Patients Undergoing Photodynamic Therapy Using Porfimer Sodium
Conditions
Brief summary
The purpose of this study was to evaluate the safety and efficacy of afamelanotide (previously developed as CUV1647) as adjunctive therapy in patients undergoing photodynamic therapy using porfimer sodium.
Interventions
DRUGAfamelanotide
DRUGPlacebo
Sponsors
Clinuvel Pharmaceuticals Limited
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
QUADRUPLE (Subject, Caregiver, Investigator, Outcomes Assessor)
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
Inclusion criteria
* Male or female Caucasian subjects undergoing photodynamic therapy with porfimer sodium; * Aged greater than 18 years; * Written informed consent prior to the performance of any study-specific procedure.
Exclusion criteria
* Known allergy or hypersensitivity to afamelanotide or the polymer contained in the implant; * Non-Caucasian patients; * Personal history of melanoma or dysplastic nevus syndrome; * Current Bowen's disease, basal cell carcinoma, squamous cell carcinoma, or other malignant or premalignant skin lesions; * Female who is pregnant (confirmed by positive serum β-HCG pregnancy test prior to baseline) or lactating; * Females of child-bearing potential (pre-menopausal, not surgically sterile) not using adequate contraceptive measures (i.e. oral contraceptives, diaphragm plus spermicide, intrauterine device); * Participation in a simultaneous clinical trial for another investigational agent or within 30 days prior to the screening visit; * Patients unable to give informed consent; * Patient needs for concomitant medication with potential photosensitizing effects e.g. antibiotics like tetracyclines.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| The First Day on Which Patients Are Free From Symptoms of Phototoxicity Following Phototesting | From Day 04 to Day 90 |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Change in Quality of Life | From Day 0 to Day 20, Day 60 and Day 90 | Quality of life, as measured by the Short Form-36 Health survey (SF-36) (quality of life questionnaire). The SF-36 consists of eight scaled scores, which are the weighted sums of the questions in their section. Each scale is directly transformed into a 0-100 scale on the assumption that each question carries equal weight. The lower the score the more disability. |
Participant flow
Participants by arm
| Arm | Count |
|---|---|
| Afamelanotide Subjects visited the clinic on Day 0 (administration of afamelanotide implant and porfimer sodium), Day 2 (photodynamic therapy), and Days 20 and 90 for assessments of adverse events, concomitant medication, and the results of evaluation of phototoxicity. | 9 |
| Placebo Subjects visited the clinic on Day 0 (administration of placebo implant and porfimer sodium), Day 2 (photodynamic therapy), and Days 20 and 90 for assessments of adverse events, concomitant medication and the results of evaluation of phototoxicity. | 7 |
| Total | 16 |
Baseline characteristics
| Characteristic | Afamelanotide | Placebo | Total |
|---|---|---|---|
| Age, Continuous | 63.0 Years STANDARD_DEVIATION 9.3 | 72.6 Years STANDARD_DEVIATION 8.8 | 67.2 Years STANDARD_DEVIATION 10 |
| Sex: Female, Male Female | 0 Participants | 1 Participants | 1 Participants |
| Sex: Female, Male Male | 9 Participants | 6 Participants | 15 Participants |
Adverse events
| Event type | EG000 affected / at risk | EG001 affected / at risk |
|---|---|---|
| deaths Total, all-cause mortality | — / — | — / — |
| other Total, other adverse events | 6 / 9 | 1 / 7 |
| serious Total, serious adverse events | 2 / 9 | 3 / 7 |
Outcome results
Primary
The First Day on Which Patients Are Free From Symptoms of Phototoxicity Following Phototesting
Time frame: From Day 04 to Day 90
Population: The planned primary efficacy endpoint was the number of days for patients to be free of phototoxic reactions following phototesting. This was recorded by patients through diaries, however these diaries were not completed appropriately, thus data could not be collected. Thus, the efficacy analysis was performed using the secondary efficacy endpoint, change in quality of life as measured by SF-36.
