Phase 3
N=281
A Study of Palifermin for the Reduction of Oral Mucositis in Subjects With Multiple Myeloma
Multiple Myeloma
Bottom Line
View on ClinicalTrials.gov: NCT00434161 ↗Enrolled (actual)
281
Serious AEs
12.3%
Results posted
Mar 2015
Primary outcome: Primary: Maximum Severity of Oral Mucositis (World Health Organization (WHO) Grades 0/1, 2, 3, or 4) — 79; 36; 68; 26 Participants — p=0.188
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 3
- Interventions
- Palifermin before only (Drug); Placebo (Drug); Palifermin before and after (Drug)
- Age
- Adult, Older Adult · 18+ yrs
- Sex
- All
- Sponsor
- Swedish Orphan Biovitrum
- Primary completion
- Mar 2009
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Maximum Severity of Oral Mucositis (World Health Organization (WHO) Grades 0/1, 2, 3, or 4) |
79; 36; 68; 26; 21; 44 | 0.188 |
| PRIMARY Incidence of Cataract Development or Progression at Month 12. |
4; 25; 10; 27; 8; 27 | — |
| SECONDARY Incidence Ulcerative Mucositis (WHO Grades 2, 3, and 4) |
56; 33; 79; 49; 24; 33 | 0.245 |
| SECONDARY Duration of Ulcerative Mucositis (WHO Grades 2, 3, and 4) |
4.78; 4.98; 7.38 | 0.095 |
| SECONDARY The Area Under the Curve (AUC) Was Calculated From the Patient-reported Outcome Mouth and Throat Soreness (MTS) Score. |
29.82; 24.55; 39.97 | 0.142 |
| SECONDARY Incidence of Cataract Development or Progression (Change of ≥0.3 in Lens Opacities Classification System III (LOCS III Score)) at Month 6. |
6; 23; 11; 30; 5; 26 | — |
| SECONDARY Incidence of an Increase Posterior Subcapsular Cataract (P), Cortical Cataract (C) and Nuclear Opalescence (NO) at Month 6 and 12 |
0; 5; 17; 47; 1; 13 | — |
| SECONDARY 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. |
0.01; 0.12; 0.14; 0.15; 0.12; 0.35 | — |
| SECONDARY 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. |
0.05; 0.25; 0.14; 0.22; 0.12; 0.35 | — |
| SECONDARY 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 |
2; 4; 15; 48; 5; 27 | — |
| SECONDARY 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. |
0; 3; 14; 49; 8; 27 | — |
| SECONDARY Incidence of Adverse Events and Laboratory Abnormalities |
56; 26; 65 | — |
| SECONDARY Overall Survival |
50.6; NA | 0.192 |
| SECONDARY Progression Free Survival |
15.1; 18.3 | 0.236 |
| SECONDARY Time Death or Disease Progression |
15.1; 18.3 | 0.372 |
| SECONDARY Incidence of Second Primary Malignancies or Other Malignancies |
1; 6 | — |
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.
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 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
Data sourced from ClinicalTrials.gov (NCT00434161). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.