Purpose
SUPPORTIVE_CARE
Enrollment
count
281
type
ACTUAL
Registered outcome plans (not posted results)
primary Outcomes
  1. description
    For the primary efficacy endpoint maximum severity of Oral Mucositis (OM) was assessed, the number of participants who had the different severity. To assess severity of OM, a 5-grade WHO scale (0, 1, 2, 3, or 4) was used. 0 = no findings or erythema only, 1= soreness present with or without erythema, 2=ulcers present but able to take solid food, 3 =ulcers present and only able to take liquids, 4 =ulcers present/not able to take anything orally.
    measure
    Maximum Severity of Oral Mucositis (World Health Organization (WHO) Grades 0/1, 2, 3, or 4)
    time Frame
    at Day 32
  2. description
    Number of participants from the primary cataract subset showing an increase from baseline of \>= 0.3 in the Lens Opacities Classification System III (LOCS III score). The LOCS III is a standard system used for grading and comparison of cataract severity and type. The ophthalmologist trained in LOCS III uses a slit lamp for examining the lens of the eye. The classification evaluates four features: posterior subcapsular cataract(P),cortical cataract(C),nuclear opalescence(NO) and nuclear color(NC). NO and NC are graded on a decimal scale of 0.1 to 6.9, based on a set of 6 standardized photographs. C and P are graded on a decimal scale of 0.1 to 5.9, based on a set of 5 standardized photographs each. In the current study, cataract development or progression was defined as an increase from baseline of ≥ 0.3 on any of the three features P, C or NO (NC is of less importance and has not been analysed further in this study).
    measure
    Incidence of Cataract Development or Progression at Month 12.
    time Frame
    12 months
secondary Outcomes
  1. description
    The incidence of ulcerative mucositis WHO grades 2, 3, and 4. Measured the number of participants who had WHO grades 2, 3, and 4: 2=ulcers present but able to take solid food, 3=ulcers present and only able to take liquids, 4=ulcers present/not able to take anything orally.
    measure
    Incidence Ulcerative Mucositis (WHO Grades 2, 3, and 4)
    time Frame
    at Day 32
  2. description
    The duration of ulcerative mucositis measured the number of days the participants had different WHO grades 2, 3, and 4: 2=ulcers present but able to take solid food, 3 =ulcers present and only able to take liquids, 4 =ulcers present/not able to take anything orally. Patients that did not have any ulcerative mucositis were given a value of 0 days.
    measure
    Duration of Ulcerative Mucositis (WHO Grades 2, 3, and 4)
    time Frame
    at Day 32
  3. description
    The mean daily scores were calculated using the subject daily assessment of Patient-reported mouth and throat soreness (MTS) on the 5 point scale with higher values in MTS indicating a worse self assessed MTS. A 5-grade WHO scale (0, 1, 2, 3, or 4). 0=no findings or erythema only, 1=soreness present with or without erythema, 2=ulcers present but able to take solid food, 3=ulcers present and only able to take liquids, 4=ulcers present/not able to take anything orally. The incidence of ulcerative mucositis WHO grades 2, 3, and 4. Measured the number of participants who had WHO grades 2, 3, and 4: 2=ulcers present but able to take solid food, 3=ulcers present and only able to take liquids, 4=ulcers present/not able to take anything orally. The area under the curve were calculated at the time points; Day(D)-2, up to Day 32.
    measure
    The Area Under the Curve (AUC) Was Calculated From the Patient-reported Outcome Mouth and Throat Soreness (MTS) Score.
    time Frame
    at Day 32
  4. description
    Number of participants from the primary cataract subset showing an increase from baseline of \>= 0.3 in the Lens Opacities Classification System III (LOCS III score). The LOCS III is a standard system used for grading and comparison of cataract severity and type. The ophthalmologist trained in LOCS III uses a slit lamp for examining the lens of the eye. The classification evaluates four features: posterior subcapsular cataract(P),cortical cataract(C),nuclear opalescence(NO) and nuclear color(NC). NO and NC are graded on a decimal scale of 0.1 to 6.9, based on a set of 6 standardized photographs. C and P are graded on a decimal scale of 0.1 to 5.9, based on a set of 5 standardized photographs each. In the current study, cataract development or progression was defined as an increase from baseline of ≥ 0.3 on any of the three features P, C or NO (NC is of less importance and has not been analysed further in this study).
    measure
    Incidence of Cataract Development or Progression (Change of ≥0.3 in Lens Opacities Classification System III (LOCS III Score)) at Month 6.
    time Frame
    6 Months
  5. description
    To assess the effect of palifermin on the incidence of cataract development or progression at Month 6 and Month 12 based on an increase of ≥ 0.3 in the Lens Opacities Classification System (LOCS III) score for Posterior (P), Cortical Cataract (C) and Nuclear Opalescence (NO). For Subcapsular cataract (P), Cortical Cataract (C) and Nuclear Opalescence (NO): at month 6 and 12 adjusted difference of rate of cataract, Palifermin - Placebo were used and the confidence interval were calculated on the adjusted difference.