Secondary
Change in Quality of Life
Quality of life, as measured by the Short Form-36 Health survey (SF-36) (quality of life questionnaire). The SF-36 consists of eight scaled scores, which are the weighted sums of the questions in their section. Each scale is directly transformed into a 0-100 scale on the assumption that each question carries equal weight. The lower the score the more disability.
Time frame: From Day 0 to Day 20, Day 60 and Day 90
| Arm | Measure | Group | Value (MEAN) |
|---|---|---|---|
| Afamelanotide | Change in Quality of Life | Day 20 - Day 0 (Physical functioning) | -5.7 Scores on a scale |
| Afamelanotide | Change in Quality of Life | Day 20 - Day 0 (Role Physical) | -14.6 Scores on a scale |
| Afamelanotide | Change in Quality of Life | Day 20 - Day 0 (Bodily Pain) | -1.4 Scores on a scale |
| Afamelanotide | Change in Quality of Life | Day 20 - Day 0 (General Health) | 2.0 Scores on a scale |
| Afamelanotide | Change in Quality of Life | Day 20 - Day 0 (Vitality) | -1.5 Scores on a scale |
| Afamelanotide | Change in Quality of Life | Day 20 - Day 0 (Social functioning) | 1.2 Scores on a scale |
| Afamelanotide | Change in Quality of Life | Day 20 - Day 0 (Role - Emotional) | -7.1 Scores on a scale |
| Afamelanotide | Change in Quality of Life | Day 20 - Day 0 (Mental Health) | 1.9 Scores on a scale |
| Afamelanotide | Change in Quality of Life | Day 20 - Day 0 (Physical Component Summary) | -2.6 Scores on a scale |
| Afamelanotide | Change in Quality of Life | Day 20 - Day 0 (Mental Component Summary) | 0.3 Scores on a scale |
| Afamelanotide | Change in Quality of Life | Day 60 - Day 0 (Physical Functioning) | -13.6 Scores on a scale |
| Afamelanotide | Change in Quality of Life | Day 60 - Day 0 (Role- Physical) | -8.9 Scores on a scale |
| Afamelanotide | Change in Quality of Life | Day 60 - Day 0 (Bodily Pain) | 2.0 Scores on a scale |
| Afamelanotide | Change in Quality of Life | Day 60 - Day 0 (General Health) | -4.2 Scores on a scale |
| Afamelanotide | Change in Quality of Life | Day 60 - Day 0 (Vitality) | 5.4 Scores on a scale |
| Afamelanotide | Change in Quality of Life | Day 60 - Day 0 (Social Functioning) | 0.0 Scores on a scale |
| Afamelanotide | Change in Quality of Life | Day 60 - Day 0 (Role- Emotional) | 6.0 Scores on a scale |
| Afamelanotide | Change in Quality of Life | Day 60 - Day 0 (Mental Health) | 2.9 Scores on a scale |
| Afamelanotide | Change in Quality of Life | Day 60 - Day 0 (Physical Component Summary) | -4.7 Scores on a scale |
| Afamelanotide | Change in Quality of Life | Day 60 - Day 0 (Mental Component Summary) | 4.3 Scores on a scale |
| Afamelanotide | Change in Quality of Life | Day 90 - Day 0 (Physical Functioning) | -13.6 Scores on a scale |
| Afamelanotide | Change in Quality of Life | Day 90 - Day 0 (Role- Physical) | 0.0 Scores on a scale |
| Afamelanotide | Change in Quality of Life | Day 90 - Day 0 (Bodily pain) | -2.7 Scores on a scale |
| Afamelanotide | Change in Quality of Life | Day 90 - Day 0 (General Health) | -4.1 Scores on a scale |
| Afamelanotide | Change in Quality of Life | Day 90 - Day 0 (Social Functioning) | 0.0 Scores on a scale |
| Afamelanotide | Change in Quality of Life | Day 90 - Day 0 (Role- Emotional) | 0.0 Scores on a scale |
| Afamelanotide | Change in Quality of Life | Day 90 - Day 0 (Mental Health) | -2.1 Scores on a scale |
| Afamelanotide | Change in Quality of Life | Day 90 - Day 0 (Physical Component Summary) | -3.0 Scores on a scale |
| Afamelanotide | Change in Quality of Life | Day 90 - Day 0 (Mental Component Summary) | 0.7 Scores on a scale |
| Placebo | Change in Quality of Life | Day 60 - Day 0 (Vitality) | -5.4 Scores on a scale |
| Placebo | Change in Quality of Life | Day 20 - Day 0 (Physical functioning) | 8.0 Scores on a scale |
| Placebo | Change in Quality of Life | Day 90 - Day 0 (Role- Emotional) | -11.7 Scores on a scale |
| Placebo | Change in Quality of Life | Day 20 - Day 0 (Role Physical) | 0.0 Scores on a scale |
| Placebo | Change in Quality of Life | Day 60 - Day 0 (Social Functioning) | -17.5 Scores on a scale |
| Placebo | Change in Quality of Life | Day 20 - Day 0 (Bodily Pain) | -11.6 Scores on a scale |
| Placebo | Change in Quality of Life | Day 90 - Day 0 (Bodily pain) | -13.8 Scores on a scale |
| Placebo | Change in Quality of Life | Day 20 - Day 0 (General Health) | -27.6 Scores on a scale |
| Placebo | Change in Quality of Life | Day 60 - Day 0 (Role- Emotional) | -10.0 Scores on a scale |
| Placebo | Change in Quality of Life | Day 20 - Day 0 (Vitality) | -7.9 Scores on a scale |
| Placebo | Change in Quality of Life | Day 90 - Day 0 (Physical Component Summary) | -5.1 Scores on a scale |
| Placebo | Change in Quality of Life | Day 20 - Day 0 (Social functioning) | 7.5 Scores on a scale |
| Placebo | Change in Quality of Life | Day 60 - Day 0 (Mental Health) | -2.0 Scores on a scale |
| Placebo | Change in Quality of Life | Day 20 - Day 0 (Role - Emotional) | 10.0 Scores on a scale |
| Placebo | Change in Quality of Life | Day 90 - Day 0 (General Health) | -32.0 Scores on a scale |
| Placebo | Change in Quality of Life | Day 20 - Day 0 (Mental Health) | 3.0 Scores on a scale |
| Placebo | Change in Quality of Life | Day 60 - Day 0 (Physical Component Summary) | -9.4 Scores on a scale |
| Placebo | Change in Quality of Life | Day 20 - Day 0 (Physical Component Summary) | -4.8 Scores on a scale |
| Placebo | Change in Quality of Life | Day 90 - Day 0 (Mental Health) | -9.0 Scores on a scale |
| Placebo | Change in Quality of Life | Day 20 - Day 0 (Mental Component Summary) | 2.7 Scores on a scale |
| Placebo | Change in Quality of Life | Day 60 - Day 0 (Mental Component Summary) | -2.5 Scores on a scale |
| Placebo | Change in Quality of Life | Day 60 - Day 0 (Physical Functioning) | -10.0 Scores on a scale |
| Placebo | Change in Quality of Life | Day 90 - Day 0 (Social Functioning) | -12.5 Scores on a scale |
| Placebo | Change in Quality of Life | Day 60 - Day 0 (Role- Physical) | -13.8 Scores on a scale |
| Placebo | Change in Quality of Life | Day 90 - Day 0 (Physical Functioning) | -5.0 Scores on a scale |
| Placebo | Change in Quality of Life | Day 60 - Day 0 (Bodily Pain) | -21.4 Scores on a scale |
| Placebo | Change in Quality of Life | Day 90 - Day 0 (Mental Component Summary) | -6.3 Scores on a scale |
| Placebo | Change in Quality of Life | Day 60 - Day 0 (General Health) | -33.0 Scores on a scale |
| Placebo | Change in Quality of Life | Day 90 - Day 0 (Role- Physical) | -3.8 Scores on a scale |