    measure
    Incidence of an Increase Posterior Subcapsular Cataract (P), Cortical Cataract (C) and Nuclear Opalescence (NO) at Month 6 and 12
    time Frame
    at Month 6 and Month 12
  6. description
    To study the change in cataract from baseline visit to months 6, three cataract main types: nuclear, cortical and posterior subcapsular measured on the Lens Opacities Classification System III (LOCS III) Scale. To assess the effect of palifermin on the incidence of cataract development or progression at Month 6 and Month 12 based on an increase of ≥ 0.3 in theLOCS III score for Posterior Subcapsular cataract (P), Cortical Cataract (C) and Nuclear Opalescence (NO). The LOCS III is a standard system used for grading and comparison of cataract severity and type. The ophthalmologist uses a slit lamp for examining the lens of the eye. The classification evaluates: P,C and NO. NO is graded on a decimal scale of 0.1 to 6.9, based on a set of 6 standardized photographs. C and P are graded on a decimal scale of 0.1 to 5.9, based on a set of 5 standardized photographs each.
    measure
    Change From Baseline in Posterior Subcapsular (P), Cortical (C) Cataract and Nuclear Opalescence (NO) on the Lens Opacities Classification System III (LOCS III) Scale at Months 6.
    time Frame
    Months 6
  7. description
    To study the change in cataract from baseline visit to months 12, regarding the three cataract main types: nuclear, cortical and posterior subcapsular measured on the Lens Opacities Classification System III (LOCS III) Scale. To assess the effect of palifermin on the incidence of cataract development or progression at Month 6 and Month 12 based on an increase of ≥ 0.3 in the Lens Opacities Classification System (LOCS III) score for Posterior Subcapsular cataract (P), Cortical Cataract (C) and Nuclear Opalescence (NO). The LOCS III is a standard system used for grading and comparison of cataract severity and type. The ophthalmologist uses a slit lamp for examining the lens of the eye. The classification evaluates: P,C and NO. NO is graded on a decimal scale of 0.1 to 6.9, based on a set of 6 standardized photographs. C and P are graded on a decimal scale of 0.1 to 5.9, based on a set of 5 standardized photographs each.
    measure
    Change From Baseline in Posterior Subcapsular (P), Cortical (C) Cataract and Nuclear Opalescence (NO) on the Lens Opacities Classification System III (LOCS III) Scale at Months 12.
    time Frame
    Months 12
  8. description
    To study if the treatment has effected on the visual acuity from baseline to months 6, by using Best Corrected Visual Acuity (BCVA) as measured by a Change of 10 Letters on the ETDRS (Early Termination Diabetic Retinopathy Study) at 4 Meters. To assess the effect of palifermin on the incidence of cataract development or progression at Month 6 and Month 12 based on an increase of ≥ 0.3 in the Lens Opacities Classification System (LOCS III) score for Posterior Subcapsular cataract (P), Cortical Cataract (C) and Nuclear Opalescence (NO).
    measure
    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 at Months 6
    time Frame
    Months 6
  9. description
    To study if the treatment has effected on the visual acuity from baseline to months 12, by using Best Corrected Visual Acuity (BCVA) as measured by a Change of 10 Letters on the ETDRS (Early Termination Diabetic Retinopathy Study) at 4 Meters. To assess the effect of palifermin on the incidence of cataract development or progression at Month 6 and Month 12 based on an increase of ≥ 0.3 in the Lens Opacities Classification System (LOCS III) score for Posterior Subcapsular cataract (P), Cortical Cataract (C) and Nuclear Opalescence (NO).
    measure
    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 at Months 12.
    time Frame
    Month 12
  10. description
    Incidence of Adverse Events CTCAE grade 3 or higher reported
    measure
    Incidence of Adverse Events and Laboratory Abnormalities
    time Frame
    at Day 32
  11. description
    Overall survival (OS) is based on death from any cause, not just the condition being treated, thus it picks up death from side effects of the treatment, and effects on survival after relapse.
    measure
    Overall Survival
    time Frame
    During long-term follow up phase (maximum of 10 years)
  12. description
    Progression-free survival (PFS) is the length of time during and after the treatment during which the disease being treated does not get worse. In this study the event for Progression-free survival was death from all causes or disease progression. Time to each event was defined as the time elapsed between the date of the first dose of investigational product, and the date of the given event.
    measure
    Progression Free Survival
    time Frame
    During long-term follow up phase (maximum of 10 years)
Full study description
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).
Source references
Source notices and limitations
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    Preserved source evidence · Not medical advice